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U.S. Food and Drug Administration. Notice: Archived Document The content in this document is provided on the FDA’s website for reference purposes only. It was current when produced, but is no longer maintained and may be outdated. . The Route to Approval. Mid-Atlantic Bio

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u s food and drug administration
U.S. Food and Drug Administration

Notice: Archived Document

The content in this document is provided on the FDA’s website for reference purposes only. It was current when produced, but is no longer maintained and may be outdated.

the route to approval

The Route to Approval

Mid-Atlantic Bio

Robert A. Yetter, Ph.D.

Associate Director for Review Management

Center for Biologics Evaluation and Research

Food and Drug Administration

booz allen hamilton
Booz Allen Hamilton

Booz Allen Hamilton

“Independent Evaluation of

FDA’s First Cycle Review Performance –

Retrospective Analysis Final Report”

January 2006


issues may be raised early but responsiveness is needed
Issues may be raised early but…responsiveness is needed
  • 71% of major deficiencies are identified at a pre-submission meeting but not resolved by first action
    • Lack of clarity on severity?
    • Belief that issue can be resolved during review?
    • Unwillingness of sponsor to comply and/or postpone submission of application?
    • Desire for a comprehensive review of application and identification of other deficiencies?
responsiveness to issues cont
Responsiveness to issues (cont.)
  • Alternative pathways are a possibility!
    • Modification or unbundling of indication
    • “Can this trial be saved?
  • Need early and open discussions on acceptable resolution pathways from FDA and sponsors
interestingly enough
Interestingly enough…
  • Compliance with FDA recommendations more critical for CMC deficiencies
  • Alternative pathways, including postmarketing study commitments, appear to be more of an option for non-CMC deficiencies
  • Note: Additional policies under development for PMC and PMR development and tracking
does it matter when you submit timing of submissions
Does it matter when you submit? Timing of submissions
  • 2-3x the number of submissions in the 4th quarter compared to any other quarter in a calendar year
  • 4th quarter applications have the lowest rate of first-cycle approval without any apparent differences in application quality
    • FDA workload?

Note: anecdotally, FDA staff believe that 4th quarter applications are of lower quality

what reviewers are saying
What Reviewers are saying:
  • Do your homework – use Guidance Documents
  • Hire consultants if you do not have in-house expertise – do not ask FDA to develop your program
  • Science, not marketing, should drive your program
avoid rookie mistakes
Avoid “Rookie Mistakes”
  • Failure to submit a complete application
    • If you have to ask “Can we submit the [fill in the blank] section of the application [fill in the length of time] after we submit the application?” you are not ready
    • Don’t submit your application prematurely according to corporate targets – multiple review cycles are NOT faster
avoid rookie mistakes1
Avoid “Rookie Mistakes”
  • Insufficient attention to details related to electronic submissions
  • Not following advice or addressing a concern
  • Persisting disagreements over issue resolution  approval delays
  • Delay or avoid meeting with FDA
use meetings wisely
Use Meetings Wisely
  • Follow the Guidance for Industry, Formal Meetings with Sponsors and Applicants for PDUFA Products (May 2009)
    • http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM153222.pdf
  • End-of-Phase 2 meetings are the most important
    • Pre-submission meetings are too late to fix the big problems
  • Pay attention to what is being said
    • Be aware of “regulatory speak”
    • Understand and consider our perspective
for a successful meeting do
For a Successful Meeting - Do
  • Provide a brief background summary
  • Assume the meeting package has been read
  • Show how study fits overall development plan
  • Focus on questions
  • Limit your presentation
  • Summarize agreements/disagreements
  • Bring hardcopy of slides for RPM
please don t
Please, Don’t -
  • Regurgitate the meeting package
  • Expect FDA to guess your critical issues
  • Request a pre-BLA before pivotal trial results shown to demonstrate efficacy and safety
  • Present issues outside proposed agenda
  • Send new data just before meeting
  • Expect evaluation of new data presented at meeting
after the meeting
After the Meeting -
  • Review agreements
  • Request FDA minutes of meeting
  • Notify FDA of any differences
  • Follow through
we re here to help you
We’re Here to Help You!
  • CBER’s Office of Communication, Outreach and Development at : 301-827-2000
  • Contact me via phone at:


  • Or contact me via e-mail at:

[email protected]

  • CBER’s webpage: http://www.fda.gov/BiologicsBloodVaccines/default.htm
  • Contacting CBER: http://www.fda.gov/AboutFDA/CentersOffices/CBER/ucm106001.htm