24 June 2016

London

Nicola Bedlington

EPF Secretary General

PROGRESS ON PATIENT INVOLVEMENT AT PAN-

EUROPEAN LEVEL

• Background on EPF

• The Bigger Picture – Access to Medicines

• The intrinsic Role of Patients

– In the policy environment

– In engaging effectively in the development of new medicines – the life cycle approach

• Reflections – new medicines and better health outcomes

Presentation Outline

• Independent, non-governmental umbrella organisation set up in 2003

• OUR VISION: All patients in the EU have equitable access to high quality, patient-centred health and social care

About the European Patients’ Forum

• OUR ROLE: United patients’ voice in EU health and social policy

• Key interlocutor with the EU Institutions

• OUR MEMBERS: disease-specific EU & national coalitions – 67 member organisations

Access and equity – a major concern

Healthcare provision must be based

on the fundamental values of equity and solidarity

Huge disparities across the EU and within countries in access to even

basic healthcare, let alone innovative treatments

Innovative treatments that add real value should be accessible to all patients, not only those who can pay

Patients engagement in the therapeutic innovation process must be set in this context – the end goal in mind

Working towards a European Partnership for equity of access to quality healthcare

The Patient Access Partnership

New momentum: EPF Election Campaign

A multi-stakeholder partnership with patients, healthcare professionals, healthcare industries, and health experts, and key decision makers

Objectives

• Join forces to measure, map and explore solutions to overcome inequities, based on individual and collective expertise

• To put the issue higher on EU political agenda

The Access Partnership

Patients as part of the solution

Patient empowerment, self management and self care

Health literacy, the right skills and competencies for all the players,including patients, and an enabling environment

Patient involvement in co-designing healthcare

Strong evidence base

Patient-centred care models: cost-effective, better health

outcomes, and patient satisfaction

Patients as experts: to identify unmet service and therapeutic

needs and point out inefficiencies and waste in systems and processes

Smart spending

where needed!

• Orphan Medicinal Products Regulation

• the Paediatric Use Regulation

• the Advanced Therapy Regulation,

• Pharmacovigilance Directive

• Revised Clinical Trials Regulation.

• Data Protection Regulation … etc…

Leading patient organisations influenced all of this legislation, patient engagement is also crucial in effective implementation, through EMA and at national level – Full circle approach

Patients’ engagement in policy

Promoting Innovation – Key dimensionsWHO Priority Medicines Report

Public Health Based R&D

Public Private Partnerships learning from

IMI

Redesigning of the Regulatory

System

New pricing and reimbursement mechanisms for

innovation

Real life data and learning from practice

Models for Stakeholder

Involvement including patients and citizens

• Benefit Risk Analysis – understanding the perspective of the patient – Weighing the psychosocial factors

– Permissiveness for patients with very serious illness ( Genetic Alliance UK)

• New IMI Call :’ Patient perspective elicitation on benefits and risks of medicinal products, from development through the entire life cycle, to inform the decision-making process by regulators and health technology assessment bodies'

Benefit - Risk

Benefit Risk Trade -Off

• Medicine use in clinical practice frequently differs widely from the (pre-approval) clinical trial settings:

• Need to bridge bench and clinical research with real-world practice, also for HTA

• Electronic Health Records – patients’ ownership of data

• Role of Registries

• IMI – Big Data for Better Health Outcomes Programme – better patient engagement

Real Life Data

• More transparency on pricing mechanisms

• Exploration of meaningful alternatives to external reference pricing

• The value of ERP as a policy tool for regulating prices warrants further examination……. LSE Report 2012

• The scope of ‘value –based pricing’ – Role of Patients in assessing that value

• EPF+ EURORDIS letter to national competent authorities-

‘ table for price negotiations’ – scale up of early dialogue involving payers - moving forward

Innovation in pricing

• Distributed in advance on Informal Health Council 15 April – ongoing discussions

• Key concerns regarding current pricing models –which contribute to inequities

• A patient perspective on the ‘value’ of innovation

• Core principles for the pharma industry, and political decision-makers to ensure equitable access to treatment based on need, not means, + long-term sustainability of our health systems

Value and Pricing

EPF Statement

• New socio-economic realities – post crisis

• New environments, new settings

• Traditional approaches no longer fit for purpose

• Need to ‘innovate the innovation process’

– More flexibility, simplification

– New models and pilots

– Targeted populations – stratified medicines

– Advanced digital technology

Better outcomes for less cost….

requires patient engagement

More efficiency in drug development

Dire need for breakthrough therapies – no room for inertia

• Early access to innovative medicines – the potential of adaptive pathways

• Early dialogue with all stakeholders concerned –predictability – clarity

• Use of Real World Data

• ADAPT SMART – IMI project led by EMA

• An enabling platform for the coordination of Medicines Adaptive Pathways to Patients (MAPPs) activities.

• Beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span in a sustainable fashion.

Adaptive Pathways

Evidence v. Access Trade -Off

Main challenges in involving patients in HTA are:

– Lack of an agreed method

– Lack of resources/capacity

– Not knowing the best stages to involve patients in HTA

– Patient evidence not being credible enough

Need for

• a framework and methodology for patient involvement in HTA

• education/training for patient representatives and the

HTA community

• New EU Joint Action on HTA – new opportunities

Health Technology Assessment

• Patient Empowerment -political strategy towards quality and sustainable health systems of the future

• Patient engagement in new medicines is seen a pre-requisite. This is complemented by patient organisations’ vital advocacy role at EU level

• IMI contributing to a more structured, systematic approach throughout the EU– Training and education is vital – more patient experts

are needed and ongoing continuous patient education is key

– New IMI Patient Engagement Strategy

• All of this must be set in the wider equity and access context – Innovation and Solidarity

Reflections

/europeanpatientsforum

/eupatientsforum

More informationwww.eu-patient.euinfo@eu-patient.eu

THANK YOU FOR YOUR ATTENTION!

Follow us on Social Media!

/eupatient

eu-patient.eu/blog

24 June 2016

EUPATIWhat have we delivered?

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.

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 demonstrateda clear need & willingness of patient advocates to be an active partner in medicines research and development

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 ofhealth-related research

Trial protocol design, informed consent, ethical

review, marketing authorization, value

assessment, health policy

What is EUPATI?

A Public Private Partnership within theInnovative Medicines Initiative Joint Undertaking*

A 5-year project, launched in February 2012

A patient-led project coordinated by theEuropean Patients’ Forum, with EGAN, EURORDIS and EATG in key roles

A strong multi-stakeholder consortium ofpatients’ organisations, academia, NGOs andindustry – 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 a 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 Patient ExpertsTraining Course-- for expert patients

EUPATI is developing education targetedat different levels

100 patient

experts

12.000patient

advocates

100.000individuals

EUPATI Educational Toolbox ( 7 languages)-- for patient advocates

EUPATI ( 7 languages)Internet Library-- for the health-interested public

English FrenchGerman

SpanishPolishItalian Russian

English

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

24

+ P

atie

nts

‘ role

s a

nd

responsib

ilities

EUPATI Progress Update

Preparation PhaseM1-18

Confirmation PhaseM19-48

Sustain PhaseM49-60

WP1 Coordination

ProjectInfrastructure

Project Management, Communication, Evaluation, Finances

WP2Network Implem.

Establishment of EUPATI Network

Public conferences, Regional Workshops,EUPATI Network, EUPATI National Platforms

WP3 Needs Assessment& Gap An.

Needs analysis, Review of material,

focus groups

Quantitative survey,Literature review

WP4 Content Dev.

SyllabusEditorial process, Content Development, translation

Refinement, quality control

WP5 IT Infrastr.

Design, development and support of technical infrastructure for eLearning/IT Platform

WP6 Deploy-ment & Quality

Assurance

First course

performed

EUPATI IT Platformlaunch

Deployment, dissemination,

quality assessm‘t

WP7 Sustainability

Research on Patient Partnership models

Best practice guideline dev.,Recommendation of new

teaching methods

Development ofcode of conduct

for patientinvolvement

Sustainability concept dev.New technologies andfuture remit strategy

Project Plan

4 and half years of EUPATI:Much has been done! …For example events in Frankfurt 2012, Barcelona 2013, Rome 2013, Warsaw 2014…

EUPATI assessed patient & publicattitudes towards medicines R&D

Review of existing information resources;230 resources reviewed.

Review of research literature; 12600 titlesand abstracts; 134 studies included

Public survey; 6931 responses in GB, Spain, Poland, Italy, France and Germany

Qualitative research with patients and thepublic; n=125 people

Qualitative research with stakeholders; n=56 (incl. policy makers, pharmaceuticalindustry, clinical research professionals)

Patient advocate survey; 468 responsesacross Europe

Patient advocate survey focused on course delivery; 41 responses

+ 3 peer reviewed

publications to come

First results published

in BMJ Open:

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

EUPATI National Platforms

EUPATI National Platforms...

bring all stakeholders together in countries

address educational needs in R&D

disseminate EUPATI’s trainingmaterial to patient organisations

ENP updates

Country Established

Austria 3 Oct 2015

Belgium summer 2016

France 16 June 2015

Germany 20 Jan 2016

Ireland 16 Sept 2014

Italy 10 April 2015

Luxembourg 17 Dec 2014

Malta 30 April 2014

Poland summer 2016

Spain 4 Nov 2014

Switzerland 3 Feb 2016

United Kingdom 6 March 2014

Country Established

Denmark 30 March 2016

Slovakia 20 Feb 2016

Serbia 06 April 2016

Netherlands Pat & Aca reps

Portugal NLT established

Greece NLT established

Romania NLT established

12 EUPATI countries*7 new EUPATI countries**

*There is progress in Belgium and we hope to soon identify a patient chair.

