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ECONOMICREGULATORYETHICAL & SOCIALPATIENTS PUBLIC COMMUNICATION

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Overview 1

Summary of recommendations 3

Patients’ Needs 5

Ethical and Societal Aspects 11

Economic Impact 19

Regulation 25

Communication 31

Participants 37

Although very promising, nanomedicine may add new

dimensions to many ethical, societal and economic

issues. For the promises to be realised and to

achieve the maximum benefit of nanomedical

innovations for everyone, the way has to be

paved for a safe, integrated and

responsible approach to nanomedicine.

This will also be a necessary condition

for the sustainable competitiveness of

nanomedical research and

development in Europe, and of its

healthcare industry. It is therefore of

primary importance to understand the

possible impacts and consequences of

nanomedicine in advance.

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• Patients’ Needs – Our research showsthat patients want nanomedicine and they want toknow more about it from reliable sources. TheEuropean Commission, national governments, andtrade and research associations all have a role toplay in ensuring dialogue with, and informationprovision to, patients.

• Ethical and Societal Aspects -Ethical engagement with nanomedicine needs tobegin with the very concept of 'nanomedicine', aword that now groups diverse research activitiestogether. Nanomedical researchers, physicians,patients, and policy makers will all benefit when, onthe basis of philosophical and social analysis, theprogramme and purpose of nanomedicine arebetter understood and more clearly defined.

• Economic Impact - Reliable data isneeded to predict the impact of nanomedicine onhealthcare costs and benefits, and market growth.This information is required to enable the EMEA tomake decisions on early interventions and nationalauthorities to make reimbursement decisions.

• Regulation - A proactive regulatory systemis required that ensures better coordination andharmonisation of regulatory procedures, earlydialogue with users and stakeholders, and takesaccount of the economic cost implications ofregulation. Given its recently enhanced role, itseems reasonable to suggest that DG SANCOshould take the lead in encouraging European levelregulatory bodies to achieve this aim. At MemberState level, national governments shouldencourage national regulatory bodies to takesimilar action.

• Communication – The EuropeanCommission should provide credible andaccessible sources of balanced informationabout nanomedicine, to facilitate understandingand dialogue.

* Ref. Economic ImpactWorking Group

Nanomedical applications are not just atheoretical possibility – for example, the RoundTable has identified 45 products that are alreadyon the market . However, the field ofnanomedicine is still a relatively new one. Thismeans that as this report is published, Europe isat an ideal moment to consider the impacts andconsequences of nanomedicine, as well asaction required as a result.

Several factors serve to underline the particularrelevance and timeliness of this report. Therecently enhanced role of DG SANCO, and theEuropean Commission’s anticipated strategicAction Plan for Nanosciences andNanotechnologies 2010-2015, rank amongthese. Another factor is the scale and depth ofimpact that nanomedicine may deliver, whichshould not be underestimated. Nanomedicine isenabling us to take a significant step forward inunderstanding and treating disease, by shiftingattention to the molecular level.

As well as adding another layer to healthcare,nanomedicine also presents us with arguablythe best case study of changing business modelsin terms of the move from curative to preventivemedicine. In the context of health being viewedincreasingly as ‘well-being’ rather than ‘absenceof disease’, nanomedicine may have much tocontribute.

Nanomedicine is also covering new ground inthat it is combining many previouslyunconnected disciplines, such as economics,supply chain and insurance, to name but a few.This convergence of different fields means thatwe need to be very clear about where and howwe start thinking about the impact ofnanomedicine.

For all of these reasons, not taking action nowwould be a wasted opportunity. By doing nothing,we would significantly risk blocking thedevelopment of innovative procedures in Europe,to the detriment of European research anddevelopment, the healthcare industry, and, mostimportantly, patients.

It’s never too early to act until it’s too late.

The NanoMed Round Table brought together expert stakeholders from acrossEurope within five Working Groups, each of which considered a specific field highlyrelevant to decision-making regarding nanomedical innovations.

1 US National Institutes ofHealth:http://nihroadmap.nih.gov/nanomedicine

2 The European MedicinesAgency, whose “mainresponsibility is theprotection and promotion ofpublic and animal health,through the evaluation andsupervision of medicinesfor human and veterinaryuse”: www.ema.europa.eu

3 The European CommissionDirectorate General forHealth and Consumers

The Groups’ key findings were as follows:

P A report on the nanomedicine economic, regulatory, ethical and social environment

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The European Commission should:• establish a platform to provide credible and accessible

sources of balanced information on the methods, benefitsand risks of nanomedicine.

• investigate funding opportunities for EU level trade andresearch associations to produce lay information usingstakeholder dialogue on nanomedicine research.

• support patient organisations to investigate whethernanomedicine is currently/potentially useful in treatingtheir condition (with a view to their providing balancedinformation to patients if so), and provide funding toidentify and understand the issues that it raises for them.

• develop communication guidelines for the variousnanomedicine stakeholders, and provide good practiceexamples.

The European Commission, national governments, industryand independent grant organisations should allocate asignificant percentage of financial resources in the field ofnanomedicine to public communication.

The European Commission and national funding agenciesshould integrate public involvement in decision making onpriorities in research funding and encourage, train andreward scientists in public engagement activities.

Patient organisations should be involved in the work of theEuropean Technology Platform on Nanomedicine and the“NANOfutures” European Technology Integration andInnovation Platform in Nanotechnology initiative, and beencouraged to participate in governmental stakeholder fora.Patients’ involvement in EU and national policy makingprocesses should also be institutionalised.

Parliamentarians at EU and national level should conductmulti-stakeholder hearings, to deliberate in a comparativemanner the value of basic nanomedical research forprevention, diagnosis and therapy of disease.

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The European Commission and national governmentsshould:

• integrate the deliberation of ethical and societal issueswith consideration of the feasibility of particulardevelopments in nanomedicine.

• encourage social science and humanities research thatgoes beyond the risks and benefits of medical innovationsto include the consideration of promises, hopes andanxieties.

• support the development of a broader range ofmethodologies for research on the environmental, healthand social implications, and on the ethical, legal and socialaspects, of nanomedicine.

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The European Commission and national governmentsshould:

• together with the European Science Foundation and theEuropean Research Council, initiate a deliberative processabout the possibly distinctive features of nanomedicinethat include its social and ethical dimensions. This processshould inform the setting of EU Framework Programmeand national research agendas.

• in consultation with patient organisations, continue todiscuss priorities for nanomedicine and fund theirresearch and development, encouraging the integration ofnational nanomedicine strategies to ensurecomplementarity.

• establish partially public-funded technology-specificreference centres linking early development with clinicalresearch and clinical practice.

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The European Commission should: • establish and promote supporting mechanisms that boost

the effectiveness of the existing regulatory framework,especially by harmonising regulatory procedures onreporting and data collection.

• ensure the proportionate responsiveness of regulatorypolicies through engagement and partnership with usersand stakeholders, as well as taking into account factors thatdiffer between Member States (e.g. national regulatoryinfrastructures and cultures), and monitoring health andenvironmental impact.

• support institutional mechanisms that facilitate a commonperspective regarding clarity, objectivity and commonpractice for credibility and authority.

• regulators should take into consideration the economicimplications of regulation for nanomedicine and the fundingsupport needed for access to regulatory expertise and extracompliance investment, especially for SMEs.

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Companies and clinicians should produce data based onwell-defined criteria of cost-effectiveness for the economicevaluation of nanotechnology-based innovations in clinicaltrials and health technology assessment studies.

The European Commission should: • together with national governments, promote and support

projects in partnership with key stakeholders, to assessthe cost-effectiveness of nanomedical innovations as earlyas possible.

• launch a health economics project to assess the economicimpact and emergence of new cost models relating tonanotechnological innovations in preventive medicineand the monitoring of chronic diseases..

• provide funding to examine access issues relating tonanomedicine products.

• support further research to work with bodies representingclinicians to gauge awareness of novel possibilities arisingfrom nanomedicine and incorporate these into clinicalpractice.

• national reimbursement agencies and public and privateinsurers should establish a European working group toconsider the future impact of innovative approaches inhealthcare systems with nanotechnology as a case study.

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The European Commission should: • to improve European and international strategies

for maximising the positive economic impact ofnanomedicine.

• together with national governments, involvelow-income countries in developing a fair andsustainable global policy on benefit sharing innanomedicine.

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Patients have relatively low levels of knowledgeand awareness of nanomedicine, but they wouldlike more information.Despite this low level of knowledge there arehigh levels of support among patients fornanomedicine products and research.Patients have clear views on how and fromwhom they would like to receive this information.

Patients do not think that nanomedicine isinherently unsafe, but there is a lack of clearunderstanding about the potential safety aspects.Nanomedicine is an opportunity that patientswant to see embraced. Equally, there is asignificant and time-critical opportunity to informpatients about it.

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There is low awareness and knowledge ofnanomedicine among patients and patientorganisations, even in condition areas where it isbeing used. The overwhelming majority of patientswould like to receive more information onnanomedicine, ideally via the internet, from patientorganisations and/or clinicians.

oÉÅçããÉåÇ~íáçå NThe European Technology Platform on Nanomedicine and“NANOfutures” European Technology Integration andInnovation Platform in Nanotechnology, together with theEuropean Patients’ Forum, should explore the involvement ofpatient organisations in the work of the Platforms, to provideinput from patients’ perspective and improve patients’understanding of nanomedicine. For these reasons, patientorganisations’ views should also actively be sought as part ofany other nanomedicine strategy development at EU level.

oÉÅçããÉåÇ~íáçå OThe European Commission has produced much usefulinformation on the internet on nanotechnology aimed at thegeneral public, e.g. short films, leaflets and brochures. Itshould organise the production of similar lay information onnanomedicine, which could be used by patient organisations,clinicians and the media.

oÉÅçããÉåÇ~íáçå PEuropean-level trade and research associations shouldproduce lay information using stakeholder dialogue onnanomedicine research. National-level trade and researchassociations should disseminate this information in theircountry’s language(s) to patient organisations, media and

bodies representing clinicians. The European Commissionshould investigate what existing programmes should orcould fund this, and/or develop new funding streams whererequired, e.g. science and society programmes or EuropeanScience Foundation.

oÉÅçããÉåÇ~íáçå QPatient organisations should be supported by the EuropeanCommission to investigate whether nanomedicine iscurrently or potentially useful in treating their condition, andif so to enable engagement in nanomedicine with a view toproviding balanced information to patients and carers.

oÉÅçããÉåÇ~íáçå RThe European Commission, working with the EuropeanPatients’ Forum, should identify and engage with patientorganisations that already communicate to their memberson nanomedicine, and work with them to produce a ‘bestpractice’ case study and toolkit (e.g. FAQs, webpages, shortfiles) to help guide and support other patient organisations.

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The majority of patients view nanomedicine as a technologythat could address many unmet medical needs and theysupport nanomedicine research.

oÉÅçããÉåÇ~íáçå SThe European Commission and national governments inconsultation with patient organisations should continue todiscuss priorities for nanomedicine and fund their researchand development. To ensure complementarity and avoidduplication, the European Commission should encourage theintegration of national nanomedicine strategies.

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Patients do not view nanomedicine as inherently unsafe, butthere is a lack of clear understanding about the potentialsafety aspects that may be unique to it. This should beaddressed through appropriate safeguards and thecommunication of information. Patients have mixed views onwhether nanomedicine is different from other new types ofmedical research.

oÉÅçããÉåÇ~íáçå TWhere governments have organised stakeholder fora todiscuss the potential risks and benefits of nanotechnologiesincluding nanomedicine, they should encourage patientorganisations to participate. Where such fora do not exist,governments should urgently consider establishing them.

oÉÅçããÉåÇ~íáçå UThe European Commission should provide funding for focusgroups and other participatory methods (e.g. citizenconferences, interviews, surveys) in a number of Europeancountries, to identify and understand the spectrum of issuesthat nanomedicine raises for patients and their families.

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Further research is desirable on access to nanomedicineproducts and clinical preparedness.

oÉÅçããÉåÇ~íáçå VThe European Commission should provide funding for aproject examining access issues relating to nanomedicineproducts (diagnostic and therapeutic).

oÉÅçããÉåÇ~íáçå NMThe European Commission should support further researchto work with bodies representing clinicians and otherhealthcare professionals to: a) gauge awareness of novelpossibilities arising from nanomedicine and incorporatethese into clinical practice, and b) assist CPD/CME(Continuous Professional Development/Continuing MedicalEducation) to anticipate novel possibilities and formulateappropriate responses.

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The findings of this pilot study are based on the results ofresearch undertaken between June and September 2009.This consisted of an online questionnaire, to which 87 peopleresponded, 12 interviews conducted face-to-face or bytelephone and one focus group held with the West Cumbriabranch of the Parkinson’s Disease Society, which wasattended by 17 Parkinson’s patients and carers.

i. Current Levels of Awareness

As the European Commission states: “(n)anomedicine, theapplication of nanotechnology to health, raises highexpectations for millions of patients for better, more efficientand affordable healthcare and has the potential of deliveringpromising solutions to many illnesses.” In view of this,arguably the most notable finding from the researchconducted by the Patients’ Needs Working Group was the lowlevel of awareness and knowledge among patients andpatient organisations of nanomedicine and its uses. It ispossible that the people who took part in the research weremore informed than average, but awareness and knowledgewere nonetheless low.

1 http://cordis.europa.eu/nanotechnology/nanomedicine.htm#challenge 2 This varied by type of respondent, from around 70 per cent of the patient organisation leaders (8 out of 11), half of the patients who responded and none of the

carers/relatives who responded to that question.

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Two-thirds of interviewees had never heard of nanomedicine,whereas the remainder had heard of it but had not realiseduntil the interview that it was nanomedicine. Furthermore,over half of the interviewees and 52 out of 85 online surveyrespondents had not heard of any use of nanomedicine intheir particular condition areas. This could be attributed tostraightforward lack of awareness of current nanomedicineresearch and development. On the other hand, it could bebecause nanomedicine is not yet being used in manycondition areas.

Parkinson’s Disease is one condition where nanomedicine isalready in use, so one might expect awareness to be higheramong people affected by or interested in this condition.Indeed, of those survey respondents who said they had heardof nanomedicine being used in their condition area, just under90 per cent (25 out of 29 respondents) gave Parkinson’sDisease as their condition area. In comparison, over half ofthose who had not heard of nanomedicine being used in theircondition area listed a range of conditions other thanParkinson’s.

However, this does not explain the full picture because justover half of the total number of survey respondents affectedby/interested in Parkinson’s nonetheless said that they hadnot heard of nanomedicine being used in their condition area.In the focus group no-one had heard of nanotechnology or itsuse in their condition.

There were also geographic variations. For example, all Swissrespondents were affected by/interested in Parkinson’s(thanks to the enthusiastic participation of a Swiss patientgroup), and a very high 20 out of 29 of these were aware ofnanomedicine being used in their condition area. Thiscompared to a much lower 4 out of 29 of the UK respondentsaffected by/interested in Parkinson’s. There were mixedresponses from countries with lower overall numbers ofrespondents, but due to the small numbers involved noconclusions can be drawn from this.

