FDA Modernization Act of 1997 Workshop

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FDA Modernization Act of 1997 Workshop

• Im From the FDA and Im Here to Help You and
  Me
• Kathryn C. Zoon, Ph.D., CBER
• San Francisco, CA
• March 4, 1998

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General Areas of Cooperation to Ensure Successful
Implementation of FDAMA

• Development of Regulations and Guidance Documents
• Implementation of PDUFA II
• Implementation of BLA and Fast-Track
• International Harmonization

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FDAMA REGULATION AND GUIDANCE DOCUMENTS

• Participate in the FDA Workshops and Public
  Meetings, e.g. radiopharmaceuticals, Biologics
  License Regulation
• Send in your comments to the dockets on the
  various guidances and regulations to maximize
  your input
• Invite us to your workshops to discuss important
  issues regarding these initiatives

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CBER LEAD IN FDAMA REGULATION/GUIDANCE
INITIATIVES

• RADIOPHARMACEUTICALS-New requirements for review
  of Radiopharmaceuticals (122) PR-May 20, 1998,
  FR-May 20, 1999
• POSTMARKETING STUDIES-revise 314 and 601 to
  require annual progress reports (130)
• BIOLOGICS MODERNIZATION-regulations for BLAs
  (123)
• FAST TRACK-policies and procedures on fast track
  products (112) Nov. 21, 1998

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CBER LEAD IN OTHER FDAMA ACTIVITIES

• Postmarketing Studies-Publish Status Annually
  (130)
• Fast Track disseminate to physicians and others
  a description of the provisions applicable to
  fast track products (112)
• Modernization of regulation MAPP/SOP to
  minimize differences between CDER and CBER review
  and approval (123)
• PDUFA II Implementation CBER/CDER co-lead

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IMPLEMENTATION OF PDUFA II

• GROUP A
• Review Performance
• Discipline Reviews/Informational Request Letters
• Two Level Resubmissions Class 1 and 2
• GROUP B
• Meeting Management
• GROUP C
• Clinical Hold Responses
• GROUP D
• Major Dispute Resolution
• Special Protocol Agreements
• GROUP E
• Electronic Submissions

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SUGGESTIONS FOR GROUP A

• Take advantage of meetings with CBER
• Prepare a clear, well-organized submission
• Use ICH and FDA Guidance Documents
• Use new BLA/NDA Form 356h
• Use common Technical Document Format/Content
  under Document (ICH)
• Be ready for inspection when the BLA is submitted
• Maximize efficiency by submitting complete
  responses
• Limit calls to reviewers to only those necessary

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SUGGESTIONS FOR GROUP B MEETING MANAGEMENT

• Direct all meeting requests to Application
  Division of appropriate office
• Schedule meetings when enough information is
  available to make the discussions meaningful
• Work with the assigned CBER Point of Contact
  regarding the meeting
• Justify the urgency for emergency meetings
• Schedule separate meetings for manufacturing,
  clinical, and or establishment if an agenda has
  extensive discussion of more than one topic the
  meetings can be consecutive with short breaks for
  change of staff

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SUGGESTIONS FOR GROUP B MEETING MANAGEMENT

• Meeting packages including adequate of copies
  should be submitted as soon as possible once the
  meeting is scheduled for an adequate review -
  they should be received not less than two weeks
  for Type A and C meetings and one month for Type
  B meeting
• Formal presentations should be limited to allow
  time to focus on questions/issues that need to be
  addressed

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SUGGESTIONS FOR GROUP B MEETING
MANAGEMENT(Continued)

• Telephone or Video Conferences may be held in
  lieu of face-to-face meetings
• A meeting summary will be available on request to
  the sponsor within 30 calendar days suggest you
  get a copy
• Sponsor may submit to CBER a memo summarizing
  their understanding of the issues discussed
  suggest you give us a copy

