PDUFA PDUFA-V PDUFA PDUFA-V V V Confidential Do not distribute - PDF document

As Presented at the NCCS Cancer Policy Roundtable By Dr. David E. Wheadon October 18-19, 2011 PDUFA PDUFA-V PDUFA PDUFA-V V V Confidential – Do not distribute Overview of PDUFA-V Proposals � Basic structure of the human drug review program, including FDA’s high review standards for safety and efficacy, remains unchanged � New provisions provide FDA with tools to make safe and effective new medicines available to patients in a more efficient, consistent, and timely manner 2 1

As Presented at the NCCS Cancer Policy Roundtable By Dr. David E. Wheadon October 18-19, 2011 Overview of PDUFA-V Proposals � Enhanced review model for New Molecular Entities (NME) � Increases efficiency and predictability � Improves upon current review performance* by enhancing scientific communication and feedback � Includes independent third party assessment of the Program’s effect � User fee funding for Regulatory Science, Expediting Drug Development, and Patient Safety � Advance development of drugs for rare diseases � Advance FDA capabilities for biomarker qualification, pharmaco-genomics, patient-reported outcomes, and meta-analysis evaluations � Promote innovation through enhanced communication � Develop and implement structured benefit-risk framework; patient-focused drug development � REMS standardization and Sentinel � Electronic regulatory submissions (eCTD) and data standards * FY2010: 9/13 months for priority/standard application; ~35% of application approved in first cycle . 3 Overview of PDUFA-V Proposals Enhanced NME NDA/Original BLA Review Program PDUFA-IV 9 months median time to approval (Priority NDA/BLA; FY 2010) Additional FDA 6 months FDA review review time (~3 months) NDA/BLA PDUFA Goal FDA Approval Submission PDUFA-V 8 months planned FDA review time (Priority NDA/BLA) 2 months 6 months FDA review validation NDA/BLA PDUFA Goal Submission FDA Feedback 4 2

As Presented at the NCCS Cancer Policy Roundtable By Dr. David E. Wheadon October 18-19, 2011 Overview of PDUFA-V Proposals Enhanced NME NDA/Original BLA Review Program Priority Application Total time (months) 0 3 4 5 6 7 8 1 2 -2 -1 0 1 2 3 4 5 6 PDUFA clock Pre- 60-day Validation Period Primary Review Period Wrap-up submission Advisory Committee a b c d e Application Application Regulatory Internal FDA mid-cycle Review completed receipt filed Action issued meeting completed AC meeting conducted 1 2 3 4 Pre-NDA/ 74-day Mid-cycle Substantive Late-cycle BLA meeting letter communication meeting with signatory authority following post internal FDA completion of primary and meeting secondary reviews x PDUFA-V Enhancements x Select FDA review milestone 5 Overview of PDUFA-V Proposals Enhancing Regulatory Science & Patient Safety Overview of Regulatory Science and Patient Safety Goals � Advance development of drugs for rare diseases � Advance biomarker qualification & pharmacogenomics � Ensure quality of patient-reported outcomes � Ensure quality in meta-analysis � Enhanced FDA/sponsor communications during drug development � Implement Benefit/Risk framework, including patient-focused drug development � REMS standardization & Sentinel � Electronic regulatory submissions (eCTD) and data standards 6 3

As Presented at the NCCS Cancer Policy Roundtable By Dr. David E. Wheadon October 18-19, 2011 Overview of PDUFA-V Proposals � Basic structure of the human drug review program, including FDA’s high review standards for safety and efficacy, remains unchanged � New provisions provide FDA with tools to make safe and effective new medicines available to patients in a more efficient, consistent, and timely manner 7 4