The research suggests that the actual use of nanomedicine ina particular condition area does not guarantee that patientswill automatically be aware of this, although it might make itmore likely. For conditions where the use of nanomedicine isfurther along the horizon, it is therefore unsurprising thatlevels of awareness are significantly lower. It also suggeststhat in most countries there may be considerable room forimprovement in raising patients’ awareness of nanomedicine.

ii. Raising Patients’ Awareness of Nanomedicine

Another salient finding from the Patients’ Needs WorkingGroup’s research - and arguably an unsurprising one giventhe preceding section - was that 80 per cent (68 out of 85respondents) of survey respondents said they would like toreceive more information about nanomedicine. Thepercentage was even higher for some types of respondents:all patient organisation leaders who responded said that theywould like to receive more information, as did almost 90 percent of carers (7 out of 8).

Of those survey respondents who said that they would likemore information on nanomedicine, over 80 per cent (57 outof 68) said they would like to receive this information on theinternet. Over half of the patients and carers at the focusgroup agreed, as did all the patients and carers interviewedfor the qualitative research, with almost 30 per cent wantingpaper-based information. Internet, followed by paper-basedinformation, were also the two top choices when responseswere also analysed by type of respondent.

With regard to the sort of information that patients want, in thewords of some of the interviewees, this should include:• “factual information in lay terms” - there was consensus

among interviewees that the information should bepresented in a lay format to enable patients to makeinformed decisions on their treatment;

• “updates on where the research is currently at”; and• “pros and cons”.

Views on the desired sources of this information were mixed.Over 60 per cent (41 out of 68)3 of survey respondents whowanted to receive more information thought that it shouldcome from clinicians. This is possibly because doctors areconsistently viewed as trusted sources of information.Similarly, over half of the focus group attendees thought thatthe information should come from health care professionals(Specialist Nurses and GPs).

Just over half of survey respondents and interviewees saidthat information should come from patient organisations. Thisfigure rose to three quarters of the focus group attendees,possibly due to the fact that all those present were alreadymembers of a patient organisation, whereas many of thosewho responded to the survey were not.

A third of people participating in the online survey and overhalf of attendees at the focus group wanted to see the mediaproviding information on nanomedicine. Just over threequarters of those surveyed would not look to their national

3 N.B. respondents were able to choose more than one option in the section of the survey on information provision, so overall responses may total more than 100 per cent.

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government to provide information on nanomedicine, and only9 out of 68 respondents said that they would look to theEuropean Institutions. This means that other stakeholdersneed to work harder to ensure that they provide patients withthis information.

This raises the question as to how to improve levels ofawareness among patient organisations and clinicians, notleast given the demands on their resources. Given theirpotential to reach the widest audience, the media also need tobe provided with appropriate information to ensure balancedreporting.

It seems reasonable to suggest that those who are actuallyconducting nanomedicine research and/or developingnanomedicine products are best placed to inform thoseaudiences from whom patients wish to receive informationabout nanomedicine. In turn, informed patients would be ableto feed back into the research agenda, making thisinformation process beneficial to all stakeholders concerned.This is illustrated by Figure 1 below.

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With this in mind, the European Technology Platform (ETP)Nanomedicine seems to be ideally placed to contribute toimproved information provision to patient organisations (andother key audiences). It is a current initiative led by industryand established together with the European Commission, andit brings together a wide range of large industry, SMEs,industrial associations, research institutions, academia andhospitals from across Europe. The ETP has identifieddissemination of knowledge, ethical and safety concerns and“input from other stakeholders like insurance companies orpatient organisations” as playing an important role in thepriority topics on which it is focussing, so it should be involvingthem. The “NANOfutures” European Technology Integrationand Innovation Platform in Nanotechnology (ETIP) initiativecurrently being established should play a similar role.

oÉÅçããÉåÇ~íáçå NThe European Technology Platform on Nanomedicine and“NANOfutures” European Technology Integration andInnovation Platform in Nanotechnology, together with theEuropean Patients’ Forum, should explore the involvement ofpatient organisations in the work of the Platforms, to provideinput from patients’ perspective and improve patients’understanding of nanomedicine. For these reasons, patientorganisations’ views should also actively be sought as part ofany other nanomedicine strategy development at EU level.

However, to ensure that information on nanomedicine iscommunicated as widely as possible steps should be taken bya number of key stakeholders.

oÉÅçããÉåÇ~íáçå OThe European Commission has produced much usefulinformation on the internet on nanotechnology aimed at thegeneral public, e.g. short films, leaflets and brochures. Itshould organise the production of similar lay information onnanomedicine, which could be used by patient organisations,clinicians and the media.

National governments should also take steps to increase theirdialogue with patient organisations on the development anduse of nanomedicine. This is covered in more detail byRecommendation 7. In addition, industry and researchassociations have an important role to play.

oÉÅçããÉåÇ~íáçå PEuropean-level trade and research associations shouldproduce lay information using stakeholder dialogue onnanomedicine research. National-level trade and researchassociations should disseminate this information in theircountry’s language(s) to patient organisations, media andbodies representing clinicians. The European Commissionshould investigate what existing programmes should orcould fund this, and/or develop new funding streams whererequired, e.g. science and society programmes or EuropeanScience Foundation.

However, to be as effective as possible, improving patientawareness of nanomedicine needs to be a two-way process.This is reliant on patient organisations also taking someresponsibility for informing first themselves (e.g. with theassistance of their scientific/medical advisers) andsubsequently both patients and their carers.

oÉÅçããÉåÇ~íáçå QPatient organisations should be supported by the EuropeanCommission to investigate whether nanomedicine is currentlyor potentially useful in treating their condition, and if so toenable engagement in nanomedicine with a view to providingbalanced information to patients and carers.

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1 http://www.etp-nanomedicine.eu/public/about/objectives-mission 2 Nanotechnology-based diagnostics including imaging; targeted drug delivery and release; and regenerative medicine.

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oÉÅçããÉåÇ~íáçå RThe European Commission, working with the EuropeanPatients’ Forum, should identify and engage with patientorganisations that already communicate to their memberson nanomedicine, and work with them to produce a ‘bestpractice’ case study and toolkit (e.g. FAQs, webpages, shortfiles) to help guide and support other patient organisations.

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i. Use of Nanotechnology in Medicine

As part of the Patients’ Needs Working Group’s quantitativeresearch, survey respondents were asked whether theysupported the use of nanotechnology in medicine, on a scaleof 1 to 5, with 1 being “not at all” and 5 being “strongly”. Over60 per cent, 53 out of 85 respondents, supported the use ofnanotechnology in medicine “strongly” or “quite strongly” (5and 4 respectively), with over 40 per cent of those who repliedto this question supporting it “strongly”. Fewer than 10 percent selected 1 or 2.

Interestingly, prior awareness or knowledge of nanomedicinedid not appear to make a significant difference to levels ofsupport. In fact, of those survey respondents supportingnanomedicine “strongly”, a higher proportion – over half - hadnot heard of nanomedicine being used in their condition area.

The interviews conducted as part of the Working Group’sresearch suggest that the reason for this could be patients’willingness in some cases to accept higher levels of risk tofind a cure or treatment. Indeed, the majority of intervieweesthought that nanomedicine would be useful in the treatmentof their condition. However, there was confusion as to hownanomedicine could be useful.

ii. Nanomedicine Research

The Patients’ Needs Working Group’s research indicates thatthe majority of patients view nanomedicine as a technologythat could address many unmet medical needs and thatpatients want to see nanomedicine research taking place.

Support for nanomedicine research was also high. 53 out of85 respondents to the survey question on research said that,within current spending, they would like to see morenanomedicine research into their condition. Among leaders ofpatient organisations and carers/relatives support forresearch support was also high: 8 out of 11 and 5 out of 8respectively.

The qualitative research undertaken for this report suggeststhat support for nanomedicine research increases with theprovision of information. Based on the information given to

them in a slide presentation, over 90 per cent of the 12patients and carers interviewed said that research intonanomedicine should be continued, not only in their conditionarea but in all areas.

oÉÅçããÉåÇ~íáçå SThe European Commission and national governments inconsultation with patient organisations should continue todiscuss priorities for nanomedicine and fund their researchand development. To ensure complementarity and avoidduplication, the European Commission should encourage theintegration of national nanomedicine strategies.

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i. Safety and Risk

Compared to other areas of questioning, views on the safety ofnanomedicine were more mixed. On a scale of 1 to 5, with 1being “not safe at all” and 5 being “very safe”, 24 out of 85(almost 30 per cent) of respondents chose 4, with 13 of the 85respondents opting for 3. Notably, the highest proportion (26respondents) selected “don’t know”.

One survey respondent specifically commented: “I believe thatnanomedicine, like other technologies, has great potentialprobably both for good and for harm. Proper, but reasonableand balanced, controls will be needed to get the maximumbenefit with the minimum harm.”

Likewise, the qualitative research also elicited a wide range ofresponses on the issue of safety. Although many of theinterviewees had concerns about the development of theresearch, based on the information presented to them theymade a risk judgement and supported the technology withappropriate regulation.

Prior awareness of nanomedicine appeared to influence viewson safety: it was notable that the interviewees who wereconcerned about the safety of nanomedicine did not haveprior knowledge of nanomedicine. Similarly, of those surveyedwho “didn’t know” whether nanomedicine was safe, ten timesas many had not previously been aware of nanomedicinebeing used in their condition area than had been aware of this.

It could therefore be concluded from the Patients’ NeedsWorking Group’s research that patients do not viewnanomedicine as inherently unsafe. Nonetheless there is alack of clear understanding about the potential safety aspectsthat may be unique to nanomedicine. This should beaddressed not only through appropriate safeguards but alsothe communication of information. As one interviewee put it:“I would like for someone to get across what the advantagesand disadvantages are to patient groups”. This could be

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addressed by Recommendations 1 to 4 as well asRecommendation 7, which would have the dual effect ofimproving patients’ understanding of the risks and benefits,and also providing an official conduit for giving their views togovernment.

oÉÅçããÉåÇ~íáçå TWhere governments have organised stakeholder fora todiscuss the potential risks and benefits of nanotechnologiesincluding nanomedicine, they should encourage patientorganisations to participate. Where such fora do not exist,governments should urgently consider establishing them.

ii. Other Observations

The Working Group’s qualititative research indicated thatthere were mixed views among patients in terms of whethernanomedicine was regarded as different from any other newtypes of medical research. Just over half of the intervieweesthought that nanomedicine was simply the next step inmedical research, whereas others thought that it was “a newbranch” of medical research and “completely different interms of technology”.

Due to patient groups’ financial constraints, the WorkingGroup was unable to focus in greater detail on ethical issues.However, this is an important area on which it would behelpful to gain further insight.

oÉÅçããÉåÇ~íáçå UThe European Commission should provide funding for focusgroups and other participatory methods (e.g. citizenconferences, interviews, surveys) in a number of Europeancountries, to identify and understand the spectrum of issuesthat nanomedicine raises for patients and their families.

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i. Access to Nanomedicine Products

The timescale and funding of this project did not permit theWorking Group to address issues surrounding access tonanomedicine products. However, given patients’ evidentsupport for nanomedicine and its potential to address unmetmedical needs, this is an area that needs to be exploredfurther, in order to establish:• Whether current systems for Health Technology

Assessment (HTA) and reimbursement processes cancope effectively with nanotech-derived products fordiagnosis and therapy; and

• For patients, whether equity between diseases and acrossnational boundaries can be achieved.

`çåÅäìÇáåÖ=`çããÉåíë=It is important to recognise that this report is verymuch a snapshot of a particular point in time.Patients’ views on nanomedicine are likely to changeas nanomedicine technology develops, soengagement with patient organisations needs to beundertaken on a sustained basis.

Crucially, however, nanomedicine is a novel area ofmedicine and as such it is a difficult area for patientorganisations to explore. They have limited financesand are often run by volunteers or a small numberof staff, so the opportunity for them to engage onissues such as this is constrained by resourcesrather than by willingness.

Consequently, if public bodies wish to engage withpatient organisations on nanomedicine, as well asbeing sustained, consultation also needs to berealistically organised and funded.

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oÉÅçããÉåÇ~íáçå VThe European Commission should provide funding for aproject examining access issues relating to nanomedicineproducts (diagnostic and therapeutic).

ii. Clinical Preparedness

A very small number of medical professionals responded tothe Patients’ Needs Working Group’s online survey. Due to thesmall sample size and the fact that this was beyond theWorking Group’s remit, no conclusions have been reached onawareness of and support for nanomedicine among medicalprofessionals.

However, having an informed clinical community is key, notleast given the high importance that patients place onreceiving information on nanomedicine from clinicians (seesection above on ‘Raising Patients’ Awareness ofNanomedicine’).

oÉÅçããÉåÇ~íáçå NMThe European Commission should support further researchto work with bodies representing clinicians and otherhealthcare professionals to: a) gauge awareness of novelpossibilities arising from nanomedicine and incorporatethese into clinical practice, and b) assist CPD/CME(Continuous Professional Development/Continuing MedicalEducation) to anticipate novel possibilities and formulateappropriate responses.

NN A report on the nanomedicine economic, regulatory, ethical and social environment

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Ethical engagement with nanomedicine beginswith the very concept of ‘nanomedicine’, a wordthat now groups diverse research activitiestogether. Nanomedical researchers, physicians,patients and policy makers will all benefit when,on the basis of philosophical and social analysis,the programme and purpose of nanomedicineare better understood and more clearly defined.

The discussion of ethical and societal questionsabout nanomedicine must be informed byconsideration of the feasibility of nanomedicineapplications.As well as the significance of nanomedicalinnovations, promises, hopes and anxietiesshould also be considered.

Such questions focus on applications (e.g. new diagnostictools or brain-machine interfaces) which are envisioned byvarious promoters of nanomedicine and which resonatepowerfully within popular culture. Their discussion reliesupon the assumption that such applications are realistic in theshort or medium term.

oÉÅçããÉåÇ~íáçå NSince the discussion of implications of hypothetical butunlikely products is unhelpful, the European Commissionand national governments should integrate the deliberationof ethical and societal issues with consideration of thefeasibility of particular nanomedicine developments. Thisrequires assessment of the visions driving nanotechnologicaldevelopments, as well as historically and theoreticallyinformed analyses of the cultural tendencies, societalaspirations, and commercial trends that influencenanotechnology for medicine and health.

oÉÅçããÉåÇ~íáçå OThe European Commission and national governmentsshould support the development of a broader range ofmethodologies for research on the environmental, healthand social (EHS) implications and the ethical, legal and socialaspects (ELSA) of nanomedicine. This requires the explicitformulation of the criteria used to determine what is relevantfor public deliberation.

Scientific and Technological Ambitions of Nanomedicine

Nanomedical research is hugely ambitious. In the search tounderstand and treat disease, it shifts attention beyond thecellular to the molecular level, and seeks to control a pathwayfrom the molecular level right up to patients’ and physicians’daily routines. Analysis of the scientific and technologicalambitions of nanomedical research directs us to a view ofnanomedicine as, primarily, basic research on theunderstanding, diagnosis and treatment of disease.

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Questions which hitherto have become central toreflection on nanomedicine include:• The ‘right not to know’ – The development of

nanotechnologically-enabled diagnostic tools islikely dramatically to widen the gap betweendiagnostic capability and available therapies.What rights and means do citizens have not toknow, or make known, the results of diagnostictests?

• The ethics of human enhancement - Howshould we determine the permissibility andregulation of interventions that could be appliedbeyond the therapeutic contexts to enhanceindividuals’ physical and mental capabilities?

