Welcome to OTAT Learn (Office of Tissues and Advanced Therapies; previously OCTGT Learn), the Center for Biologics, Evaluation and Research's (CBER) web page for industry education. CBER ensures the safety, purity, potency, and effectiveness of biological products, including vaccines and allergenics, blood and blood products, and cells, tissues, and gene therapies for the prevention, diagnosis, and treatment of human diseases, conditions, or injuries. OTAT-regulated products include gene therapy, tumor vaccines, xenotransplantation, stem cells, human tissue for transplantation, combination products, bioengineered tissues and certain medical devices.
The presenters are OTAT staff and therefore, as employees, have claimed no interests, financial or otherwise, with products that may be shown in any of the presentations.
REdI Biologics Track 2021
Presenters in the biologics track discuss the following topics: Expedited Programs, Regenerative Medicine, Genetically Modified Cells, CMC for In Vivo Gene Therapy Products, Preclinical Development for Gene Therapies, Genetically Modified Cellular and Cancer
OTAT Learn Course List:
Listed below are the courses OTAT currently offers. Additional online courses may be developed and will be posted upon completion.
You may submit suggestions for future courses to OCTGTLearn@fda.hhs.gov.
- OTAT Processing of Formal Meeting Requests
Mehran Azari and Danielle Bauman discuss how OTAT processes formal meeting requests. Topics include receipt and evaluation of the request, scheduling and confirmation, submitting the briefing package, meeting preparation from receipt of the package through internal meeting and preliminary responses, conduct of the meeting, meeting summaries and best practices.
- Original IND Applications – Behind the Scenes
Nadia Whitt provides a behind the scenes look at Original IND processing and review in OTAT. Topics include how to title an IND, process flow from receipt through review to decision, and best practices.
- Formal Meetings PDUFA Products for Between the FDA and Sponsors or Applicants of Industry
Candace Jarvis provides a description of the timelines and requirements for requesting a meeting with CBER’s Office of Cellular, Tissue, and Gene Therapies consistent with the agreements set in place in the PDUFA V negotiations.
- Biologic License Applications to OCTGT
Ramani Sista Ph.D., RAC, CQA gives an overview of the type of inflation and format to include in the submission of a Biologics License Application to CBER’s Office of Cellular, Tissue, and Gene Therapies....
- Regulatory Obligations for Investigator-Sponsored Research
Pat Holobaugh, MS discusses the regulatory requirements for an investigator who is also a sponsor of an IND or IDE.
- Early-Phase Trials of Cellular and Gene Therapies
Steve Winitsky, MD discusses the clinical risks of cellular and gene therapy products, and reviews some considerations for the design of early-phase trials of these products.
- Pediatric Clinical Trials
Steve Winitsky, MD discusses the regulation and design of pediatric clinical trials.
- The Target Product Profile
Bindu George, MD presents the Target Product Profile (TPP) and describes how the TPP can facilitate product development.
- Fast Track (FT) for Products Regulated in OCTGT
Bindu George, MD discusses Fast Track requests and designation for OCTGT products, including cellular and gene therapies.
- IND Safety Reporting
Bindu George, MD provides basic information regarding the FDA safety reporting requirements for INDs.
- Data Monitoring Committees
Ke Liu, MD, PhD describes the history, establishment, operation, and responsibilities of Data Monitoring Committees (DMCs).
- Endpoint Assessment and Adjudication Committees (EAACs)
Ke Liu, MD, PhD presents situations when an EAAC is useful, discusses potential bias in endpoint assessment, and describes the EAAC charter and operation.
- Introduction and Scope of OCTGT
Patrick Riggins introduces the Office of Cellular, Tissue and Gene Therapies and provides a scope of what the office does.
- The Chemistry, Manufacturing and Controls (CMC) Section of a Gene Therapy IND
In this presentation, Andrew Byrnes explains the basics of how to put together the CMC section of a gene therapy IND, particularly for Phase 1 trials.
- Advanced Topics: Successful Development of Quality Cell and Gene Therapy Products
Denise Gavin aims to guide manufacturers toward successful development of quality cell and gene therapy products, in this presentation.
- Cellular Therapy Products
Keith Wonnacott discusses information that is needed to prepare an investigational new drug application for a cellular therapy product.
- "361" Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps)
Michelle McClure, Ph.D. provides an overview of the regulatory approach for “361” human cells, tissues, and cellular and tissue-based products (HCT/Ps).
- Advanced Topics: Design and Analysis of Shedding Studies
Zenobia Taraporewala Ph.D. provides an overview of the design and analysis of shedding studies that are conducted before licensure for OCTGT-regulated virus or bacteria-based gene therapy and oncolytic products.
- Advanced Topics: Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines and Related Recombinant Viral or Microbial Products
Michael Havert, Ph.D., reviews the requirements for an environmental assessment for gene therapies, vectored vaccines and related recombinant viral and microbial products.
- Determining Eligibility of a Donor of Human Cells, Tissues, or Cellular or Tissue-Based Products (HCT/Ps)
Michelle McClure, Ph.D., reviews the process for determining the eligibility of potential donors of human cells, tissues, and cellular and tissue-based products (HCT/Ps).
- Preclinical Considerations for Products Regulated in OCTGT
Allen Wensky, Ph.D. provides a basic overview of preclinical considerations that make up one of the three key elements of an IND submission.
- Early-Phase Trials of Cellular and Gene Therapies
Steve Winitsky, MD's presentation reviews some of the major clinical risks associated with cellular and gene therapy products and will discuss some considerations for clinical protocol design that are relevant to these products.
- Special Protocol Assessment (SPA)
Steve Winitsky, MD provides some background and information on a 2016 draft guidance document related to the Special Protocol Assessment (SPA) process.
- Breakthrough Therapy Designation
Ke Liu, MD, PhD, provides an overview of FDA’s expedited programs for serious conditions (FDA guidance 2014), focusing on Breakthrough Therapy Designation (BTD).
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