Innovative Medicines Initiative: The European response to the innovation challenge in the pharmaceutical sector Michel Goldman, MD, PhD Executive Director

The Productivity Gap in Pharma R&D 60 $55 50 50 45 40 40 35 30 New Drug Approvals Pharma R&D ($ billions) 30 25 20 20 15 10 10 5 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 Source: Burrill & Company; US Food and Drug Administration. 2

The Decline of Pharmaceutical Industry in Europe USA Best selling medicines R&D Sites (2002-2006) 10 20 Closed New 5 10 1992 2005 Source: EFPIA & IFPMA

Innovative Medicines Initiative: Joining Forces in the Healthcare Sector 2 Billion Euro 1 Billion € 1 Billion € Public Private Partnership

Core objectives defined in 2007 To overcome research bottlenecks in drug development through collaborative approaches To increase investments in the biopharmaceutical sector and provide socio-economic benefits across Europe To contribute to the health of European citizens

Key Hurdles in R&D Knowledge fragmentation Insufficient mechanistic understanding Disease heterogeneity Multitude of unvalidated models Lack of predictive biomarkers for drug efficacy/ safety Insufficient pharmacovigilance tools Need for improved manufacturing Late involvement of regulators Insufficient involvement of patients Inappropriate clinical designs Insufficient incentives for industry

Private Investment ‘n kind (€1 billion) A Typical IMI Consortium EFPIA Private Investment ‘n kind (€1 billion) Pharma 4 Pharma 1 Pharma 5 Pharma 2 Pharma 6 Pharma 3 SMALL AND MEDIUM-SIZED ENTERPRISES ACADEMIA EU Public Funding cash (€1 billion) PATIENTS’ ORGANISATIONS HOSPITALS REGULATORS

Key Concepts Non-competitive research for EFPIA companies Competitive calls for IMI beneficiaries Open collaboration in public-private consortia

Science Translational Medicine (2011), 76: 1-2

IMI Executive Office as a Neutral Third-Party Ensures that projects are carried out in the common interest of all stakeholders Guarantees fair conditions for knowledge exploitation and dissemination Facilitates the dialog between industry, patients’ organizations and regulatory agencies

Nature Medicine 18:341, 2012

Intellectual property Facilitation of Call 2 and Call 3 agreements (“honest broker”) IMI Policy recognized as “model” for PPPs Science Translational Medicine 22 February 2012

Overview of IMI projects * Small and medium-sized enterprises

Non-Competitive Research Key Deliverables of Non-Competitive Research Establishment of common databases New tools for identification of drug targets Standardization of models and assays for drug efficacy and safety Patient reported outcomes Classification of diseases

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