Florence Paterson, Ecole des Mines, France

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 l’innovation,
UMR CNRS 7185 - Mines-ParisTech
Paris, France

2. 2010 Survey on Patient
Organisations and Research
results from a joint collaboration between
Fabrizia Bignami
EURORDIS Paloma Tejada
François Faurisson
Julia Fitzgerald
and the
Anja Helm
Madeleine Akrich
Centre de sociologie Vololona Rabeharisoa
de l’innovation Florence Paterson
Frédéric Vergnaud
Michel Callon

3. A few words on the works of the Centre de
sociologie de l’innovation
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 patient’s 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
2/3 of (non umbrella) POs
have less than 500 members
Number of members
Building its membership:
100 a long-term process
80 •44% of the POs
60 created before 1970
40 33% have more than 3000
21% members.
20 15% 13% 13%
6%
0 •31% of the POs
# POs created between 2000
50
9
0
9
9
9
-9
49
99
00
99
<
50
-2
>3
0-
0-
and 2009 have 50 to
10
50
00
10
150 members.

10. High diversity in POs’ annual budgets
25 000 €
3 000 € 100 000 €
0€
¼ of POs have less ¼ of POs have more
than 3 000 € than 100 000 €
10% less than 300 €
5% more than 1 200 000 €
6% no budget

11. Diseases and clinical domains
110 diseases from various clinical domains
(1.3 million patients specifically represented)
Multisystemic Dermatology
Neurology n=30
n=38 n=23
Musculoskeletal Ophthalmology Metabolic
n=22 n=12 n=13
Neuromuscular Oncology Cardiovascular
n=27 n=12 n=14
Haematology Others Umbrella
n=10 n=59 n=15
15 Umbrella organisations from 10 countries
(20 million patients represented)

12. Integration of external scientific competences
within the POs structure
56% of the POs have a
Danemark 22
Scientific Committee (174
Sweden 36 out of 309)
NL 44 A very common device
Spain 46 within French POs
Belgium 47 Greater proportion among
older POs: 80% of the POs
Ireland 50
created before 1980 vs.
UK 59 60% after 1980
Italy 60
45% of the Scientific
Germany 69 Committees contribute to
the allocation of financial
France 90
resources for research
0 20 40 60 80 100

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
54% Epidemiology / Natural history of the disease
46% Human and Social Science
24% Assistance technologies / Daily life
19% Research infrastructures

15. Types of financial support
(37% of the Pos)
77% Initiating and financing a specific research project
75% Co-financing the operating budget of a specific research project
54% Financing the acquisition of a specific research equipment (centrifuge,
computer, etc)
47% Financing a fellowship for a young researcher
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
% of POs % of POs
POs’ annual total
funding Date of creation funding
budget
research research
Budget > 300.000 € 81% Before 1970 70%
Budget between Between 1970 - 1979 61%
50% - 56%
30.000 to 300.000 €
Between 1980-1989 47%
Budget between
32% - 33%
5000 to 30.000 €
Between 1990 -1999 35%
Budget < 5000 € 11% -14% Between 2000-2009 30%

17. POs’ support to research
Non-financial support to research
76% Actions aiming at creating links between patients, researchers and physicians
57% Helping to identify patients to participate in clinical trials
49% Providing information and counseling for potential participants in clinical trials
48% Defining research projects by highlighting patients' needs and expectations
45% Collaboration in clinical trials design
30% Participation in scientific committees within institutions
28% Launching campaigns for the collection of biological samples from patients
Financial support to research: 37%

18. POs’ experience of
collaboration
with the research
community

19. POs’ experience of collaboration
with researchers
Relationship with research
15 12 34 19 5
teams (Close or distant)
NA
Quality of research results ++
22 13 38 71
(Encouraging or not) +
-
Information on research --
15 16 33 13 7
results (Satisfying or poor)
0 20 40 60 80 100

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
Therapeutics 49
Diagnosis 40
Basic research 35 Strong priority
44 Absolute priority
Epidem iology
Hum an and social sciences 43
Assistive technologies 36
Infrastructures 36
0 10 20 30 40 50 60

23. Obstacles to the advance of
research
Lack of recognition of your disease 21 19 13 11
Lack of multidisciplinary research 18 25 15 12
Lack of coordination between specialists major
23 28 12 10
(researchers and clinicians) important
minor
Difficulties to identify patients 21 32 17 12
none
Low number of clinicians specialised in your
39 29 9 5
disease/s
Low number of researchers working on
37 30 9 7
your disease/s
0% 20% 40% 60% 80% 100%

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.