Industry seeks clarity on FDA’s standards recognition process for regenerative medicines

Regulatory NewsRegulatory News | 21 September 2022 | By

Commenters on a recent draft guidance released by the US Food and Drug Administration (FDA)’s Center for Biologics Evaluation and Research (CBER) support the idea of creating a voluntary consensus standards (VCS) recognition program for regenerative medicine therapies (RMTs) but want more details from the agency on the types of VCS recognized and the process for engaging FDA through the program.
As the RMT product scientific and manufacturing landscape evolves, recognizing appropriate standards in developing RMT products that fall under CBER’s purview can aid in product development by reducing the need for unique methods for individual products, as well as the documentation a developer needs to submit, the agency noted.
CBER’s VCS program would apply to allogenic and autologous cell therapies, therapeutic tissue engineering products, products involving human cell and tissue and most combination products with RMTs, as well as human gene therapies like genetically modified cells and xenogeneic cell products, FDA wrote.
The VCS standard recognition program for RMTs as outlined in the draft guidance, is modeled after the formal standards and conformity assessment program (S-CAP) for medical devices. The program would have subject matter experts in the Office of Tissue and Advanced Therapies review RMT-related VCS bodies and standards as being useful in whole or in part to industry and CBER staff. Each VCS identified in this way would then be listed on CBER’s website with details from the Center on how it recognizes the standard. (RELATED: CBER outlines recognition process for regenerative medicine therapy standards, Regulatory Focus 17 June 2022)
“By leveraging stakeholders’ efforts to develop standards, FDA can eliminate costs to the Federal government associated with the development of government-unique standards and promote international harmonization of standards that are acceptable to FDA,” the authors of the draft guidance wrote.
Response to draft guidance
In general, stakeholders responded to the draft guidance with support for FDA’s efforts to make it easier to meet regulatory requirements and improve predictability for RMT products. The International Society for Cell & Gene Therapy (ISCT) requested more information in its comment to FDA on how the agency defines what interested stakeholders qualify to participate in the program and more details on the appeals progress, due process and consensus for the VCS program.
In their comments, life science non-profit organization Biocom California asked FDA to provide additional details on how “scientifically sound” standards can be included in the VCS program. “Biocom California suggests that the agency provide specific, objective criteria and clarifying examples in the guidance document,” they wrote. “Furthermore, we ask the FDA to please include additional language explaining how these criteria will be utilized during CBER’s VCS evaluation process.”
The organization also expressed concern that applying a “unified approach” to VCS for a broad range of RMT products may be difficult for stakeholders. “We encourage the agency to consider the applicability of a standard for a particular RMT when evaluating how manufacturers utilize VCSs, especially in the case where deviations from the standard are made and supported by a scientific rationale and data,” they said.
Writing to FDA, the Standards Coordinating Body (SCB) offered up comments from a recent educational webinar they held to educate participants on the draft guidance. Participants in SCB’s webinar wanted more information on how FDA defines a VCS, such as whether it follows a definition from an American National Standards Institute accredited organization, how it determines whether a VCS is relevant, the specific therapies included in the VCS program and educational opportunities for stakeholders. Like ISCT, the participants of SCB’s educational webinar also wanted more information on how to engage with FDA within the context of this program.
The Association for the Advancement of Blood and Biotherapies (AABB), a signatory to SCB’s comments to FDA, expressed its hope to see the agency adopt its own Standards for Cellular Therapy Services “to ensure the safety, quality, and consistency of RMT products by recognizing standards that promote comparability and process controls.”
The Foundation for the Accreditation of Cellular Therapy (FACT) also noted it supports SCB’s comments and “strongly supports” the creation of a VCS program for RMT at CBER. FACT also echoed Biocom California’s concerns about the variability of RMT products and how certain standards may need to differ based on the type of product.
“We suggest that acknowledgement of the differences between the S-CAP programs for medical devices and the relevant standards for biologic systems be built into the recognition program for RMT. Consideration within the guidance of the complexity and variability of human cells, tissues, and cellular and tissue-based products used as starting material, in addition to the novelty of RMT products, is suggested,” FACT wrote.
Biotechnology Innovation Organization said it supported the draft guidance and use of VCS in general, but noted FDA should still be aware that new standards do not impact product quality, product safety or innovation. They also said the standards should change along with emerging science, technology and clinical experience. In addition, standards that apply across RMT products should leave room for various modality-specific considerations like adeno-associated virus vectors and CAR-T cells, they explained.
The American Association of Tissue Banks (AATB) and AATB’s Tissue Policy Group, commenting jointly to FDA, requested this VCS program be applied to not just RMT products but all human cells, tissues, and cellular and tissue-based products that require a Biologics License Application. “[T]he Agency has the authority to go beyond the strict statutory requirement and can opt to include additional regulated products within the voluntary consensus program,” AATB and its Tissue Policy Group wrote, arguing that including all these related products rather than singling out RMTs “is a better delineation of covered products and, as such, provides greater regulatory clarity.”
Draft guidance
Guidance comments


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