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Creating the Path for Innovative New Therapies Raymond L. Woosley, MD, PhD

Creating the Path for Innovative New Therapies Raymond L. Woosley, MD, PhD President, The Critical Path Institute. Goals. Overview of the PDUFA Impact Critical Path Initiative Purpose of the Critical Path Institute 4. A model for new relationships 5. Proposal for a PDUFA initiative.

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Creating the Path for Innovative New Therapies Raymond L. Woosley, MD, PhD

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  1. Creating the Path for Innovative New Therapies Raymond L. Woosley, MD, PhD President, The Critical Path Institute

  2. Goals • Overview of the PDUFA Impact • Critical Path Initiative • Purpose of the Critical Path Institute 4. A model for new relationships 5. Proposal for a PDUFA initiative

  3. FDA Review Efficiency Has Increased… * Prior to 1992, therapeutic gain was classified as type A, B, or C (defined below). Starting in 1992 Priority and Standard designation was used to represent therapeutic potential for new drug approvals.

  4. 60 35 30 50 Approvals 25 40 20 30 15 No. of Drugs Approved 20 10 10 5 0 0 1990 1991 1992 1993 1994 1995 1996 1997 1998 1999 2000 2001 2002 Sources: Washington Analysis, LLC and PhRMA for 1990-2000; PhRMA website for 2001-2 Product Approvals Increased Transiently…

  5. New Drug Submissions Increased Transiently… * for NMEs submitted prior to 1992, type A and type B applications are counted as Priority review and type C applications are counted as Standard review.

  6. 10 year Trend in Biomedical R&D Spending US Pharmaceutical R&D Total NIH Budget

  7. 10 year Trend in New Applications to FDA New Drug Applications New Biological Applications

  8. Success Rates Remain Low Nature Reviews: Drug Discovery, 3 (8): 711, 2004

  9. The Critical Path Initiative

  10. CPI calls for Innovations and Collaboration in the Development Phases Discovery Critical Path to Market

  11. Response to CPI Overwhelming support: Industry (Internal CPI task forces) NIH Collaborations (NCI, NHGRI) The Critical Path Institute Academia CDDS and JETS at UCSF MIT Center for Biomed. Innovation ISIS at Indiana Univ. ECG Warehouse at Duke Univ. NIPTE (11 univ. manufacturing partners)

  12. Why “Critical Path Institute”

  13. C-Path Vision and Mission A non-profit, publicly-funded Institute that serves as a “neutral ground” for scientists from the FDA, academia and the pharmaceutical industry to accelerate the development of safe medical products. C-Path will develop tools not drugs

  14. Neutral Ground A proven concept…….. Moffett Center, U. Illinois, Chicago The NCFST is a unique research consortium composed of scientists from academia, the FDA and food-related industries. The Center provides a neutral ground where industry, academia and the FDA scientists address food safety issues.

  15. C-Path Programswww.C-Path.org SMARTER FASTER SAFER

  16. Toxicogenomic Cross-Validation Consortia • Pharmaceutical companies have created innovative tests to predict drug toxicity • Data from these tests cannot be submitted as evidence of safety for new drugs because they have not been “independently validated” • Companies will disclose their methods so they can test and validate one another’s methods • Focus areas: liver, kidney, muscle, nerve • C-Path will gather the data and submit to FDA

  17. Toxicogenomic Cross-Validation Consortia Company • “A” • “B” • “C” • “D” • “E” • “F” • FDA • SRI • UA • TGen • Pharmaceutical Consortium

  18. C-Path: “Neutral Territory” • Basic Principles: • Publicly funded, no direct funding from medical product companies • Industry consortia funding is possible with transparency and oversight • Project Specific consortia funding • Oversight Board • FDA • Industry • Consumer/Patient Representatives

  19. Major Impediments for CPI • Lack of funding for FDA participation • Lack of funding for method development and validation • Lack of process to prioritize and coordinate CPI activities • Lack of “laboratory” for testing new methods, biomarkers, etc

  20. A Proposal to Advance Drug Development: 1. FDA Funding for CPI • Small percent increase in PFUFA fees for FDA/CPI • Match from Congressional appropriations

  21. A Proposal to Advance Drug Development: 2. Funding for Methods Development/Validation • Industry consortia operating with FDA advisors on “neutral ground” • PDUFA grants/contracts for work mutually agreed upon by CPI Steering Committee

  22. A Proposal to Advance Drug Development: 3. CPI Steering Committee Moffett Center Model • FDA representatives • Industry representatives • Consumer/Patient reps • Independent scientists/experts

  23. A Proposal to Advance Drug Development: 4. CPI Testing Environment • Life threatening illnesses • Orphan drug development (Congressionally mandated to assist in development) • Personalized medicine

  24. Summary The regulators and the regulated need “neutral ground” where they can work together to improve the process of drug development. PDUFA could be the catalyst for change.

  25. Thank You!

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