Therapies that use autologous or allogeneic stem cells are examples of early personalized therapies. Removing cells from a patient and reintroducing them to the same or a matched patient, for example, as done in bone marrow transplants or peripheral blood stem cell transplantation, has provided benefits to many patients and are effective treatments for leukemia, lymphoma, myelodysplasia, multiple myeloma, and certain immune deficiency diseases.

However, these life-saving therapies are not without risk. In August 2016, the US Food and Drug Administration (FDA) released a statement that due to some “unscrupulous actors”, FDA would be increasing oversight of clinics and providers that offer stem cell therapies. See “Statement from FDA Commissioner Scott Gottlieb, M.D. on the FDA’s new policy steps and enforcement of stem cell therapies and regenerative medicine.” (Statement) As stated by FDA in 2016:

“At the same time we take steps to prevent unscrupulous actors from being able to deceive patients and potentially harm their health, we also need to make sure that the vast majority of responsible product developers know where the regulatory lines governing this new field are drawn. The FDA must advance an efficient and least burdensome framework as a way to help new products remain compliant with the law through a regulatory structure that does not become a barrier to beneficial new innovation.”

Statement at page 1.

Consistent with the FDA’s 2016 announcement, the agency has now released two new final and two new draft guidance documents that provide a “comprehensive regenerative medicine policy framework to spur innovation, efficient access to potentially transformative products, while ensuring safety & efficacy.” See “FDA announces comprehensive regenerative medicine policy framework.” (Summary Document). The framework is stated to provide the following public benefits:

1. A risk–based regulatory approach to describe what products will be regulated as drugs, devices, and/or biological products;

2. A science-based process to ensure the safety and effectiveness of stem cell therapies;

3. A risk-based framework for how the FDA will focus enforcement actions against products that raise significant safety concerns; and

4. A means to support innovators bring new and effective therapies to patients and drive further advances in regenerative medicine.

Summary Document at page 1.

Two New Final Guidance Documents

The first final guidance document is intended to provide greater clarity around when cell- and tissue-based products would be excepted from regulation, i.e., “if they are removed from and implanted into the same individual within the same surgical procedure and remain in their original form.” Summary Document on page 2. The second final guidance document is intended to help stakeholders better understand how existing regulatory criteria define “minimal manipulation” and “homologous use.” Id. As explained in the Statement, these two concepts are critical to determining whether a stem cell product or procedure is subject to FDA’s premarket approval requirements.

Two New Draft Guidance Documents

The first of the two new draft guidance documents is intended to “address how the FDA intends to simplify and streamline its application of the regulatory requirements for devices used in the recovery, isolation, and delivery of regenerative medicine advanced therapies.” Summary Document at page 3. The second draft guidance document describes several expedited programs to speed therapies to patients.

Moving Forward

The FDA’s new final and draft guidance documents signal the government’s continued commitment to personalized therapies and in particular, regenerative medicine. They also seek to guarantee that current and future therapies are safer and effective for the patients.