The FDA has issued two final guidances and two draft guidances, all designed to articulate the agency’s approach to developing and overseeing novel cellular therapies and other regenerative medicine products.

The agency said its suite of four guidance documents constituted a risk-based and science-based policy framework approach designed to support innovative product development while clarifying the FDA’s authority, its enforcement priorities against products deemed to raise potential significant safety concerns.

The two final guidances are designed to clarify the FDA’s interpretation of the risk-based criteria manufacturers must use to determine whether a product is subject to the FDA’s premarket review.

The first guidance is intended to spell out more clearly when cell- and tissue-based products are excepted from the established regulations if they are removed from and implanted into the same individual within the same surgical procedure and remain in their original form. The guidance is a final version of a draft issued under the same title—“Same Surgical Procedure Exception under 21 CFR 1271.15(b): Questions and Answers Regarding the Scope of the Exception”—in October 2014.

The second final guidance is designed to explain to stakeholders—defined as “human cells, tissues, and cellular and tissue-based product (HCT/P) manufacturers, healthcare providers, and FDA staff”— how existing regulatory criteria apply to their products by clarifying the agency’s interpretations of two existing regulatory definitions, “minimal manipulation” and “homologous use.”

The guidance is a final version of a draft issued in December 2014 under the same title, “Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use.”

The FDA has also issued two draft guidances. “Evaluation of Devices Used with Regenerative Medicine Advanced Therapies” is meant to address how the FDA plans to simplify and streamline its application of the regulatory requirements for devices used in the recovery, isolation, and delivery of regenerative medicine advanced therapies (RMATs), a designation created by the 21st Century Cures Act and which includes combination products. According to the draft guidance, devices intended for use with a specific RMAT may, together with the RMAT, be considered a combination product.

The second draft guidance, “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions,” details the expedited programs that may be available to sponsors of regenerative medicine therapies, including the RMAT designation, Priority Review, and Accelerated Approval. The draft guidance describes the regenerative medicine therapies that may be eligible for RMAT designation. They include cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products, as well as gene therapies that lead to a durable modification of cells or tissues, including genetically modified cells.

Support, and a Caveat, from ISCT

“This field is dynamic and complex. As such, it has presented unique challenges to researchers, healthcare providers, and the FDA as we seek to provide a clear pathway for those developing new therapies in this promising field, while making sure that the FDA meets its obligation to ensure the safety and efficacy of the medical products that patients rely upon,” FDA Commissioner Scott Gottlieb, M.D., said in a statement.

The FDA’s framework has won support from the International Society for Cellular Therapy (ISCT), a global professional society of clinicians, researchers, regulatory specialists, technologists, and industry partners in the cell therapy sector—with a caveat.

“ISCT welcomes the efforts of the FDA to provide additional regulatory clarification for all those operating in the cell therapy field. However, it is essential that the FDA is now cautious and measured in its introduction and application of the framework,” ISCT president Catherine Bollard, M.D.,  said in a statement issued today by the Society.

“The FDA needs to balance bringing those operating outside the regulatory pathways to compliance, taking action against those that remain outside the licensing and regulatory frameworks, and continuing to foster the ongoing innovation and considerable potential for the majority of the sector operating within the regulatory frameworks.”

Bollard cautioned that “an overzealous regulatory application could increase development and manufacturing bureaucracy and costs and time to market, and delay the validation of products and facilities.

“Ultimately, though, it is patients that are the customers of cell and gene therapies. They have a fundamental right to expect a scientific and clinical rationale for a proposed treatment or therapy they might receive,” Bollard added.

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