ucd rare disease module 2017 - dr derick mitchell - march 28th 2017
TRANSCRIPT
PATIENTS – SCIENCE – INDUSTRY
UCD Rare Disease Module, March 28th, 2017
Derick Mitchell, PhD
Overview• Context
• Environment IPPOSI exists in / was created to address• IPPOSI – aims/objectives• Rare diseases in Ireland & Europe
• Centres of Expertise – European Reference Networks
• Orphan drugs & technologies
• Your feedback
May you live in changing times….
Clara’s Story• At birth, Clara was diagnosed with
Achondroplasia - a rare bone disease that no one knew about in the local hospital.
• With no experts in the local area, Inés, Clara’s mum, became the expert, directing the medical teams
• Clara had to go through months of countless exams and medical appointments
• She had three surgeries, one of which was life threatening and possibly avoidable
• Meanwhile, Inés had to cope with the worry, take numerous days off work and leave behind her 3 year-old son with his grandmother
Slide courtesy of A. Daly, EURORDIS
Patient‘s organisations have unique insights in “real life“ and “real needs“ of patients:
Clinical Research DesignPriority SettingResearch Policy
Building a new environment for health care and research
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In a changing system - the two loops theory
Trailblazers
Protectors
Firefighters
Illuminators
Platform
IPPOSI? Who?IPPOSI
A patient-led organisation
that works with patients, government, industry,
& science to put patients at the heart of health
innovation
“Turning Patients into
Partners”
IPPOSI Members (examples)
100+ Patient Organisations
100+ Scientific/Regulatory/Agencies
(200+ individuals)
20 Industry members
Partnership ApproachSc
ience
Industry
Other Stakeholders
Patient Experts
• Encourage open, constructive dialogue and interaction
• Highlight value that patients can bring
• Develop new opportunities for patients to influence decision-making
Patient-led activities
Health HacksWorkshopsConferencesConsultationsRound-tablesWorking GroupsTraining Days
Rare DiseaseseHealthClinical ResearchHealth InformationHealth EconomicsPatient RegistriesBiobanking
Rare Diseases in Europe
• Rare diseases in EU = 5 in 10,000
• 6-7,000 rare diseases found (Orphanet)
• Up to 6% - 8% of EU population affected at one point in life = 30 million people in the EU are affected or will be affected by a Rare Disease
• Republic of Ireland = 157,000 have a Rare Disease • 200,000 in the population yet to be diagnosed
Rare Diseases in European policy
Early 2000s• Rare Diseases not a recognised as a public health priority and there
were no public health policies for RD
2017• EU regulations on medicines: orphan, paediatric, advanced therapies,
(Regulation EC NO 141/2000)• EU Health Strategy (2008-2013): Rare Diseases = a strategic objective• EU Research FrameWork Programme (2007- 2013): RD = a priority• National Plans on Rare Diseases
EU Council Recommendation on Action in the Field of Rare Diseases (June 2009)
• This recommendation is important because it calls for concerted action at EU and national level in order to:
• Ensure that rare diseases are adequately coded and classified
• Enhance research in the field of rare diseases
• Identify Centres of Expertise by the end of 2013 and foster their participation into European Reference Networks
• Support the pooling of expertise at European level
• Share assessments on the clinical added value of orphan drugs
• Foster patient empowerment by involving patients and their representatives at all stages of the decision making process
• Ensure the sustainability of infrastructures developed for rare diseases
EU Committee of Experts on Rare Diseases ‘EUCERD’
• Multi-stakeholder• Provided an opportunity to enhance the partnership
between all rare disease stakeholders - patient representatives, academic experts, industry, national and European decision makers
• Comprehensive• Provided the opportunity to discuss, promote and guide EU
policy in all main fields of action relevant for rare diseases
Rare Diseases in Ireland
• National Plan – Ireland 2014-2018 (published May 2015)
• Patient Involvement – key to success • Member of the steering committee to develop the plan
• IPPOSI Head of subgroup on Orphan Drugs• Member of the plan Implementation Oversight Committee
• June 2015• Launch of the National Rare Disease Office – Mater Hospital
www.rarediseases.ie
Centres of ExpertiseEUROPEAN REFERENCE NETWORKS
ADDRESSING THE NEED
Integrated action and collaboration
The traditional short duration of contracts hampers the sustainability of research
