Implementation of the CTD: CBER

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Implementation of the CTDCBERS Perspective

• May 8, 2001
• Joan Wilmarth Blair, M.A.
• International Affairs Advisor
• CBER/FDA

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International Conference on Harmonisation of
Technical Requirements for the
Registration of Pharmaceuticals for
Human Use
3
International Conference on Harmonisation of
Technical Requirements for the Registration
of Pharmaceuticals for Human Use
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BIOLOGICAL PRODUCTSREGULATED BY CBER
Vaccines
Plasma Derivatives
Allergenic Extracts
Monoclonal Antibodies
Biotech DerivedTherapeutics
BloodComponents
Peptides
Whole Blood
SomaticCell GeneTherapy
Blood Related Devices
Tissues
Xenotransplantation
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Q6B Specifications,Test Procedures
Acceptance Criteria for Biotechnological/ Biolo
gical Products
Scope apply to proteins polypeptides,
their derivatives, products of which they are
components (e.g., conjugates) does not cover
antibiotics, synthetic peptides polypeptides,
heparins, vitamins, cell metabolites, DNA
products, allergenic extracts, conventional
vaccines, cells, whole blood, and cellular blood
components
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BIOLOGICAL PRODUCTSREGULATED BY CBER
Vaccines
Plasma Derivatives
Allergenic Extracts
Monoclonal Antibodies
Biotech DerivedTherapeutics
BloodComponents
Peptides
ICH
Whole Blood
SomaticCell GeneTherapy
Blood Related Devices
Tissues
Xenotransplantation
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CTD application format only applicable to BLAs
for this subset of products?
Vaccines
Plasma Derivatives
Allergenic Extracts
Monoclonal Antibodies
Biotech DerivedTherapeutics
BloodComponents
Peptides
ICH CTD?
Whole Blood
SomaticCell GeneTherapy
Blood Related Devices
Tissues
Xenotransplantation
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BLA - Section 601.2Application for Biologics
Licenses

• Describes content of application
• Describes procedures for filing

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NDA - Section 314.50Content and Format of
Application

• Describes form and content, e.g.,
• Application form
• Numbers of copies (archival copy)
• Index
• Summaries
• Case report tabulations
• Case report forms
• Labeling
• etc.

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The question is NOT Will CBER be accepting the
CTD instead of the BLA?
The question being asked is Will the CTD
application format be applicable to all BLAs?
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Application Format - Regulatory Requirements?

• NDA - Section 314.50
• Describes content of application
• Describes procedures for filing
• Describes format

• BLA - Section 601.2
• Describes content of application
• Describes procedures for filing
• DOES NOT describe format

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Migration from ELA/PLA to BLA Construct
BLA reqments and Form 356h format

• ELA/PLA
• Reqments

Issue guidance for content format for
product categories
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BLA/Form 356h ? Issuance of Guidances

• 8/96 Therapeutic rDNA-derived products and
  monoclonal antibody products for in vivo use
• 1/97 Autologous somatic cell therapy products
• 1/99 Vaccines related products
• 2/99 Plasma derivatives, animal plasma,
  serum-derived products
• 3/99 Biological in-vitro diagnostics
• 4/99 Allergenic extracts or allergen patch
  tests
• 5/99 Human blood blood components

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Formatting of BLA to CTD Construct
CTD BLA
Revise guidance for new format
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CBERs Acceptance of CTD

• Question of acceptance of CTD format prior to
  issuance of guidance
• Use of CTD format not precluded in advance of
  guidance
• URGE early communication -- assure all required
  content included in submission

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CBERs CTD Implementation Process

• Revise CMC guidances to map CTD to BLA
• Specified Products currently in process
• Staff training
• Workshops likely

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Integration of CTD with Applications for Product
Approval