conference 3 "outcomes of 15 national conferences, "

Istituto Superiore di SanitàRome (Italy)

Avril Daly

Representing EURORDIS as a Partner in EUROPLAN

Chief Executive Officer of Fighting Blindness (Ireland)

Chair of the Genetic & Rare Disorders Organisation (GRDO)

13 May 2011, Eurordis General Assembly – Amsterdam

Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)

National Conferences

OBJECTIVES, METHODOLOGY and TOOLS

KEY FACTS & FIGURES and MAIN RESULTS

KEY MESAGES for each THEME

KEY QUESTIONS for PANEL DISCUSSION


EUROPLAN WP8

Title: “Promoting the recommendations for Rare Diseases National Plans and presenting the Commission Communication on Rare Diseases”.

WP leader: EURORDIS

Funding: 60% EC Contribution + 40% EURORDIS


Commission Communication and Council

Recommendations on RD ensure that common policy

guidelines are shared everywhere in the EU

EUROPLAN Rrecommendations on RD National Plan within

EU MS link national efforts with a common strategy at EU level

Presenting the Commission Communication and Council Recommendations on RD

WP 8 objectives reflect this double combined approach

Reality check + Promoting the EUROPLAN

Recommendations on RD National Plans

EUROPLAN WP8 OBJECTIVES


EUROPLAN WP8

EURORDIS supported the organisation of 15 National Conferences (instead10 planned in initial

contract) on Promoting National Plans or Strategies for Rare

Diseases based on the Commission Communication , the Council

Recommendations and EUROPLAN Recommendations and monitoring indicators

14 National Alliances and 1 Foundation for RDs, selected following a Call for Proposals

6 Advisors appointed by EURORDIS following an Expression of Interests: Simona Bellagambi, UNIAMO, Italy Britta Berglund, Rare Diseases Sweden, Sweden Avril Daly, GRDO, Ireland Dorica Dan, RONARD, Romania Mirjam Mann, ACHSE, Germany Christel Nourissier, Alliance Maladies Rares, France


WP8 EURORDIS’ ADVISORS

MoU signed between EURORDIS and Advisors’ organisations where the reciprocal engagements are laid out

Advisors in charge of 2 to 3 national conferences

Liaise with Conference organisers / National Alliances: key contacts throughout the entire project

Liaise with EURORDIS and other Advisors to exchange best practices and difficulties

Supervise the Conference Agenda setting

Support to Conference Report drafting


METHODOLOGY (1)

A methodology was developed for the organisation of the 15 National Conferences based on the following:

Promote Patient-run Multi-stakeholders approach Ensure that all interests at stake in each country were

involved in the debate that should eventually lead to advance the national policy on RDs

Promote EU & MSs integration + MSs exchanges Discuss the challenges of the transferability of the EU

Recommendations

A ‘supranational’ coordination was created while ensuring a close touch with the national situation (EURORDIS staff and 6 Advisors)


METHODOLOGY (2)

Result-oriented / transferability and national situation Conducive to deliver suggestions to improve the existing

country policy on RDs, while analysing the transferability of European guidelines in the national system.

Common Framework for all Conferences. Same format for each conference Same layout and design of presentations Same documents downloadable on EURORDIS’ website Same format for final reports

Harmonised model of discussion, tackling the same issues, same concepts and terminology is strengthening the unity of RDs’ policy and its supporting community


EUROPLAN CONFERENCESCOMMON FORMAT

Each National Conference is configured with Plenary Session and Workshops

Each Workshop is devoted to a specific Theme and reports to the plenary on that Theme

Conference organisers decided how each Theme and Sub-Theme is dealt with in the National Conference and on practical solutions or proposals emerged in that area

Therefore the Conference Final Report is based on the Workshops’ reports and cover the proposal emerged from each of the Workshops


EUROPLAN NATIONAL CONFERENCES - THEMES

THEME 1: Methodology and Governance of a National Plan or National Strategy

THEME 2: Adequate Definition, Codification and Inventorying of Rare Diseases THEME 3: Research on Rare Diseases THEME 4: Standards of Care for RDs, Centres of

