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Regulations and Science: Common Sense Approaches to Accelerating Patient Access to Novel Gene and Cell Therapies

Presented at the 24th annual meeting of the American Society of Gene and Cell Therapy Regulations and Science: Common Sense Approaches to Accelerating Patient Access to Novel Gene and Cell Therapies Abstract number: 840 Session: Clinical Translation of Gene and Cell Therapies Date and time: Tuesday, May 11, 2021 8:00 – 10:00 a.m

Author Block: Tanya M. Scharton-Kersten, Tirzah Cherian Pathicheril Kersten Compliance Services, LLC, Dobbs Ferry, NY
Disclosure Block: T.M. Scharton-Kersten: None.

New products plus predicate data equals faster product development for cell and gene therapies. Informatics related to Clinical Trial and Marketing Applications is an emerging, often overlooked tool in product development that can be used by all sponsors - academic, industry, non-profit to accelerate gene and cell therapy therapeutic interventions. In 2019 FDA anticipated that by 2020 it would be receiving more than 200 INDs per year and by 2025 would be approving 10 to 20 cell and gene therapy products a year based on an assessment of the current pipeline and the clinical success rates of these products (1) . The use of converging technologies for different gene and cell therapy indications is an unprecedented opportunity for leveraging predicate data for clinical trial initiation and product licensure. Robust compilation of evidence-based data for safety, efficacy and quality facilitates patient access to therapeutic interventions. Sponsors that are not aware of predicate CMC, clinical and non-clinical data are at risk of performing unnecessary development activities, delay in timelines for concept to IND/CTA to BLA/MAA submission, inefficient use of sponsor and health authority resources and, most importantly, delay of patient access to new medicines. Example questions that can be readily addressed for most programs, by any SME in an organization include: How many trials were conducted to license product X, what country was the phase 1 trial conducted in, what cell line was the product manufactured in, what is the dosage form, what is the final container, what is the storage condition for drug product, how long can it be stored, what changes were made to the product during development, which changes required additional non-clinical or clinical studies? Strategies for compiling what is known and not known in the public domain for gene and cell therapies are available and accessible but may be underutilized due to lack of awareness of the databases and regulatory repositories available on the internet. Both the US and European regulatory agencies (FDA and EMA) provide public information on most aspects of the approval process for Gene Therapy, Cell Therapy and Vaccines in their public assessment reports (EPAR in Europe and SBAR in the US). Relevant details on the Phase 1, 2 and 3 clinical studies including the number of subjects and endpoints, a description of the manufacturing process, and the animal studies (safety, biodistribution and efficacy) are listed for each product with redaction of proprietary details. Issues encountered during product development, risks highlighted during the marketing application review, and commitments between the manufacturer-sponsor and the health authority are highlighted. Transcripts of advisory committee meetings where product risks are discussed with experts, the sponsor-manufacturer, and the health authority are provided in the US and provide rationales for reviewer concerns and thought processes during product application review. These health authority repositories complement the international clinical trial databases, company investor reports for public traded companies, grant agency repositories, and the growing number of scientific literature databases available through the internet. Example resources: FDA: Europe: (1) Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies (January 2019).