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SUGGESTIONS FOR GROUP C CLINICAL HOLD RESPONSES

• Please designate in correspondence when the
  response is a complete response (cover letter)
• To maximize the agencys efficiency and decision
  making in the initial package, include all the
  information needed for us to make a decision to
  allow a trial to proceed

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SUGGESTIONS FOR GROUP D

• Please try to resolve disputes in the supervisory
  chain first
• CBERs ombudsperson is currently David Feigal
• Please ensure protocols are complete and
  well-designed from a pre-clinical, clinical
  and/or statistical perspective and adequate
  information is given to the agency to efficiently
  assess

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SUGGESTIONS FOR GROUP E ELECTRONIC SUBMISSIONS

• For now-please submit electronic submission
  concurrenly with hard copy
• Please use the agency standards for electronic
  submissions we do not accept customized CALAs as
  of 8/97
• CBER will issue three guidance documents on
  electronic submissions soon and will have a
  public workshop this Spring with industry
• Eight to ten months before submission date meet
  with staff to discuss structure and content of
  electronic submission

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SUGGESTIONS FOR GROUP E ELECTRONIC SUBMISSIONS

• Four to six months before the submission, arrange
  a demonstration of the structure and content of
  the planned submission for the staff
• 30 days before submission, provide a mock-up
  version of the electronic application for testing
  on our networks and a letter confirming the
  planned submission date
• On day of submission, provide all the
  certifications and information requested
• Please check with docket 92-0251 which lists
  which documents we will receive without paper

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IMPLEMENTATION OF THE BLA

• The proposed Regulation will be out shortly,
  please comment
• A workshop will be held during the comment period
  
• Roll out of the BLA new form 356h is available
  and release of CMC Guidance documents for each
  product class is in progress

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IMPLEMENTATION OF BLA

• Currently available Guidances
• In Final
• For the Submission of Chemistry, Manufacturing
  and Controls and Establishment Description
  Information for Autologous Somatic Cell Therapy
  Products
• For the Submission of Chemistry, Manufacturing,
  and Controls Information for Synthetic Peptide
  Substance
• For the Submission of Chemistry, Manufacturing,
  and Controls Information for a Therapeutic
  Recombinant DNA-Derived Product or a Monoclonal
  Antibody Product for In Vivo Use

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IMPLEMENTATION OF BLA(Continued)

• In Draft
• For the Submission of Chemistry, Manufacturing
  and Controls and Establishment Description
  Information for Human Plasma-Derived Biological
  Products or Animal Plasma or Serum-Derived
  Products
• For the Submission of Chemistry, Manufacturing,
  and Controls Information for Synthetic Peptide
  Substance (revision of currently available
  guidance)

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IMPLEMENTATION OF BLA(Continued)

• Coming Soon as Draft for Comment
• For the Submission of Chemistry, Manufacturing
  and Controls and Establishment Description
  Information for a Vaccine or Related Product
• For the Submission of Chemistry, Manufacturing
  and Controls and Establishment Description
  Information for a Biological In Vitro Diagnostic
  Product

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IMPLEMENTATION OF BLA(Continued)

• For the Submission of Chemistry, Manufacturing
  and Controls and Establishment Description
  Information for Limulus Amebocyte Lysate
• For the Submission of Chemistry, Manufacturing
  and Controls and Establishment Description
  Information for Allergenic Products
• For the Submission of Chemistry, Manufacturing
  and Controls and Establishment Description
  Information for Blood and Blood Components

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IMPLEMENTATION OF FAST-TRACK

• Is available for use now, the agency is working
  very hard on policies and procedures so as to
  minimize confusion (Spring release)
• Suggestions for implementations what would be
  helpful to you?
• Identify areas where you would like the agency to
  focus with respect to surrogate endpoints

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INTERNATIONAL HARMONIZATION

• Get more involved in international harmonization
  efforts
• Harmonize prospectively in many of the new
  biotech areas
• Support FDA science-based decision making and
  research to make effective and efficient policy
  decision