Welcome to OTP Learn (Office of Therapeutic Products; previously OTAT 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. OTP-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 OTP 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
OTP Learn Course List:
Listed below are the courses OTP 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.
- IND Decisions: Safe to Proceed, Clinical Hold, and Partial Hold
Danielle Bauman discusses the decisions made for IND applications including Clinical Hold and Partial Hold. Topics include the hold regulations, frequent reasons for hold, the hold process, and how to respond to a hold letter.
- Overview of Office of Therapeutic Products (OTP) Regulated Products
Jennifer Albert provides an overview of OTP and the products regulated in OTP. Topics include the role and current organization of OTP, the types of products regulated and reviewed in OTP, the divisions and branches within OTP, and the responsibilities of each of those divisions.
- Original Investigational New Drug (IND) Content and Format
Niloofar Kennedy discusses the content and format of an Original IND in OTP. Topics include details on the required forms, cross-referencing, CMC content, pharmacology/toxicology content, and clinical content. Also covered is brief overview of the IND receipt and 30-day safety review process as well as best practices.
- Regenerative Medicine Advanced Therapy (RMAT) Designation
Cara Pardon discusses RMAT Designation. The topics include criteria for designation, how to submit a request for designation, contents of the request, how the requests are processed and reviewed in OTP, decision options for granting or denying the request, and best practices.
- Breakthrough Therapy (BT) Designation
Cara Pardon discusses Breakthrough Therapy Designation. The topics include criteria for designation, how to submit a request for designation, contents of the request, how the requests are processed and reviewed in OTP, decision options for granting or denying the request, and best practices.
- Pre-Biologics License Application (Pre-BLA) Meeting for Original BLAs - An Overview
- Crystal Melendez provides an overview of the Pre-Biologics License Application (Pre-BLA) meeting for original BLAs. Topics include the purpose of the meeting, expectations of this type of meeting, and the format. Details on some discipline specific content requirements are also discussed.
- Original Biologics License Application Overview
Mara Miller provides an overview of the original Biologics License Application (BLA) process in OTP. Topics include BLA review timelines and meetings under "The Program", filing procedures, regulatory actions, and best practices.
- OTP Processing of Formal Meeting Requests
Mehran Azari and Danielle Bauman discuss how OTP 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 OTP. 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 Tissues and Advanced Therapies consistent with the agreements set in place in the PDUFA V negotiations.
- 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 OTP 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.
- 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 OTP-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 OPT
Allen Wensky, Ph.D. provides a basic overview of preclinical considerations that make up one of the three key elements of an IND submission.
- 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 (BT) 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).