Kaisa Immonen EPF Director of Policy CIOMS Working Group XI: Patient involvement in the development and safe use of medicines Kaisa Immonen EPF Director of Policy @eupatientsforum Geneva, 20 April 2018

Patients as partners = meeting unmet needs Need to involve patients throughout the “chain” Innovation on “what matters most” for patients Patients’ priorities -> co-designed research, regulation, HTA, pricing/reimbursement, RWE Right priorities / needs Maximum impact & added value unique value of patient perspective – particularly when it comes to identifying needs, and added (therapeutic) value – only patients know which outcomes matter most to patients Patients focus on issues that researchers, companies or regulators may not identify as important Patients’ priorities for research are often different – better alignment of innovation with real needs contributes to quality of life and health outcomes – and value for health systems! Broader/different perspective (patients’ experiential knowledge, “patient expertise”) Involve patients throughout the “chain”: from priority-setting for research , to co-designing and participating in the assessment of clinical trials; to Cost-benefit and effectiveness evaluation (through HTA), to Pricing & reimbursement decisions . BUT currently it often comes TOO LATE – need to move “upstream” – whatever the activity Involvement also leads to better quality research results Strengthen the trust and acceptance of research Produce results that can be used to effectively improve practice in health care for the benefit of the patients. Alignment of innovation with needs Strengthened trust, increased take-up “Upstreaming” for greater impact

PE Mainstreaming patient involvement HTA Unmet needs R&D EDs Clinical development Regulatory HTA Post-market RWE PhV …. and in the wider healthcare system: Outcomes measurement, “performance” assessment Service evaluation & co- design Self-care support, peer support, community engagement Capacity-building and patient training Advising on good practice …

Barriers to meaningful involvement Lack of recognition of unique value Agendas driven by other interests Asymmetric power relations Lack of understanding of “good” PI Lack of common, recognised methodologies for PI Lack of (perceived) capacity on patients’ part Lack of resources of patient organisations Perceptions of bias

Interests… everyone has them Patient groups have legitimate relations with industry This does not mean that patients organisations and industry always share the same agenda Everyone has biases – including academic researchers, commercial sponsors, public health experts/consumer representatives, regulators, governments... 100% public funding =/= lack of any conflict of interest Patient representatives can and do wear many “hats” Increasing professionalism and maturity in patient movement in the last years: Codes of ethics, principles for conduct & collaboration Transparency as a fundamental principle Diversification of funding, good financial management & governance

How to manage diverse interests No funding is ever free of potential “strings” – and no patient organisation can operate on “pure, clean air” Recognising => managing Patient community recognises this: Codes of ethics, explicit principles for collaboration Transparency is fundamental, diversification of funding, good governance Individual patient experts should be treated like other experts No “representative”/”independent” patients = no patient perspective = no problem? EPF: Code of Ethics & Framework for Cooperation with industry + a diversified funding base, unrestricted funding + strong commitment to transparency and independence EPF transparency guidelines for Patient organisations (2018)

EPF’s engagement We channel our members’ expertise & connect patient organisations and other stakeholders We advise on meaningful patient involvement: EMA, WHO, OECD, HCP organisations… Two parallel initiatives to develop “patient-led framework on good practice” by 2021: Across Health system: service co-design / evaluation, health research, patient- driven technology solutions Criteria, guidance, mapping of GPs Patient involvement in pharmaceutical R&D – through IMI PARADIGM

Lessons learnt from EPF “Value+” project Fully patient-led project (2008-9) developed a framework for “meaningful” patient involvement & practical tools “Meaningful” = recognising patients’ specific expertise & valuing it Involvement must be planned, resourced & evaluated Quality of the experiences of all parties during the activity should be evaluated  learning & improvement

EMA definition of "patient organisation" Patients’ organisations are defined as not-for profit organisations which are patient focused, and whereby patients and/or informal carers (the latter when patients are unable to represent themselves) represent a majority of members in governing bodies These could be: general umbrella organisations (e.g. representing either European specific disease organisations and/or national umbrella organisations) European disease specific organisations (i.e. representing national organisations or individual patients on acute and/or chronic diseases).

The Value+ model Meaningful Patient Involvement = patients take an active role in activities or decisions that will have consequences for the patient community, because of their specific knowledge and relevant experience as patients. The involvement must be planned, appropriately resourced, carried out, and evaluated from the perspectives of: The participating patients or patient organisations Other participating organisations and funding bodies The quality of their experiences during the involvement process The opposite of tokenism Value+ Toolkit and Handbook offer guidance how to realise this