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 1

Confidential – Do not distribute

PDUFA PDUFA-V V PDUFA PDUFA-V V

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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

As Presented at the NCCS Cancer Policy Roundtable By Dr. David E. Wheadon October 18-19, 2011 2

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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. 4

Overview of PDUFA-V Proposals Enhanced NME NDA/Original BLA Review Program

FDA Approval

6 months FDA review

NDA/BLA Submission PDUFA Goal

Additional FDA review time (~3 months) 9 months median time to approval (Priority NDA/BLA; FY 2010) 6 months FDA review

NDA/BLA Submission FDA Feedback PDUFA Goal

8 months planned FDA review time (Priority NDA/BLA)

2 months validation

PDUFA-IV PDUFA-V

As Presented at the NCCS Cancer Policy Roundtable By Dr. David E. Wheadon October 18-19, 2011 3

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Overview of PDUFA-V Proposals Enhanced NME NDA/Original BLA Review Program

Total time

(months)

PDUFA clock

2 3 1 4 2 5 3 8 6 7 5

Primary Review Period 60-day Validation Period Advisory Committee Wrap-up

Pre- submission

Priority Application

x PDUFA-V Enhancements Select FDA review milestone x Application receipt

74-day letter Pre-NDA/ BLA meeting a

Internal FDA mid-cycle meeting completed

c Mid-cycle communication post internal FDA meeting

Regulatory Action issued

e

Review completed AC meeting conducted

d

Application filed

b 2 1 3 4 Substantive Late-cycle meeting with signatory authority following completion of primary and secondary reviews

• 2
• 1

6 4 6

Overview of PDUFA-V Proposals Enhancing Regulatory Science & Patient Safety

• Advance development of drugs for rare diseases
• Implement Benefit/Risk framework, including patient-focused

drug development

• Electronic regulatory submissions (eCTD) and data standards
• REMS standardization & Sentinel
• Ensure quality in meta-analysis
• Ensure quality of patient-reported outcomes
• Advance biomarker qualification & pharmacogenomics

Overview of Regulatory Science and Patient Safety Goals

• Enhanced FDA/sponsor communications during drug development

As Presented at the NCCS Cancer Policy Roundtable By Dr. David E. Wheadon October 18-19, 2011 4

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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