Public private partnerships to encourage patient access

www.efpia.eu

5th SFE SFUS Conference

Challenges for efficient health care in

Central and Eastern Europe

Belgrade, 10th October 2015

www.efpia.eu

How does IMI work?

3www.efpia.eu

The Innovative Medicines Initiative:

the largest public-private partnership for

health research worldwide

In total €5 bn until 2014 – half from EU FP7 & Horizon 2020 R&D funding, half from Industry

4www.efpia.eu

Scientific excellence

Market access

Patient access

Rewarding Systems

The Pillars of Innovation

5www.efpia.eu

Elias A. Zerhouni, President Global R&D, Sanofi

Editorial in Science Translational Medicine, January 2014

‘We must acknowledge that no

single institution, company,

university, country, or government

has a monopoly on innovation.’

6www.efpia.eu

From First to Last Mile

CollaborationPrioritisation

Why IMI?

Flexibility

7www.efpia.eu

Prescribers

Patients

Big and Small Companies

Payers

Regulators

Academia

Primary & Special care

Social security

IMI breaks silos and enables the ecosystem

8www.efpia.eu

IMI – The European Added Value

9www.efpia.eu

IMI: Europe’s partnership for health

10www.efpia.eu

IMI Project Process

11www.efpia.eu

Since 2008 the pharmaceutical

industry has already committed

€ 1.2 Billion in kind to 70+

projects

www.efpia.eu

IMI Achievements

13www.efpia.eu

Over 7 000 experts

working for:

collective intelligence

networks

improved R&D

productivity

innovative approaches

to unmet medical

needs

An international, cross-sector community

14www.efpia.eu

IMI stimulates collaboration across EUCo-authorship between IMI-supported researchers Calls 1-4

Pre IMI funding award Post IMI funding award

Data & analysis: Thomson Reuters (Custom Analytics & Engineered Solutions)

15www.efpia.eu

Mapping Collaborative Networks

Data & analysis: Thomson Reuters Custom Analytics & IP Solutions

15

http://imi.efpia.eu/imi2/create-your-imi2

16www.efpia.eu

Collaboration delivers excellenceIMI Citation Impact, Source: Thomson Reuters analysis, 2015

vs. . ave age

17www.efpia.eu

Collaboration delivers excellence

INTERNATIONALcollaboration

50%of IMI publications

Collaboration BETWEEN PUBLIC AND PRIVATE

SECTORS 61% of IMI publications

collaboration BETWEEN INSTITUTIONS

75%of IMI publications

0

1

2

3

Cross-institution Cross-sector International

Citation impactCollaboration impact on quality

Collaborative Non-collaborative

17

Thompson Reuters - 2014

18www.efpia.eu

Citation index

IMI projects deliver excellent science

19www.efpia.eu

• EUPATI provides scientifically reliable, objective, comprehensive information to patients on medicines research and development.

• It will increase the capacities and capabilities of patients and patient organisations to be effective advocates and advisors in medicines research, e.g. in clinical trials, with regulatory authorities and in ethics committees.

EUPATIEuropean Patients‘ Academy on Therapeutic Innovation

20www.efpia.eu

New access models that balance healthcare budgets imperatives with businesssustainability for products that are supposed to be shelved and only used as lastresort (antibiotics).

Join forces between public and private partners to

bring new antimicrobials closer to patients

share information and

boost research on improving the uptake of antibiotics into Gram-negative bacteria

New Drugs for Bad Bugs - ND4BB

iABC

21www.efpia.eu

Reduce antimicrobial resistance through responsible antibiotic use

Identify how, through new economic models, to incentivise the discovery anddevelopment of new novel antibiotics for use now and in the future

Define what constitutes responsible antibiotic use, forecast future resistancetrends to inform public health priorities and estimate the real value of newantibiotics based on the economic and health costs of growing antimicrobialresistance

Engage with all interested stakeholders during the 3 year project, with fulltransparency to develop and test new economic models for antibioticdevelopment and use to reinvigorate investment in this area.