Expertise, European Reference Networks, Orphan

Drugs THEME 5: Patient Empowerment and Specialised

Services

Horizontal themes THEME 6: Sustainability of infrastructures and activities

in the field of RDs

THEME 7: Gathering the Expertise on Rare Diseases at the

EU level


www.eurordis.org National Rare Disease Policy


TOOLS on EURORDIS’WEBSITEEUROPLAN & National Conferences

I. EU Policy Documents on Rare Diseases (EC Comm. RDs; Council Recos on RDs…)

II. EUROPLAN Guidance Documents for the National Plans (Recommendations, Indicators…)

III. EURORDIS Guidance Documents for the National Conferences: Conference Agenda Layout & Content outline Guidelines for WG Chairs and Rapporteurs Template Reports Conference Reports EURORDIS Policy Fact Sheets, Key Presentations,

etc…IV. EUROPLAN National Conferences’ Final Reports

and Conferences « Photo Wall »


EUROPLAN NATIONAL CONFERENCE REPORTS

Each Final Report includes:

Report of the discussion and outcomes of the Working Groups (themes)An overall assessment of the usefulness of the EUROPLAN Recommendations for the advancement of a national strategy in the country.The transferability of the EUROPLAN Recommendations in your country.

This assessment results from the collection of the evaluation carried out at the end of each Workshop

and included in the Workshop reports…


15 National Conferences

KEY FACTS & FIGURES

MAIN RESULTS

KEY MESAGES by THEME


KEYS FACTS & FIGURES (1)



COUNTRY CITY ORGANISERNUMBER of

PARTICIPANTSDATE ADVISOR

BULGARIA Plovdiv NAPRD National Alliance of People with RD

352 28-29-30 May Dorica Dan

CROATIA Dubrovnik Croatian Society for RD 180 17-18-19 September Christel Nourissier

DENMARK Copenhagen Rare Disorders Denmark 81 19 November Britta Berglund

FRANCE Paris Alliance Maladies Rares 153 30 September Christel Nourissier

GERMANY Berlin ACHSE 190 13-14 October Mirjam Mann

GREECE Athens PESPA 199 26-27 November Simona Bellagambi

HUNGARY Budapest HUFERDIS 138 15-16 October Dorica Dan

IRELAND Dublin GRDO 169 20 January 2011 Avril Daly

ITALY Florence UNIAMO 120 11-12-13 November Simona Bellagambi

NETHERLANDS The Hague VSOP 80 18-19-20 November Mirjam Mann

POLAND Krakow Foundation MATIO 64 22 October Christel Nourissier

ROMANIA Bucarest RONARD 178 18-19 June Dorica Dan

SPAIN Burgos FEDER 116 5-6 November Simona Bellagambi

SWEDEN Stockholm Sällsynta diagnoser 84 11 November Britta Berglund

UNITED KINGDOM

Manchester Genetic Alliance UK - Rare Diseases UK

86 16 November Avril Daly



15 Conferences in a range of different countries:

Northern and Southern countries. New and old EU Members States, and even a EU

candidate country, Croatia. Countries of different sizes, from the largest EU

countries (Germany) to smaller countries like Denmark or Ireland.

All sorts of health systems were represented: from centralised ones (France) to systems with a greater degree of decentralisation (Italy, Spain, Germany and UK).

Spill-over effect in countries outside the scope of EUROPLAN Project: conferences in Austria, Finland, Switzerland, Russia, Armenia, Georgia.



Over 2200 participants with representatives of main stakeholders groups (so distributed by % of attendance):

Public authorities: 14% (up to 22% in Spain)

Academics /researchers: 20% (up to 30% in Hungary)

Healthcare professionals: 14 % (up to 29% in France)

Industry: 10% (up to 22% in UK)

Patients: 39% (between 33% and 44%)


MAIN RESULTS about NATIONAL CONFERENCES (1)

1. Has effectively help present the two new EU key policy texts on RD - Commission Communication & Council Recommendation on Rare Diseases – reaching broader national audiences

2. Helped to make RD communities more aware that national efforts are “embedded” into a more comprehensive and long-term approach integrating EU and national levels.

3. Specific recommended actions and national measures have been integrated into the debate and the language of stakeholders participating to the Conferences .

4. Altogether, EUROPLAN Recommendations were broadly endorsed or validated and in many cases amended by the national audiences.



6. There is a great variability among and within countries about the type of services provided to rare disease patients and the accessibility to these services. Hence, national conferences were modulated to propose a common format while, at the same time, leaving a certain degree of flexibility to the organisers to respond to the most pressing questions arising from their specific national context.

7. The EUROPLAN Conference provided an invaluable opportunity to have the all or most national stakeholders sitting around the same table to discuss the same agenda.



8. The whole experience of EUROPLAN proved to be an outstanding awareness-raising exercise in the field of rare diseases at the national level and created momentum on Rare Disease National Plans across Europe.

9. This momentum should not be lost: the results of the EUROPLAN Conferences should converge into the work carried out in the country and serve as a “blue print” to develop/implement the National Plans.

10.To conclude: the National Conferences proved instrumental to achieve the expected outputs of the EUROPLAN project.


KEY MESSAGES by AREA

1. It is essential to concentrate policy actions on RDs around a National Plan (NP) or Strategy, to address specificities and to optimise resource and actions.

2. Integration of the measures of the NP into the national health system is crucial, as rare disease patients need to become part of the health and social system planning.

3. Mapping existing resources is a necessary process virtually in all countries.

4. Drawing up a NP should be an inclusive process i.e. a with a body responsible for the development and the implementation of the NP composed of all stakeholders including patients.

Area 1 – Methodology and Governance



5. NPs must transcend sectors, be comprehensive and include healthcare and social provisions.

6. NPs cannot function without resources attached. Sustaining the Rare Disease Strategy will be very important, particularly in terms of embedding the strategy into everyday healthcare services.

7. A monitoring system must be integrated into the Plan with a suitable representation of qualified patients and possibly using as a reference the EUROPLAN Indicators (to be refined based on experience).

Area 1 – Methodology and Governance



1. The EU definition of RDs is supported although with a few exceptions .

2. While awaiting ICD-11, it is suggested to introduce existing coding systems such as the ICD10 or the Orphanet code, possibly with cross-referring systems.

3. Day-to-day registration of rare diseases should be organised in such a way that the workflow, as far as possible, is integrated into existing administrative systems.

4. Patient registries sustainability must be achieved by optimising resources and reducing fragmentation and overlapping.

Area 2 – Definition, coding and inventorying



1. RDs must be a national priority in medical research and ad hoc national research measures should be dedicated to RDs.

2. RD research programmes must be supported primarily by adequate public funds, even though public-private partnerships were not excluded.

3. RD research should be multidisciplinary and cover all research areas, but chiefly focus on translational research.

4. Socio-economic research (i.e. research on quality of life, living conditions, etc.) was highlighted in most National Conferences as extremely important and should become a national priority.

Area 3 – Research on RDs



5. Qualified patients advocates must become fully-fledged research partners.

6. Centres of Expertise must play a pivotal role in closing the gap between research and care.

7. All National Conferences called for the creation of quality patient registries as a primary objective and a basic requirement in the development of RD research.

8. Multi-centre national and international studies are an absolute necessity for the organisation of clinical trials.

Area 3 – Research on RDs



1. Mapping out CoE (Centres of Expertise) is a necessary exercise, prior to any planning on CoE.

2. National Conferences called uniformly for common quality standards for the designation and the accreditation of CoE.

3. Proximity of care is a recurring theme with answers modulated according to the size of the country and the healthcare system existing therein.

– In smaller countries, CoE for all diseases is inappropriate, still valid for some diseases, when expertise exist + networks of medical experts could be established, while promoting cross-border cooperation at the same time.

– In larger countries or decentralised countries, vertical systems (local/regional/national) are preferable.

Area 4 – Centres of Expertise and European Reference Networks



4. Integration between medical and social levels must be strengthened further, with a necessary reinforcement of the social level.

5. Services acknowledged by Centres of Expertise as essential to provide to patients should be recognised and reimbursed.

6. Healthcare pathways are defined and adopted, based on best practices and expertise at national and international. “Case managers” should dedicate their attention to individual cases and follow them all along the healthcare pathway.

