IMI2: Strategic research agenda and overall approach

Hugh Laverty Senior Scientific Project Manager

Introducing IMI2: Vienna 16 July 2014

Pharma

Regulators

EU Pricing

Generics

HC Reform

Rising

R&D cost

Declining R&D

productivity

Patent cliff

The way in which pharmaceutical companies develop new medicines is changing

New approaches needed

“Deciphering the complexity of human diseases and finding safe, cost-effective solutions that help people live healthier lives requires collaboration across scientific and medical communities throughout the health care ecosystem. Indeed, we must acknowledge that no single institution, company, university, country, or government has a monopoly on innovation.”

Innovative Medicines Initiative: Joining Forces in the Healthcare Sector

The biggest public/private partnership in Life Science aiming to:

• Make drug R&D processes in Europe more innovative and efficient

• Enhance Europe’s competitiveness

• Address key societal challenges

Features:

• 1:1 funding, joint decision making

• All EU funds go to SMEs, academia, patient organisations and regulatory agencies

• Large pharmaceutical industry, represented by EFPIA, contributes in-kind

The IMI Portfolio

The IMI Community

REGULATORS ON BOARD OF

12 PROJECTS

50% of projects have

REGULATORY AUTHORITIES representatives in Scientific

Advisory Boards

61% of projects

reported some form of

PATIENT INVOLVEMENT

14 regulators

135 SMEs

23 patient

org.

410 EFPIA teams

714 academic &

research teams

> 6000 researchers 46 projects Calls 1-8

The measures of success

SUCCESS

New models developed &

published

Setting new standards

In house implementation

by industry

Impact on regulatory guidelines

Better Science = Better Decisions

IMI2

New approaches needed

“Deciphering the complexity of human diseases and finding safe, cost-effective solutions that help people live healthier lives requires collaboration across scientific and medical communities throughout the health care ecosystem. Indeed, we must acknowledge that no single institution, company, university, country, or government has a monopoly on innovation.”

The Vision For IMI2 – The Right Prevention And Treatment For The Right Patient At The Right Time

Biologically

heterogeneous

patient population

Graphic adapted from C. Carini, C. Fratazzi, Eur. Pharm. Rev. 2008, 2, 39-45

effective

not effective

Adverse events

Trial and Error

effective

not effective

adverse events

Dx Test

A

B

C e.g.

biomarker

Information based treatment decisions

vs

11

Science is driving advances in diagnosis: breast cancer is actually 10 different diseases

“A landmark study has reclassified the country’s most common cancer in breakthrough research that could revolutionise the way we treat breast tumours… scientists found breast cancer could be classified into 10 different broad types according to their common genetic features.”

http://www.nhs.uk/news/2012/04april/Pages/breast-cancer-genetic-diversity-mapped.aspx

Thursday April 19 2012

Make Drug R&D processes in Europe more efficient and effective and enhance Europe’s competitiveness in the Pharma sector

Idea generation

Primary focus of early IMI calls

2007 SRA

Basic research

and non-clinical

testing

Shift to also addressing challenges in in society and healthcare

2011 SRA

Human testing Regulatory

Approval

HTA and

Pharmacovigi-

lance

Daily

Medical

practice

IMI 2 includes real life medical practice

2013 SRA

The Evolution of IMI: From bottlenecks in industry

– to bottlenecks in Industry and Society

Objectives of IMI2 – what the regulation says

• Increase the success rate in clinical trials

• Where possible, reduce the time to reach clinical proof of concept in medicine development

• Develop new therapies for diseases for which there is a high unmet need and limited market incentives

• Develop diagnostic and treatment biomarkers for diseases clearly linked to clinical relevance and approved by regulators;

• Reduce the failure rate of vaccine candidates in phase III clinical trials through new biomarkers for initial efficacy and safety checks;

• Provide support for the development of tools, standards and approaches to assess efficacy, safety and quality of regulated health products.

