FDA STAFF MANUAL GUIDES, VOLUME I - ORGANIZATIONS AND FUNCTIONS
FOOD AND DRUG ADMINISTRATION
OFFICE OF MEDICAL PRODUCTS AND TOBACCO
CENTER FOR BIOLOGICS EVALUATION AND RESEARCH
OFFICE OF CELLULAR, TISSUE AND GENE THERAPIES
Effective Date: 07/08/2011
1. OFFICE OF CELLULAR, TISSUE AND GENE THERAPIES (DKKBL).
A. Plans and conducts research related to the development, manufacture, testing, and activities of cellular, tissue, and gene therapy products, including those related to AIDS and those prepared by genetic engineering and synthetic procedures, in order to develop and maintain a scientific base for establishing standards designed to ensure the continued safety, purity, potency and effectiveness of cellular, tissue, and gene therapy products.
B. Performs functions regarding cells and analogous products, tissues and analogous products, and biological gene therapy products.
C. Develops policy and procedures governing the pre-market approval review and evaluation of cellular, tissue, and gene therapy products in keeping with the provisions of the PHS Act and applicable provisions of the FFD&C Act.
D. Reviews, evaluates and takes appropriate action on investigational new drug applications (INDs) related to therapeutic products and amendments or supplements to these applications. Actions include, but are not limited to, approval or disapproval of research plans and protocols, modifications, and restrictions.
E. Performs the investigational device exemption (IDE) review process for devices related to cellular, tissue, and gene therapy products regulated by the Office, and develops related policy.
F. Reviews, evaluates, and takes appropriate action on product applications submitted by manufacturers of cellular, tissue, and gene therapy products, including labeling, and proposes written and reference standards for cellular, tissue, and gene therapy products.
G. In cooperation with CBER components, as appropriate, tests products submitted for release by manufacturers.
H. In coordination with the Office of Compliance and Biologics Quality, evaluates clinical experience and reports of adverse events as necessary.
I. Participates in inspections of manufacturing facilities for compliance with applicable standards.
J. Reviews, evaluates, and takes appropriate action on recommendations concerning denial of license applications for products.
K. Administers applicable provisions of the FFD&C Act as they pertain to certain devices and drugs that are under the jurisdiction of the Office and cooperates with other Agency components and outside organizations on a variety of issues related to these products.
L. Develops, organizes and maintains quality assurance and quality control for reviews of INDS, PMAS, BLAS and other regulatory activities for products regulated by the Office.
M. Develops, organizes and maintains quality assurance and quality control for the conduct of research in support of standards development to assure the continued safety, purity and potency of products regulated by the Office.
2. REGULATORY MANAGEMENT STAFF (DKKBL1).
A. Conducts administrative and regulatory screening of all investigational new drug applications (INDs), investigational device exemptions (IDEs), new drug applications (NDAs), biologics license applications (BLAs), premarket approvals (PMAs) for medical devices, and related submissions for products regulated by the Office.
B. Coordinates review of all INDs and IDEs. Serves as primary contact between IND/IDE sponsors and the Office. Monitors and tracks all regulatory actions for Office INDs/IDEs and ensures consistency of actions with applicable regulations and policies.
C. Coordinates processing and review of BLA/PMA/NDA marketing applications and supplements for the Office and ensures that actions are consistent with applicable regulations and policies.
D. Reviews regulations and guidelines setting forth administrative and regulatory applications for procedures in the biological licensing area. Develops policies and procedures applicable to the review of INDs/IDEs and BLA/PMA/NDA marketing applications.
E. Monitors and evaluates labeling of biological products, drugs, and devices assigned to the Office. Assists in evaluating the adequacy of product package inserts for products regulated by the Office.
F. Conducts or coordinates for the Office, written and oral communication with sponsors/manufacturers. Coordinates, schedules, and conducts all formal meetings between sponsors/manufacturers and Office personnel.
G. Conducts, in coordination with other Agency components, continuing surveillance and medical evaluation of clinical experience reports submitted under the IND and IDE reporting requirements.
H. Maintains databases on IND/IDE and marketing application review activities and provides information to Center and Agency management, manufacturers, and consumers.
I. Provides staff support and regulatory guidance and advice on biologics, drugs, and devices to Agency committees and provides advice and guidance to other government agencies, manufacturers, and consumers on issues related to this Office.
3. AUTHORITY AND EFFECTIVE DATE.
The functional statements for this Office were approved by the Secretary of Health and Human Services on July 8, 2011.