The role of patients and healthcare providers in translational medicine, presented by Jan Geissler at the European Commission's Personalized Medicine Conference 2016 on 1 June 2016 in Brussels

1. Jan Geissler
Director EUPATI
Co-founder, CML Advocates Network
The role of patients and
healthcare providers in
translational medicine

2. Patients are excited about progress,
but getting there needs real change
 Molecular targets
 Genome sequencing
 Translational research
 Personalized medicine
 Small trial populations
 Companion diagnostics
 HTA, QoL, endpoints,
comparators
 Healthcare budgets
vs drug pricing
2
 New trial designs,
recruitment strat.,
licensing schemes,
drug/diagnostics
 New collaboration
models
 Patient involvement

3. 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, Paul et al (2010)

4. 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?
1 2 3 4
Iain Chalmers, Paul Glasziou, The Lancet, 15 June 2009, doi:10.1016/S0140-6736(09)60329-9

5. 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
Translational medicine needs multidisciplinary
approach of „patients involved“
Patients

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

7. 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
considerations
Health Technology
Assessment
Protocol
Synopsis
Research
priorities
Setting
research
priorities:
Information to
trial participants
Research conduct and
operations
Regulatory affairs
Dissemination,
communication,
post-approval
Source: Geissler, Ryll, Leto, Uhlenhopp
EPALCO/EUPATI (2015, unpublished)
Fundraising
for research
Ethics Review

8. Collaboration needs
cultural change,
empowered patients,
and education how to put
“involvement” into practice.

9. That’s why EUPATI is needed.
 Public Private Partnership
funded by IMI*
 5-year, patient-led project,
launched 2012
 Strong multi-stakeholder
consortium of patients’
organisations, academia,
NGOs and industry – 33
organisations
 The key pan-European
initiative to build
competencies & expert
capacity among patients
and the health-interested
public

10. No research about us
without us.
Jan Geissler
jan@patientsacademy.eu