October 29, 2020
In 2017, the U.S. Food and Drug Administration (FDA) published a comprehensive regenerative medicine policy framework to spur innovation and efficient access to safe and effective regenerative medicine products. For nearly three years, the agency has exercised enforcement discretion to allow more time for manufacturers of human cells, tissues, and cellular and tissue-based products (HCT/Ps) to determine which regulations apply to their products. In May 2021, this period of enforcement discretion will end. See our post from September 24, 2020, for a comprehensive understanding of this framework.
Both regenerative medicine product manufacturers and clinicians who offer cellular therapies need to understand how their products are regulated. Manufacturers and clinicians can save valuable time and resources by investing in risk mitigation strategies today.
Risk Mitigation for Regenerative Medicine Product Manufacturers
It is important for manufacturers who have already launched regenerative medicine products or plan to do so to ensure their products are correctly classified. If not appropriately addressed, these concerns could affect the continuity of existing operations or the effectiveness of future regulatory submissions.
We recommend that manufacturers evaluate their products to understand whether they will be regulated under Section 361 or Section 351 of the Public Health Service Act (PHS Act). Products that qualify as HCT/Ps under Section 361 criteria will be subject only to the rules intended to minimize the introduction, transmission, and spread of communicable disease as outlined by Section 361 of the PHS Act and 21 CFR 1271.10. Products that do not qualify will be regulated as drugs, biologics, or combination products under Section 351.
Because Section 351 regulated products are considered higher risk than Section 361 products (i.e., HCT/Ps), these products hold more risk management requirements, including the need for premarket approval. Notably, FDA has specifically asserted its authority to regulate such products, including taking action in the courts. Thus, careful classification is an important part of risk management that encompasses both regulatory and legal risks to manufacturers for these types of products.
Risk Mitigation for Clinicians Who Offer Cellular Therapies
Clinics performing cell therapies may also be considered manufacturers of HCT/Ps under certain circumstances. Clinicians and care providers who offer cellular therapies may be unaware that their work may fall under these regulations as manufacturers. The following considerations can help medical care providers determine whether or not a service that involves cellular therapy may be an FDA-regulated product.
- Regarding the practice of medicine, do the cells or tissues qualify under the "Same Surgical Procedure Exception" under 21 CFR 1271.15(b)?
- Is the cellular therapy being offered intended to be implanted into the same person?
- If so, how are the harvested cells being handled and manipulated?
- Is the risk posed to the patient from more-than-minimal manipulation and addition of other agents greater than what is allowed by HCT/P designation?
Further details about these considerations can be found here.
How Exponent Can Help
Exponent's multidisciplinary team of biomedical engineers, health scientists, and regulatory consultants can help regenerative medicine product manufacturers and clinicians who offer cellular therapies build risk mitigation plans. Our experts have deep knowledge of regenerative medicine products, the development of unique testing parameters for these products, and FDA regulation.
We can help clients implement the tiered, risk-based approach that FDA uses to determine whether or not a product is considered an HCT/P under Section 361. In the event that a client's product does not qualify as an HCT/P, Exponent can support mechanical, chemical, and human factors testing that may be required for regulatory submissions. We can also help clients understand what changes to manufacturing, marketing, or other go-to-market activities might be required to maintain regulatory compliance after May 2021.