1. Results of a survey on the role of Patient Groups in Research and their priorities for the future Florence Paterson Centre de sociologie de linnovation, UMR CNRS 7185 - Mines-ParisTech Paris, France

2. 2010 Survey on Patient Organisations and Research

• EURORDIS

• • and the

• • Centre de sociologie de linnovation

• Fabrizia Bignami

• Paloma Tejada

• Franois Faurisson

• Julia Fitzgerald

• Anja Helm

• Madeleine Akrich

• Vololona Rabeharisoa

• Florence Paterson

• Frdric Vergnaud

• Michel Callon

results from a joint collaboration between

3. A few words on the works of the Centre de sociologie de linnovation

• Contribution to Science and Technology Studies

• • understanding the rise of technical democracy

• Forms of participation of civil society organizations in scientific and technical activities and debates

• impact on the modes of production of knowledge

• and on scientific practices

• upshot on the emergence and the increase of collective mobilizations around science issues

4. Research questions

• Lay knowledge and expertise

• POs expertise: to what extend is the claim to hold an expertise based on the mobilization of experiential knowledge coupled with the acquisition of certified knowledge?

• In what form do patients collectives mobilize experiential knowledge & certified knowledge: alignment, opposition or conjunction?

• The role of knowledge in the governance of medical and health policies, and the contribution of patient organizations

• Mechanisms through which POs voice their concerns in health-policy making

• The ways POs promote their expertise capacity in health policy-making

5. Aims of the Survey

• To evaluate POs support to research: in what ways and to what extent

• To learn about POs experience of collaboration with researchers

• To collect POs opinion on priorities and obstacles for rare disease research

6. Survey population

• Questionnaire sent to 772 POs

• 309 responses from 29 European countries

• (178 member + 131 non member POs)

• = 40% response rate

7. General characteristics of participating POs

• Age of the POs

• Financial resources

• Diseases and clinical domains concerned

• Existence of internal Scientific Committees

8. Rare diseases POs: newly created organisations Number of years active

• • 37% exist for 10 to 15 years

• • 22% exist for less than 5 years

9. Membership Number of members 2/3 of (non umbrella) POs have less than 500 members Building its membership: a long-term process

• • 44% of the POs created before 1970 have more than 3000 members .

• • 31% of the POs created between 2000 and 2009 have 50 to 150 members .

10. High diversity in POs annual budgets

• of POs have less than 3 000

• 10% less than 300

• 6% no budget

of POs have more than 100 000 5% more than 1 200 000 100 000 3 000 25 000 0

11. Diseases and clinical domains

• 110 diseases from various clinical domains

• (1.3 million patients specifically represented)

15 Umbrella organisations from 10 countries (20 million patients represented) Neurology n=30 Ophthalmology n= 12 Oncology n=12 Others n=59 Dermatology n=23 Metabolic n=13 Cardiovascular n=14 Umbrella n=15 Multisystemic n=38 Musculoskeletal n=22 Neuromuscular n=27 Haematology n=10

12. Integration of external scientific competences within the POs structure

• 56% of the POs have a Scientific Committee (174 out of 309)

• A very common device within French POs

• Greater proportion among older POs: 80% of the POs created before 1980 vs. 60% after 1980

• 45% of the Scientific Committees contribute to the allocation of financial resources for research

13. POs support to Research

• 37 % of POs fund research

• but

• POs non-financial support to research, less visible, provides specific resources

14. Areas of research funded 81 % Basic research 57% Therapeutics 56% Diagnosis 46% Human and Social Science 19% Research infrastructures 54% Epidemiology / Natural history of the disease 24% Assistance technologies / Daily life

15. Types of financial support (37% of the Pos) 47% Financing a fellowship for a young researcher 54% Financing the acquisition of a specific research equipment (centrifuge, computer, etc) 77% Initiating and financing a specific research project 75% Co-financing the operating budget of a specific research project 39% Co-financing meetings of researchers / clinicians 30% Co-financing training of researchers / clinicians

16. Financial support to research is linked to the POs budgets and age POs annual total budget % of POs funding research Budget > 300.000 81% Budget between 30.000 to 300.000 50% - 56% Budget between 5000 to 30.000 32% - 33% Budget < 5000 11% -14% Date of creation % of POs funding research Before 1970 70% Between 1970 - 1979 61% Between 1980-1989 47% Between 1990 -1999 35% Between 2000-2009 30%

17. POs support to research 76% Actions aiming at creating links between patients, researchers and physicians 49% Providing information and counseling for potential participants in clinical trials 57% Helping to identify patients to participate in clinical trials 30% Participation in scientific committees within institutions 45% Collaboration in clinical trials design 48% Defining research projects by highlighting patients' needs and expectations 28% Launching campaigns for the collection of biological samples from patients

• Non-financial support to research

• Financial support to research: 37%

18. POs experience of collaboration with the research community

19. POs experience of collaboration with researchers

20. Priorities and obstacles to the development of research

21. Advances observed by the POs in different research areas (5 last years)

• Major vs. important advances

• Basic research 15% vs. 27%

• Diagnosis 13% vs. 24%

• Minor advances

• Human and social sciences 40%

• Assistive technologies 38%

• Epidemiology 38%

22. Degree of priority that should be given when allocating public funds & research areas

23. Obstacles to the advance of research

24. Conclusion

• • POs efforts for creating collectives of researchers, clinicians & POs around the specific diseases they are concerned with appears as being for them a priority action in the support / development of research.

• • POs support to research concern a large range of research areas (basic, therapeutic, social and human sciences).

• • Although financial support to research is the more visible form of support, a large part of their involvement consist in activities providing non-financial support to research.

• • POs have limited budgets, but rare disease research benefits from their specific resources: contacts and trust between members & their expertise on the diseases they are concerned with.