PDUFA V. New Review Program for NME NDAs and Original BLAs. Enhanced Communications with FDA and Sponsors. Beth Duvall

Transcription

1 PDUFA V New Review Program for NME NDAs and Original BLAs Enhanced Communications with FDA and Sponsors Beth Duvall Assoc Director for Regulatory Affairs FDA/CDER/Office of New Drugs September 25,

2 Program for Enhanced Review Transparency and Communication for NME NDAs and Original BLAs ( The Program )

3 The Program: Goals Improve the efficiency and effectiveness of the first cycle review process Decrease the number of review cycles necessary for approval without changing the standards for approval Ensure that patients have timely access to safe, effective, and high quality drugs 3

4 The Program: Basics All NMEs and original BLAs received between 10/1/12 and 9/30/17 Includes resubmissions after RTF; excludes file over protest after RTF Does apply to rolling NDAs/BLAs Does not apply to supplemental applications Applicants must uphold agreements re: complete application and late submission of certain minor components within 30 days Incomplete applications subject to RTF decision Complete and readily located lists of clinical and manufacturing sites expected upon submission 4

5 The Program: Highlights What s New/Different for NME Program reviews? What s the Same for NME Program reviews? 6/10 mo clocks begin at 60d filing date User fees, other than usual yearly increases Agreement on complete application at pre-submission meeting Late submission of certain minor components w/in 30 days of application receipt Mid-cycle communication 60-day filing period RTF and S/P review criteria 74-day letters Late-cycle meeting, including FDA meeting package PMRs/PMCs + labeling discussions 5

6 Shift of the PDUFA Clock Work Time PDUFA Clock Time Submit Filing Primary reviews & consults due Review clock starts at submission PDUFA clock starts at filing date Extends total work time Time for primary and secondary reviews remains the same Time for late cycle activities Issues found during review Issues found during inspections AC feedback 6 6

7 Complete Application An application is considered complete if the initial submission contains all the necessary information to allow for a comprehensive review and decision on the proposed labeling (e.g., indications), the required data to assess the safety of the drug, including any required long-term safety data and pediatric plan, and the information needed to evaluate and approve manufacturing facilities and proposed specifications. 7

8 Late Submission Components These submissions must be of a type that would not be expected to materially impact the ability of the review team to begin its review. 8

9 Mid-Cycle Communication Internal FDA meeting held that is intended to discuss current status of review Communication (telecon) to applicant w/in 14 days of internal meeting that describes: Any significant issues identified; Any information requests; Any major safety concerns or preliminary REMS; Proposed date for late-cycle meeting Updates on AC meeting plans, if planned Other projected review milestone dates Minutes of telecon will be sent to applicant 9

10 Late-Cycle Meetings Meeting with applicant to: share information, plan for AC, and plan for remainder of review FDA attendees include signatory authority FDA briefing package that includes/references: FDA background package for AC mtg*, if planned Memo from review team that outlines substantive issues + AC discussion points Discipline review letters (or date noted if previously sent) Current assessment of REMS or other risk management actions *AC staff will continue to send package 10

11 Late-Cycle Meetings (cont) Potential topics for discussion include: Major application deficiencies to date Issues for AC discussion, if planned Potential for REMS or other risk-management PMRs/PMCs Information requests, including more data Meeting is not: A decisional meeting Intended to resolve disagreements between FDA and applicant 11

12 Inspection Goals Inspections (GCP, GLP, and GMP) complete by: 6 mos (P) of original receipt date 10 mos (S) of original receipt date Additional 2 months allows for time to address deficiencies 12

13 Program Assessments The Program will be assessed by an independent contractor who will evaluate applications as they are being reviewed Eastern Research Group = contractor Interim Assessment (3/2015) Final Assessment (12/2016) Assessments will be published in the Federal Register Public meetings will be held on each assessment to discuss findings and share feedback 13

14 Program Summary Additional 2 months of Program clock intended to: Provide time for greater transparency of review Provide additional time for FDA to complete complex review work (e.g., AC meetings, REMS, PMRs/PMCs, inspectional deficiencies) Improve the efficiency of the first cycle review and decrease the number of review cycles necessary for approval Contractor to assess the quality and efficiency of regulatory review programs 14

15 Program Activities to Date (as of 8/31/13) Actions: 3, all approvals MCCs: 27 LCMs: 12 Positive feedback from industry 15

16 Enhanced Communications Between FDA and Sponsors During Drug Development

17 Enhanced Communication Team (ECT) New CDER/OND-based team Focused on enhancing communication between FDA and industry during drug development Liaison staff Training staff Tasks include: Identifying and disseminating best practices for enhanced communication Developing training programs for review staff and sponsors 17

18 Enhanced Communication Team CDER/OND contact A contact for general questions or jurisdictional questions Secondary contact for facilitating sponsor communication with review division when problems exist Application-specific or scientific questions OND division RPM 18

19 Enhanced Communication Team Examples of general or jurisdictional questions: What is the mailing address for regulatory submissions? Which review division should I submit an IND for an inhaled insulin product? Who should I contact to request a pre-ind meeting for a drug being developed for the treatment of MS? Where on the FDA Web site can I find guidance on electronic submissions? Please consult our Web site resources first 19

20 Enhanced Communication Team Examples of problems communicating with review division Review Division has not responded to a request within a reasonable time frame, or the expected time frame (e.g., response to special protocol assessment request w/in 45 days, response to meeting request w/in 14/21 days) Review Division has not responded within 30 days to a simple or clarifying question or been referred to the formal meeting process Liaison staff is not responsible for providing answers to application-specific or scientific questions 20

21 Enhanced Communication Team When and how to engage the Enhanced Communication Team as communication facilitators: Step 1 allow RPM to respond within reasonable or expected time frame Step 2 if no response, follow-up with division management Step 3 if still no response, contact ECT Include pertinent information in to identify specific communication problem 21

22 Enhanced Communication Team FDA Deliverables: Establish staff by end of FY2013 Best practices (Draft Guidance 2Q FY2015) Training component, internal and external Teach a man to fish 22

23 ECT Activities to Date (as of 8/31/13) 91 inquiries received: General/Jurisdictional: 89 (54 are IND-related questions) Communication Facilitator: 2 Training underway Soliciting best practices for draft guidance 23

24 Resources PDUFA V Web page PDUFA V Goals Letter Enhanced Communication Elizabeth.Duvall@fda.hhs.gov 24