• Demographic effects - The Nano CancerInitiative of the US National Institutes of Healthhas claimed that, by 2015, no one will suffer ordie from cancer. Although this seems unrealistic,such a drastic decrease of mortality would havea considerable impact on social securitysystems.

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Moreover, the lack of a clear-cut definition of ‘nanomedicine’could be an opportunity to configure nanomedical research soas to mark a departure from ‘business as usual’. Someprominent actors in the field suggest that the novelty ofnanomedicine is its adoption of a ‘bottom-up’ approach whichdraws on principles of self-organisation and on systemstheory more generally. Accordingly, nanomedicine might bedefined, for funding purposes, as medical research groundedin systems biology. The notion of the system encompassesmany orders of magnitude, from the molecular level all theway up to societies and healthcare services, so this definitionaffords a unique opportunity to incorporate ethical andsocietal perspectives into the research process.

oÉÅçããÉåÇ~íáçå PThe European Commission’s Research Directorate-General,the European Research Council and the European ScienceFoundation together with national funding agencies andresearch ministries, should initiate a deliberative processabout the possibly distinctive features of nanomedicine thatinclude its social and ethical dimensions. This process mightinclude the development of roadmaps and should inform thesetting of EU Framework Programme and national researchagendas, and engage the scientific communities and theirgoverning bodies, including academies of science.

Nanomedicine is the subject of high expectations andconsiderable public investment, but has yet to prove itself. Inorder to assess the value of basic nanomedical research, wemight ask where a nanomedical approach is most productiveand beneficial for researchers, healthcare systems andsocieties.

oÉÅçããÉåÇ~íáçå QParliamentarians at EU and Member State level shouldconduct multi-stakeholder hearings that encompass publicand world health advocates and patient groups. The aimshould be to deliberate in a comparative manner the value ofbasic nanomedical research for prevention, diagnosis andtherapy of disease. Scientific input should be provided fromfields as diverse as medical anthropology, international law,bioethics and cancer research. Such hearings will focus andstrengthen nanomedical research and raise public awarenessof its possibilities and expectations.

Changing Conceptions of Medicine and Health

Health used to be defined as ‘normal functioning’, or as theabsence of disease, but is now increasingly viewed as ‘well-being’, or as living to the fullness of one’s capacities.Irrespective of whether and when nanomedical research livesup to its scientific and technological ambitions, it is already apart of these developments. A host of issues arises wherecommercial interests and medical research intersect.Nanotechnologically-enabled diagnostic tools, for example,expand possibilities of self-diagnosis and self-treatment,contributing to a reorganisation of medical expertise. At thesame time, the marketing of diagnostic capabilities empowersindividuals but also exploits their vulnerabilities and anxieties.

oÉÅçããÉåÇ~íáçå RThe European Commission and national governments shouldencourage social science and humanities research that goesbeyond the risks and benefits of medical innovations to includethe consideration of promises, hopes and anxieties. Suchresearch should assess the emerging divisions of roles andresponsibilities between patients, physicians, hospitals, e-health information systems, insurers and consumers. Itshould also identify safeguards that could and should beprovided by public health care systems.1 This includes calls for research on foresight and technology assessment of

nanomedical developments, as well as the design and evaluationof public engagement exercises.

Low-income countries must be involved in thedevelopment of fair and sustainable globalpolicy on benefit sharing in nanomedicine.

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NP A report on the nanomedicine economic, regulatory, ethical and social environment

If health is defined as the absence of disease or as normalfunctioning, it is an unevenly distributed public good. However,when it is defined more broadly as living to the fullness ofone’s capacities, the gap between rich and poor countrieswidens further, broadening responsibilities and opportunitiesfor benefit sharing.

oÉÅçããÉåÇ~íáçå SThe European Commission and national governmentsshould create ways of involving low-income countries in thedevelopment of a fair and sustainable global policy on benefitsharing in nanomedicine. Avenues for this might includeinternational organisations such as UNESCO and bodieswhich work towards and monitor the implementation of theUnited Nations Millennium Development Goals .

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There have been many calls for an ethics of nanomedicine,and a number of authors and reports have discussed whatthis should look like or have surveyed the field as it currentlystands. These should be understood as early attempts tocome to terms with the emerging field of ‘nanomedicine’(given that nanomedicine does not involve new forms ofmedical practice, a more appropriate label might be‘nanotechnological research for medicine and health’ ). Aswith all ethics for new and emerging science and technology(NEST-ethics ), these first attempts at identifying ethical andsocietal issues follow familiar patterns. The Working Grouptherefore began its work by reflecting on the various ways inwhich nanomedicine has so far been approached.

For example, discussion of nanomedicine has often involvedfar-reaching visions promising entirely new ways ofdiagnosing, preventing, and treating disease. While it seemsobvious that such new techniques will present new ethicaland societal challenges, if the visions concern only thealleviation of human suffering it becomes difficult to see whatthese challenges might be. Accordingly, there is a tendency tosimply call for further research on prospective technologyassessment and potential ethical and societal issues. Aresponse in the opposite direction imagines extreme ethicaland societal problems which might appear if the most radicalpromises were fulfilled, such as the ‘problem’ of a worldwithout disease, in which people live much longer and currentsocial security systems would be overtaxed. Such speculation

requires excessive credulity about the likely success ofnanomedicine, and has led to discussion of the pitfalls of‘speculative ethics’ of nanotechnologies and nanomedicalresearch.

One of the central questions in this debate is thereforewhether ethical and societal issues should be discovered byimagining (and then discussing the ethics of) those diagnosticcapabilities or therapeutic methods which are more or lesslikely. However, instead of trying to imagine possibleoutcomes and future applications, another approach might beto consider what we bring to nanomedicine.

The debate about speculative ethics shows that ethical andsocietal issues arising from nanomedicine cannot beconsidered in isolation from questions of what we know anddon’t know, or of how feasible different scenarios are.Accordingly, the Working Group recommends a process ofintegration of ethics and epistemology, sometimes referred toas vision assessment. This process requires more than theidentification of particular technological trends, insteadinvolving full consideration of how societies change alongsidetheir technologies, and the development of detailed socio-technical scenarios. Debate about speculative ethics alsoshows that there are different ways of approaching the ethicaland societal dimensions of nanomedicine. The Working Groupencourages the development and pursuit of alternativemethods to those which currently dominate debate.

oÉÅçããÉåÇ~íáçå NSince the discussion of implications of hypothetical butunlikely products is unhelpful, the European Commissionand national governments should integrate the deliberationof ethical and societal issues with consideration of thefeasibility of particular nanomedicine developments. Thisrequires assessment of the visions driving nanotechnologicaldevelopments, as well as historically and theoreticallyinformed analyses of the cultural tendencies, societalaspirations, and commercial trends that influencenanotechnology for medicine and health.

oÉÅçããÉåÇ~íáçå OThe European Commission and national governmentsshould support the development of a broader range ofmethodologies for research on the environmental, healthand social (EHS) implications and the ethical, legal and socialaspects (ELSA) of nanomedicine. This requires the explicitformulation of the criteria used to determine what is relevantfor public deliberation.

2 United Nations Educational, Scientific and Cultural Organization3 www.un.org/millenniumgoals4 E.g., European Group on Ethics 2007, Bruce 2006, Ach & Siep 2006, Ach & Lüttenberg 2008, Bawa & Johnson 2007, Ebbesen & Jensen 2006, Godman 2008, Gordijn 2 25 2005, Grunwald 2005, Haker 2008, Horner 2005, Khushf 2007, Lenk & Biller-Andorno 2007, Weil 2003.6 European Commission 2006, European Science Foundation 2005, Murday, J. S.; Siegel, R. W.; Stein, J.; Wright, J. F. 2009, NanoBio-Raise 2008.Swierstra & Rip 2007.

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The Working Group identified a number of considerationswhich might be used to recommend or reject particularapproaches: • To what degree is the approach speculative, as opposed to

grounded in sound judgements of feasibility?• How concrete is the approach’s orientation towards future

applications, compared to its concrete focus on thepriorities, values and expectations that currently motivateresearch and research funding?

• Is the main goal of the chosen method to achievepreparedness in the face of uncertainty about the future, orto empower citizens to deliberate current choices inresearch and development?

• Does the research seek to give expression to the concernsof stakeholders, publics, and citizens, or to develop findingswhich are historically and theoretically informed bybioethicists, historians of medicine, political theorists,sociologists and philosophers of science?

The Working Group followed its own advice on the importanceof new approaches, choosing not merely to collate existingconcerns around nanomedicine but rather to highlight areasof ethical questioning resulting from analysis of trends andexpectations driving contemporary nanomedicine. The Grouptherefore sought to look beyond basic research, not so as tofocus on potential futures, but rather by consideringnanotechnologies for medicine and healthcare as thecontinuation of long-term developments within healthcare.The ethical and social significance of nanomedicine lies in itsinvitation to question the place of medical research inmedicine, healthcare, and society at large. In doing this it alsochallenges us to consider alternative ways of configuringscience, technology and medicine so as best to supporthuman flourishing.

In adopting this approach the Working Group is suggesting analternative to what is known as ‘consequentialism’.Consequentialist ethics evaluates actions by considering theirconsequences or effects. According to consequentialism,technologies are judged by their outcomes or applications – ifthese are, for the most part, beneficial, then the technology isgood. However, consequentialism is only one of a number ofapproaches to ethics, and a controversial one at that. Otherapproaches include ‘virtue ethics’, which emphasises thecharacter of persons, institutions or traditions, and‘deontology’, which is based on principles and rules aboutgood actions.

Nanomedicine consolidates particular trends, expressescertain scientific ideas, pursues particular economic interests,and makes visionary promises: all of these carry values,norms, and ideas of the good life. These deserve ethicalconsideration, but are ignored within consequentialistapproaches. In other words, whether good or ill comes fromnanomedicine depends greatly on how the researchenterprise is framed and conceptualised. The principles thatlead us to dedicate public funds to nanomedical research andthe character of European knowledge societies are thereforeimportant features for any ethical analysis of nanomedicine.

The Working Group chose to take this comprehensive view,attempting to consider the forces shaping the development ofnanomedicine. It placed the programmes and promises ofnanomedicine in the context of long-term trends which haveshaped – and will continue to shape – entire healthcaresystems. It did this by drawing on academic expertise fromdisciplines including bioethics, human rights, philosophy ofmedicine, medical anthropology, philosophy of science andtechnology, history of medicine and public health.

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Given the sheer breadth of research activities which the term‘nanomedicine’ incorporates, the Working Group suggeststhat ‘nanotechnology for medicine and health’ is moreappropriate. However, in doing so we may be giving up toosoon: perhaps there is a way of using the term wherebynanomedicine appears as a new way of conducting researchand of practicing medicine. Indeed, it might be a vehicle forachieving a new and better alignment of research andpractice.

The Working Group proposes, then, that instead of merelyobserving what goes by the name of nanomedicine we takeseriously the notion that nanomedicine might be anopportunity to do things differently. Discussion ofnanomedicine can act as an invitation to researchers,physicians, patients, policy makers and publics to formallyarticulate their expectations of nanotechnology for medicineand health (e.g. as in the NIH Roadmap initiative in the USA ).

7 European Group on Ethics 2007, Sections 5.4.2 and 5.10.8 National Science and Technology Council Report 1999, European Commission 2006, Max Planck Gesellschaft 2009.9 DEEPEN 2009, Nordmann & Rip 2009, Roache 2008.10 Brune, Ernst & Grunwald 2006, Grunwald 2005.11 Rip & te Kulve 2008.

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NR A report on the nanomedicine economic, regulatory, ethical and social environment

molecular processes deep inside the cell. Taken to itsextreme, this reduction of ordinary observable phenomenato fundamental physical processes amounts to the claimthat “psychiatry is merely applied physics.” Arguably, thiscould place nanomedicine on a path towards diminishingreturns.

ii. If we take seriously the (supposed) novelty of an emergingfield of ‘nanomedicine’, this field might be viewed as ahighly interdisciplinary science of complex systems. Thisscience is based not only on an integrated physiologicalunderstanding of a tumour, for example, but issimultaneously based in stakeholder platforms answeringto public interests. This new science might reorient thereductionist natural science tradition described in (i) abovetowards a multi-level approach to medical research. Ifhealth comprises not just physical but also mental andsocial well-being, then social and ethical considerations arenot ‘add-ons’ to clinical nanomedicine but integral to it.

By deliberating these alternative pathways, patient groups,policy makers, scientists and health professionals can explorewhether nanomedicine might create opportunities tointegrate scientific understanding, medical practice, andethical wisdom. Depending on the route taken, medicalprofessionals could find themselves merely at the receivingend of technological development, or in a context in whichmedical experience and ethical considerations informresearch in unprecedented ways, e.g. as outlined in thefollowing four points:

• If the novelty of nanomedicine is defined only in terms oftechnical intervention at the nanoscale, nanomedicine is‘business as usual’ simply taken to the next level ofphysiological understanding. Philosophical and socialanalysis has pointed out the pitfalls of this approach, e.g.the move from the cellular to the molecular level widensthe already existing distance between medical researchand practice. This ever-increasing distance poses problemsfor technological and economic development as well asunbridgeable discrepancies between the definition of adisease at the molecular level and the symptomaticexperience of the disease in a therapeutic context.

• Associating nanomedicine with a novel way of thinkingenables it to become a research enterprise dedicated, in anintegrated manner, to the formation and re-formation ofhuman well-being. The well-used notions of ‘bottom-upengineering’, ‘systems and integrative biology’, ‘harnessingself-organisation’, and ‘complexity’ suggest that central tothis novel mode of thought is the behaviour and thedynamics of systems. If it were explicitly grounded in

oÉÅçããÉåÇ~íáçå PThe European Commission’s Research Directorate-General,the European Research Council and the European ScienceFoundation together with national funding agencies andresearch ministries, should initiate a deliberative processabout the possibly distinctive features of nanomedicine thatinclude its social and ethical dimensions. This process mightinclude the development of roadmaps and should inform thesetting of EU Framework Programme and national researchagendas, and engage the scientific communities and theirgoverning bodies, including academies of science.

The Working Group arrived at such questions about thenature and purpose of nanomedicine on the basis of itsanalysis of nanomedical ambitions. At first glance, theseresemble the ambitions of rational drug design, in which thedevelopment of new therapies is based on preciseunderstandings of disease processes. In practice, theseambitions were never realised: the deliberate molecularconstruction of pharmaceutical agents proved less successfulthan traditional randomised search processes. Similarly,while genomics promised the discovery of the genesresponsible for specific diseases it remains – several yearsafter the conclusion of the Human Genome Project –questionable whether these ambitions will be fulfilled.Nanomedicine has also promised to provide new diagnostictools and therapeutic interventions based on understandingand control of molecular processes in the cell. Will it suffer thesame fate – that of exaggerated expectations – as rationaldrug design and genomics?

Based on such considerations the Working Groupcharacterised nanomedicine in the following way:Nanomedical research shifts attention beyond the cellular tothe molecular level in the search for basic processesfundamental to the understanding and treatment of disease.It seeks to control a pathway which stretches from themolecular level all the way up to the daily routines of patientsand physicians: it is thus a hugely ambitious project, and anyscientific success may not, in the short or medium term,translate into significant innovations. On the other hand, itmay be more productive than precursors such as rationaldrug design or genomics.

