Patient Advocates in Cancer Research: European Patients’ Perspective, presented by Jan Geissler (Twitter @jangeissler) at ISOQOL 19th Annual Conference, Budapast, 26 Oct 2012

1. Patient Advocates
in Cancer Research:
European Patients‟ Perspective
ISOQOL 19th ANNUAL CONFERENCE, BUDAPEST, 26 OCT 2012
JAN GEISSLER
Director, European Patients’ Academy on Therapeutic Innovation (EUPATI)
Co-founder, CML Advocates Network / Leukemia Patient Advocates Foundation
Secretary, European Forum For Good Clinical Practice
Member, EU Committee of Experts for Rare Diseases (EUCERD)

2. We„re excited about progress in
tackling cancer… but…
 Molecular targets/pathways New approaches
needed
 Genome sequencing
 Drug 
 Translational research Drug+Pathway
 Personalized medicine +Biomarker
• Small trial populations  New trial design
• Biomarkers and recruitment
 HTA, QoL, endpoints,  New collaboration
comparators models
 Healthcare budgets vs
 Patients in
drug pricing research
2

3. There is no „magic bullet“
for most patients yet
 “Success stories” available only to
small numbers of rare cancers
 Patients in urgent need of
therapeutic innovation
5-year survival in cancer (USA, 2008)
National Cancer Institute RareCare (Gatta et al, 2012)

4. Patients with rare cancers
face specific challenges
1. Prevention and screening mostly irrelevant
2. Late or incorrect diagnosis
3. Experienced doctor not available locally,
lack of access to clinical expertise
4. Lack of appropriate therapies
5. Slowness of research (lack
of trials & commercial interest),
6. Facing stigma and inequity
7. Lack of information & local
patient groups

5. In addition, public misperception of
clinical trials delay research
?
Lack of public confidence…
= delayed / lack of trial recruitment
= delayed generation of meaningful
data
= slow progress, lack of research
in Europe

6. What patients want to know
about clinical trials
 Knowing that trials exist at all
 Benefits of participation in trials:
• Personal benefit: Innovative treatment,
closer monitoring, better QoL,
chance for response, "last resort"
• Altruistic reasons: Achieving progress
beyond their own case

7. What patients need to know
about RISKS in clinical trials
 Medical risk of uncertain therapies
 Quality of life, unpleasant diagnostics
 Paediatric trials: administration
 Influence on family life (intensity of care)
 Financial impact (travel), ability to work (patient & carers)
 Fertility
 Protection from stigma and discrimination (use of data)
 Patient groups: the only stakeholder with the
big picture on QoL of a patient population

8. Doctor/patient perception on
impact on QoL differs
Detecting Myeloma, ways to shortening an often painful and tedious patient odyssey: results from an international
survey. Myeloma Euronet (2009). 314 physicians & nurses, 260 patients & carers, 43 countries

9. Doctor/patient perception on
management of side effects differs
Detecting Myeloma, ways to shortening an often painful and tedious patient odyssey: results from an international
survey. Myeloma Euronet (2009). 314 physicians & nurses, 260 patients & carers, 43 countries

10. Patients – as partners of
researchers
“ More needs to be done: rare cancers will never be a
priority unless the patients make it one. Patients
themselves must therefore play a larger role in
driving forward the search for therapies. They are „
able to see connections that have eluded scientists.

11. Collaborative cancer research:
More participation of patient groups
 Before research starts
• Influencing public health / research policy
• Identification of indications, therapies, patient population, gaps
• Uncovering ethical and risk/benefit dilemmas
• Assessing endpoints (PFS vs OS)
 While research is done
• Managing of expectations: hope or hype Driving force
• Patient recruitment, compliance Co-researcher
• Side effect monitoring
Reviewer
• Patient & public confidence in clinical research
Advisor
 After conclusion of research
Information provider
• Quality of life monitoring in “the field”
• Assessment of (cost-)effectiveness Research subject
• Improving compliance Source: PatientPartner FP7
Project (2010)

12. Opportunities improving
informed consent
 Patient-driven
optimization
of informed
consent
 ToC, Glossary,
study flow chart,
terminology,
video
EudraCT-Nr. 2010-024262-22, „TIGER“ Study in CML

13. CML Advocates Network:
Community-driven adherence
research
 Pilot survey completed in 2011,
phase II in Sept 2012 in
12 languages
Goal:
 CML treatment adherence
(intentional, non-intentional)
 Interaction with physician
 (Use of) adherence tools
 1000 patients in 3 weeks,
3000 until EOY2012

14. Patient-driven biobanking in cancer
• PATH - Patients Tumorbank of Hope
– Initiated by breast cancer patient group
MAMAZONE (DE)
– Samples stored for research and individual use
– Collected >5475 samples in 7 breast centers to date
• The GIST Collaborative Tissue Bank
– Initiated by the Life Raft Group (USA)
– Patients receive mutational testing
on the Primary Mutation

15. Self-reported QoL data accelerating
clinical discovery
 “the first real-time, real-
world, open and non-
blinded, patient-driven trial”
 …"Although observational
studies using un-blinded data
are not a substitute for double-
blind randomized control trials,
this study reached the same
conclusion as subsequent
randomized trials”
 …”Accelerating clinical
discovery and evaluating the
effectiveness of drugs already
in use."

16. Unmet need of patient and public on
information on medicines R&D
 Patients…
• seek up-to-date, credible, understandable information
about innovation in treatments
• are largely unaware about clinical trials, translational
research, personalized medicine, pharmaco-economics,
their key role
 Patient advocates…
• like to advise on protocol design, informed consent,
ethical review, marketing authorization, value
assessment, health policy
• lack the education and training required to participate
as a partner in medicines R&D

17. EUPATI: Empowering
patients on medical R&D
 Launched Feb 2012, runs for 5 years,
29 consortium members,
PPP of EU Commission and EFPIA
 will develop and provide, objective, credible,
correct, up-to-date knowledge
about medicines R&D
 will build competencies
& expert capacity among patients & public
 will facilitate patient involvement in R&D to support
industry, academia, authorities and ethics committees

18. Areas covered by EUPATI
1. Medicines development process
from research to approval
2. Personalized and predictive medicine
3. Drug safety and risk/benefit assessment
of medicines
4. Pharmaco-economics, health economics and health
technology assessment
5. Design and objectives of clinical trials
(& roles of stakeholders) …and NOT:
develop indication-
6. Patients roles & responsibilities in or therapy-specific
medicines development information!

19. Audiences: advocacy leaders
and the public at large
100
EUPATI Certificate patient
Training Programme advocates
English
French
12.000 German
EUPATI Educational patient Spanish
Toolbox advocates Polish
Italian
Russian
EUPATI 100.000
Internet Library individuals

20. Summary
 Key goal of research:
To get better answers to cancer patients more quickly.
 Patient participation makes research more successful
• make design of trials patient-centric
• communication of trials with patients,
improving "informed consent"
• improve quality of life assessment
 Training of patient groups to foster cooperation with
clinicians, cancer researchers, authorities, industry is
essential!

21. Get to know us!
Web:
www.patientsacademy.eu
Jan Geissler
EUPATI Director
Twitter: @eupatients
jan@patientsacademy.eu as well as: