European Patients Academy:

Outline & Status Update

22 Nov 2016

The project is receiving support from the Innovative Medicines Initiative Joint Undertaking under grant agreement n 115334, resources of which are composed
of financial contribution from the European Union's Seventh Framework Programme (FP7/2007-2013) and EFPIA companies.
Outline of the presentation

Why EUPATI?

What is EUPATI?

EUPATI Progress Update

Why EUPATI?
Health research & policy is changing
at a fast pace
Innovation transforms the lives of
patients with serious, lifelong conditions:

Molecular targets/pathways Window of

Genome sequencing, opportunity
Translational research trial design
Personalized medicine relationship
Small trial populations between
Biomarkers, companion diagnostics researchers,
Need for post-marketing data regulators,
Health Technology Assessment, industry,
QoL, endpoints, comparators
patients
BUT long term pressure on health budgets
here to stay
4
Patients as partners: partnership model
requires a paradigm shift, and more
training for patients and advocates

Patient roles in
Medicines R&D
(academia + industry)

Driving force

Co-researcher
Competent Authorities Policy
Makers/Research
Reviewer Policy

Advisor

Info provider

Research subject

HTA Research Ethics

Source: PatientPartner Agencies/Committees Committees
FP7 Project (2010)

5
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
Patients have a key role in all aspects of
health-related research

Competent
authorities
Policy makers
Trial protocol design, /Research Policy
informed consent, ethical
review, marketing
authorization, value Driving force
Public assessment, health policy
Co-researcher

Reviewer

Advisor

Info provider

Research subject

Clinical
Research Ethics HTA agencies Research
Committees & committees
EUPATI is needed because

Patients
seek up-to-date, credible, understandable information about
innovation in treatments
are largely unaware about clinical trials, translational research,
personalized medicine, pharmaco-economics etc and their key role

Patient advocates
have an increasingly complex and professional task of advising on
protocol design, informed consent, ethical review, marketing
authorization, value assessment, health policy
are often self-taught and have gaps in the education and training
required to participate as an equal partner in medicines R&D

Prior to EUPATI the FP7-funded PatientPartner project demonstrated

a clear need & willingness of patient advocates to be an active
partner in medicines research and development
What is EUPATI?
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
The EUPATI objectives are directly
contributing to this paradigm shift
Key objectives:

1. Develop and disseminate objective, credible, correct and

up-to-date public knowledge about medicines R&D

2. Build competencies & expert capacity among patients &

public

3. Facilitate patient involvement in R&D to collaborate in

academic research, industry research, authorities and ethics
committees

and NOT:
develop indication- or therapy-specific information!
EUPATI is developing education targeted
at different levels

100
EUPATI Patient Experts
Training Course
patient English
-- for expert patients experts

12.000
EUPATI Educational
patient
English
Toolbox
-- for patient advocates advocates French
German
Spanish
Polish
EUPATI 100.000 Italian
Internet Library individuals
-- for the health-interested public Russian
EUPATI is developing education targeted
at different levels

100
EUPATI Patient Experts e-learning
patient Classroom style face to
Training Course face teaching
-- for expert patients experts

Slide shows,
EUPATI Educational
12.000 factsheets, videos and
Toolbox patient webinars, glossary
-- for patient advocates advocates 'print ready' content

Encyclopaedia
Short video
documentaries
EUPATI 100.000 Patient interviews
Internet Library individuals micro-lectures (as
-- for the health-interested public podcasts)
Images, diagrams
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

+ Patients roles and

responsibilities
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
14
Reflecting European diversity:
7 languages, 12 countries

7 most frequently
12 countries:
spoken languages:
UK, Ireland, Malta, France,
English Luxemburg, the francophone
French Belgium, Germany, Austria,
Switzerland, Spain, Italy and Poland,
German plus Russian-speaking population in
Spanish Central and Eastern Europe

Polish Plus additional countries and

Italian languages in preparation by the
community, e.g. Danish,
Russian Romanian,
Truly patient-led: 4 major pan-European
patient associations in key roles

EUPATI Project Coordination

>50 umbrella patient organisations.

Additional
Linking national and regional patient partners in other
alliances patient
organisations
and "members
of members"
via
>600 rare disease organisations in "EUPATI
>45 countries Network"

>100 members in over 30 countries

16
Strong consortium
& strong governance

Coordinated by patients (EPF)

Leading pan-EU patient umbrella
groups involved in all key activities
Strong impetus from key academic partners
and research organisations
Industry expertise in medicines R&D

Advisory bodies & codes committed to

ensure independence and good
governance
EMA, Swissmedic, MHRA, BfArM, AIFA
Key experts in bioethics, genetics, HTA,
economics, evidence based med,
patient advocacy
 Strong governance in a patient-led,
collaborative public private partnership
Co-led by Coordinator is
patients patient advocate,
5 organisations
Regulatory Advisory
Panel Project Steering Committee
EMA, BfArM, AIFA, (Project coordinator, 1 representative of
Patient Swissmedic, MHRA each Consortium Partner)
co-chair, PO Innovative
members Project Medicines
Advisory Board Run by Initiative
Cochrane, academia, patient org Joint Undertaking
ethics, media, patients
50% Project Secretariat / WP1
patients EUPATI Director, Coordinator,
Ethics Panel Assistant, Communication Officer

WP2: Network WP3: Needs WP5: IT WP6:

WP4: Content WP7:
Implementation Assessment, Infrastructure Deployment &
Development Sustainability
Gap Analysis Quality Control

Patient-led WP co-led WP led

Patient by patients WP co-led
National by patients,
focuss groups +authors, by patients
Platforms user-testing
+reviewers

18
Uniqueness of the partnership

Unique and unprecedented partnership between patient

organisations, other public partners and pharmaceutical industry
based on the philosophy of the IMI
Unique in IMI that EUPATI is patient- and not industry-led
Opportunity to establish an effective, transparent and
credible partnership
Exceptional learning experience for industry representatives
to work with patient organisations in a constructive and
reflective manner, which could serve as a role model in other
environments as well
Making best use of industry expertise in medicines R&D,
incl. e.g. the legislative environment and development of ITP as
required by law

Opportunity to strengthen the voice of the patients in the

search for innovation and new medicines

Chance to reduce fear, lack of trust and misconception among

patients regarding the pharmaceutical industry
Public license model guarantees
ownership and re-use by the public

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,
all within the boundaries of copyright law on non-commercial basis
Authorship and licensing needs to stay intact and mentioned on all
derivatives
Similarly applied by e.g. Wikipedia, Google,
and many others

See http://creativecommons.org/licenses/by/3.0/
EUPATI Progress Update
 Project Plan

IMI Project EUPATI Programme

2017+
Preparation Phase Confirmation Phase Sustain Phase EUPATI
M1-18 M19-48 M49-60 Programme

WP1 Project Project Management, Communication, Coordination

Coordination Infrastructure Evaluation, Finances

Establishment Public conferences, Regional Workshops,

WP2
Network Implem. of EUPATI National platforms
Network EUPATI Network, EUPATI National Platforms

WP3 Needs Needs analysis, Quantitative survey,

Assessment & Review of material,
Gap An. focus groups Literature review

Editorial process, Content Refinemen

WP4 Content
Dev. Syllabus t, quality Content updates
Development, translation control

TODAY
WP5 Design, development and support of technical IT Platform
IT Infrastr.
infrastructure for eLearning/IT Platform
First Deployment, EUPATI Course #3,
WP6 Deploy- EUPATI IT
course dissemination,
ment & Quality
performe
Platform quality Toolbox, Capacity
Assurance launch assessmt building
d

Development of
WP7 Research on Patient
Best practice guideline dev.,
Recommendation of new teaching
code of conduct
Sustainability concept dev.
New technologies and EUPATI FUTURES
Sustainability Partnership models for patient future remit strategy
methods
involvement Team
Three years of EUPATI:
Much has been done!
For example events in Frankfurt 2012,
Barcelona 2013, Rome 2013, Warsaw 2014
EUPATI assessed patient & public
attitudes towards medicines R&D

Review of existing information resources;

230 resources reviewed.
First results published
Review of research literature; 12600 titles in BMJ Open:
and abstracts; 134 studies included

Public survey; 6931 responses in GB,

Spain, Poland, Italy, France and Germany
Patient advocate survey; 468 responses
across Europe
Patient advocate survey focused on
course delivery; 41 responses

Qualitative research with patients and the + 3 peer reviewed

public; n=125 people publications to come
Qualitative research with stakeholders;
n=56 (incl. policy makers, pharmaceutical
industry, clinical research professionals)
The EUPATI Network & (Social) Media
is growing

TOPRA article, Nature Medicine article

~1.200 EUPATI Network Members
>6.500 website visits per month
~1.300 Newsletter subscribers
>1.300 Twitter followers; >1.600 tweets
>900 Facebook friends
>550 LinkedIn members
Engage in
18+ EUPATI National Platforms
EUPATI National Platforms...
bring all stakeholders together in
countries
address educational needs in R&D
disseminate EUPATIs training
material to patient organisations

National platforms set up in AT, FR,

DE, IE, IT, LU, MT, PL, ES, CH, UK,
DK, SK, PT
Additional platform initiatives
ongoing in NO, GR, RU,
SRB, BE
 What are ENPs doing?

ENP workshops & ENP MoUs with

ENPs on the TV
conferences partners

ENPs in the ENP learning ENP social media

papers webinars campaigns
Patient Expert Training Course

+ +
Online self-learning 2 Face-to-face events Patient involvement
forum

150-175 hours of e-learning

and 8 days for two Face-to-Face meetings
over a period of 14 months
EUPATI Patient Expert Training Course:
Rollout of course modules
 Courses

1st EUPATIs Patient Experts Training Toolbox

Course kicked off on 6 Oct 2014 Internet Library

>200 Applications for 2

courses with
~50 trainees each

Patient advocates from

30 countries
and 28 disease areas
enrolled

1st course graduated in

Dec 2015
EUPATI Course #1 completed,
course #2 just running

42 graduates
completed 1st Course
56 trainees from
32 countries
currently in training
Graduation on
13 Dec 2016
Involvement of EUPATI
Fellows and trainees
evidenced in R&D
projects, regulators,
ethics
Course #2 Trainees by Disease Area
Mental Health
Epidermolysis bullosa (EB)
Disability
Genetic Disorders
Muscular Dystrophy
Neurofybromatosis
Rett Syndrome
Cystic Fibrosis
Incontinence
Myelodysplastic Syndromes
Coeliacy
Adrenoleukodystrophy
Lung conditions
Aspergers syndrome/Fibromyalge and Chronic Fatigue Syndrome
Sight loss
Gaucher Disease
Allergy and airways diseases
Hereditary haemorrhagic telangiectasia (hht)
Achondroplasia
Parkinson's Disease
Lupus
Melanoma
Myeloma
Hepatitis C/HIV/STDs
Endometriosis
Haemophilia/hematology
Diabetes
Leukemia
Multiple Sclerosis
Rare diseases
Rheumatic diseases/Arthritis
Cancer
0 1 2 3 4 5 6
True impact: EUPATI Patient Expert Training Course is
generating patient leaders and advisers to pharmaceutical
research, regulatory and HTA authorities

5272%

4472%

852%
1240%
48%

Source: EUPATI Survey of EUPATI Fellows of Course #1, June 2016 (ongoing, n=25)
EUPATI is inducing patient involvement
in research and development
For example:
1. in industry (their in medicines R&D, e.g. standard operating
procedures, R&D processes, frameworks)
2. in academic groups (study groups, medical conferences)
3. in patient organisations (capacity building programmes, Community
Advisory Boards)
4. in funding entities (IMI Patient Engagement Strategy)
5. in regulators (patient involvement pilots)
6. in ethics committees (patient involvement in committees)

Our trainees and Fellows have become ambassadors, advisers,

contributors, knowledgeable partners!
What our EUPATI trainees say about the
EUPATI Patient Expert Training Course

arnas Narbutas from Lithuania

(Lithuanian Cancer Patient Coalition, POLA):
10 days of superbly engaging discussions with 50
capable patient advocates is the capacity building
experience equal to none.

Begonya Nafra from Spain

(Hospital Sant Joan de Du, Barcelona):
Its essential that patients and their families are
involved in research on rare diseases. They have
an immense expertise, and their needs must be
taken into account.

35
What our EUPATI trainees say about the
EUPATI Patient Expert Training Course

Birgit Bauer from Germany

(MS patient and Blogger about Multiple Sclerosis):
() I use my gained knowledge for articles,
blogposts and for talks. I now have better arguments
in discussion panels and understand the different
perspectives of different stakeholders.

Maria Piggin from England

(PNH Support):
I have been provided with tools which give me
confidence to assert the patients' voice. Each process
examined highlights how a patient advocate can add
value.
36
What our EUPATI trainees say about the
EUPATI Patient Expert Training Course

Margaret Graham McDonald from Scotland

(Healthcare Improvement Scotland):
The EUPATI course has harnessed the skills I
needed to play an effective and active part in
Research & Development with confidence.

Stefano Mazzariol from Italy

(Parent Project Onlus):
I would like to work with stakeholders to bring
patients closer to research. I wish that all patients
could benefit from the knowledge that I am acquiring
in this course.
37
Multi-stakeholder development and review
of the EUPATI materials
Internal production and review process
Content Editorial
Expert
Production Group
Review
by experts Review
Created by experts from different stakeholders
EUPATI Editorial Group (comprising Patient Organisations, Academia,
Industry, NGOs) collectively reviewed each topic to ensure content is
consistent, avoids unnecessary duplication and is fit for intended
audience/purpose.

External testing and review process

Ethics
Project Advisor Regulatory Health Literacy
User Testing Committee Release
Review Expert Review Expert Review
Review

user-tested with target audience

reviewed by independent Project Advisory Board members and
Ethics Committee members
Relevant topics reviewed by independent regulatory experts and HTA
experts
edited for appropriate language by health literacy experts
EUPATI Toolbox on Medicines R&D
in 7 languages

Fact sheets,
detailed papers,
PPTs, videos,
illustrations,
glossary.

In English,
French, Italian,
Spanish,
German, Polish,
Russian.

39
EUPATI Toolbox on Medicines R&D
in 7+ languages

In English,
French, Italian,
Spanish,
German, Polish,
Russian

63.200 unique users

in 86.900 visits
(Jan-Nov 2016)

Visitors from 149

countries

40
The Patients Academy: Patient Advocate Toolbox
Formats

PowerPoints
Articles

Infographics Fact sheets

EUPATI Mini-Course Starter Kits:
Resource to prepare and run mini-courses
Core set of PPT slides, outlining a specific area of R&D and how patients
can get involved, plus additional links of EUPATI Toolbox resources, case
studies and exercises, plus Trainers Manual
Patient involvement in
Setting research priorities Protocol design
Ethics Committees Product information, informed consent and
Data Monitoring Committees patient information to trial participants
Medicines safety
Trial Steering Committees
Health Technology Assessment
Scientific advice
EUPATI has collected, analysed and
published successful PPPs

Collected and analysed examples of

successful public-private partnerships
(PPP) between patient organisations,
academia and industry.
348 collaborations studied assessed on
robust criteria
53 were selected as successful models, a
further 23 considered as relevant references.
Catalogue of PPPs and report were publicly
released in January 2014,
see www.patientsacademy.eu/ppp

43
EUPATI Guidances (frameworks) to
support the interaction in R&D
EUPATI has developed guidances for the interaction of
patient organisations with stakeholders
in industry-led R&D
in HTA bodies
in regulatory processes
in ethics committees

Consultation ended 26/9/2016,

Publication in December 2016
EUPATI.eu > Resources > Guidances
EUPATI Guidance for patient involvement
and interaction Why?

Patients & patient organisations to be involved more widely

include early & post-approval stages,
not confined to clinical development
Overarching guidance on meaningful and ethical interaction
is missing in many areas, especially in R&D, causing insecurity
Existing codes/regulations hold mostly legal & ethical content,
i.e. collaboration, communication, funding
-- do not cover patient involvement in R&D
e.g. EFPIA, EATG code, PCWP framework, ECAB,
EMA framework, HTAi
Language needs to be more directive towards patient involvement
Clear default statement that collaboration is allowed unless
expressly forbidden
 EUPATI-trained advocates are the baseline
resource for many R&D-related patient
involvement initiatives
Reg.
Value chain Novel research Pre-
Phase I-III HTA
Post MA activities
projects / TPP clinic Approval (Real World Data, PV, )

Big Data for Better Outcomes

(IMI2 start Q2 2016 )

Subject Patient perspective in B/R

assessment (IMI2 start Q1 2016)

ADAPT SMART (MAPPS) (IMI2- start 1.7.2015)

multistakeholder coordination platform

PATIENT FOCUSED MEDICINES DEVELOPMENT (PFMD)

EUPATI
Well-trained / empowered patients
Page 46
2016 and beyond:
Time to harvest the fruits
EUPATI as a PPP has pioneered a paradigm shift
towards patient involvement in medicines R&D:
Established a true, trustful, unpolitical partnership
between patient organisations, science and
industry
Developed Patient Expert Training Course,
trained 98 Patient Experts
Deployed EUPATI Toolbox and EUPATI Internet
Library in 7 languages to more than 50.000 individuals
Released EUPATI material under Creative Commons
Established ~18 EUPATI National Platforms
(launched in AT, FR, DE, IE, IT, MT, ES, CH, UK,
additionally emerging in DK, SL, SR, NL, PT, GR,
under construction in BE, LUX, PO)
Developed guidance and identified best practice
Established EUPATI as quality brand for patient
education
 EUPATI will continue as
the EUPATI 2017+ Programme
EPF-led programme: Coordination and management in the PPP spirit
Exploitation of the EUPATI Toolbox in all languages
Toolbox-based Starter Kits for community-run trainings / capacity
building, based on the Toolbox
Content updates (ca. 10 Toolbox elements updated per year)
IT platform (Toolbox, Moodle, Project Tools)
Evaluation and evolution (EUPATI Futures Team)
EUPATI National Platforms & coordination support
Patient Expert Training Course 2017-2018

2-stage
IMI Call 1: application, EUPATI IMI Project 2012-2016: EUPATI
not funded consortium patient-led PPP of patients, academia, NGOs and industry Programme
building

2010 2011 2012 2013 2014 2015 2016 2017 20

can really make a unique
difference to patient
empowerment and to
medicines R&D.

You can help us to make

it a success.

Jan Geissler
jan@patientsacademy.eu