For integrated healthcare solutions

• Alignment with Horizon 2020 objectives of the Health challenge

• Addressing healthcare priorities identified by the WHO 2013 report on priority medicines for Europe and the world

• Strategic Research Agenda aimed at progressing the vision of stratified medicines: prevention, treatment and health management

• Entire cycle from discovery, through development to healthcare delivery and access models

• Collaboration across sectors to harness all knowledge and technologies which can contribute to IMI2 vision - diagnostics, imaging, IT, medical devices, …

Strategic Research Agenda

Comprehensive framework for a 10-year programme

Prepared with input from 80+ organisations

Project ideas from industry and third parties will be screened against it

http://goo.gl/jqMP9g

2013: WHO report on priority medicines for Europe and the World: societal challenge reflected in the IMI2 SRA

0

2

4

6

8

10

12

Isch

aem

ic h

eart

dis

eas

e

Ce

reb

rova

scu

lar

Un

ipo

lar

dep

ress

ion

Dia

be

tes

mel

litu

s

Hea

rin

g lo

ss

CO

PD

HIV

/AID

S

Alc

oh

ol u

se d

iso

rde

rs

Low

er r

esp

irat

ory

infe

ctio

ns

Alz

he

ime

r an

d o

ther

dem

enti

as

Trac

hea

, bro

nch

us,

lun

g ca

nce

r

Ost

eoar

thri

tis

Cir

rho

sis

of

the

live

r

Co

lon

an

d r

ect

um

can

cer

Bre

ast

can

cer

Pre

mat

uri

ty a

nd

low

bir

th w

eig

ht

Bir

th a

sph

yxia

an

d b

irth

tra

um

a

Neo

nat

al in

fect

ion

s an

d o

ther

…

Mal

aria

Tub

erc

ulo

sis

WHO report: Percentage of DALYs for top 20 high burden diseases and conditions

Europe

World

2

Table of Contents

1. Introduction and Background .................................................................................................... 9 1.1. The Strategic Research Agenda (SRA) ................................................................................. 9 1.2. The aim and vision of IMI2 – The right prevention and treatment, to the right patients at

the right time .............................................................................................................. 10 1.3. Building on the strengths of Europe .................................................................................. 11 1.4. Learning from the Innovative Medicines Initiative (IMI) ................................................... 13

2.Establishing the Research Priorities for IMI2 ............................................................................. 15 2.1. Challenges facing the healthcare ecosystem of today ...................................................... 15 2.2. The role of Research & Development in addressing healthcare challenges ..................... 16 2.3. Regulatory, health technology assessment and healthcare delivery challenges .............. 18

3.Research Objectives of IMI2 ..................................................................................................... 19 3.1. Four major axes of research .............................................................................................. 19 3.1.1. Axis 1: Target validation and biomarker research (efficacy and safety) ........................... 19 3.1.2. Axis 2: Adoption of innovative clinical trial paradigms ..................................................... 21 3.1.3. Axis 3: Innovative Medicines ............................................................................................. 23 3.1.4. Axis 4 : Patient tailored adherence programmes .............................................................. 24

4.Enabling Technologies ............................................................................................................. 28 4.1. Excellence in Data Management ................................................................................ 28

5.Implementation strategies ....................................................................................................... 29 5.1. Education and Training required to implement new scientific advances ......................... 29 5.2. Excellence in clinical trial implementation ........................................................................ 31

6.European Health Priorities to be addressed by IMI2 ................................................................. 32 6.1. Antimicrobial resistance .................................................................................................... 32 6.2. Osteoarthritis .................................................................................................................... 34 6.3. Cardiovascular diseases ..................................................................................................... 37 6.4. Diabetes ............................................................................................................................. 39 6.5. Neurodegenerative diseases ............................................................................................. 41 6.6. Psychiatric diseases ........................................................................................................... 44 6.7. Respiratory diseases .......................................................................................................... 46 6.8. Autoimmune diseases ....................................................................................................... 48 6.9. Ageing-associated diseases ............................................................................................... 50 6.10. Oncology ............................................................................................................................ 52 6.11. Rare/Orphan Diseases ....................................................................................................... 55 6.12. Vaccines ............................................................................................................................. 58