If we understand the scientific and technological ambitions ofnanomedicine in these terms, there are at least twoalternative ways of framing it that deserve deliberation:i. Nanomedicine is part of a long tradition within the natural

sciences towards analysis at deeper and deeperphysiological levels. In this view, causes of and treatmentsfor diseases should not be sought within environmentalconditions, or even at the cellular level, but at the level of

12 NIH Roadmap for Medical Research 2006, Khushf 2008.13 NanoBioRaise 2008.14 Adam 2007.

15 An attitude encountered by one of the members of the Working Group.16 Ahn, Tewari, Poon & Phillips 2006.17 Ref. report by the Economic Impact Working Group.

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systems biology, for example, nanomedicine would not be‘business as usual’ because it would not be concernedmerely with processes at the nanoscale but withinteractions between different levels and scales as well. Itsambition would be to integrate observations at thenanoscale with those of humans in their environment. Itcould also seek to integrate the search for therapies withethical and social conceptions of the good life.

• If nanomedicine is ‘business as usual’, the notion of‘personalised medicine’ is misleading. By definition, aperson is far more than a specific collection of genes but asocial being with a unique biography. To offer ‘personalised’treatment, then, requires more comprehensiveunderstanding and appreciation than can be acquired bylooking at genes, molecules and cells.

• Nanomedicine’s scientific and technological ambitionssuggest that it is not a short-term investment withimmediate returns. A commitment to nanomedicine is acommitment to basic medical research, with the tenuousand long-term prospects of diagnostic and therapeuticbenefits that this typically entails. Depending on whichperspective one adopts, nanomedicine is either a long-term investment demonstrating the commitment ofaffluent societies to the best medical care for their citizens,or a misguided investment with little effect on sustaininghealthy living globally.

Taken together, the considerations outlined above suggestthat it is wrong to take nanomedicine as a given or to assumethat it is only a matter of time until its benefits areforthcoming. Instead, the Working Group suggests thatnanomedicine has yet to prove itself and should be evaluatedin light of comparisons with other areas of health research,especially with regard to its overall contribution to prevention,diagnosis and therapy of disease and to the creation andpreservation of conditions for healthy living.

oÉÅçããÉåÇ~íáçå QParliamentarians at EU and Member State level shouldconduct multi-stakeholder hearings that encompass publicand world health advocates and patient groups. The aimshould be to deliberate in a comparative manner the value ofbasic nanomedical research for prevention, diagnosis andtherapy of disease. Scientific input should be provided fromfields as diverse as medical anthropology, international law,bioethics and cancer research. Such hearings will focus andstrengthen nanomedical research and raise publicawareness of its possibilities and expectations.

Ethical engagement with nanomedicine therefore begins withthe very concept of ‘nanomedicine’, a word that now groupstogether diverse research activities. Nanomedicalresearchers, physicians, patients and policy makers will allbenefit when, on the basis of philosophical and social analysis,the programme and purpose of nanomedicine are betterunderstood and more clearly defined.

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If medicine is the maintenance and restoration of health, thenethical, legal, and societal considerations of nanomedicinewould at first glance appear to be confined to the narrowrange of issues that arise in the process of moving peoplefrom an (undesirable) state of disease to a (desirable) state ofhealth. However, health is no longer defined only as theabsence of disease, but as well-being more generally. Thisbroadening of scope opens the door to many challenges, suchas the issue of human enhancement.

Nanomedical research is unlikely to introduce radically newtechniques for human enhancement. The ethics of humanenhancement are most fruitfully discussed not with regard toimagined nanomedical breakthroughs but by consideringexisting techniques such as sports drug-use or cosmeticsurgery. These exist within a framework of ongoing trends,which are also taken forward by nanomedical research.Through an awareness of these trends, nanomedicine can beunderstood as incorporating broader notions of health ashuman wellness. Health used to be defined as ‘normalfunctioning’, or as the absence of disease; it is nowincreasingly viewed as ‘well-being’ or as living to the fullnessof one’s capacities. Irrespective of whether and whennanomedical research lives up to its scientific andtechnological ambitions, it is already a part of thesedevelopments. Areas such as e-health, point-of-carediagnostics, antibacterials, and neutraceuticals envisagenanotechnologies for medicine and health as part of awellness market addressed to patient-consumers.

An analysis of nanomedicine in this context of changingconceptions of health and disease calls for consideration ofthe patient as consumer and in particular as a consumer ofnanotechnologically-enabled diagnostic tools. Thesediagnostic tools have so far mostly been discussed in terms of‘the right not to know’ and the increasing gap betweendiagnosis and therapy. This narrow focus needs to bewidened, however, as so-called ‘point-of-care diagnostics’

18 Khushf 2007, Khushf 2008, Engel 1977.19 Weckert 2008.21 Müller 2009.

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NT A report on the nanomedicine economic, regulatory, ethical and social environment

also provide scope for more expansive self-monitoring byhealthy people and broaden possibilities of self-diagnosis andtreatment which are already changing the definition ofmedical expertise. In the context of ‘e-health’, for example, thescience- and technology-driven move towards point-of-carediagnostics and the commercial exploitation of anxieties arecoming together in new divisions of labour in healthcaresystems. These developments require careful monitoring andanalysis, in particular the assessment of established andemerging divisions of labour amongst patients, physicians,hospitals and insurers. What are the implicit values drivingthese divisions of labour, and what should be done to ensuregood practice?

oÉÅçããÉåÇ~íáçå RThe European Commission and national governmentsshould encourage social science and humanities researchthat goes beyond the risks and benefits of medicalinnovations to include the consideration of promises, hopesand anxieties. Such research should assess the emergingdivisions of roles and responsibilities between patients,physicians, hospitals, e-health information systems, insurersand consumers. It should also identify safeguards that couldand should be provided by public health care systems.

Once health is broadly defined as well-being, the responsibilityof benefit sharing with low-income countries is alsobroadened, creating opportunities for new modes of globalinteraction. The responsibility of benefit sharing is currentlyframed as compensation, i.e. medical research anddevelopment need to make up for past injustices, for example.If we understand health as well-being, however, macro-ethical considerations lead to wider, human rights-basednotions of benefit-sharing. These ultimately aim at therestoration of human flourishing (as measured byimprovements in quality of life or public health infrastructure).Such debates raise the question of how to define theresponsibility of states or transnational entities towards low-income countries.

oÉÅçããÉåÇ~íáçå SThe European Commission and national governmentsshould create ways of involving low-income countries in thedevelopment of a fair and sustainable global policy on benefitsharing in nanomedicine. Avenues for this might includeinternational organisations such as UNESCO and bodieswhich work towards and monitor the implementation of theUnited Nations Millennium Development Goals.

22 Hastings Center Report 1996.23 European Group on Ethics 2007, section 5.6.24 E.g., European Commission 2006, p. 24.25 Boenink 2009. 26 Presentation by Susana Vidal at Working Group meeting.

`çåÅäìÇáåÖ=`çããÉåíëWcìêíÜÉê=fëëìÉë=Ñçê`çåëáÇÉê~íáçåThe Working Group had much more to say than hasbeen possible to include in this report, havingdiscussed issues such as:• the ‘metaphysical research programme’ of

nanomedical research, its implicit assumptionsabout the human body, health and illness, andtechnology and nature, and how these mightchallenge more traditional conceptions;

• regenerative medicine and tissue engineering, theintegrity of the patient’s body and the changingcharacter of medicine as it moves from the art ofhealing to the engineering of replaceable body parts;and

• the development of new analytic tools which canquickly obtain overwhelming amounts of data from adrop of blood. How might this ‘data overload’ alterthe concept of ‘meaningful information’ for patientsand doctors? What is the value of information? Arethere issues of confidentiality and privacy related tosuch techniques?

By highlighting the recommendations in this report,the Working Group has signalled that ethical andsocietal deliberation confronts a far wider range ofissues than those of safety and risk that are generallyrecognised. At the same time it has emphasised anapproach in which ethics goes beyond questions ofrisk not by looking into the distant future, but by askinghow nanomedical research intensifies current trendsand engages with choices that are before us today.

For a desperately ill patient, hope is a realachievement. Societies also hope for a better future,and often pin these hopes on emerging technologies.Ethical and social analysis of emerging technologies,however, should be careful not to take as fact what atthis point is only a matter of hope. Such analysis musttake hopes and concerns about the future seriously,but do so without second-guessing what the future willhold. We should ask of nanomedicine what trends itcontinues, problems it intensifies, and promises itholds out. In this way we can evaluate what measureof public support these promises deserve.

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NV A report on the nanomedicine economic, regulatory, ethical and social environment

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Reliable data is needed to predict the impact ofnanomedicine on healthcare costs and benefits,and market growth. If urgent action is not taken, the current lack ofdata and economic models will hinder thedevelopment of nanomedicine in Europe.

Early health economics assessment is crucial torecognise the potential applications ofnanomedical innovations as early as possible andtherefore enable maximum patient benefit.The future impact of nanotechnology innovationsin healthcare systems, and their added value inpreventive medicine, should be assessed.

The Importance of Assessing the Economic Impact ofNanomedicineThe potential of nanomedicine to diagnose, treat and preventdiseases has been recognised, but its cost-effectiveness is notwell developed in the public framework. Since each MemberState applies different reimbursement rules, the lack of dataand economic models will hamper the development ofnanomedicine in Europe.

How Should the Economic Impact of Nanomedicine beEvaluated?Nanotechnology will be considered in health economics only ifit brings significant added therapeutic value. Data on clinicaleffectiveness must therefore be acquired prior to any attemptto evaluate economic significance. Societal and economiccriteria (going far beyond the cost of treatment) also need tobe taken into account.

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Addressing the Current Lack of dataUp to now health economics studies do not cover innovationscoming from nanotechnology, so data regarding the economicassessment of nanotechnological innovations in thehealthcare sector is scarce.

oÉÅçããÉåÇ~íáçå NCompanies and clinicians should produce data based onwell-defined criteria of cost-effectiveness for the economicevaluation of nanotechnology-based innovations in clinicaltrials and health technology assessment studies.

Early Health Economics AssessmentEarly medico-economic assessment is critical to recognise asearly as possible (ideally at the pre-clinical stage) the potentialapplications of nanomedical innovations; this enablesassessment of maximised therapeutic value for patients.

oÉÅçããÉåÇ~íáçå OThe European Commission and national governmentsshould promote and support projects in partnership withhealth economists, technology developers, clinicalresearchers, healthcare providers and patient associations,with comparisons across Europe, to assess the costeffectiveness of nanomedical innovations as early aspossible.

Reimbursement IssuesWhen added therapeutic value is clearly demonstrated, e.g. inthe case of unmet medical need, reimbursement generallyoccurs. However, nanotechnology is most often considered asone innovation among others, without recognising its deepimpact on the definition of medical practices.

oÉÅçããÉåÇ~íáçå PNational reimbursement agencies, along with public andprivate insurers, should establish a European working groupto consider the future impact of innovative approaches inhealthcare systems across Europe, taking nanotechnology asa case study.

Preventive Medicine and Monitoring of Chronic DiseaseNanotechnology applications may offer new solutions forpreventive medicine and the growing need for diagnostic andmonitoring tools. The added value of nanotechnology forconsumer-driven markets in healthcare and well-being mustalso be considered.

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1 Annex 1 provides some snapshots about the most promising aspects of nanotechnology in medicine.2 The main conclusions of the Working Group’s data collection is provided in Annex 1, and key references are outlined in Annex 2.3 VDI, Bionest, Business Insights, Visiongain.4 US Food and Drug Administration (FDA) and some national databases.

Action is required to ensure the effectiveorganisation of nanomedicine in Europe andEurope’s competitiveness in the face ofworldwide competition.

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oÉÅçããÉåÇ~íáçå QThe European Commission should launch a health-economics project to assess the economic impact andemergence of new cost models relating to nanotechnologicalinnovations in preventive medicine and the monitoring ofchronic diseases.

Effective Organisation of Nanomedicine in EuropeThis is critical to avoid false investments, optimise clinicalbenefits and therefore to maximise economic impact.

oÉÅçããÉåÇ~íáçå RThe European Commission and national governments(especially health and research ministries and bodies) shouldestablish technology-specific reference centres linking earlydevelopment with clinical research and clinical practice.These centres should be partially publicly-funded.

Worldwide Competition in NanomedicineEurope’s competitiveness must be ensured at every level ofnanomedicine investment, from basic research toreimbursement.

oÉÅçããÉåÇ~íáçå SThe European Commission should share the conclusions ofthe NanoMed Roundtable with international expert groups inorder to improve European and international strategies formaximising the positive economic impact of nanomedicine.

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In addition to its main applications in diagnosis, drug deliveryand various therapeutic effects, nanotechnology is also ourbest hope for regenerative medicine, with significantachievements realised in recent years in the laboratory.Nanotechnology is also of great interest for cancer diagnosisand treatment, both in preventive and curative approaches.

This has been recognised in Europe with the EU projectNano2Life (Network of Excellence), which has establisheddetailed objectives for nanobiotechnology development,starting with clinical needs in the field of neurosciences andoncology.

However, the cost-effectiveness of nanomedicine (for allfields of application, including drug development) is not welldeveloped within the public framework. This could hamperthe development of nanomedicine in general, and in Europein particular.

Since each EU Member State applies different rules forreimbursement, the lack of data and economic modelsconstitutes a weakness that will hinder or even block thedevelopment of innovative procedures.

In the short-term this would particularly affect cancerdiagnostics and treatments. Longer term, the absence ofeconomic models and aggregated data regarding the cost-effectiveness of nanomedicine could endanger the potential of

5 www.clinicaltrials.gov 6 A liposome is a tiny bubble made out of the same material as a cell membrane. Liposomes

can be filled with drugs, and used to deliver drugs for cancer and other diseases.(http://en.wikipedia.org/wiki/Liposomal)

7 Bionest 20088 Visiongain 20069 Frost and Sullivan 200710Lux Research 200911Bionest 2008

ON A report on the nanomedicine economic, regulatory, ethical and social environment

nanotechnology to prevent/treat other diseases (includingrare genetic diseases, neurodegenerative diseases andchronic diseases, for example), as well as the promisingapproaches provided by nanobioengineering in regenerativemedicine.

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• Commercialised products - Except for certain surveys , it isalmost impossible to retrieve nanomedicines and medicaldevices integrating nano features from existing approveddrugs and devices databases . The “nano” feature is simplynot indicated. However, the Working Group counted about45 products commercialised in 2008 (developed mainly byUS companies), of which only two products werediagnostics.

• Pipeline and products under development - Informationabout product pipelines and products under development isscarce and not comprehensive. Consultants often refer tocompanies active in nanotechnology, developing variousapplications including healthcare products. Communicationis therefore targeted towards investors. There is no publicinformation about products under development by largecompanies in the healthcare sector, e.g. pharmaceutical,diagnostics or medical devices companies.

The Working Group’s preliminary research identified about 60products under development, although this figure is anestimate, since the group did not have access to primarysources (companies). A preliminary search of the US NationalInstitutes of Health’s clinical trials database revealed 69results containing the word “nanoparticle” and 372 resultscontaining the words “liposome or liposomal” .

