Jan Geissler, EUPATI: Presentation at workshop "Patient involvement in medicines development - paradigm shift towards true patient impact in regulatory science?" at the University of Copenhagen on 6 May 2015

1. Setting the scene, current
status and future plans:
a Patient Perspective
Jan Geissler
Copenhagen, 6 May 2015
Director, European Patients‘ Academy (EUPATI)
Vice President, Leukemia Patient Advocates Foundation
jan@patientsacademy.eu, Twitter @jangeissler

2. Patients need innovation –
Medical landscape is changing at a fast pace
2
 Molecular targets/pathways
 Genome sequencing
 Translational research
 Personalized medicine
• Small trial populations
• Biomarkers, companion diagnostics
 Need for post-marketing data
 Health Technology Assessment,
QoL, endpoints, comparators
 BUT long term pressure on health
budgets – here to stay…
Window of
opportunity
New
collaboration
models

3. We need more „magic bullets“:
Not yet there for most patients
 We are excited about what happens in
CML, Melanoma, NSC Lung Cancer, …
 “Success stories” only for small
populations and “best ages” yet
 Patients need both
incremental
& breakthrough
innovation
RareCare (Gatta et al, 2012)

4. How to address >200 cancers, >5.200 rare diseases
in times of decreasing medical R&D productivity?
Structure
of DNA
Restriction
enzymes
DNA
sequencing
Recombinant
DNA
Human
insulin
Dolly
the sheep
Human
genome v1milestones
Source: Nature – How to improve R&D productivity: the pharmaceutical industry's grand challenge

5. Over 30% of trial
interventions not
sufficiently described
Over 50% of planned
study outcomes not
reported
Most new research not
interpreted in the
context of systematic
assessment of other
relevant evidence
Unbiased and
usable report?
85% research waste = over $85 billion / year
“Avoidable waste in the production and reporting
of research evidence”
Low priority
questions
addressed
Important outcomes
not assessed
Clinicians and
patients not
involved in setting
research agendas
Questions
relevant
to clinicians &
patients?
Over 50% studies
designed without
reference to
systematic reviews of
existing evidence
Over 50% of studies fail
to take adequate steps
to reduce biases, e.g.
unconcealed treatment
allocation
Appropriate
design and
methods?
Over 50% of studies
never published in full
Biased under-reporting
of studies with
disappointing results
Accessible
full publication?
Iain Chalmers, Paul Glasziou, The Lancet, 15 June 2009, doi:10.1016/S0140-6736(09)60329-9

6. Patients have a unique perspective on symptoms and
side effects – may differ from HCP’s
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

7. “We need to do the right things,
not just doing things right.”
Bettina Ryll,
European Melanoma Patient
Advocate, 28 Feb 2014
http://is.gd/bettinaryll

8. Early involvement may create highest impact,
but involvement today is mostly at late phase
Courtesy of B. Ryll (2014)

9. Practical “Roadmap” on patient involvement in R&D:
Research design
and Planning
Design of Protocol:
Informed Consent Study reporting
Post-study
communication
Patient Info
Leaflet
Trial steering committee
Investigators Meeting:
Level of expertise in the disease area required:
mediumhigh
Data Monitoring CommitteePractical
con-
siderations
Health Technology
Assessment
Protocol
Synopsis:
design,
comparators
Research
priorities
Setting
research
priorities:
Fundraising
Information to
trial participants
Research conduct and
operations
Source: Geissler, Ryll,
EPALCO (2014, unpublished)
Regulatory affairs
Dissemination,
communication,
post-approval

10. Public
Research Ethics
Committees
Competent
authorities
Policy makers
/Research Policy
HTA agencies
& committees
Research subject
Info provider
Advisor
Reviewer
Co-researcher
Driving force
Clinical
Research
Patients have a key role in all aspects of
health-related research
Trial protocol design,
informed consent, ethical
review, marketing
authorization, value
assessment, health policy

11. Education is key to increase the number
of empowered patient advocates in R&D
 Launched Feb ’12, runs for 5 years,
30 consortium members,
Funded by Innovative Medicines Initiative
 Patient-led public private partnership of
patients, academia, NGOs and industry
 will build competencies
& expert capacity to facilitate patient
involvement in R&D to collaborate in
academic research, industry research,
authorities and ethics committees
 First 50 patients in training, 50 more next
year, educational material in 7 languages
rolled out end of 2015 www.patientsacademy.eu

12. No research about us
without us!
Jan Geißler
jan@patientsacademy.eu
Twitter @jangeissler