7. Mechanisms should be found to recognise and reimburse the interventions of paramedical and other specialist professionals involved in the care of RD patients.




8. Promoting the participation of CoEs in European Reference Networks (ERN) should be integrated in the NP and become a quality criterion of the performance of each CoE.

9. Cross-border healthcare should be promoted, especially for small countries where the existing resources cannot cover all RDs and mobility of patients appears both as a need and a concern.

10. Screening policies must be supported by robust legal frameworks and by reliable implementation and evaluation. National Conferences are calling to extend the current neonatal screening programmes and to coordinate them.




1. Activities to promote information and increase awareness on rare diseases to the public at large must be boosted.

2. Information sources for patients and professionals should be validated to ensure a consistent quality level.

3. To make healthcare professionals acquainted with rare diseases, training of professional doctors should start during the medical degree course and continue during their careers, with different degrees of specialisation.

4. Guidelines should be developed to align actions performed at different levels of care and by different healthcare professionals, with specific information for patients, families, caregivers, social workers and teachers.

Area 5 – Gathering expertise at EU level (Information and training)



1. Collaboration among EU countries and EU authorities is necessary to overcome the bottlenecks created by the scarce and fragmented expertise on orphan drugs at national level. The participation to the Common Scientific Assessment of the Clinical Added Value of ODs is widely supported as the right mechanisms to make this cooperation work in practice.

2. In order to manage compassionate provision of orphan drugs, systems of ‘temporary authorisations’ could be granted under certain conditions.

3. It is important to make compulsory at the national level the collection of data on the efficacy and tolerance of medicines under compassionate or early access and off-label use.

Area 5 – Gathering expertise at EU level (Orphan Drugs)



1. Firstly, patient empowerment is a holistic approach to each individual citizen.

2. Involvement of patient groups representatives in decision-making processes can be only achieved when patients are really enabled to participate to such processes. This means that patients need to be qualified to become fully-fledged partners in decision-making debates which affect them directly

3. The importance of specialised services for RD patients for patients and families must be recognised and financially supported by the community - in particular Respite Care Services and Therapeutic Recreative Programmes.

Area 6 – Patient Empowerment



1. Need to devise strategies and mechanisms to recognise and integrate RD patients into existing social services (rehabilitation, access to social services and financial supports, integration into school and workplaces), while recognising their specificities and providing quality services in response to their needs.

2. Help lines should be included in the provisions of a NP on RD and financially supported to provide personalised information and orientation. Help lines should be set up according to quality standards, such as those developed in the framework of European Network of Help Lines.

Area 6 – Patient Empowerment


ACKNOWLEDGEMENTS TO

• EURORDIS Advisors:

Simona Bellagambi, Britta Berglund,

Avril Daly, Dorica Dan,

Mirjam Mann, Christel Nourrissier

• EURORDIS Staff:

Yann Le Cam as Chief ExecutiveValentina Bottarelli as our Project Manager in EuroPlanAriane Weinman as support to Managers & AdvisorsJustine Evans as Web Content ManagerPatrice Régnier as Finance Director

• The 14 National Alliances and Fundacio Matjo of Poland who organised National Conferences

• ISS colleagues and all EuroPlan Partners


PANEL DISCUSSION

KEY QUESTIONS FOR DISCUSSIONon

IMPACT and USEFULNESS of NATIONAL CONFERENCES

and SUGGESTIONS for 2012-2014


KEY QUESTIONS FOR DISCUSSION

Were the national conferences useful:In promoting national plans in these 15 countries?

To disseminate and promote the understanding of the EU policy framework on rare diseases?

To bring all stakeholders together?

To do a reality check of EUROPLAN Recommendations?


KEY QUESTIONS FOR DISCUSSION

Overall, are the EUROPLAN Recommendations relevant, understood and well accepted?

Can we sort out a common core of recommendations and other recommendations which need to be further adapted to national situations?

Which lessons can we learn from these 15 conferences regarding adjustment national strategies on rare diseases according to country population size and GNP dedicated to health?


EUROPLAN

THANK YOU!

conference 3 "outcomes of 15 national conferences, "

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