UCD Rare Disease Module 2017 - Dr Derick Mitchell - March 28th 2017
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Medical students taking the elective course in rare diseases are provided a number of patient perspectives throughout the module. This is what IPPOSI presented in 2017.
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UCD Rare Disease Module 2017 - Dr Derick Mitchell - March 28th 2017
- 1. PATIENTS â SCIENCE â INDUSTRY UCD Rare Disease Module, March 28th, 2017 Derick Mitchell, PhD
- 2. Overview ⢠Context ⢠Environment IPPOSI exists in / was created to address ⢠IPPOSI â aims/objectives ⢠Rare diseases in Ireland & Europe ⢠Centres of Expertise â European Reference Networks ⢠Orphan drugs & technologies ⢠Your feedback
- 3. May you live in changing timesâŚ.
- 4. Claraâs 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, InĂŠs, Claraâs 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, InĂŠs 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
- 5. Patientâs organisations have unique insights in âreal lifeâ and âreal needsâ of patients: Clinical Research Design Priority Setting Research Policy Building a new environment for health care and research 5
- 6. In a changing system - the two loops theory Trailblazers Protectors Firefighters Illuminators Platform
- 8. IPPOSI? Who?IPPOSI A patient-led organisation that works with patients, government, industry, & science to put patients at the heart of health innovation
- 9. IPPOSI Members (examples) 100+ Patient Organisations 100+ Scientific/Regulatory/Agencies (200+ individuals) 20 Industry members
- 10. Partnership Approach 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
- 11. Patient-led activities Health Hacks Workshops Conferences Consultations Round-tables Working Groups Training Days Rare Diseases eHealth Clinical Research Health Information Health Economics Patient Registries Biobanking
- 12. 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
- 13. 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
- 14. 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
- 15. EU Committee of Experts on Rare Diseases âEUCERDâ ⢠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
- 16. Rare Diseases in Ireland ⢠National Plan â Ireland 2014-2018 (published May 2015) ⢠Patient Involvement â 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 â Mater Hospital www.rarediseases.ie
- 17. Centres of Expertise EUROPEAN REFERENCE NETWORKS
- 18. 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
- 19. 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
- 20. 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 ď 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 ď 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: ď 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 ď Clustering training and capacity building activities aimed to patients but also to young scientists, clinicians etc. Slide courtesy of A. Daly, EURORDIS
- 21. ERNs & Research Infrastructure 21 Always between a patient and a doctor in a hospital Data sharing is the key to unlock the potential of ERNs Optimising the use of existing knowledge Generating new knowledge Aiming for clinical excellence and improved patients outcomes Research Infrastructure Clinical Research Networks European Reference Networks Slide courtesy of A. Daly, EURORDIS
- 22. Orphan Drugs
- 23. What we knowâŚ. ⢠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
- 24. What we donât knowâŚ.. ⢠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?
- 25. 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
- 27. 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
- 28. European Patients Academy on Therapeutic Innovation (EUPATI) www.eupati.eu
- 29. 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.
- 30. Irish Patient Education Programme
- 31. 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 âperson-centred coordinated careâ that is shared, understood and used by service users and staff at all levels of the health service in Ireland
Hinweis der Redaktion
- Training required to get the expertise required to contribute to research & development projects
- A patient-led platform that provides a structured way of facilitating interaction between patients, government, industry, science and academia to put patients at the heart of policy and medicines development. In pursuit of this we hold workshops, discussion groups, training days and conferences on policy, legislation and regulation around the development of new medicines, products, devices and diagnostics for unmet medical needs.
- Ireland behind in facilitating access
- Across all hospital settings HSE guidelines for prescribing? How are they being monitored to ensure patient safety and quality healthcare?
- Health Information and Quality Authority (HIQA) official HTA agency HIQA conduct HTAâs on surgical/diagnostic/therapeutic interventions The National Centre for Pharmacoeconomics (NCPE) is tasked by HIQA and the Health Service Executive to perform assessments on Pharmaceutical Products
- HIQA have patient involvement on the advisory board of the particular HTA that theyâre doing. And thatâs a good example of patient involvement right the way through. And one of the outcomes from that is, itâs great to talk to us. You know, when youâre at that point where the HTA is nearly complete and a decision has to be made on access by the government to bring patients in. Thatâs very positive. List of products under NCPE rapid review available on IPPOSI website A lot of people see payers and the regulatory system were barriers. Thatâs historically been the case where patients not being engaged, or communicted to have gone on joe Duffy. So therefore, the expert patient â the one living with the condition â is really important to get into the process. We think itâs important for them to understand what health technology is about. We think itâs important for them to gather evidence to back up their views. Youâve really got to understand the process and follow it most carefully. We believe that the more opportunities patients get to engage, the more understanding that they have about being part of a very difficult and complex process. And one with which patients should engage in. And the more they engaged in it, the more they understand why the processes and agencies were there and the challenges that they faced themselves.