This document discusses the benefits of patient involvement in health research and development. It notes that innovation is transforming lives but more breakthroughs are still needed. Patients can provide unique perspectives to improve trial design and address unmet needs. However, public distrust and lack of knowledge limit patient participation in research. The document advocates for greater patient involvement at all stages of research, from setting priorities to disseminating results. It highlights the EUPATI initiative which aims to educate patients and the public about medicines research through training courses, educational tools, and national platforms. The goal is empowering patients as partners in research.

1. Jan Geissler
Director EUPATI
Co-founder, CML Advocates Network
The benefits of patient
involvement in research
and development

2. Health research & policy is changing
at a fast pace
Innovation transforms the lives of
patients with serious, lifelong conditions:
 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
 trial design
 relationship
between
researchers,
regulators,
industry,
patients
2

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”
Iain Chalmers, Paul Glasziou, The Lancet, 15 June 2009, doi:10.1016/S0140-6736(09)60329-9
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

6. Patients have a unique perspective.
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. There is public distrust and a lack of
knowledge about research in the lay public
 Only 6-12% of cancer patients
participate in clinical studies
 75% of Phase II-IV studies delayed due to slow
patient recruitment
 Bad image one reason for delayed generation of
meaningful clinical data

8. Patients as partners of research:
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.
“
„

9. Fantastic examples of patient
involvement in R&D already today

10. Patients' organisations key role in
building a new environment for the
development of new medicines
 Patient‘s organisations have unique insights in
„real life“ and „real needs“ of patients:
• Gap analysis in research priorities
• Clinical trial design
• Priority setting
• Research policy
 Training required to get expertise required to
contribute to medicines research & development
(R&D) projects
Research subject
Info provider
Advisor
Reviewer
Co-researcher
Driving force
Source: PatientPartner FP7
Project (2010) 10

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

12. 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

13. 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

14. What is EUPATI?
 A Public Private Partnership within the
Innovative Medicines Initiative Joint
Undertaking*
 A 5-year project, launched in February 2012
 A patient-led project coordinated by the
European Patients’ Forum, with EGAN,
EURORDIS and EATG in key roles
 A 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
* Resources are composed of financial contribution from the European Union's Seventh Framework Programme and in-kind
and financial contributions from EFPIA companies

15. EUPATI empowers patients with education
in key areas of medicines R&D
Educate and train patients and patient advocates with objective, credible,
correct and up-to-date information about:
1. Discovery of Medicines & Planning of
Medicine Development
2. Non-Clinical Testing and Pharmaceutical Development
3. Exploratory and Confirmatory
Clinical Development
4. Clinical Trials
5. Regulatory Affairs, Medicinal Product Safety, Pharmacovigilance and
Pharmaco-epidemiology
6. HTA principles and practices

16. EUPATI Patient Experts
Training Course
-- for expert patients
EUPATI is developing education targeted
at different levels
100
patient
experts
12.000
patient
advocates
100.000
individuals
EUPATI Educational
Toolbox
-- for patient advocates
EUPATI
Internet Library
-- for the health-interested public
English
French
German
Spanish
Polish
Italian
Russian
English

17. EUPATI’s Patient Experts Training Course:
Course 1 in final phase, course 2 kicked off
 Open to patients, carers,
patient advocates and
volunteers
 ~200 fully qualified
applications per course,
 Two course cycles
• Oct 2014 - Nov 2015
(53 trainees)
• Oct 2015 - Nov 2016 (60
trainees)
 Participants from 28
countries and 32 disease
areas

18. EUPATI Course 2 trainees by disease area
0 1 2 3 4 5 6
Cancer
Rheumatic diseases/Arthritis
Rare diseases
Multiple Sclerosis
Leukemia
Diabetes
Haemophilia/hematology
Endometriosis
Hepatitis C/HIV/STDs
Myeloma
Melanoma
Lupus
Parkinson's Disease
Achondroplasia
Hereditary haemorrhagic telangiectasia (hht)
Allergy and airways diseases
Gaucher Disease
Sight loss
Asperger´s syndrome/Fibromyalge and Chronic Fatigue Syndrome
Lung conditions
Adrenoleukodystrophy
Coeliacy
Myelodysplastic Syndromes
Incontinence
Cystic Fibrosis
Rett Syndrome
Neurofybromatosis
Muscular Dystrophy
Genetic Disorders
Disability
Epidermolysis bullosa (EB)
Mental Health

19. The EUPATI Advocate Toolbox
in 7 languages at www.eupati.eu
19
Fact sheets, detailed
papers, PPTs,
videos, illustrations
in English, French,
Italian, Spanish,
German, Polish,
Russian

20. The Patients’ Academy: Patient Advocate Toolbox
– Formats www.eupati.eu
Infographics
Articles
PowerPoints
Fact sheets

21.  What we bring in and what we produce
will be owned by the public
 EUPATI material will be provided under the
"Creative Commons License"
 Content can be copied, distributed, edited, remixed, and
built upon, for non-commercial use
 Authorship and licensing needs to stay intact and
mentioned on all derivatives
See http://creativecommons.org/licenses/by/3.0/
Public license model guarantees
ownership and re-use by advocates

22. EUPATI National Platforms
The EUPATI National Platforms...
 ensure EUPATI understands educational needs in R&D
on the national level
 disseminate EUPATI’s existing training material and
information on the national level
 raise public interest about EUPATI in 12 countries
 identify training faculty, logistics and financial support
on the national level
UK IE CH ES FR IT LU MT AT DE PL BE
(+ SK, DK, …)

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