• Market surveys - Absolute values of markets are verydifficult to forecast. A detailed analysis of pipelines andreimbursement issues would be required to do so, but thisis never addressed by market surveys. However, theWorking Group purchased excerpts of three surveys thathave been evaluated as more reliable than others. Themost recent of these estimated the value of the final (global)market of nanomedicine drug delivery products at US$4,181 million in 2007. Estimations of the value ofnanomedicine’s future markets are US$ 19,262 million in2011 and US$ 27,700 million in 2014.

The most interesting strategic analysis considers thatproducts are enabled by nanotechnology, whatever theapplication. Therefore the market of nanotechnology forhealthcare comprises nanomaterials, nanointermediatesand final products (integrating nano features).Nanomedicine is definitely not a market segment.

• Companies - The Working Group identified a preliminarylist of companies involved in nanomedicine products: 19 inGermany, 12 in France and 9 in the UK .

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i. Clinical Effectiveness

Nanotechnology will be considered in health economics only ifit brings a significant added therapeutic value, which dependson each particular situation (from unmet medical need toincremental improvement of patient care). Therefore, it is ofprimary importance to acquire data on the clinicaleffectiveness of nanotechnology, prior to any attempt toevaluate economic relevance.

ii. Societal and Economic Criteria

The economic impact of nanomedicine has to be consideredin a general model that integrates relevant criteria going farbeyond the cost of treatment itself, and asks the followingthree questions:

1. To what extent does nanotechnology contribute to newhealth products and services, and what is the future wealthgenerated?

2. How much does nanotechnology contribute to a change ofcost models of the health systems?

3. How much are the two previous questions influenced by theregulatory framework, which basically is a result of socialand political pressure?

An ideal model to assess the economic impact ofnanomedicine would integrate parameters relating to societaland economic criteria:

• Wealth created by nanomedical innovations

• Industrial property value (patents and licences).• Markets and turnover for companies, due to new

products and services.• Existing jobs and new jobs in companies and services.

• Costs relating to regulation for all stakeholders

(e.g. regulatory agencies, reimbursement agencies,companies, technology developers)

• Additional costs relating to the establishment of newprocedures (e.g. combination products), evaluation ofmaterials, and relevance of manufacturing standards.

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12This results from the Economic Impact Working Group’s discussions rather thanacademic review.

13Ref. Regulation Working Group of the NanoMed Round Table.

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• Economic costs and benefits in the healthcare system• Public and private “out of pocket” expenditures for the

new treatment.• Cost-benefit-ratio of standard treatment compared to

the nanomedicine treatment.• Patients’ access to innovative (often expensive) diagnostic

procedures and treatments through nationalreimbursement systems.

• Costs relating to breakthrough innovation (if innovationcreates new costs).

• Costs relating to assessing so far potential/unknownlong-term effects.

• Economic benefits induced by health innovation (e.g.efficacy, fewer side effects, reduction of length of hospitalstay, added month/years of life, added quality of life) andtransformation of social benefits into economic benefits.

• The power of public perception• Towards nanotechnology, including other applications

such as cosmetics, food, consumer products.• In terms of health expectations, e.g. policies, access,

costs.

Analysing the economic impact of nanomedicine is thereforecomplex. Nanotechnology produces incremental innovationin medicine but also more radical changes in diagnostics andtherapeutics, pushing away the boundaries between curativeand pre-emptive medicine.

Evaluating the economic impact of such medical innovationscannot be done without a clear analysis of healthcare cycles.For example, an increased cost in early diagnostics can bevery effective later in terms of benefits.

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i. Addressing the Current Lack of Data

Health economics is a complex area. There are differenthealthcare systems in Europe, with different criteria (e.g.Quality Adjusted Life Years in the UK and cost/benefit inGermany and France) with various reimbursement tracks andprocedures . Data aggregation is therefore difficult.

Health economics studies currently do not considerinnovation coming from nanotechnology, so data regardingthe economic assessment of nanotechnological innovationsin the healthcare sector is scarce.

The Working Group conducted a systematic search on publicHealth Technology Assessment databases but did not find anysurvey or evaluation regarding nanomedicine,nanotechnology or nanoparticles. The Working Group did findthree examples of economic assessment related to liposomal

doxorubicin (for ovarian cancer) and amphotericin B (anantifungal prophylaxis for neutropenic patients). Theseproducts have been used for many years in clinical practice,but in the case of amphotericin B the Working Group foundtwo surveys (both based on efficacy/cost analysis and for thesame medical objectives) that reached opposite conclusionsas to whether it was economically worth providing theproduct. This shows that the criteria that are needed forevaluation can vary significantly (e.g. what is a severe sideeffect?) and that this does not make economic evaluation aneasy task.

Health economists need data from clinical research andpractical application in order to conduct an economicevaluation of any nanomedical innovation.

oÉÅçããÉåÇ~íáçå NCompanies and clinicians should produce data based onwell-defined criteria of cost-effectiveness for the economicevaluation of nanotechnology-based innovations in clinicaltrials and health technology assessment studies.

ii. Early Health Economics Assessment for Nanomedicine

The Working Group concluded that: “It’s always too early toassess until suddenly it’s too late”.

Nanotechnology researchers may not have the rightunderstanding of the added therapeutic value of theirinnovation. Without blocking developments, it is important torecognise as early as possible (ideally at the pre-clinical stage)the potential applications of a given nanomedical innovation.This early recognition of applications would enableassessment of maximised therapeutic value for patients,which could take the form of efficacy, effectiveness, outpatientrather than inpatient treatment, shorter hospital stays, fewerdrugs, patients’ acceptance of, and compliance with, thetreatment. In turn, this will define the future access tomarkets, and therefore market volumes.

Commercial market surveys most often considernanomedicine market volumes without any reference to itsreal therapeutic added value. Even when the assumption isaddressed (e.g. one market analysis considered by theWorking Group considers that nanotechnology, as an“enabling” technology, will progressively penetrate existingmarkets), it is never based on a precise market segmentation,which is fundamental to provide reliable market studies.

oÉÅçããÉåÇ~íáçå OThe European Commission and national governmentsshould promote and support projects in partnership withhealth economists, technology developers, clinicalresearchers, healthcare providers and patient associations,

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16 Lux Research 200914www.ifeg.de 15Patients with an abnormally low number of white blood cells

OP A report on the nanomedicine economic, regulatory, ethical and social environment

with comparisons across Europe, to assess the costeffectiveness of nanomedical innovations as early aspossible.

The implementation of Recommendation 2 could lead to acontinuous health-economic assessment of nanotechnologyin medicine, for instance in the shape of specific projects, apermanent working group, or an exchange platformconnected to the European Technology Platform (ETP) onNanomedicine or “NANOfutures” European TechnologyIntegration and Innovation Platform in Nanotechnology. Thisearly medico-economic assessment is critical to makemarket projections more reliable.

iii.Reimbursement Issues

Several national agencies responsible for guidelines leadingto reimbursement decisions were invited to take part in theWorking Group’s discussions but were unfortunately unable tojoin us (e.g. Haute Autorité de Santé – HAS (France), NationalInstitute for Health and Clinical Excellence – NICE (UK),Institute for Quality and Efficiency in Health Care – IQWiG(Germany)). Therefore our recommendations do not take intoaccount the position of these stakeholders, who might have adifferent idea of nanomedical innovations.

When added therapeutic value is clearly demonstrated, e.g.in the case of an unmet medical need, reimbursementgenerally occurs. When a balance between the cost oftreatment and induced benefits (e.g. quality of life, short/longterm side effects, hospital costs) needs to be established,each country manages its economic compromises followingspecific guidelines.

However, nanotechnology is most often considered as oneinnovation among others, without really recognising its deepimpact on the definition of medical practices.

oÉÅçããÉåÇ~íáçå PNational reimbursement agencies, along with public andprivate insurers, should establish a European working groupto consider the future impact of innovative approaches inhealthcare systems across Europe, taking nanotechnology asa case study.

The objective of this working group could be to analyse indetail, in a forward-looking approach, the social and economicconsequences of nanomedical innovations and theirimplications for health policies in the Member States.

iv.Preventive Medicine and Monitoring of Chronic Disease

Nanotechnology is most often considered as being of greatinterest for early diagnostics and targeted therapies, in thecurrent scheme of curative medicine. In addition,

nanotechnology applications may offer new solutions forpreventive medicine and the growing need for diagnostic andmonitoring tools, especially for patients with chronicdiseases but also for healthy people. Nanotechnology canalso bring innovation in miniaturisation and functionalcomplexity for point of care or wearable devices.

Consequently, new business models will begin to emerge.Many Framework Programme 7 (FP7) projects address thischallenge, but the added value of nanotechnology for moreconsumer-driven markets in healthcare and well-beingmust also be considered.

oÉÅçããÉåÇ~íáçå QThe European Commission should launch a health-economics project to assess the economic impact andemergence of new cost models relating to nanotechnologicalinnovations in preventive medicine and the monitoring ofchronic diseases.

v. Effective Organisation of Nanomedicine in Europe

A good organisation of nanomedicine in Europe is of criticalimportance to avoid false investments, optimise clinicalbenefits and therefore to maximise economic impact. Theideal configuration of centres of reference (or networks) couldbe as follows:• Basic research and early technology development in public

or private research institutes (as in the NanomedicineCenters of Excellence in the USA).

• ‘Technosensitive’ clinical researchers (i.e. researchers whoare interested in and can understand technology, especiallynanotechnology) recruited in clinical networks (e.g. theEuropean Organisation for the Research and Treatment ofCancer) to develop clinical research about nanomedicine,working closely with nanotechnology researchers andpharmaceutical/medical technology companies.

• Support from experts in health economics assessment,dealing with specific studies in selected areas.

• All stakeholders in the three previous points taking part in athorough safety assessment (covering, for example, toxicity,side effects, ecotoxicity and long term effects) in order tobalance possible benefits.

• Patient associations participating as leading observers andadvisers from the inception of technology developments.

• Representatives of the fields of ethics, regulation andcommunication need also to be involved from the outset.

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oÉÅçããÉåÇ~íáçå RThe European Commission and national governments(especially health and research ministries and bodies) shouldestablish technology-specific reference centres linking earlydevelopment with clinical research and clinical practice.These centres should be partially publicly-funded.

In order to facilitate Recommendation 5, the results of theNanoMed Round Table should be disseminated to existingnetworks and projects related to nanomedicine, including:

• Nanomedicine ETP (European Technology Platform)• CLINAM (European Foundation for Clinical Nanomedicine)• ERANET nanomedicine• Euronanobio FP7 project• EORTC (European Organisation for the Research and

Treatment of Cancer)• EMIL (European Network on Molecular Imaging)• Conticanet FP6 network (Connective Tissue Cancer

Network)• European JTI IMI (Joint Technology Initiative in medicines:

Innovative Medicines Initiative)

vi.Worldwide Competition in Nanomedicine

In parallel with the recommendations outlined above, it isequally important to take account of the international contextof nanomedicine investment in order to ensure Europe’scompetitiveness in the face of worldwide competition. Thisapplies to every level of nanomedicine investment: basicresearch, clinical research, economic, social and ethicalevaluation and reimbursement.

It is clear that the importance of nanomedicine has beenrecognised internationally. For example, in the USA theNational Institute of Cancer has established an Alliance forNanotechnology in Cancer, which drives many researchprojects to maximise benefits for patients.

Due to the financial and time limitations of this project, theWorking Group was unable to focus in detail on theinternational context. However, this is an important area andthere are key issues on which it would be helpful to gainfurther insight, such as the consequences of the currentevolution of the American healthcare system, and whetherthe strategies of leading US companies in Europe will change.

oÉÅçããÉåÇ~íáçå SThe European Commission should share the conclusions ofthe NanoMed Roundtable with international expert groups inorder to improve European and international strategies formaximising the positive economic impact of nanomedicine.

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OR A report on the nanomedicine economic, regulatory, ethical and social environment

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In order to enable responsible innovation innanomedicine, the regulatory framework shouldfacilitate a scientifically-based societal learningprocess, i.e. by being flexible enough to allowsociety to acquire, exchange and accumulateknowledge and experience in dealing with a newtechnology.

For effective implementation of the existingregulatory framework, there is a need for bettercoordination and harmonisation of regulatoryprocedures, especially those on reporting anddata collection.

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Better coordination and harmonisation of existingregulatory procedures is urgently needed tofacilitate data collection and improve regulatoryclarity. Priorities are the clarifying of the regulatorypathway for ‘combination products’ (which bear thefeatures of different medical products and evenfood or cosmetic products), defining commonterminology and relevant data, and promoting datacollection efficiency.

oÉÅçããÉåÇ~íáçå NAt the current development stage, regulatory policy shouldfocus on promoting the harmonisation and responsiveness ofexisting regulatory systems. The European Commissionshould establish and promote supporting mechanisms thatboost the effectiveness and responsiveness of the existingregulatory framework.

oÉÅçããÉåÇ~íáçå OThe European Commission should strengthen its efforts toclarify the regulatory pathway and classification ofcombination products in the EU, and actively seekinternational collaboration to improve consistency betweendifferent jurisdictions.

oÉÅçããÉåÇ~íáçå PThe European Commission should strengthen its efforts toclarify the regulatory pathway and classification ofcombination products in the EU, and actively seekinternational collaboration to improve consistency betweendifferent jurisdictions.

oÉÅçããÉåÇ~íáçå QThe European Commission should take advantage of themerits of recent medical product regulation initiatives (e.g.seek international collaboration in establishing commonreporting schemes to promote the efficiency andeffectiveness of product authorisation), and use the Cross-border Healthcare Directive to facilitate data collection oncommon clinical issues and boost expertise for the clinicalapplication of nanomedicine.

Ensuring the Responsiveness of Regulatory Policies

This requires, among other measures, meaningfulengagement with users and stakeholders.

oÉÅçããÉåÇ~íáçå RThe European Commission should establish and promoteearly dialogue with the different stakeholders on regulatoryissues concerning nanomedicine, and ensure the regulatoryframework for nanomedicine is grounded in users’experience.

oÉÅçããÉåÇ~íáçå SPatients’ involvement in the nanomedicine policy makingprocess should be institutionalised at both EU and nationallevels.

oÉÅçããÉåÇ~íáçå TThe European Commission should facilitate the accessibilityof regulatory expertise by establishing user-friendlymechanisms which encourage early dialogue with regulatorybodies and regulatory partnership in order to facilitateconsensus on data requirements.

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oÉÅçããÉåÇ~íáçå UThe European Commission should:• consider the appropriate application of the subsidiarity

principle in regulating nanomedicine, taking intoconsideration national regulatory infrastructures andcultures. The need for capacity building at national levelshould also be addressed in EU regulatory policy.

• ensure regulatory policies take into account factors suchas the situation in different Member States, different sizesof companies and different types and applications ofnanotechnologies.

oÉÅçããÉåÇ~íáçå VThe European Commission should ensure continued effortsto address and monitor the health and environmental impactof nanomedicine, including improving awareness ofenvironmental, health and safety (EHS) issues ofnanomaterials, both in hospitals and nanomedicinecompanies.

Establishing Mechanisms of Credibility for ResponsibleInnovation

Mechanisms that offer credibility and authority are needed tosupport and encourage responsible practice in thedevelopment of innovative products.

oÉÅçããÉåÇ~íáçå NMThe European Commission should support institutionalmechanisms that facilitate a common perspective withregard to clarity, objectivity, and common practice forcredibility and authority, e.g. joint efforts on development oftesting protocols, standards and best practice.

