Implementation of the CTD: CBER’S Perspective
May 8, 2001
Joan Wilmarth Blair, M.A.
International Affairs Advisor CBER/FDA
C B E R
International Conference on Harmonisation of Technical Requirements for the Registration of Pharmaceuticals for Human Use
International Conference on Harmonisation of Technical Requirements for the Registration of
Pharmaceuticals for
Human Use
BIOLOGICAL PRODUCTS REGULATED BY CBER
Plasma Derivatives Vaccines Monoclonal Antibodies Biotech Derived Therapeutics
Allergenic Extracts
Blood Components Whole Blood
Blood Related Devices
Peptides
Somatic Cell & Gene Therapy Xenotransplantation
Tissues
Q6B: Specifications,Test Procedures & Acceptance Criteria for Biotechnological/ Biological 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”
BIOLOGICAL PRODUCTS REGULATED BY CBER
Plasma Derivatives Vaccines Monoclonal Antibodies Biotech Derived Therapeutics
ICH
Allergenic Extracts
Blood Components Whole Blood
Blood Related Devices
Peptides
Somatic Cell & Gene Therapy Xenotransplantation
Tissues
CTD application format only applicable to BLAs for this subset of products?
Plasma Derivatives Vaccines Monoclonal Antibodies Biotech Derived Therapeutics
ICH CTD?
Allergenic Extracts
Blood Components Whole Blood
Blood Related Devices
Peptides
Somatic Cell & Gene Therapy
Tissues
Xenotransplantation
BLA - Section 601.2 Application for Biologics Licenses
Describes content of application Describes procedures for filing
NDA - Section 314.50 Content 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.
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?”
Application Format - Regulatory Requirements?
NDA - Section 314.50
BLA - Section 601.2
Describes content of application
Describes content of application
Describes procedures for filing
Describes format
Describes procedures for filing
DOES NOT describe format
Migration from ELA/PLA to BLA Construct
ELA/PLA Req’ments
Issue guidance for content & format for product categories
BLA req’ments and Form 356h format
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
Formatting of BLA to CTD Construct
BLA Content Req’ments • “CMC” guidance • “CMC” guidance • “CMC” guidance
Revise guidance for new format
CTD BLA
CBER’s 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
CBER’s CTD Implementation Process
Revise
“CMC” guidances to map CTD to BLA
“Specified Products” currently in process training Workshops likely
Staff
Integration of CTD with Applications for Product Approval