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Printable Handouts
Navigable Slide Index
- Introduction
- FDA Organization
- Examples of Cellular and Gene Therapy Products
- Drug and Biologic Product Laws in The United States
- Investigational New Drug (IND) Application
- Medical Product Development Overview
- Regulatory Considerations
- FDA/Sponsor interactions in Product Programs
- Examples of Review Timelines
- Development Programs for Cellular and Gene Therapies: IND Trends in 2002-2020
- Considerations for Cellular and Gene Therapy Product Development
- Risk-Based Approach to Product Quality
- Product Quality Testing: Examples
- Genetically Modified Cells: Process Complexity
- Considerations for Preclinical Development
- Protection of Human Subjects in Clinical Research
- Gene Therapies: Ethical Considerations
- Considerations for Product Dosing
- Dosing initiation in First-In-Human Studies
- Considerations for Clinical Program Design: Efficacy
- Examples of Safety Concerns with Cellular and Gene Therapy Products
- Tumorigenicity: Risk Reduction Through All Stages of Development
- Considerations for Clinical Program Design: Safety
- Conclusions
- Additional Guidance Documents
Topics Covered
- Cellular and gene therapy products
- U.S. laws and regulations
- FDA review
- Product development considerations
- Regulatory considerations
- Product manufacturing stages and quality testing
- Safety, efficacy and ethical considerations for clinical program design
- FDA Guidance documents
Links
Categories:
External Links
- Guidance for Industry: Expedited Programs for Serious Conditions – Drugs and Biologics
- Guidance for Industry: Expedited Programs for RMAT for Serious Conditions
- Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products; Draft Guidance for Industry
- INTERACT program
- Expectations for Biodistribution Assessments for Gene Therapy Products, IPRP
- Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products, 2013
- NIH Recombinant DNA Advisory Committee
- Informed Consent Guidance for Human Gene Transfer Trials
- Guidance for Industry: Rare Diseases – Natural History Studies for Drug Development
- Guidance for Industry: Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products, June 2015
- Guidance for Industry: Long-Term Follow-Up Administration of Human Gene Therapy Products, January 2020
- Cellular & Gene Therapy Guidances
Talk Citation
Lapteva, L. (2021, November 28). Development and regulation of cellular and gene therapy products: FDA perspective [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved October 11, 2024, from https://doi.org/10.69645/NGRE8239.Export Citation (RIS)
Publication History
Financial Disclosures
- Dr. Larissa Lapteva has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
Development and regulation of cellular and gene therapy products: FDA perspective
Published on November 28, 2021
51 min
A selection of talks on Genetics & Epigenetics
Transcript
Please wait while the transcript is being prepared...
0:00
My name is Larissa Lapteva, and I am the Associate Director
in the Division of Clinical Evaluation, Pharmacology and Toxicology in
the Office of Tissues and Advanced Therapies in the Center for Biologics Evaluation and Research
at the Food and Drug Administration.
Today, I will give you an overview of the regulatory approach to the development of
cellular and gene therapies aiming to treat diseases, in serious medical conditions.
In the United States, cellular and gene therapy products are regulated
by the Food and Drug Administration (or FDA).
0:37
At the FDA, the oversight of medical products for human and veterinary use is accomplished
through the work of various centers.
For example, there is a Center for Biologics, a Center for Drugs, and a Center for Devices,
among others.
The expertise for the development and regulation of cellular and gene therapies
aimed to treat diseases currently resides in the Center for Biologics Evaluation and Research,
specifically in the Office of Tissues and Advanced Therapies.
When a new product is developed, a multidisciplinary team from the FDA side
supports that product development program.
The team would often include pharmacologists, toxicologists, chemists, and (for example)
for gene therapies it may include biologists with backgrounds in genetics, as well as physicians,
statisticians, and other professionals whose input is useful
for successful development of a new treatment.
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