Innovative Medicines Initiative: EU Partnership for Health

EARLIER ACCESS TO INNOVATION

Richard Bergström EFPIA Director-General

ISPOR Europe, Amsterdam, 2014

Pipelines are full – patients are waiting

Product Company Lead therapy area Pharmacology class NPV ( $bn)

Palbociclib Pfizer Breast cancer CDK 4 & 6 inhibitor 8.67

Evolocumab Amgen Hyperlipidaemia Anti-PCSK9 MAb 5.44

Interferon-free combo AbbVie Hepatitis C PNSD3/4A- p1rotesa sem & NSa5kA ienhibi wtor ay for t5.2h7 e next wave of blockbuster launches

Brexpiprazole Otsuka Holdings Schizophrenia, depression 5-HT1A & D2 agonist & 5-HT2 antagonist 2.64

Cosentyx Novartis Psoriasis Anti-IL-17A MAb 2.50

Toujeo Sanofi Type I and II diabetes Insulin analogue 2.26

RLX030 Novartis Congestive heart failure RXFP agonist 2.23 V503 Merck & Co Cervical cancer prophylaxis AHPV vaccine 2.17 Asfotase Alfa Alexion Pharmaceuticals Hypophosphatasia Alkaline phosphatase enzyme therapy 2.16

Lynparza AstraZeneca Ovarian cancer PARP inhibitor 2.11

Lenvatinib Eisai Thyroid cancer VEGFr tyrosine kinase inhibitor 1.93

Natpara NPS Pharmaceuticals Hypoparathyroidism Parathyroid hormone 1.29

Blinatumomab Amgen Acute lymphocytic leukaemia Anti-CD19 MAb 1.25

Farydak Novartis Multiple myeloma Pan-deacetylase inhibitor 1.09

Aripiprazole lauroxil Alkermes Schizophrenia 5-HT1A & D2 agonist & 5-HT2 antagonist 1.09

Product Company Lead therapy area Pharmacology class NPV ( $bn)

Kalydeco + lumacaftor Vertex Cystic fibrosis CFTR corrector 13.83

LCZ696 Novartis Heart failure AT1 antagonist & neprilysin inhibitor 12.45

Obeticholic acid Intercept Liver diseases FXR agonist 7.84

Source Evaluate Pharma report Fovista Ophthotech Macular degeneration Anti-PDGF-B aptamer 6.19

MPDL3280A Roche Lung cancer Anti-PD-L1 MAb 5.41 MEDI4736 AstraZeneca Lung cancer Anti-PD-L1 & CTLA4 MAb 5.33

PB272 (neratinib) Puma Biotechnology Breast cancer pan-HER inhibitor 4.81

MK-5172/MK-8742 Merck & Co Hepatitis C NS3/4A & NS5A inhibitor 3.87

AZD9291 AstraZeneca Lung cancer EGFR inhibitor 3.55

Bosatria GlaxoSmithKline Asthma IL-5 MAb 3.09

Abemaciclib Eli Lilly Breast, lung cancer CDK 4 & 6 inhibitor 3.02

Dupilumab Sanofi Eczema/dermatitis IL-4 & IL-13 antagonist 2.97

Selexipag Actelion Pulmonary hypertension PGI-2 receptor agonist 2.36

Dengue vaccine Sanofi Dengue fever Dengue fever vaccine 2.35 HEALTH & GROWTH 2

1 A revolutionary period in R&D

 Wealth of Data  High-speed, low-cost genomic sequencing  Increased availability of human tissue samples  Increased access to clinical trial data, and electronic medical records  New Technologies  Stem cells, cell and gene therapy, RNAi…  Advance in medical devices, imaging, robotics  M-Health  Increased input of health stakeholders  Doctors, Patients, Regulators

Two Pillars to Reinvent R&D

Translational Medicine Networked Innovation

Based on Outstanding Science

2 Innovative Medicines Initiative: EU Partnership for Health

2014 – 2024 €3,276bn Public/Private = EU + Pharma + Other sectors

3 IMI projects

The MAPPs "Ecosystem”

Continuously expanding pre- and post- approval data sets

Adaptive HTA Patient Centric and benefit/risk INPUT: reimbursement assessment OUTPUT:

Multiple More Sources MAPPs timely of Data access to Obtained needed Responsive new Real Time Prescription clinical trial practice design medicines

Modeling of impact on business and society

4 Strategic Research Agenda

Comprehensive framework for a 10-year programme

Prepared with input from 80+ organisations (internet and targeted)

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

http://goo.gl/jqMP9g

IMI2 scientific programme: First five big themes

Therapeutic Areas and Cross-cutting Themes Differentiating Enablers for all themes

1. Neuro-degeneration Towards early and effective patient access Prevent and treat dementia and other neurodegenerative to innovative prevention and treatment diseases solutions (MAPPs): 2. Immune-mediated disease • Target validation based on human biology Advance immunological understanding to deliver new • Stratified medicine, precision medicine medicines and new and better vaccines • Innovation in clinical trials 3. Metabolic disorders • Data generation and interpretation (knowledge Tackle all phases of disease and its complications, including prevention and early interception management) 4. Infection control • Prevention, disease interception, patient adherence (incl. societal acceptance of Multidrug resistance (including antimicrobials, antivirals, vaccines) vaccines) and develop new and better vaccines 5. Translational Safety • Effect on medical practice and outcomes (health/disease management) Predictors of safety and development of point of care for safety biomarkers • Regulatory framework (including New human biology platform to predict toxicity and safety pharmacovigilance) during early drug development • Patient access

All about adaptive pathways to patients!