1. 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

2. 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

3. 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 …

4. 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

5. 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

6. 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)

7. 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

8. 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

10. 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).

11. 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