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NA LRA Watchdog - August 2022

  


North America Legal and Regulatory Affairs

Watchdog Update
August 2022

Kevin Bosse, PhD, RAC
Elizabeth George, MS
Nationwide Children's Hospital
Columbus, OH, United States

Co-Editors:
Yossi Schwartz MD, MPH
Mount Sinai Health System
New York NY, United States

Crystal Ruff MBA, PhD
Takeda
London, United Kingdom


Regulatory Aspects of the ISCT 2022 Anual Meeting

Overview:

Over 1,600 global researchers, technologists, industry members, and regulators attended and presented at the International Society for Cell & Gene Therapy (ISCT) 2022 Annual Meeting.

The well-rounded meeting format continued this year, featuring multiple regulatory focused sessions and including regulatory considerations throughout, with an additional session centered around manufacturing and clinical application.

New to the meeting template this year were roundtable talks, designed to promote audience participation. The roundtables discussed relevant topics such as chain of custody, donor eligibility, and risks with attention to maintaining regulatory compliance. The broad background of the roundtable panelists added to the depth of discussion.

Regulatory perspectives were represented by informative speakers from around the world with targeted discussion presenting and contrasting regional regulations. Throughout the meeting, engaged stakeholders considered a desire for harmonization and a pursuit of industry consensus for practices in the development of cell and gene therapies.

Key Highlights:

Regulatory Considerations throughout Product Lifecycle

Regulatory topics included design of IND enabling preclinical studies, cGMP processes, clinical trials outcomes, and considerations for manufacturing maturation. In addition, ISCT has been a field leader in discussion of unproven cell therapies, and ISCT 2022 also featured sessions on ethics relating to Cell and Gene Therapy and unlicensed cell therapies. Meeting participants had multiple opportunities to hear from and interact with researchers and industry representatives regarding licensed products, who shared their knowledge from clinical trials through marketing. These experiences included general information regarding regulatory interactions and utilization of tools such as expedited pathways designed to help facilitate accelerated approvals of qualifying Cell and Gene Therapy products.

A session dedicated to Expanded Access and Hospital Exemption featured Wilson Bryan, MD, Director of the Office of Tissues and Advanced Therapies at the Food and Drug Administration’s Center for Biologics Evaluation and Research as well as legal and regulatory experts. Cell and Gene Therapy products are often designed for severe and life-threatening indications for which there is an unmet clinical need and thus positioned to potentially satisfy requirements for each program, both of which are designed to connect patients with potentially life-saving treatment. However, speakers also reiterated the importance of the clinical trial process through which safety and efficacy data necessary for an application for marketing approval is compiled. This concentration on treatment access to unapproved cell therapies is consistent with the remit of ISCT’s new working group, announced in February 2022, to identify and address practical, ethical, and regulatory issues that arise from Expanded Access.

Manufacturing Documentation

As so many of the new Cell and Gene Therapy technologies are truly first-in-human, regulatory approval is a major hurdle, with manufacturing often being the critical gateway. Multiple sessions on Chemistry, Manufacturing, and Control (CMC) were featured during the ISCT 2022 meeting. Speakers and roundtable discussion emphasized the importance of planning CMC considerations such as potency assays and scalability throughout product development in order to avoid additional delays or unplanned expenses in the path to marketing approval and production of potential life-saving therapies.

Roundtable discussion regarding donor eligibility and testing of starting materials included minimum regulatory requirements as well as additional considerations which may lead to generation of additional quality related eligibility and acceptance criteria. Input from stakeholders representing researchers, manufacturers, and collection facilities enhanced discussion regarding eligibility testing in the context of autologous products, allogeneic third-party products, and critical materials used in manufacturing. A session outlining practices for ensuring proper chain of identity and chain of custody included not only the regulatory and logistical considerations regarding product tracking and transport between facilities but also practices for protection of identifiable patient information.

Safety, quality, and regulatory considerations were considered through roundtable discussions in the context of in-process and release sterility testing, planning for potency testing, and development of stability programs. Audience engagement allowed for specific manufacturing and compliance questions and responses. Presentations also included considerations relating to changes made to the manufacturing process or product through product lifecycle focusing on the planning, resources, and documentation to support comparability.

Integrating Regulatory Topics into Training

For the final day, ISCT offered a technologist workshop on the FUNdamentals of Cell Product Manufacturing, featuring regulatory talks. J Wade Akins, MS, MT(ASCP) SBB, CQA (ASQ), QA Specialist, NIH Clinical Center Department of Transfusion Medicine (DTM), presented information on Quality Assurance and Regulatory. Kevin Bosse PhD, RAC, Director of the Office of Research Regulatory Affairs at Nationwide Children’s Hospital and co-chair of the ISCT Early Stage Professionals Committee provided content on the role of manufacturing in regulatory submissions.

Summary:

Overall, the meeting highlighted regulatory framework and considerations that impact regulatory compliance for Cell and Gene Therapy products throughout product development and product lifecycle. Collective discussion promoted pursuit of industry guidelines and regulatory harmonization, which can help to enable ISCT aims of improving lives through safe and effective cell and gene therapies.

 

*views represented are contributors’ own and do not necessarily represent those of their employers

 

This newsletter includes links to issues and activities that might be of interest both on the Health Canada as well as the FDA websites.  Please note that you can follow HC and FDA on Facebook and Twitter. You can also subscribe to receive emails from the FDA at https://www.fda.gov/about-fda/contact-fda/get-email-updates

Health Canada

What’s new in Biologics, radiopharmaceuticals and genetic therapies can be found at: https://www.canada.ca/en/health-canada/services/drugs-health-products/biologics-radio-pharmaceuticals-genetic-therapies/what-new-biologics-radiopharmaceuticals-genetic-therapies-health-canada.html

Information for health product manufacturers and distributors in relation to COVID-19:  https://www.canada.ca/en/health-canada/services/drugs-health-products/covid19-industry.html

FDA

What’s new for Biologics including Approval and Determination Letters:
https://www.fda.gov/vaccines-blood-biologics/news-events-biologics/whats-new-biologics

COVID-19 Information and Resources:
https://www.fda.gov/emergency-preparedness-and-response/counterterrorism-and-emerging-threats/coronavirus-disease-2019-covid-19

Guidance Documents:
https://www.fda.gov/regulatory-information/search-fda-guidance-documents

Upcoming Conferences:
https://www.fda.gov/vaccines-blood-biologics/news-events-biologics

Updated Approvals and Listings:

Complete List of Licensed Products and Establishments

Complete List of Substantially Equivalent 510(k) Device Applications

Complete List of Currently Approved Premarket Approvals (PMAs)

Complete List of Currently Approved NDA and ANDA Application Submissions

2021 Biological Approvals



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