• Better coordination and better optimisation of research activities, including standards and tools, and research funding
• Reinforced public funding both at the EU and national levels• In this framework, the potential of ERNs to embed and boost research activities should be
exploited
The lack of coordination in initiatives, tools and standards causes a waste of limited resources
To better coordinate both policy and funding within a strategic framework
To ensure the long-term continuity of research and limit the loss of precious funds
Slide courtesy of A. Daly, EURORDIS
HOW PATIENTS CAN DELIVER
Full involvement of expert & empowered patients as actors of research
• co-funding, leveraging & private-public partnerships
• identification of unmet research needs
• gap analysis
• definition of the research strategy
• clinical trial design
• identification of participants for clinical trials
• source of data
Slide courtesy of A. Daly, EURORDIS
WHERE THERE ARE CHALLENGES THERE ARE OPPORTUNITIES
• Science has taken enormous steps over the last 10-15 years scientific and technological advances, mostly but not only limited to genetics
Opportunity to address societal and ethical questions that these advances raise, not least of all how to address the deepening of inequalities across countries & regions in access to results of scientific research, i.e. improved care & therapies
• Cooperation in the field of research can bring about significant results as shown by voluntary initiatives such as E-Rare (funding), and IRDiRC
We need nhanced and structured cooperation in research
• Full integration of ERNs into national healthcare systems. Creation of the ERNs that will pool of experts, patients, researchers:
Will enable the creation of Clinical Research Networks for Rare Diseases strategically aligned with the ERNs
• Increasingly complex system in the field of rare disease research
Clustering training and capacity building activities aimed to patients but also to young scientists, clinicians etc.
Slide courtesy of A. Daly, EURORDIS
ERNs & Research Infrastructure
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Always between a patient and a doctor in a hospital Data sharing is the key to unlock the potential of ERNsOptimising the use of existing knowledgeGenerating new knowledgeAiming for clinical excellence and improved patients outcomes
Research Infrastructure
Clinical Research Networks
European Reference Networks
Slide courtesy of A. Daly, EURORDIS
Orphan Drugs
What we know….
• Irish health system is unique + requires an Irish solution
• 2014: 9 ODs assessed, majority eventually approved
• High budget impact = decisions take longer
• No funding process in place
What we don’t know…..
• What the final decisions are based on?
• How many ODs are currently available in Ireland?
• What reimbursement of ODs actually costs?
• How ODs are being prescribed/monitored?
IPPOSI views on Orphan Drugs (ODs)
• Separate assessment system with different criteria for assessing OD in Ireland
• Criteria should consider • where patient perspective can be incorporated• the cost to the state of not treating someone
• Transparency and Communication
Health Technology Assessment
Active Partnership with HIQA + NCPE• HIQA have patient involvement on the advisory
board of individual HTAs
• Annual HTA Training for patients through IPPOSI
• Acknowledgement that data provided by patient organisations is invaluable to NCPE assessments
• Irish patient groups exploring measuring patient outcomes with the NCPE
European Patients Academy on Therapeutic Innovation (EUPATI) www.eupati.eu
Roles change as a result of EUPATI course- creating patient leaders Role Before EUPATI After
Member of patient organisation, not actively involved 17% 2%
Active role in a patient organisation 62% 71%
Leadership role in a patient organisation 62% 71%
Employee of a patient organisation 25% 23%
Volunteer role in a patient organisation 60% 67%
Presenting at conferences, workshops etc. 63% 83%
Advising a pharmaceutical company 13% 44%
Advising a regulatory agency 21% 42%
Advising a reimbursement agency 4% 8%
EUPATI Fellows are increasingly taking leadership roles and are engaging with pharma, regulators and HTA bodies.
Role changes also imply identity shifts.
Irish Patient Education Programme
Patient Narrative Project
Person-Centered, Coordinated Care
• A set of generic descriptors for what person-centred, coordinated care looks and feels like from the Irish service user perspective
• A definition for ‘person-centred coordinated care’ that is shared, understood and used by service users and staff at all levels of the health service in Ireland