From genes to jeans : Challenges on the road to Personalized Medicine

1. Challenges for Industry Developers Alicia Granados, MD Head Evidence Definition / Global HTA Global Medical Affairs Genzyme Corporation EPTA Conference October 23th , 2012, Barcelona, Catalonia From genes to jeans : Challenges on the road to Personalized Medicine

2. Outline • The context: Health System’s Pressures and Drivers • Personalize Medicine and Health Technology Assessment • Current Challenges for Industry developers • Final reflections

3. Health Systems under pressure Public expectations ; Socio-demographic changes Changes in patterns of disease and risk factors Changes in scientific knowledge Health Care Policy Makers & Providers Globalisation of information on health care systems Pressures for financial sustainability and transparency Patient Participation Spreading awareness of the need for evidence base decision making.

4. Health Systems under pressure Increased Scientific Scrutiny of Innovations Pricing Pressures “Generic and Biosimilars Threats Unsustainable R &D Models Health Industries Eroded Society Trust Patent Loses Uncertain Pipeline Obsolete Commercial Models Inflexible Cost Structure .

5. INNOVATION IN BIOMEDICAL SCIENCE Molecular Biology (Complex System Biology) Cell, gene and enzyme therapy Tissue engineering Modulators of aging Personalize/Stratified medicine (molecular diagnostic and gene research) Changing paradigm of biomedical and health science research Health Systems drivers (1)

6. Health systems drivers (2) COVERAGE AND PROVISION OF HEALTH CARE • Governance vsManagerialism • Turning doctors into leaders • Quality, Efficiency …but Solidarity as ethical imperatives • Public Private Partnership under the umbrella of Social Responsibility • Rising pressure for HC accountability • Health Technology Assessment (HTA)

7. Health Technology Assessment: current meaning for Health Policy Makers and Providers

8. Health Technology Assessment: current meaning for Health Industry

9. Innovations: Current Realities Innovations which can’t demonstrate that in addition of being scientific novelties, are able to provide relative superior therapeutic added value. compared with already available alternatives , or standards of care, are facing growing difficulties in entering or being maintained in the health care market

10. Higher probability of desired outcome with a drug Low probability of untoward side effects Preventivestrategies Focusedtherapies Potential better health outcomes Need for changing research and healthcare provision paradigm and relationships., Needs for holistic HTA: clinical, economic, social and ethical assessment, including patients views. Scientific challenges (lack of validated molecular markers and consensus on what kind of evidence do we need to prove their value for HTAs? How do we get it? Who should pay for it? Operational issues. Economic challenges (poorly aligned incentives), Ethical dilemmas not systematically addressed Personalized Medicine . Impact / difficulties

11. Personalized Medicine . Impact / difficulties • Genomics tech can generate massive amount of information • Needs for alignment: pharma and diagnostics companies • New business models • The discovery of genes associate with specific diseases provide hopes in the diagnostic and treatment of rare diseases and subpopulations of common diseases • Lack of clarity on how to evaluate clinical validity and utility for decision making • Needs for a new regulatory framework • Health care and Industry “silos” • The global recession has forced governments to reconcile budgetary challenges: Efficiency vs Solidarity?

12. Therapies for Rare Diseases Challenges for Treatment Developers: Patient Access

13. Therapies for Rare Diseases: Challenges for Treatment Developers Industry Receiving Inconsistent Messages from Health Systems on What they Value on Drug Development Plans Impact

14. Therapies for Rare Diseases. Challenges for Treatment Developers: measuring outcomes • Small and geographically dispersed populations present recruitment challenges for clinical trials • Low prevalence limits the ability to perform multiple studies • The heterogeneity of rare diseases pose challenges to uniform treatment paradigms and to study design • The slow progression of disease means measurable effects may take years • Surrogate endpoints are often more apparent than direct clinical outcomes in rare diseases • Regulatory agencies requests not always aligned, neither HTAs • Post Approval Commitments to perform more studies to further understand clinical outcomes of therapies require continuous significant investments

15. Final reflections 1: How to keep Health Care Systems sustainable in a changing scientific and social context? • Avoiding “silo” approach • Effective Colaboration (PPP) • Deliver the highest quality of health care a system can afford. • Vision for integrated care • Social, organizational and policy innovation (not only technological innovation)

16. Final reflections 2: How to keep Health Care Systems sustainable in a changing scientific and social context

17. In the era of networks…. Health Systems sustainability will mostly depend on the degree of health care stakeholders co-responsability Final reflections 3

18. THANKS! alicia.granados@genzyme.com alicia.granados@uab.cat