Recommend to governments and policy makers around the world appropriateoptions to resolve the crisis

ND4BB: DRIVE-ABDriving reinvestment in research and development

and responsible antibiotic use

22www.efpia.eu

Flexibility – many collaboration models

Ebola +

European Lead Factory

ND4BBAdapt Smart

Safe-T

EU AIMS

23www.efpia.eu

already paved the way

to new treatments or

prevention of pain, autism,

diabetes, schizophrenia,

asthma, rheumatoid arthritis

and geriatric medicines

24www.efpia.eu

Impact on Regulatory Framework

25www.efpia.eu

Established robust validated models for Alzheimer, Diabetes, Schizophrenia, Asthma

Developed clinically relevant biomarkers for Alzheimer, Diabetes, Schizophrenia, Asthma

Established robust tools for drug safety prediction, prevention and monitoring

Establishment and regulatory submission of key standards and tools for drug development in infectious diseases, COPD, diabetes

Improved clinical trial design (shorter/smaller trials) and process in schizophrenia, pain, autism

Co-funding of antibiotics and vaccines development

Projects launched and planned on use of real life data and alignment of regulators and payers data requirement

Uptake by Regulators has started (guidance, biomarkers

IMI already delivers on Innovation and Health

www.efpia.eu

IMI and patient access

27www.efpia.eu

Addressing parameters for early and effective patient

access

Differentiating enablers/platform tools

Reimbursement/patient access

Regulatory framework

Prevention, disease interception and adherence

Data generation and interpretation

Overarching Theme of IMI 2 Programme

28www.efpia.eu

• STEMBANCC & EBiSC (stem cells, biobanking)

• PROactive (Patient Reported Outcomes in COPD)

• UBIOPRED (Patient Reported Outasthma)

• WEB-RADR (Web based AE reporting)

• EU AIMS (autism)

• ADAPT SMART (adaptive pathways, managing uncertainties)

• EHR4CR (EHR, patient recruitment)

• NEWMEDS ( CT depression & schizophrenia)

• SUMMIT (personalised approach to diabetes)

• Patient Preference elicitation in B/R

Biomarkers

Outcomes

Stem cells Disease

classification

Enablers of Adaptive Pathways

Patient recruitment

IMI projects generate tools for personalised medicines

29www.efpia.eu

The wider landscape of EU projects driving Patient Involvement in R&D

EUPATI• Delivers patient expert and lay audience

training in 7 languages• Empowers patients on R&D;

National platforms in 12 countries;• Guidance for patient involvement in R&D

PFMD(Patient focused Med. Dev.)Coordination activity for systematic involvement in industry R&D;global perspective

Patient-Smart(IMI2, start 01/2016)Web-based knowledge repository for patient interaction with industry and other stakeholders match-making, best practice, guidance

IMI2 ADAPT SMARTCoordination of “Medicines Adaptive Pathways to Patients” (MAPPS) in Europe Lead: EMA patient organisations involvedStarting 1 September 2015

Other upcoming IMI2 projects• Patient perspective in B/R asessment Big

Data for better outcomes• Data Protection / Informed Consent

…….

EUPATI Material

Existing Community Training Initiativese.g. EURORDIS Summer SchoolEATG STEP-UP / ECABERS EPAPEPF Capacity Building

30www.efpia.eu

A Sustainable Multi-stakeholder Approach from Research to Treatment(SMART): A Structured Patient Engagement Knowledge Platform (PEKH) forImproved Outcomes".

New IMI2 project; currently preparing the Full Project Proposal, due 28September; project start planned for Q1/2016; budget: ca. 16 mio Euro

Current partners: 5 EFPIA companies (MSD, Pfizer, UCB, Bayer, Sanofi), 15public partners incl. Eurordis, EPF, EFGCP, EATG, IPPOSI)

Goal: Web-based platform to facilitate patient engagement in therapeuticinnovation- good practice, standards, norms – and ‘ match-making’

A systematic and structured approach, tested in real-life pilotswith long term perspective

IT Infrastructure focussed project complementary to EUPATI including thepotential to implement the EUPATI WP7 guidance concept for patientinvolvement

Participation of more EFPIA companies (from EUPATI) would be extremlyhelpful

IMI2 PATIENT SMART

30

31www.efpia.eu

Improve the framework for Adaptive Pathways

Identify relevant MAPPs activities (pilots, case

studies) and build up of a repository

Identify the scientific challenges and opportunities

related to MAPPs implementation

Support new IMI 2 research and innovation actions

by facilitating the inclusion of MAPPs enablers

Conduct horizon scanning and gap analysis and

advice on future research activities to IMI

Status

Approved as “IMI2 Coordination and Support

Action”

Detailed project plan agreed, contracts under

negotiation

Coordinated by EMA

IMI2 ADAPT SMARTAccelerated Development of Appropriate Patient Therapies

Budget: ca. 7 mio Euro

Start Date – End Date: 01/08/2015 – 31/12/2017

• AbbVie Deutschland GmbH & Co. KG• Bayer Pharma AG• Boehringer Ingelheim Int’l GmbH• Merck KGaA