The Economic Implications of Regulation

The cost of accessing regulatory expertise and meetingregulatory requirements can present significant barriers tobringing innovative nanomedicine to market.

oÉÅçããÉåÇ~íáçå NNRegulators should take into consideration the economicimplications of regulation for nanomedicine, including impacton timeline, insurability and the funding support needed foraccess to regulatory expertise and extra complianceinvestment, especially for SMEs and academic institutions.

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The behaviour and functionality of many chemicals atnanoscale may differ substantially from their behaviour inbulk. This offers new opportunities and a wider diversity ofapplications, but may also raise new risks and challenges. Anongoing question is whether existing regulations are adequatefor managing the new risks, while not unduly constrainingresearch and innovation.

A prefix such as “nano-” does not necessarily imply novelty inscientific research or technological application. Even wherethere may be novelty, this does not imply either an automaticneed for regulation, or an absence of coverage by existingregulation. The breadth of applications of nanotechnologiesimplies that some or all of these will be in fields that arealready significantly regulated at national and Europeanlevels, as illustrated by the existing literature. However, withthe current limitation of scientific knowledge, a regulatoryframework that aims to enable responsible innovation is

OT A report on the nanomedicine economic, regulatory, ethical and social environment

needed, rather than a traditional reactive regulatoryapproach. This will facilitate scientifically-based societallearning, by being flexible enough to allow society as a wholethe chance to acquire, exchange and accumulate knowledgeand experience in dealing with a new technology.

The Regulation Working Group considers that no specificneed for new legislation can be identified at the present time.However, several implementation issues have been identifiedas primary regulatory concerns. Mechanisms to boost theeffectiveness and responsiveness of the existing regulatoryframework and to establish credibility for responsibleinnovation are of greatest importance and are therefore thefocus of this report.

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Better coordination and harmonisation of existing regulatoryprocedures is urgently needed in order to facilitate datacollection and improve regulatory clarity. This is crucial foradvancing knowledge on safety, reducing disproportionateregulatory burden and improving the accessibility of newmedicinal products.

oÉÅçããÉåÇ~íáçå NAt the current development stage, regulatory policy shouldfocus on promoting the harmonisation and responsiveness ofexisting regulatory systems. The European Commissionshould establish and promote supporting mechanisms thatboost the effectiveness and responsiveness of the existingregulatory framework.

Three immediate and priority targets for coordination andharmonisation are outlined in further detail below. These are:• clarifying the regulatory pathway; • defining common terminology and relevant data; and• promoting data collection efficiency.

i. Clarifying the Regulatory Pathway

Nanomedicine provides great opportunities to integratemedicine, medical devices and other medical products (e.g.drug/device, device/implant). Many of the products will be‘combination products’ or ‘borderline products’ that bear thefeatures of different medical products and even products ofadjacent areas such as food and cosmetics.

However, regulations on healthcare have generally beensector-specific. A recurrent problem in regulation is,

therefore, the overlap (or gap) between the regulatorysystems and frameworks for different sectors. For example,EU legislation differentiates clearly between medicinalproducts and medical devices. For medicinal products there isa highly centralised system governing legal access to market,whereas medical devices are regulated under New ApproachDirectives, a more decentralised system.

In both systems, risk management is a key requirementguarding the quality and safety of products on the market.However, the regulatory approaches for risk assessment inthese two fields differ (see Annex 1 for more information).Since new medicinal products – including those enabled bynanotechnologies – are increasingly resulting fromconverging technologies, levels of regulatory oversight onthese products depend to a large extent on regulatory policyon ‘combination’ and ‘borderline’ products.

There is also inconsistency in new regulations onnanotechnology-enabled products in the adjacent areas ofcosmetics and novel food. Food has been the subject of muchregulatory debate, now including the regulation of foodadditives. The area of cosmetics is also carefully regulated,but is the subject of quite intense research and innovationwhich may raise new problems relating to consumer health.

Clarity in product classification is of primary importance forensuring adequate regulatory oversight and estimatingdevelopment cost. However, there is yet no sharedunderstanding of what a ‘combination product’ is and how itshould be regulated. It is not yet clear whether nanoscaleproducts, or products that contain nanoscale materials, raiseany novel questions relevant to their classification as biologicalproducts, devices, drugs, or combination products.

It is therefore not clear whether the current regulatoryapproach concerning combination products is able to addressappropriately the regulatory needs of nanomedical products.Along with the inconsistency in new regulations onnanotechnology-enabled products in the adjacent areas ofcosmetics and novel food, this means that collaborationbetween the regulatory bodies at both European andinternational levels is crucial in improving consistency andclarity in regulation.

oÉÅçããÉåÇ~íáçå OThe European Commission should strengthen its efforts toclarify the regulatory pathway and classification ofcombination products in the EU, and actively seekinternational collaboration to improve consistency betweendifferent jurisdictions.

1 The UK report “Nanoscience and nanotechnologies: opportunities and uncertainties”, July 2004, RS Policy document 19/04, The Royal Society and The Royal Academy of Engineering, points outthat “some chemicals are more toxic when in the form of nanoparticles or nanotubes … safety assessments based on the testing of a larger form of a chemical cannot be used to infer the safety ofchemicals in the form of nanoparticles”. Consequently the adequacy of existing chemicals regulation should be appraised. The UK’s “Overview” of regulation presents an analysis of regulatorygaps. See “An Overview of the Framework of Current Regulation affecting the Development and Marketing of Nanomaterials”, Report to the UK Department of Trade and Industry, December 2006.

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ii. Defining Common Terminology and Relevant Data

Nanotechnology governance presents two types of regulatoryissues:1. generic issues originating from the special features of

nanotechnologies; and 2. issues concerning general deficiencies of current

regulatory systems that cause problems for all applicableproducts including nanomedical products.

In the second of the two points above, it is more appropriate toaddress the issues as general issues concerning all theapplicable products instead of seeing them asnanotechnology issues. This report focuses on the question“What is 'new' about nanomedicine?” and therefore genericregulatory issues of nanomedicine. In order to identify suchissues, there is an urgent need to establish common groundsfor enabling comparable testing results, e.g. agreed commonterminology and testing endpoints. Several internationalorganisations have devoted significant efforts to this (seeAnnexes 5 and 6 for more details).

A primary question concerning medicinal products is how togenerate the ‘evidence’ required to fulfil the safety and efficacyrequirements in existing regulatory frameworks. Currentregulation for medicinal products is well known for itsstringency and long history of using risk/benefit comparisonas evaluation tools. However, when it comes to innovativenanotechnology products, it is not clear what methods anddata should be considered appropriate and satisfactory forfulfilling the legal requirements of safety and efficacy.

There is, therefore, an urgent need to improve the sharedunderstanding on the nature of the data that are needed toestablish safety and efficacy. The data requirementsconcerning clinical endpoints for evaluation of effect of theproducts are crucially important in ensuring safety andbenefits for patients, as well as regulatory clarity forcompanies and the insurance industry.

oÉÅçããÉåÇ~íáçå PThe European Commission should devote efforts to definingcommon terminology and relevant data in nanomedicine,actively supporting the clarification of data requirementsconcerning safety, efficacy and clinical endpoints for theevaluation of effect of the products.

iii. Promoting Data Collection Efficiency

To promote the efficiency of data collection and reduceunnecessary duplication of reporting procedures, regulatorybodies should take advantage of the merits of several of the

latest initiatives in medical product regulation. These includethe common reporting schemes established through theagreement between the US Food and Drug Administration(FDA) and the European Medicines Agency (EMEA) on orphandrug authorisation, as well as the cross-border data collectionprocedure established in the Cross-border HealthcareDirective.

The EU has the advantage of data-generating capacity fromall its Member States. Expert members of the NanoMedRound Table identified the cross-border data collection oncommon clinical issues as an important competitiveadvantage for the EU. The forthcoming Cross-borderHealthcare Directive will create opportunities in data sharingthrough the establishment of ‘centres of reference’ that willcreate critical mass for research and boost expertise for theclinical application of new medical products.

oÉÅçããÉåÇ~íáçå QThe European Commission should take advantage of themerits of recent medical product regulation initiatives (e.g.seek international collaboration in establishing commonreporting schemes to promote the efficiency andeffectiveness of product authorisation), and use the Cross-border Healthcare Directive to facilitate data collection oncommon clinical issues and boost expertise for the clinicalapplication of nanomedicine.

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The Regulation Working Group has identified the followingapproaches as important steps to ensure the responsivenessof regulatory policies concerning nanomedicine:• engagement;• regulatory partnership;• differentiated considerations; and• environmental, health and safety (EHS) risk management.

i. Engagement

The regulatory framework must be grounded in users’experience and regulatory policies communicated withstakeholders at early stages (see Annexes 2 and 3 for moreinformation).

oÉÅçããÉåÇ~íáçå RThe European Commission should establish and promoteearly dialogue with the different stakeholders on regulatoryissues concerning nanomedicine, and ensure the regulatoryframework for nanomedicine is grounded in users’experience.

2 The European Commission has recently updated two guidance documents: 1) MEDDEV 2.1/3 rev 3 Borderline products, drug-delivery products and medical devices incorporating, asan integral part, an ancillary medicinal substance or an ancillary human blood derivative; 2)Manual on Borderline and Classification in the Community Regulatory Framework forMedical Devices.

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3 Workplace Exposure to Nanoparticles, EASHW, 2009, based on materials published up toNovember 2008.

OV A report on the nanomedicine economic, regulatory, ethical and social environment

oÉÅçããÉåÇ~íáçå SPatients’ involvement in the nanomedicine policy makingprocess should be institutionalised at both EU and nationallevels.

ii. Regulatory Partnership

Opportunities for companies to have early dialogue withregulatory bodies (including notifying bodies on issuesconcerning what data would be considered appropriate andsufficient) will contribute significantly to regulatory clarity andfacilitate mutual learning on the nature of the data required.Such a process will encourage the development of consensusabout data to be generated and shared, therefore facilitatingprogress towards licensing.

oÉÅçããÉåÇ~íáçå TThe European Commission should facilitate the accessibilityof regulatory expertise by establishing user-friendlymechanisms which encourage early dialogue with regulatorybodies and regulatory partnership in order to facilitateconsensus on data requirements.

iii. Differentiated Considerations

The Regulation Working Group has concerns regarding thecapacity of national regulatory authorities and the need fordifferentiated policy considerations.

oÉÅçããÉåÇ~íáçå UThe European Commission should:• consider the appropriate application of the subsidiarity

principle in regulating nanomedicine, taking intoconsideration national regulatory infrastructures andcultures. The need for capacity building at national levelshould also be addressed in EU regulatory policy.

• ensure regulatory policies take into account factors suchas the situation in different Member States, different sizesof companies and different types and applications ofnanotechnologies.

iv. Environmental, Health and Safety (EHS) RiskManagement

The safety of workers who may have the most exposure tonanomaterials is a major focus for regulation. This has beenaddressed in a comprehensive recent report by the EuropeanAgency for Safety and Health at Work . The current principlesof risk assessment are judged to be in general appropriate.However, the validation of in vitro methods and thedevelopment of a testing strategy remain future tasks.Classification and labelling as well as occupational exposurelimits, which are derived from toxicological data, are

appropriate instruments for managing risks resulting fromexposure to nanomaterials. Critically, however, these dependon the availability of toxicity studies.

A review of the recent literature on workplace exposure tonanoparticles indicates that several statutory instruments arein place to ensure an appropriate level of protection ofworkers. Currently there is discussion of how to considerappropriately the broad variety of nanomaterials within theseregulations.

In addition, the Community strategy on health and safety atwork for the period 2007 – 2012 includes nanotechnology asan important topic to be worked on in the context of theidentification of new, emerging risks, and specific topics havebeen identified as priorities for future actions and activities.

Hospitals and nanotechnology companies have responsibilityfor their workers’ safety. Evidence from around the worldsuggests wide diversity in the standards expected or imposed,but intense reporting and electronic media maintain acontinuing pressure for improvement, if not conformity.Medical waste disposal procedures normally have to followvery strict regulation. However, the environmental impact ofmedical waste is a relatively new topic in regulation, and ischaracterised by many unknowns. The Working Groupbelieves that most of the hospitals that are working withnanomaterials are not aware of their potential environmentalimpacts.

oÉÅçããÉåÇ~íáçå VThe European Commission should ensure continued effortsto address and monitor the health and environmental impactof nanomedicine, including improving awareness ofenvironmental, health and safety (EHS) issues ofnanomaterials, both in hospitals and nanomedicinecompanies.

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Given the hype about new technologies, and both the anxietiesand over-expectations that the general public may have as aresult, there is a need for society to establish and promotemechanisms that offer credibility and authority. This supportsand encourages responsible practice in the development ofinnovative products. Institutional mechanisms that facilitate acommon perspective with regard to clarity, objectivity, andcommon practice for credibility and authority will help to guideand facilitate responsible innovation.

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4 The general framework is provided by the regulation on occupational safety and health of workers (EU Directive 89/391/EEC) and specifically for chemical safety by the directive onthe protection of the health and safety of workers from the risks related to chemical agents at work (Directive 98/24/EC). Substance-specific regulation is intended by the biocideDirective (Directive 98/8/EC) and Regulation 1907/2006 (REACH)..

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oÉÅçããÉåÇ~íáçå NMThe European Commission should support institutionalmechanisms that facilitate a common perspective withregard to clarity, objectivity, and common practice forcredibility and authority, e.g. joint efforts on development oftesting protocols, standards and best practice.

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Regulation has significant economic implications forcompanies’ competitiveness and the availability of products. Itis therefore important to find the balance between patients’need for speedy access to innovative medical products andsociety’s general need for safety.

For example, on the one hand the expensive high stringencyclassification (class III) may have a significant impact on smalland medium-sized enterprises’ (SMEs’) research anddevelopment strategies and investments in beneficialproducts with niche markets. On the other hand, theassessment of biological and medical devices that is based onthe application of voluntary or general (non-specific)standards may not be sufficient to address the safety issues ofborderline products.

For SMEs, access to regulatory expertise and the cost ofmeeting regulatory requirements present significantbarriers to bringing innovative and potentially beneficialnanomedicine products to market.

Regulation also has considerable impact on the insurability ofenvironmental, health and safety risks and legal risk such asliability involved in the development of nanomedicine. In lightof what the insurance industry has learned to date throughmonitoring activities, it would appear that there is currently nourgent need to tighten underwriting. Nor, however, shouldone sound the ‘all clear’. Nanotechnology, in its immensediversity, is developing rapidly. While current questions as toits potential risks will be answered, new ones will alsoemerge (see Annex 4 for more information).

oÉÅçããÉåÇ~íáçå NNRegulators should take into consideration the economicimplications of regulation for nanomedicine, including impacton timeline, insurability and the funding support needed foraccess to regulatory expertise and extra complianceinvestment, especially for SMEs and academic institutions.

