New Tools & Pathways. Raymond L. Woosley, MD, PhD President and CEO Critical Path Institute

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1 -Diagnostic Codevelopment for Tuberculosis New Tools & Pathways Raymond L. Woosley, MD, PhD President and CEO Critical Path Institute

2 Acknowledgements FDA, Office of Critical Path Initiatives Rachel Behrman, MD Leonard Sachs, MD FDA, Center for Evaluation and Research Janet Woodcock, MD C-Path Jeff Cossman, MD Anthony, PhD Maryellen demars, PhD Marietta

3 Critical Path Institute A non-profit, c0-founded by the FDA and Arizona for the sole purpose of serving as a neutral third party to work on the FDA s critical path initiative. Neutral Funding Pre-competitive Focus: Process, not products FDA Acceptance of new methods

4 C-Path s Goal To help create the infrastructure and the FDA-qualified methods so that.. (As with HIV/AIDS) When a new, important treatment or cure is found, it can be developed in less than three years with a >95% chance of success. Not 15 years with a 5% chance of success.

5 It is possible AIDS Development Zidovudine (AZT) Didanosine (DDI) Zalcitabine (DDC) Stavudine (D4T) 3TC Saquinavir Ritonavir Indinvair Mean IND to NDA (yrs) years NDA to Approval (mos) months

6 New Pathways for Change VXDS - Voluntary X Data Submission Genomic Data Warfarin dosing Biomarker Data Preclinical Safety BQR - Biomarker Qualification Review Quantitative Disease Progression Models

7 Biomarker Qualification Predictive Safety Testing Consortium (PSTC) Planning Phase Execution Phase FDA Review Phase Coordinating Committee Planning & Writing Groups Work Methods FDA FDA Working Scope & Results Submission Review Groups Document Sharing VXDS BQR Qualified Preclinical Safety Tests Greater Safety

8 PSTC Members Advisors: FDA, EMEA

9 An International Sharing Endeavor PSTC Convenes 190 Scientists Every 2 Weeks

10 Kidney Working Group Progress Creatinine & BUN do not detect subtle drug injury Twenty three new kidney biomarkers: Extremely Sensitive Seven had excellent data for submission to FDA and EMEA

11 FDA Decision: Biomarkers Qualified

12 EMEA Decision: Biomarkers Qualified

13 Biomarker Qualification Predictive Safety Testing Consortium (PSTC) Planning Phase Execution Phase FDA Review Phase Coordinating Committee Planning & Writing Groups Work Methods FDA FDA Working Scope & Results Submission Review Groups Document Sharing VXDS BQR Qualified Preclinical Safety Tests Greater Safety

14 Model-based Development

15 Clinical Trial Simulation Applications in 2008 End of Phase 2A meetings Labeling Claims Pediatric approvals (Trileptal) Sub-group approvals (Zoledronate) REMS (Busulfan, peds) Approval Calcium channel blocker Larry Lesko, PhD, CDER, 2008

16 FDA s Disease Models Non-small cell Lung Cancer Alzheimer s Disease Parkinson s Disease Based on NDA data - Not current Limited Value

17 Critical First Step: Data Stds Clinical Data Interchange Standards Consortium (CDISC) CDISC Standard Data Elements for Tuberculosis

18 Global consensus FDA s Critical Path & IMI recommendations: Support of collaborations with transparency Sharing placebo data Establishing disease models Focus on a major disease with unmet need Include regulatory agencies in design Share failures Larry Lesko, PhD, CDER, 2008

19 Development Candidate Development Path Proof of Concept Proof of Clinical Utility (Clinical Trials) Biomarker Assay? Diagnostic Development Path

20 Development Candidate Development Path Proof of Concept Proof of Clinical Utility (Clinical Trials) Biomarker Where do we use these new tools? Assay? Diagnostic Development Path

21 Diagnostic Co-development Candidate Development Path Proof of Concept Proof of Clinical Utility (Clinical Trials) Biomarker DATA SHARING Disease Progression Model (In Silico) Assay? Companion Diagnostic Development Path

22 Diagnostic Co-development Candidate Development Path Proof of Concept Proof of Clinical Utility (Clinical Trials) Biomarker DATA SHARING Disease Progression Model (In Silico) Assay? Companion Diagnostic Development Path

23 Diagnostic Co-development Candidate Development Path Proof of Concept Proof of Clinical Utility (Clinical Trials) Biomarker DATA SHARING Disease Progression Model (In Silico) ASSAY STANDARDS Assay USDS Performance Certification (Laboratory) Companion Diagnostic Development Path

24 Diagnostic Co-development Candidate Development Path Proof of Concept Proof of Clinical Utility (Clinical Trials) Biomarker DATA SHARING Disease Progression Model (In Silico) Assay ASSAY STANDARDS USDS Performance Certification (Laboratory) Indication of Clinical Utility (Based on M&S) Companion Diagnostic Development Path

25 Diagnostic Co-development Candidate Development Path Proof of Concept Proof of Clinical Utility (Clinical Trials) Biomarker DATA SHARING Disease Progression Model (In Silico) Assay ASSAY STANDARDS USDS Performance Certification (Laboratory) Indication of Clinical Utility (Based on M&S) Companion Diagnostic Development Path

26 Diagnostic Co-development Candidate Development Path Proof of Concept Proof of Clinical Utility (Clinical Trials) Biomarker DATA SHARING Disease Progression Model (In Silico) FDA Approved STRATEGY Assay ASSAY STANDARDS USDS Performance Certification (Laboratory) Indication of Clinical Utility (Based on M&S) Companion Diagnostic Development Path