FDA guidance on regenerative medicine has been issued covering the allowable uses of novel cellular therapies, when the use of such therapies is exempt from FDA regulation, and how the FDA’s enforcement policy concentrates on new therapies that have significant safety issues. The FDA guidance on regenerative medicine is spread over two final guidance documents and two drafts.
The guidance documents cover therapies that use harvested stem cells to treat the same patient from whom the stem cells were removed, and other patients who have been matched with the donor of the cells. While these therapies have, in some cases, had highly beneficial results, the treatments are not without risks. The FDA does not want to hamper development of new therapies, but must ensure that any new treatments are safe and effective before they are used on patients.
The FDA points out that these novel therapies have been used by certain ‘unscrupulous actors’, and in the summer of 2016, the FDA said it would be increasing oversight on the use of these therapies. The aim of the increased oversight was to prevent patients from being deceived and to ensure that the new therapies are used in a responsible manner.
The new guidance introduces a new framework covering the use of regenerative medicine, which the FDA says will help to ensure products and techniques remain compliant, while not limiting innovation.
The new FDA guidance on regenerative medicine detail a “comprehensive regenerative medicine policy framework to spur innovation, efficient access to potentially transformative products, while ensuring safety & efficacy,” according to a recent FDA announcement.
The framework details the FDA’s risk–based regulatory approach to the regulation of regenerative medicine, which includes drugs, therapies involving the use of biological products, and medical devices. The FDA requires a science-based process to be adopted to ensure stem cell therapies are both safe and effective.
The FDA’s guidance will help to support innovators to drive advances in regenerative medicine, and explains its enforcement policy, which is centered on products and therapies that raise significant safety concerns.
The final FDA guidance on regenerative medicine explains when cell and tissue-based therapies are excepted from FDA regulation – When tissues are removed from one individual and re-introduced into the same individual during the same surgical procedure, with the cells and tissues remaining in their original form.
The second document clarifies to stakeholders, healthcare providers, and FDA staff the terms “minimal manipulation” and “homologous use,” and how existing regulations apply.
These final guidance documents – “Same Surgical Procedure Exception under 21 CFR 1271.15(b): Questions and Answers Regarding the Scope of the Exception” and “Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use,” are final versions of draft guidance documents first issued in late 2014.
The two draft guidance documents – “Evaluation of Devices Used with Regenerative Medicine Advanced Therapies” and “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions” – spell out how the FDA intends to streamline and simplify the application of regulatory requirements for devices used the recovery, isolation, and delivery of regenerative medicine advanced therapies, and detail some of the FDA’s expedited programs that will help to bring new therapies to patients more rapidly.