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This report draws on a wide range of literature surveyed, anda broad spectrum of conclusions advocated by differentauthors or groups. However, it is important to recognise thatthe situation is evolving continuously, with the progressworldwide of scientific knowledge, and the continuing effortsto bring the new knowledge into application in various fields.Hazards may exist in some research activities, and in someapplications: risk management suggests the use of“appropriate” regulations, which covers several possibilities:

• It may be that existing regulations can be interpreted tocover the new, nanotechnological application, even if thereis no explicit language covering the specific use. It would beunhelpful (although unfortunately not unprecedented) forregulations to be drafted in great detail, as such regulationcarries the risk of rapid obsolescence, and inhibits researchand innovation.

• At the opposite extreme from over-detailed regulations,there is the simple possibility of a general moratorium. Thisis quickly drafted, and apparently a “safe option” – if onlyone side of the safety balance is considered. However, it hasmaximal adverse effect on incentives for research andinnovation, and clearly reduces or eliminates a country’sability to compete in the new sectors and activities whichmay be opened up by the progress of knowledge andtechnique. The history of the over-regulation of geneticallymodified organisms (GMOs) in the EU provides an example.The report endorsed by the European Parliament in April2009 appears to tend in this direction: it is based onextensive research, which in political debate becomesreduced to the simplistic, memorable (and potentiallydangerous) formula (used in the debate on REACH) of “nodata, no market”.

The Regulation Working Group concludes that, while someregulation is clearly necessary, it should be appliedproportionately and limitedly. New legislation may well notbe required if the situations arising can be covered byintelligent interpretation of existing texts, and/or standards,codes of practice, guidelines, and informal but enforceableagreements. The concept of proportionateness and limitationmay include:• the concept of “case-by-case” decisions, and monitory

oversight, to facilitate learning;• the use of “sunset clauses” to end or adapt regulations if

not explicitly renewed.

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5 European Parliament, report on regulatory aspects of nanomaterials (2008/2208(INI)).

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Public engagement and deliberation aboutnanomedicine at all levels is fundamental if weas a society want to profit from the hopes andpromises invested in nanomedicine. Credible and accessible sources of balancedinformation must be provided, together with forato debate and discuss questions about needs,risks, benefits and ethical and social issuesrelating to nanomedicine to facilitateunderstanding and dialogue.

Communications know-how needs to be fosteredamong nanomedicine stakeholders, otherwisethe development, introduction and application ofinnovative nanomedical treatments will besignificantly delayed or hindered.Good communication needs adequate financing– this should be built into all nanomedicinefunding.

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There is much uncertainty in defining the term‘nanomedicine’, not least because this relativelynew field is transdisciplinary, blurring establishedboundaries and combining previously unconnectedfields. When considering this report’srecommendations this uncertainty must be takeninto account, as public engagement anddeliberation can only be effective if there isagreement on a common foundation on which tobuild dialogue.

For the purpose of this report ‘communication’ also needs tobe defined. The goal of any nanomedicine communicationstrategy must be to initiate public debate, deliberating on bothpersonal and social consequences of innovations andacknowledging that a simple discussion of risks and benefitsshould only be a starting point for dealing with the complexconcerns of a technology-dependent society. Furthermore,the communication requirements of individual members ofthe public can greatly differ, so it is vital for any successfulcommunication strategy to take the range of understandingsand expectations into account.

The Importance of Communicating About NanomedicineCommunicating about nanomedicine is increasinglyimportant because of its significant potential to:

• raise societal and ethical questions concerning risks andbenefits; and

• have an emotional and material impact (arising frombenefits and uncertainties) due to the large number ofpeople affected.

However, there is still a considerable lack of public awarenessabout nanomedicine. At the same time, social, ethical andlegal questions are being raised concerning safety,environmental and long-term effects. The best way to answerthe demands of an inquiring public is to develop andimplement a strategic approach for active public debate.

Any communication strategy needs to take into account thesignificant changes in communication methods; for example,the internet and new media are replacing doctors as theprimary source of medical information. Communicationpractices must therefore be continuously innovative in order toreach as wide and varied an audience as possible.

Providing Reliable Information About NanomedicineIn order to enable patients to make informed decisions, thepublic needs access to reliable and credible sources ofinformation and fora to debate and discuss questions aboutneeds, risks and benefits as well as ethical and social issuesrelated to nanomedicine.

oÉÅçããÉåÇ~íáçå NThe European Commission should establish a platform toprovide credible and accessible sources of balancedinformation on the methods, benefits and risks ofnanomedicine, focusing on the communication needs ofpatients and the medical community.

Establishing such a platform would make it easier for thepublic to understand fundamental nanomedical issues, andwould also support the communication tasks of stakeholderssuch as doctors, patient groups and health insurers.

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Providing Know-How for Communication AboutNanomedicine

The majority of current nanomedical research anddevelopment is conducted by small or medium-sizedenterprises (SMEs) which often do not have the know-how,staff time and financial resources required for initiating andimplementing successful communication strategies. This cansignificantly delay or hinder the development, introduction andapplication of innovative nanomedical treatments, to thedetriment of nanomedicine’s medical and economic impact inEurope.

The communication aspects of the related fields of food andenvironment must be carefully differentiated, or negativetendencies concerning these fields may have detrimentaleffects on nanomedicine.

oÉÅçããÉåÇ~íáçå OThe European Commission should develop communicationguidelines for the various nanomedicine stakeholders andprovide good practice examples. Particular care should betaken to differentiate between the communication aspects ofthe related fields of food, environment and nanomedicine.

Ensuring Sufficient Resources for Communication AboutNanomedicineInefficient or inadequate communication about nanomedicinecan lead, on a small scale, to the rejection of an individualtreatment method. On a larger scale, a number of such casesof rejection could lead to distrust and fear of the entire field.The question is how to finance communication in a field whichis struggling to define itself and which has long developmenttimes and therefore long investment return times, meaningthat profits are currently negligible.

oÉÅçããÉåÇ~íáçå PThe European Commission, national governments, industryand independent grant organisations should allocate asignificant percentage of financial resources in the field ofnanomedicine to public communication. The goal of thisfinancial allocation should be to encourage publicengagement, foster dialogue and move beyond the simplediscussion of risks versus benefits.

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i. Definition of Nanomedicine for Communication Purposes

From a communication point of view it is interesting to notethat neither the participants of the Set-up Round Tablemeeting nor the members of the Communication WorkingGroup were able to agree on a precise definition of the term'nanomedicine'. Suggestions ranged from the simple'application of nanosciences to medicine', through theelaborate definition used in the NanoBio-RAISE project, whichdifferentiated between 'nanomedicine and medicalnanotechnologies', to the widespread public perception of'nanomedicine is whatever calls itself nanomedicine'.

The difficulty in agreeing on a precise definition in itselfdemonstrates a significant amount of uncertainty indelineating this relatively new field of medicine. One of themain reasons for this is that nanomedicine istransdisciplinary, blurring established boundaries andcombining previously unconnected fields.

When considering this report’s recommendations thisuncertainty has to be taken into account, as publicengagement and deliberation can only be effective if the

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various parties can agree on a common foundation on whichto commence a dialogue. This basic tenet also applies to otherissues. The Working Group deemed one potential source ofdisagreement to be the discrepancy between nanomedicalvisions and the current state-of-the-art. The hopes andexpectations raised by the former are generally in no waylikely to be met by the latter. However, instead of viewing thisin a negative light, it should be regarded as a possible startingpoint for a public deliberation on nanomedicine.

The necessity for using the term 'nanomedicine' may need tobe questioned. For researchers and companies the use of thisterm can be very important, as it can help in fund-raising,marketing, product placement or the establishment of acorporate identity, among many things. At the other end of thescale the individual patient is mainly interested in theeffectiveness of a therapy ('Does it work?'), and whether it canbe used to treat the patient's condition ('Will it work for me?').At this point the 'nanomedicine' terminology of a noveltherapeutic method becomes a minor concern. For patients,the difficulties of defining and delineating nanomedicine aretherefore no longer an issue, which leads to the question ofwhether efforts to initiate and conduct a public debate basedon the term 'nanomedicine' are superfluous.

ii. Defining Communication

For the purpose of this report, ‘communication’ needs to beconsidered comprehensively. The simple act of transmittingand exchanging information by various means has beenextensively shown to be insufficient to address current needs.The desired outcome of nanomedicine communication shouldnot simply be one of maximising the benefits to society whilstminimising the risks. The debate should also be reconfiguredso that it better characterises what is at stake in emergingtechnologies.

As the DEEPEN project report states: "lay reactions tonanotechnology are complex. Public responses to thetechnology, or even to particular applications, are not simplyeither positive or negative; rather, pros and cons are seen asintermingled and often inseparable. Laypeople are also notcontent with weighing up risks and benefits. Their concernsand enthusiasms go beyond this narrow framing toencompass anything from the dangers of perfection to theproblematic nature of controlling life."

The goal of any nanomedicine communication strategy musttherefore be to initiate public debate, deliberating on bothpersonal and social consequences of innovations andacknowledging that a simple discussion of risks and benefitsshould only be regarded as a starting point for dealing withthe complex concerns of a technology-dependent society.

At the same time it is important to note that different actors

and stakeholders will themselves have different definitions ofthe term 'communication'. In the extreme the term couldmean things as distant as propaganda and education. It istherefore essential to separate, for example:• communication with peers/industry/students/policy

makers (a form of marketing with science: seduce, sell,convince);

• communication with the media (inform, avoidsensationalism); or

• communication with the public (explain, evaluate, debate,deliberate).

These different definitions have to be kept in mind whendiscussing and planning a comprehensive communicationstrategy in order to avoid misunderstandings between thevarious stakeholders. It is also essential to take into accountthe communication requirements and needs of the variousstakeholder groups. For example, various studies, includingthat conducted by the NanoMed Round Table’s Patients’Needs Working Group , have shown that the level ofknowledge about nanomedicine is very low among the publicin general.

It is also important to note that the communicationrequirements of each individual member of the generalpublic can greatly differ, ranging from the simple request forbasic information on a specific therapy to a need for fully-fledged deliberation on ethical, social and regulatory aspects.It is therefore vital for any successful communication strategyto take this range of varying expectations into account.

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The findings and recommendations in this report are basedon a thorough analysis of existing case studies, documents,and construction of scenarios on the possible consequencesand impacts of various communication methods in the field ofnanomedicine. The Working Group’s discussions werefocused around two main case studies chosen for their widerange of communication aspects:• the communication strategy of the nanomedical company

'MagForce Nanotechnologies AG' (see Annex 1); and• a study of 'Visions of Nanomedicine' (see Annex 2).

The Working Group also took into account patients’ needs andethical, regulatory, social and economic issues.

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1 Davies, S., Macnaghten, P. and Kearnes, M. (ed.) 2009. Reconfiguring Responsibility: Lessons for Public Policy (Part 1 of the report on Deepening Debate on Nanotechnology).Durham: Durham University.

2 Ref. Patients’ Needs Working Group report.

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i. The Importance of Communicating About Nanomedicine

Communicating about nanomedicine is increasinglyimportant because of its significant potential to:• raise societal and ethical questions concerning risks and

benefits due to the predicted use of active nanomaterials innovel treatments and, subsequently, their use within thehuman body; and

• have an emotional and material impact (arising frombenefits and uncertainties) due to the large number ofimmediately and peripherally affected people.

Although the term 'nanomedicine' has been used by expertsand stakeholders to describe the application ofnanotechnology to health and medicine for over ten years,there is still a considerable lack of public awarenessconcerning this field. Despite high expectations of futuredevelopments to enable better, more efficient and affordablehealthcare for millions of patients and promise noveltreatments for many illnesses, the general public is oftenuninformed not only of these potential benefits, but of theentire field of nanomedicine.

However, even among informed members of the public,perceptions vary significantly. On the one hand the abundanceof visions, both realistic and unrealistic, has raised hopeswhich stakeholders may well not be able to meet in the nearfuture, due to the time required for the development andmarketing of novel treatment methods. At the same time,social, ethical and legal questions are being raised concerningissues of safety, environmental and long-term effects, toname but a few. In particular, the use of treatments based onactive nanomaterials such as therapies with cancer-targetingparticles or theranostic approaches have high potential forcontroversy. The best way of answering the demands of aninquiring public is to realise the need for active public debateand to develop and implement strategic approaches to thisend.

The potential of nanomedicine to be the field of applicationabout which important societal and ethical questions are firstraised, means that any public reaction to nanomedicine islikely to reflect on nanotechnological applications outside ofthe medical field. However, one of the inherent dangers is thatthe nanomedical community will not be able to fulfil theexpectations of a hopeful public before it becomesdisillusioned. A comprehensive debate on the visions, likelytimelines, risks and benefits of nanomedicine is, therefore,also vital to the future of nanotechnology.

Although easy to state and recommend, facilitating such acomprehensive debate is not a trivial task. Public engagementis challenging, and reactions can often differ greatly from thedesired outcome. It is therefore necessary to work to

understand the complexity of public 'attitudes' and tocontinuously adapt communication strategies accordingly.

As expert groups and governments have repeatedly stated, itis also important for scientists, businesses and otherstakeholders to ‘earn’ public trust in order to be ablesuccessfully to engage the public in dialogue, for example onhow to develop and regulate nanotechnologies.

Furthermore, there is a need for governments and fundersacross Europe to respond to past public dialogues and expertgroup recommendations in order to prevent overreactions topotential hazards and risks, which would severely limit thepossibility of public dialogue and potentially lead to the wholearea of nanotechnologies being rejected by the public andNGOs. In view of this, special attention should be paid tocommunication in the fields of health and environmentalimpacts of nanoparticles, as substantial amounts of fundinggo into commercialising them.

Finally, any communication strategy that is developed needsto take into account the significant changes in communicationmethods that have occurred over the past years. For example,the importance of doctors as the primary source of medicalinformation is steadily declining, being replaced by theinternet and new media. This development is questionable, asthe quality of this information is highly variable on the onehand, whilst on the other hand the sheer volume ofinformation is often confusing or overwhelming to the averagepatient. Both these aspects can lead to confusion andmisunderstanding. Nevertheless it is expected that patientsgive their informed consent before initiating a medicaltreatment, often based on this confusing or overwhelminginformation. This is a discrepancy which needs to beaddressed. In order to enable patients to make informeddecisions, the public not only needs access to a reliable andcredible source of information, but also to fora to debate anddiscuss questions concerning needs, risks, benefits, as wellas ethical and social issues related to nanomedicine.

In conclusion, it is therefore important to be continuouslyinnovative in communication practices, especially pertainingto public engagement and debate, in order to reach as wideand varied an audience as possible.

ii. Providing Reliable Information About Nanomedicine

oÉÅçããÉåÇ~íáçå NThe European Commission should establish a platform toprovide credible and accessible sources of balancedinformation on the methods, benefits and risks ofnanomedicine, focusing on the communication needs ofpatients and the medical community.

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3 Combined diagnosis and treatment.

PR A report on the nanomedicine economic, regulatory, ethical and social environment

The term 'nanomedicine' is a generic term and can thereforegenerate misunderstanding, which is a source of mistrust anddoubt. Even experts find it difficult, if not impossible, to providea valid and accepted definition for this term. In addition, laypeople often become confused by the wealth of informationavailable and unspecified use of technical terms. This alsoleads to uncertainty and apprehension. Recommendation 1 isintended to help achieve higher clarity of message in regard tonanomedicine, and therefore counter these problems.

The platform should provide quality information whilst alsoencouraging public debate and deliberation on risks,innovations and developments in the field of nanomedicine. Itshould focus on the communication needs of those mostimmediately and personally affected by nanomedicine:patients, doctors, healthcare workers, medical students andpharmacists.

