FDA resources for cell and gene therapy product development
Cell Gene Therapy Insights 2015; 4(10), 983-994.
10.18609/cgti.2015.015
In the USA, the FDA Office of Cellular, Tissue, and Gene Therapies (OCTGT) is principally responsible for regulation of cell and gene therapy products. Based in FDA’s Center for Biologics Evaluation and Research, OCTGT’s perspective on regulating cell and gene therapy products reflects the growth and development of the field, as well as FDA experience reviewing these complex biologics. OCTGT guidance documents and the webinar series OCTGT Learn are invaluable resources for cell and gene therapy product development.
Guidance documents
FDA’s regulatory expectations and advice regarding cell and gene therapy products are outlined in a growing set of guidance documents. Selected guidance documents are listed in Table 1, grouped topically into three main categories – preclinical, manufacturing, and clinical. Four of these guidance documents together represent the foundation of FDA’s regulatory approach to cell therapy and gene therapy products, and should be read by anyone involved in developing these products. Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products (June, 2015) provides a synthesis of FDA’s advice on issues to address in early development, with references to other key guidance documents on preclinical, clinical, and manufacturing aspects. Preclinical Assessment of Investigational Cellular and Gene Therapy Products (November, 2013) discusses design, conduct, and interpretation of preclinical pharmacology/toxicology studies. Cell therapy product manufacturing and testing is covered in Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs) (April, 2008), and separately for gene therapy products in Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) (April, 2008).
OCTGT Learn
The FDA webinar series OCTGT Learn is an educational resource for cell and gene therapy product developers and investigators, discussing key regulatory topics, and providing insight into topics of particular concern to FDA OCTGT. These are points FDA OCTGT wants to make sure you understand. Selected OCTGT Learn webinar titles are shown in Table 2.
| Webinar | Topic |
|---|---|
| Target Product Profile | Role of the Target Product Profile in facilitating product development |
| Early-Phase Trials of Cellular and Gene Therapies | Clinical risks of cellular and gene therapy products, and considerations for design of early-phase trials |
| Fast Track (FT) for Products Regulated in OCTGT | OCTGT experience and advice regarding Fast Track requests and designation for cell and gene therapies, and other OCTGT-regulated products |
| Regulatory Obligations for Investigator-Sponsored Research | Regulatory requirements for an investigator who is also a sponsor of an IND or IDE |
| Pediatric Clinical Trials | Regulation and design of pediatric clinical trials |
| IND Safety Reporting | FDA safety reporting requirements for products under IND |
| Data Monitoring Committees | Data Monitoring Committees - establishment, operation, and responsibilities |
| Endpoint Assessment and Adjudication Committees | Circumstances in which an Endpoint Assessment and Adjudication Committee (EAAC) may be useful, charter and operation of an EAAC, and potential bias in endpoint assessment |
| Successful Development of Quality Cell and Gene Therapy Products | Guidance for successful development of quality cell and gene therapy products |
| Cellular Therapy Products | Information needed for cell therapy product INDs |
| The Chemistry, Manufacturing and Controls (CMC) Section of a Gene Therapy IND | Elements of the IND CMC section for gene therapy products |
| Preclinical Considerations for Products Regulated in OCTGT | Preclinical considerations for cell and gene therapy INDs |
| "361" Human Cells, Tissues, & Cellular and Tissue Based Products (HCT/Ps) | Definition of HCT/Ps and how they are regulated |
| IND Basics in OCTGT | IND submissions to OCTGT |
| Sponsor Meetings with OCTGT | Types of sponsor meetings with OCTGT |
Affiliation
Scott R Burger, MD
Advanced Cell & Gene Therapy, LLC
+1 (919) 969-1103
celltherapy@ac-gt.com