1. The ESFRI context. Clinical Trials as Instruments supporting Evidence-based Medical Practice. Dr Rafael de Andres Medina, Instituto de Salud Carlos III rdam@isciii.e s www.isciii.es, DC Day 3, Wednesday, Embassy of France to the United States of America

2. An overview of the context Washington, DC 4 - 6 June 2012 2 RDAM Clinical Trials as Instruments Supporting Evidence-based Medical Practice

3. 2006 2008  Should provide relevant scientific services.  Promote continuous innovation.  Business Plan.  Sustainability ensured by self committed member states.  Predictable Governnance.  Science is scientists´ driven.  Fair access (no self service).  External advisory Board - helicopter view.  Financing and administration is member state driven.  Used to have each own legal personnality.  If ERIC, awarded by the European Commission, VTA exempt. Washington, DC 4 - 6 June 2012 RDAM ] Biological and Medical Sciences [BMS] Research Infrastructures [RI]  ESFRI - SWGs assess the implementation and performance 3

4.  BBMRI - Biobanks Spain: ISCIII as funder with 63 biobanks´ network as Scientific partners  EATRIS - Translational Research Facilities Spain: ISCIII as funder and Health Research Institutes´ network as Scientific partners  ECRIN - Clinical trial platform Spain: ISCIII as funder with 40 CRO as Scientific partners  ELIXIR - Bioinformatics Spain: ISCIII as funder with and INB as Scientific partner  INFRAFRONTIER - Mouse Models and Archives  INSTRUCT - Structural Biology Facilities  EMBRC - Marine Biology Resources  ERINHA - High-security labs Spain: ISCIII as partner  EuroBioImaging - Cellular and Medical Imaging  EU-Openscreen - Chemical Libraries and Screening  ANAEE - Analysis and experimentation on ecosystems  ISBE  ISBE – Infrastructure for Systems Biology  EMBRC - European Marine Biological Resource Centre  MIRRI  MIRRI – Microbial resources 2006 2008 2010 ] Biological and Medical Sciences [BMS] Researhc Infrastructures [RI] RDAM Washington, DC 4 - 6 June 2012 4

5.  Health & Food.  Enviromental & Climate Change.  Energy.  Social & Cultural Innovation.  Innovation Technology Transfer.  RegionaI.  Evaluation.  Implementation. Washington, DC 4 - 6 June 2012 RDAM Strategic Working Groups [SWGs] 5

6.  Investment in independent clinical trials brings major added value for European R&D, health care systems, patients and citizens. Washington, DC 4 - 6 June 2012  Policy makers´s and public understanding and effective support. Need for independent clinical trials. Need for multinational collaboration. 6 RDAM

7. Washington, DC 4 - 6 June 2012 It applies not only for medicinal products, but also for medical devices. Other non-pharmacological interventions, e.g. rehabilitative procedures and surgical techniques, require comparative clinical trials too.  e.g. Relevant in Multi-morbidity / poly-pathology of ageing populations that will require therapeutic combinations.  The goal of evidence-based medicine. 7 RDAM

8. Washington, DC 4 - 6 June 2012  Basic sciences, particularly the new –omics methods, are leading to an important change in the nomenclature of diseases. What was considered a single disease is now dissected into various strata.  Prevention and predictive medicine. 8 RDAM Relevat for distinct diagnostic and prognostic features.

9.  Need for independent clinical trials. Independent evaluation of health interventions is required to support health authorities, develop health care policies and define clinical guidelines for healthcare professionals. “what is the best treatment option for this patient / disease ?” rather than “is this particular product effective and safe?” Highly positive return on investment for society.  Decrease in the burden of disease,  Optimized health care strategies,  Cost containment in health care systems. Independent evaluation of health interventions is required to support health authorities, develop health care policies and define clinical guidelines for healthcare professionals. “what is the best treatment option for this patient / disease ?” rather than “is this particular product effective and safe?” Highly positive return on investment for society.  Decrease in the burden of disease,  Optimized health care strategies,  Cost containment in health care systems. Washington, DC 4 - 6 June 2012 “what is the best treatment option for this patient / disease ?” rather than “is this particular product effective and safe?”. “what is the best treatment option for this patient / disease ?” rather than “is this particular product effective and safe?”.  To get robuster evidence. 9 RDAM

10.  Need for multinational collaboration. Rapid assessment of the effects of treatments.  Large and diverse populations.  Wider applicability of study results.  More successful adoption in national health care systems.  Reduce inequalities in health care.  Promotes evidence-based medical practice. Access to larger patient populations and to clinical expertise.  To study of stratified and personnalised treatment strategies.  To study on rare diseases / conditions.  Access to best appropriate medical centres. Rapid assessment of the effects of treatments.  Large and diverse populations.  Wider applicability of study results.  More successful adoption in national health care systems.  Reduce inequalities in health care.  Promotes evidence-based medical practice. Access to larger patient populations and to clinical expertise.  To study of stratified and personnalised treatment strategies.  To study on rare diseases / conditions.  Access to best appropriate medical centres. Washington, DC 4 - 6 June 2012  Room for more regulatory collaboration too. 10 RDAM

11. We need a common vision and a shared will. Washington, DC 4 - 6 June 2012 11 RDAM Many thanks for your attention !!!