**There is interest in Norway, Hungary and Macedonia.

Patient Expert Training Course

+

Online self-learning

Approx 200 hours of e-learning

and 8 days - two Face-to-Face meetings

over a period of 14 months

+

2 Face-to-face events Patient involvement

forum

EUPATI Course #1 completed, course #2 in progress

47 graduates successfully completed the 1st course

56 trainees from 32 countries currently in training

Well-educated, energized, experienced advocates constitute a pan-EU, pan-disease network

Involvement of EUPATI Fellows and trainees evidenced in R&D projects, regulatory (e.g. EMA), ethics

True impact: EUPATI Patient Expert Training Course is generating patient leaders and advisers to pharmaceutical research, regulatory and HTA authorities

Source: EUPATI Survey of EUPATI Fellows of Course #1, June 2016 (ongoing, n=25)

5272%

4472%

852%

1240%

48%

The EUPATI Advocate Toolboxin 7 languages (launch 27 January 2016)

35

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

Accessible via web and on mobile devices as well as for offline use.

All content available in English, French, Italian, Spanish, German, Polish, Russian.

EUPATI Toolbox on medicines R&D haslaunched very successfully

• 25.000 unique users in

35.000 sessions in just

4 months (by May 2016)

• Visitors from 149

countries

• Most popular topics:

0 200 400 600 800 1000 1200

Pharmacoepidemiology

Non Clinical Studies

Benefit and Risk Assessment

Safety of Medicines

Personalised Medicine

Regulatory Affairs

Types of Medicine

Pharmaceutical Development

Health Technology Assessment

Drug Discovery

Clinical Development and Trials

Basics of Medicines Development

EUPATI held a Patient organisations & industry workshop in July 2014, further workshop on POs, Regulators, Industry July 2016

Turning aspirations into reality focusing on:

Benefits and impact of involving patients and advocates in the R&D process

Common concerns, hurdles and solutions

Possible Instruments

These help develop guidance, framework, key indicators.case studies published in public domain!

EUPATI-trained advocates are the “baseline resource” for many R&D-related patient involvement initiatives

Novel research

projects / TPPPre-

clinicPhase I-III

Reg.

Approval

Patient perspective in B/R

assessment (IMI2 – start Q1 2016)

PATIENT FOCUSED MEDICINES DEVELOPMENT (PFMD)

ADAPT SMART (MAPPS) (IMI2- start 1.7.2015)multistakeholder coordination platform

Value chain

Subject

Page 38

HTAPost MA activities

(Real World Data, PV, …)

EUPATI

Well-trained / “empowered” patients

Big Data for Better Outcomes

(IMI2 – start Q2 2016 )

Why do we need to continue with EUPATIbeyond 2016?

True impact on medicines R&D and patient empowerment will happen when EUPATI’s “core” continues to educate patients (advocates) beyond 2016.

We need to train more “Expert Patients” in all disease areas and from all countries and to make maximum use of the EUPATI material

100 trained patient experts from 30 countries are not enough to participate / collaborate in R&D (in academia, industry), competent authorities, ethics committees across countries and diseases.

EUPATI needs to continue to increase awareness of R&D amongst patient community / broader public, academia, industry and authorities

EUPATI is the only dedicated training course on the process of medicines development. Patient experts are the core resource for any project on “patient involvement / centricity in R&D”

EUPATI beyond 2016: Sustainability beyond current IMI1 funding being discussed

What EUPATI could continue to doafter current funding ends:

EUPATI “Central project”• Provision of the EUPATI course:

– Run the English-language Patient Expert Training Course

– Provide the EUPATI Toolbox (IT platform)

– Provide the EUPATI Internet Library (IT platform)

– Support matchmaking of EUPATI trainees with interested parties (researchers, authorities)

• Keeping EUPATI’s material up to date, e.g. when regulation changes

– Update the course material, the Toolbox, the Internet Library(in all 7 languages)

– Continue to implement feedback of users e.g. corrections, terminology

• Extend content beyond the current syllabus (e.g. patient involvement in research)

Some coordination and support of EUPATI’s National Platforms• Coordinate the 12 National Platforms (& potentially additional countries)

• Support the conduct of local courses / other language courses

• Make sure EUPATI material derivatives do not deviate from EUPATI content

Coordination

Training

National

Plat-

forms

Content

Manage-

ment

IT

Platform

Now is the time to “harvest the fruits”

and to create a sustainable EUPATI beyond 2016!

Jan Geisslerjan@patientsacademy.eu

can really make a unique

difference to patient

empowerment and to

medicines R&D.

You can help us to make

it a success.