Prior to establishing the platform, representatives of thegroups listed above need to be consulted to determine theirneeds concerning required information, dialogue fora andinteractive elements. The inclusion of certain stakeholdergroups at such an early stage correlates with the findings of thePatients’ Needs Working Group, which recommended acommunication loop where patients could contribute what theywould like to know and how they would like this information tobe structured and made available. At a secondary level, furtherstakeholders should be included such as health insurers,regulatory bodies and pharmaceutical companies.

In order to ensure that the format of the platform remains up-to-date with new developments, both with regard to thenanomedical field as well as to public debates, the platformshould be reviewed every two or three years. The review, whichcould be organised as a recurring round table event, shouldinvolve various stakeholders and a wide range of experts in thefields of nanomedicine, communication, ethical and socialdebate, as well as representatives of doctor and patientorganisations. As an additional benefit this approach couldprovide a model for other medical fields such as regenerativemedicine.

Establishing such a platform would not only make it easier forthe general public to understand fundamental nanomedicalissues as well as specific product details, but would alsosupport the communication tasks of key actors in this fieldsuch as doctors, patient groups and health insurers.

A structured communication platform in the nanomedical fieldwould contribute to the improvement of sub-optimalcommunication between health professionals, which is asignificant cause of incidents compromising patient safety. It

would alleviate current problems of information transfer,thereby reducing future misunderstandings. This will becomeincreasingly important as nanomedical methods becomemore complex and the associated communication issuesbecome increasingly challenging as a result.

Recommendation 1 would also satisfy a number of importantpoints raised by the experts involved in the NanoMed RoundTable project, not least because it could be used to implementsome of the Patients’ Needs Working Group’srecommendations. In the initial phase Recommendations 7and 8 should be considered, whilst the information referred toin Recommendations 2 and 3 could be incorporated into thefinal platform.

iii. Providing Know-How for Communication AboutNanomedicine

oÉÅçããÉåÇ~íáçå OThe European Commission should develop communicationguidelines for the various nanomedicine stakeholders andprovide good practice examples. Particular care should betaken to differentiate between the communication aspects ofthe related fields of food, environment and nanomedicine.

Common questions concerning the subject of nanomedicineare not only 'How does it work?' or 'What are the risks?', butalso 'How reliable is the information?', 'How do they know?','Who is the messenger?' or 'Can the messenger be trusted?'

The latter five questions are directly linked to the expectationsof the questioner, and can therefore directly influenceacceptance, so a structured communication strategy is vitalto improving public understanding of and initiating abalanced public dialogue on nanomedicine. In addition, due totheir individuality, highly specialised nanomedical treatmentswill often require specifically tailored communicationapproaches. This is also often necessary due to competition,alternative treatments, patient needs and numerous otherreasons.

However, as the majority of current nanomedical researchand development is being conducted by small or medium-sized enterprises (SMEs), the actors in this field often do nothave the know-how or staff time and financial resources

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4 Ref Patients’ Needs Working Group report.5 Recommendation 2 -The European Commission has produced much useful information

on the internet on nanotechnology aimed at the general public, e.g. short films, leafletsand brochures. It should organise the production of similar lay information onnanomedicine, which could be used by patient organisations, clinicians and the media.

Recommendation 3 - European-level trade and research associations should produce layinformation using stakeholder dialogue on nanomedicine research. National-level trade andresearch associations should disseminate this information in their country’s language(s) topatient organisations, media and bodies representing clinicians. The European Commissionshould investigate what existing programmes should or could fund this, and/or develop newfunding streams where required, e.g. science and society programmes or European ScienceFoundation.Recommendation 7 - Where governments have organised stakeholder fora to discuss thepotential risks and benefits of nanotechnologies including nanomedicine, they shouldencourage patient organisations to participate. Where such fora do not exist, governmentsshould urgently consider establishing them.Recommendation 8 - The European Commission should provide funding for focus groups andother participatory methods (e.g. citizen conferences, interviews, surveys) in a number ofEuropean countries, to identify and understand the spectrum of issues that nanomedicineraises for patients and their families.

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required for initiating and implementing a successfulcommunication strategy. This can significantly delay or hinderthe development, introduction and application of innovativenanomedical treatments, with a detrimental effect on theimpact of nanomedicine in Europe, both in medical andeconomic terms.

For the above reasons, a series of communication guidelinesshould be developed by leading experts in this field. These couldbe used by research organisations, nanomedicine companiesand other stakeholders as an effective tool for initiating publicengagement whilst contributing to economic viability. Theguidelines could be compiled in the form of a matrix, containinginformation on:• which target groups need to be communicated to (e.g.

regulatory bodies, patient groups, medical associations,general public, NGOs);

• what information is required by these individual groups (e.g.technical requirements, regulatory issues, possible sideeffects, life cycle analysis); and

• at what stage of treatment, development or testing therequired information should be communicated to therespective groups.

In developing these guidelines a special emphasis must beplaced on carefully differentiating between the communicationaspects of the related fields of food, environment andnanomedicine. Although there are some common issues andoverlap in certain areas, it is highly important that thedifferences of these respective fields are communicated. If thisis not considered, any negative tendencies concerningnanotechnology in food or environment are likely to havedetrimental effects on the field of nanomedicine.

However, the development and application of communicationguidelines does not in itself guarantee the success of ananomedical method or even of nanomedicine itself. The casestudy on MagForce Nanotechnologies (see Annex 1) shows thateven a principally sound communication structure is in itself notsufficient to ensure a breakthrough of a novel treatment(obviously one of the main goals for a company developing anew product). In this case the company was not able to convincethe professional oncology community to support thedevelopment of their treatment method, despite promising testresults. This hindered the project, influencing the choice of testsubjects and causing delays in the clinical tests.

iv. Ensuring Sufficient Resources for Communication AboutNanomedicine

oÉÅçããÉåÇ~íáçå PThe European Commission, national governments, industryand independent grant organisations should allocate asignificant percentage of financial resources in the field ofnanomedicine to public communication. The goal of this

financial allocation should be to encourage publicengagement, foster dialogue and move beyond the simplediscussion of risks versus benefits.

Communication is one of the main pillars of our society. Goodcommunication causes societies to flourish, businesses toprosper and innovations to succeed. In contrast, inefficient orinadequate communication is a major source of doubt,distrust and fear, with potentially devastating results. In thecase of nanomedicine, this could lead, on a small scale,simply to the rejection of an individual treatment method. On alarge scale, however, a number of such cases of rejectioncould lead to distrust and fear of the entire field.

A key question, however, is how to finance communication in afield which on the one hand is struggling to define itself, whilston the other hand has long development times and thereforelong investment return times, meaning that profits arenegligible, currently and for the near future. In answer to thisquestion, a significant percentage of financial resources in thefield of nanomedicine should be allocated to communication –both in European and national funding programmes as wellas by industry and independent grant organisations. Thisapproach would ensure that communication expenditure isdirectly tied into the amount invested into research and wouldtherefore automatically increase with any future growth in thisfield. In addition, the informational content would always betied into current research, ensuring up-to-datecommunication.

In this context it is important to ensure that thecommunication strategies of a project are not simply focusedon disseminating the results of the project itself. Of course thisis always a vital element in any project. However, the goal ofthis financial allocation should be to go many steps further,encouraging public engagement, fostering dialogue andmoving beyond the simple discussion of risks versusbenefits. In practice, project proposals which do notdemonstrate a comprehensive communication strategyshould receive a lower evaluation or be encouraged toimprove on this point. It could also be a requisite that newcalls for proposals recommend the inclusion of projectpartners with expertise in this field.

Furthermore, businesses need encouragement and supportin engaging with the public in the ways described above. Atpresent, many nanotechnology companies are fearful ofpublic engagement, having seen what happened regardingthe issue of genetic modification, and are aiming to 'managethe message' better, rather than open up a dialogue with thepublic about developing nanotechnologies. Although at firstthis would seem to be a preferable way of preventing orlimiting overreactions in this field, in the long term it iscounter- productive to improving public understanding andinitiating a balanced public dialogue on nanotechnology ingeneral and nanomedicine in particular.

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PT A report on the nanomedicine economic, regulatory, ethical and social environment

ChairProf Sir John Beringer Formerly Pro Vice-Chancellor, University of Bristol, Symbiotic Consultancy Ltd, UKCoordinatorProf Julian Kinderlerer TU Delft, Netherlands/ University of Cape Town, South Africa/ European Group on

Ethics in Science and New Technologies ManagementDr David Bennett Delft University of Technology, The Netherlands & St Edmund’s College,

University of Cambridge, UKDr Klaus-Michael Weltring Bioanalytik-muenster, GermanySerene K. Chi TU Delft, Netherlands

tmO=m~íáÉåíëÛ=kÉÉÇëChair Alastair Kent Director, Genetic Interest Group, UKParticipantsDr David Bennett Delft University of Technology, The Netherlands & St Edmund’s College,

University of Cambridge, UKProf Dr Sir John Beringer Formerly Pro Vice-Chancellor, University of Bristol, Symbiotic Consultancy Ltd, UKAvril Daly Fighting Blindness, IrelandLaura Gilbert RapporteurMelissa Hillier Genetic Interest Group, UK Dr Mahendra Gonsalkorale Former member of European Parkinson's AssociationLorraine Le Floch Independent Patient, European Multiple Sclerosis Platform (EMSP)Sabine Lobnig European Patients ForumRod Mitchell Chairman, European Crohn's and Colitis Organization (ECCO)Liuska Sanna European Patients Forum

tmP=bíÜáÅ~ä=~åÇ=pçÅáÉí~ä=^ëéÉÅíëChairProf Alfred Nordmann Technische Universität Darmstadt, Germany & University of South Carolina, USAParticipantsDr Johann S. Ach Universität Münster, GermanyDr David Bennett Delft University of Technology, The Netherlands & St Edmund’s College, University

of Cambridge, UKProf Dr Sir John Beringer Formerly Pro Vice-Chancellor, University of Bristol, Symbiotic Consultancy Ltd, UKProf Marianne Boenink University of Twente, NetherlandsDr Donald Bruce Edinethics Ltd., Edinburgh, ScotlandDr Karin Christiansen University of Aarhus, DenmarkProf José Manuel de Cózar University of La Laguna, SpainDr Arianna Ferrari Technische Universität Darmstadt, Germany Robert Geertsma RIVM Bilthoven, NetherlandsProf Uffe Juul Jensen University of Aarhus, DenmarkJan Reinert Karlsen University of Oslo, NorwayProf George Khushf University of South Carolina, USADr Thorsten Kohl Technische Universität Darmstadt, Germany (Researcher, Rapporteur)Dr Ineke Malsch Malsch TechnoValuation, NetherlandsDr Sabine Müller Universität Bonn, GermanyProf João Arriscado Nunes University of Coimbra, PortugalProf Jan Helge Solbakk University of Oslo, NorwayProf Mariachiara Tallachini University of Milano, ItalyDr Susana Vidal UNESCO, UruguayDr Klaus-Michael Weltring Bioanalytik-muenster, Germany

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PUA report on the nanomedicine economic, regulatory, ethical and social environmentNQ

The Working Group thanks the Brocher Foundation in Geneva for hosting its second meeting at its facility for conferences about bioethical dimensions of medical research. For editorial support, the Group thanks Sarah Davies.

tmQ=bÅçåçãáÅ=fãé~ÅíChairDr Françoise Charbit CEA, France ParticipantsDr François Berger Grenoble Hospital and INSERM, FranceProf Dr Sir John Beringer Formerly Pro Vice-Chancellor, University of Bristol, Symbiotic Consultancy Ltd, UKDr Bernhard Buehrlen Fraunhofer Institute for Systems and Innovation Research, GermanyDr Giorgio Cassara Don Gnocchi Foundation, ItalyDr Paul Garassus Clinique du Tonkin, FranceDr Laurent Levy Nanobiotix, FranceDr Christine M’rini Mérieux Alliance, FranceDr Christine Muzel Philips Healthcare, NetherlandsProf Reinhard Rychlik Institute for Empirical Health Economics, Burscheid, GermanyDr Jürgen Schnekenburger University Clinic Münster, GermanyDr Paul Smit Philips Healthcare, NetherlandsDr Klaus-Michael Weltring Bioanalytik-muenster, Germany

tmR=oÉÖìä~íáçåChairMark Cantley Formerly European Commission Research Directorate-General, UKParticipantsProf John Adams University College London, UKDr Mike Adcock Durham University, UKProf Dr Sir John Beringer Formerly Pro Vice-Chancellor, University of Bristol, Symbiotic Consultancy Ltd, UKSerene K. Chi Delft University of Technology, The NetherlandsProf Ken Donaldson University of Edinburgh, UKDr Bärbel Dorbeck-Jung University of Twente, NetherlandsJoel D’Silva Leuven University, BelgiumDr Thomas K. Epprecht Swiss Re, SwitzerlandDr Steffi Friedrichs Nanotechnology Industry Association, BelgiumDr Peter Hatto CEN/ISO, UKDr Juergen Hirschfeld Bayer, Germany Dr Peter Kearns OECD, FranceProf Julian Kinderlerer Delft University of Technology, The Netherlands/ University of Cape Town, South

Africa/ European Group on Ethics in Science and New Technologies Dr Horst Siebold Siemens, Germany Prof Rogério Sá Gaspar University of Lisbon, PortugalDr Rob Slobbe Philips, NetherlandsDr Klaus-Michael Weltring Bioanalytik-muenster, GermanyDr Jiang Yu Chinese Academy of Science, China

PV A report on the nanomedicine economic, regulatory, ethical and social environment

tmS=`çããìåáÅ~íáçåChairProf Dr Wolfgang M. Heckl Deutsches Museum/ TU München, GermanyParticipantsDr David Bennett Delft University of Technology, The Netherlands & St Edmund’s College, Universityof Cambridge, UKProf Dr Sir John Beringer Formerly Pro Vice-Chancellor of University of Bristol, Symbiotic Consultancy Ltd, UKMark Cantley Formerly DG Research, UKRichard Hayhurst Hayhurst Media, UKMelissa Hillier Genetic Interest Group, UKPaul Hix Deutsches Museum, GermanyDr Andreas Jordan MagForce Nanotechnologies AG, GermanyDr Ulrich Kernbach Deutsches Museum, GermanyProf Dr Harald Krug EMPA Materials Science & Technology, SwitzerlandProf Dr Phil Macnaghten Durham University, UKHayley Birch Science Writer/Editor, UKProf Dr Harald Fuchs WWU Münster, GermanyDr Antje Grobe Risk Dialogue Foundation, SwitzerlandProf Kathy Sykes University of Bristol, UKWiebke Pohler LMU München, GermanyDr Michael Schillmeier LMU München, GermanyBart Walhout Rathenau Institute, NetherlandsDr Klaus-Michael Weltring Bioanalytik-muenster, Germany

Other ContributorsDr Simon Baconnier Connective Tissue Cancer Network, FranceDr Bruno Bonnemain Guerbet, FranceProf Gérard Duru University of Lyon, FranceDr Philippe Mourouga Sanofi Aventis, FranceDr Detlef Niese Novartis, SwitzerlandProf Dr Lode Vereeck Member of the Flemish Parliament, Belgium

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