• GlaxoSmithKline R&D Ltd• Eli Lilly & Company Ltd• Pfizer Ltd• European Medicines Agency• National Institute for Health

and Care Excellence• University of Oxford

• H. Lundbeck A/S• Novo Nordisk A/S• Danish Health and Medicines

• Amgen NV• Janssen Pharmaceutica NV• UCB Pharma SA• European Federation of

Pharmaceutical Industriesand Associations

• European Patients’ Forum

• Merck Sharp & Dohme Corp., US• Massachusetts Institute of Technology’s NEW Drug Development ParadIGmS, USA

• Stichting Top Institute Pharma• Zorginstituut Nederland

• Astellas Pharma• Bristol Myers Squibb EMEA sarl• Ipsen Pharma• Lysogene• Sanofi-Aventis R&D• European Organisation for Rare Diseases• Haute Autorité de Santé

• AstraZeneca AB

• F. Hoffmann-La Roche AG• Novartis Pharma AG

32www.efpia.eu

Big data for better outcomes programme

Goal Support the evolution towards outcomes-focused and sustainable healthcare systems, exploiting the opportunities offered by big and deep data sources

Design sets of standard outcomes and demonstrate

value

• Sets of target outcomes

• Clinical endpoints• Alignment of HC

stakeholders on the value of those outcomes..

Increase accessto high quality outcomes data

• Mapping of sources, methods and tolls for collection and harmonization

• Governance and technical standards...

Use data toimprove value of HC

delivery

• Drivers of outcomes variation

• Best clinical practices

• Methodologies to predict outcomes...

Increase patient engagement through

digital solutions

• Patient Reported Outcomes opportunities

• Profiling patients behaviors

• Tools to increase patient engagement...

Themes/Enablers

11 22 33 44

33www.efpia.eu

Better Health Outcomes

Design sets of standard outcomes and

demonstrate value

Increase accessto high quality outcomes

data

Use data toimprove value of HC

delivery

Increase patient engagement through

digital solutions

COORDINATION AND SUPPORT ACTION (CSA)

HEMATOLOGIC MALIGNANCIES

MULTIPLE SCLEROSIS

CARDIOVASCULAR

RARE CANCERS

PATIENTS' MULTI-MORBIDITIES

Themes / Enablers

11 22 33 44

Support the evolution towards outcomes-focused and sustainable healthcare systems

"Big data for better outcomes"

Coordination and operational topics

Disease- specific topics

EUROPEAN DISTRIBUTED DATA NETWORK

ROADS: ALZHEIMER'S DISEASE

www.efpia.eu

IMI Oncology and Rare Diseases

35www.efpia.eu

MultipleStakeholders:

IMI1 in Numbers

Source: IMI2 JU Annual Activity Report 2014

23 patient organisations. 14 regulators. 714 academic & research teams. 410 EFPIA

teams. 135 SMEs. More than 7,000 researchers. 61% of projects reported some form of

patient involvement. 12 regulators on boards of projects. 50% of projects have

representatives of regulatory authorities on scientific advisory boards.

IMI2 Strategic Research Agenda

Antimicrobial resistance

Osteoarthritis

Cardiovascular diseases

Diabetes

Neurodegenerative diseases

Psychiatric diseases

Respiratory diseases

Immune-mediated diseases

Ageing-associated diseases

Cancer

Rare/Orphan Diseases

Vaccines

37www.efpia.eu

OncoTrack

Methods for systematic next generation oncology biomarker

development (colon cancer)

Predect

Preclinical evaluation of drug efficacy in common solid tumours

(breast, prostate and lung cancers)

Quic-Concept

Quantative imaging in cancer: connecting cellular process with

therapy

CANCER-ID

Identification of circulating tumor cells and tumor related nucleic

acids in blood

IMI Focus on Oncology/Rare Diseases

38www.efpia.eu

Do current approaches to oncology and rare diseases appropriatelyaddress the high medical need?

What are the issues?

Could a collaborative approach involving public private partnershipssupport solutions?

IMI 2 recognises:

Rare diseases are a strategic area for more pre-competitive collaboration and public-private partnership, aiming for progressive patient access to medicines.

E-Rare-3

Seeks to establish closer contact with the Innovative Medicines Initiative (IMI) and the European Medicines Agency (EMA) to facilitate the access of researchers to these important stakeholders in drug development.

Ensure cooperation between academia, authorities and industry

Oncology and Rare Diseases

‘Neglected’ in IMI?