7.Translating research to tangible benefits for European and Global Healthcare infrastructures ... 61 8.Impact of IMI2 on the use of animals in research and development .......................................... 64 Reference List

Therapeutic Areas in IMI2 SRA (no priority order)

IMI2: Major Axes of Research

Target & Biomarker

Identification (safety & efficacy)

Innovative clinical trial paradigms

Innovative Medicines

Patient tailored

adherence programmes

Reclassification of disease by molecular means

Target Identification and validation(human biology)

Determinants of drug /vaccine Safety and efficacy

Biomarker identification/validation (precision medicine) Innovative methodologies to

evaluate treatment effect

Adoption of innovative clinical trial designs

Benefit/Risk Assessment

Innovative drug delivery methodologies

Manufacturing for personalised medicines

Healthcare delivery: focus on the treatment programmes not just the medicine

Innovative adherence programmes

Discovery and Development of novel preventative and therapeutic agents

European Health

Priorities

Drive change in delivery of medical practice

First five big themes (1)

Therapeutic Areas and Cross-cutting Themes

1. Neuro-degeneration • Successfully prevent and treat dementia and other

neurodegenerative diseases

2. Prevention and treatment of immune-mediated disease Advance immunological understanding to deliver new medicines and new and better vaccines

3. Metabolic disorders • Tackle all phases of disease and its complications,

including prevention and early interception (type 2 diabetes, obesity, dislipidemia, hypertension)

4. Infection control • Address big societal problem related to multidrug

resistance and create incentives for reinvestment (including antimicrobials, antivirals, vaccines) and develop new and better vaccines

5. Translational Safety • identification of predictors of safety and development

of point of care for safety biomarkers & Development of new human biology platform to predict toxicity and safety during early drug development

Differentiating Enablers for all themes

Towards early and effective patient access to innovative prevention and treatment solutions (Medicines Adaptive Pathways to Patients, MAPPs):

• Target validation based on human biology

• Stratified medicine, precision medicine

• Innovation in clinical trials

• Data generation and interpretation (knowledge management)

• Prevention, disease interception, patient adherence (incl. societal acceptance of vaccines)

• Effect on medical practice and outcomes (health/disease management)

• Regulatory framework (including pharmacovigilance)

• Patient access

First five big themes (2)

• Prioritisation/selection criteria • Field of unmet need

• Patient-centric approach

• The science appears ready to make a big change over the next decade

• Added value of PPP to make a difference (including collaboration with other industry sectors/technologies)

• Synergies/complementarity with similar initiatives

• While keeping focus on prioritised questions, there is

sufficient room for other projects within the SRA • e.g. Oncology; Rare/Orphan Diseases; Psychiatric Diseases; Respiratory

Diseases

IMI2 - Broad Participation to be able to set ambitious goals:

Bigger budget: >3 Billion Euro, equally shared by EU and industry

Not limited to EFPIA members: open for other industries / companies, which can contribute to the PPP goals (Healthcare IT, medical devices,…)

SME Funding: Better conditions for medium sized SMEs than in IMI1 (companies according to former EU rules to big for funding but too small to be able to afford non-funded participation)

Standardized financial framework: 100% Funding plus 25% overhead, as in general in HORIZON2020

The Role Of The Programme Office

To implement programmes and activities in the common interest of all stakeholders

To monitor the use of public funds and industry investment

To guarantee fair and reasonable conditions for optimal knowledge exploitation and dissemination

To facilitate the interaction between stakeholders, including Intellectual Property agreements

To actively communicate and promote IMI and its activities

A neutral broker:

IMI2: The First Call

Two topics:

Translational approaches to disease modifying therapy of type 1 diabetes mellitus (T1DM)

Discovery and validation of novel endpoints in dry age-related macular degeneration and diabetic retinopathy

Submission date: 12 November 2014

Questions?

Hugh.Laverty@imi.europa.eu

www.imi.europa.eu