Statistics in the FDA and Industry: Past, Present, and Future Organizers: Ken Koury, Estelle Russek-Cohen, Greg Wei Chair: Mohan Beltangady FDA/Industry Workshop September 27-29, 2005, Washington, D.C.

delete these guides from slide master before printing or giving to the client 2 Development Costs Quadrupled in 13 Years Source: Tufts Center for the Study of Drug Development $MM Innovative pharma research is very expensive and is becoming more expensive Annualized growth clinical costs ~ 11.8%, more than 5 times greater than for pre- clinical R&D - PhRMA, 2001

delete these guides from slide master before printing or giving to the client 3 The FDA Perspective FDAs analysis of the Pipeline Problem – issuing Critical Path Initiative. FDA will lead the development of a list of critical path opportunities through extensive consultation with public and private stakeholders. FDA will make internal changes to respond to crucial issues and to support high-priority research efforts.

delete these guides from slide master before printing or giving to the client 4 The Industry Perspective Improve Industry-FDA Interactions Participation in critical path opportunities Development and deployment of disease models Learning and sharing collectively –From positive experience –From failures Understand limitations and improve tools to address development challenges

delete these guides from slide master before printing or giving to the client 5 FDA and Industry We all agree on fundamental premise There is no need for unsafe drugs There is need for innovative, effective therapies that benefit patients There is a need to spend the limited clinical development resources wisely and not expose subjects to failing or less effective drugs And the patients are waiting

delete these guides from slide master before printing or giving to the client 6 Speakers David Salsburg Mary Foulkes David DeMets

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8 Challenges and Opportunities Industry Productivity Hunger for effective new medicines – for prevention and disease management Predictability of pre-clinical models Expectations from Promising new technologies Translational Medicines and biomarkers Genomics, proteomics, metabonomics, -omics Patient and Physician perspective Patient Reported Outcomes Effectiveness studies Evidence-based medicine Payors and Costs -

delete these guides from slide master before printing or giving to the client 9 Information Technologies – We continue to get better at generating lots of data – Very fast With a large enough sample, almost anything outrageous will happen. (Diaconis & Mosteller, 1989, J.Amer.Statist.Assoc.)

delete these guides from slide master before printing or giving to the client 10 Can we Leverage our Skills better? Developing Medicines Treatment Choices and Decisions Discovery: Molecules and Targets Increase our contributions and impact