Public Private collaborations to optimise translational research and pathways to patients: IMI Magda Chlebus, Director Science Policy Warsaw, 22 May 2015

EFPIA  Aim of the European Federation of Pharmaceutical Industries & Associations is to promote pharmaceutical discovery and development in Europe and to bring to the market medicinal products in order to improve human health worldwide.  EFPIA’s represents the pharmaceutical industry operating in Europe. Its direct membership includes 33 national associations and 41 leading companies. Two specialised groups within EFPIA represent vaccine manufacturers – Vaccines Europe, with 17 member companies) and European / emerging bio-pharmaceutical companies – EBE with +/- 50 member companies.  “Partners in Research” is constituted of non-pharma companies that collaborate in the IMI public-private membership. This constituent entity, created in June 2014, counts 5 members. EFPIA - Decembe r

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Evolution: science and technology  Science more exciting and promising than ever  More diverse R&D toolbox  Life science integrates more disciplines every day

Evolution: the industry (-ies?)  From vertical integration to externalisation and open collaboration  From market access to patient access and outcomes  From products to therapies  From pharma only/mainly to ecosystems where we do not always hold the keys of success

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.’

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Complementary investments: NIH and private sector in biomedical research 8 Product Idea Basic Research Translational research Clinical research U.S. Private Sector: $51 B National Institutes of Health: $31 B Tufts Center for the Study of Drug Development, Jan, 2015

IMI – Europe’s partnership for health > €5 bn Partnership €2.5 bn

Essential features for research and policy agendas  Public private partnership  Companies and public partners work together  Industry cost is not reimbursed: it is our in kind contribution  Public partners (including companies up to 500 mio turnover) cost is reimbursed by EU: grants for collaborating with industry  Industry defines the research agenda and projects  Beyond the shelf – impact on research, regulatory and medical practice  Managed by a neutral broker that allows participation of authorities and patients  “Institutionalized” – legitimacy of direction and outputs 10

IMI2 Strategic Research Agenda

Strategic Research Agenda: reduced attrition, faster patient access, improved outcomes

First five big themes Therapeutic Areas and Cross-cutting Themes  Neuro-degeneration Successfully prevent and treat dementia and other neurodegenerative diseases  Prevention and treatment of immune- mediated disease Advance immunological understanding to deliver new medicines and new and better vaccines  Metabolic disorders Tackle all phases of disease and its complications, including prevention and early interception  Infection control Address big societal problem related to multidrug resistance and develop new and better vaccines  Translational Safety 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 (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

New Big Challenges under consideration Real World Evidence and for Better Outcomes Advanced Therapies “industrialisation” Biomarker Strategy One Health TITLE OF THE POWERPOINT 14

Topic definition phase Typical IMI project life cycle Negotiation phase Stage 1 Identification of topics and willingness to collaborate by industry partners Signature of Project Agreement and Grant Agreement Submission of proposals by public consortia & evaluation by independent experts Patients’ organisations Academic research teams Regulators Hospitals SMEs Mid-size enterprises Industry consortium Stage 2 Preparation of full proposal & evaluation by independent experts/ethical panel industry consortium Applicant consortium Call launch Selected team merges with industry Definition of contractual terms Project launch!

Over researchers 59 public-private consortia An international, cross-sector community

Programme across products lifecycle New Drugs for Bad Bugs programme Topic 1: COMBACTE a) CT networks and CT design b) Clinical Development of Antibiotics Topic 2 : TRANSLOCATION Research penetration and efflux Gram- negatives Data Hub and Learning from R&D experience Topic 4: DRIVE AB Driving re- investment in R&D and Responsible use of Antibiotics Topic 5: Clinical development of antibacterial agents for Gram-negative antibiotic resistant pathogens Topic 6: Systemic Antibacterials against hospital acquired infections Topic 7: Inhalational Antibacterials in Cystic Fibrosis and COPD DISCOVERYACCESS DEVELOPMNENT CT NETWORKS Topic 3 : ENABLE Discovery & development of new drugs combatting Gram–negative infections DEVELOPMENT CT NETWORKS ND4BB Information Centre – All data generated is submitted and is accessible to all consortium partners

It works! IMI delivers on Innovation and Health - a few examples:  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)

IMI2 vision: European Partnership for Health  Addressing healthcare priorities identified by the WHO 2013 report  Strategic Research Agenda aimed at progressing the vision of stratified medicines: prevention, treatment and health management  Entire product 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, …

Need more information? EFPIA Science Policy Team Mail: IMI office Mail: National Contact Point