The FDA outlined its stance on the exploding field of regenerative medicine in a new paper,1 trying to strike a balance in stem cell research oversight to temper risk without blunting reward.

“The potential benefits to human health have spurred major progress in stem-cell biology over the past several decades,” the FDA authors wrote. “At the same time, the administration of such stem cells may be associated with serious adverse events. … It is critical to focus on efforts to facilitate the development of such therapies, rather than propagating products with dubious clinical efficacy and possible risks.”

The paper was co-authored by FDA Commissioner Scott Gottlieb, MD, and Peter Marks, MD, PhD, director of the FDA Center for Biologics Evaluation and Research.

“Working within the existing regulatory framework, the FDA will make use of all available regulatory pathways and will adopt the use of some new principles that we believe will make the appropriate premarket evaluation of stem cell-based therapies more efficient,” the authors noted. “On a large scale, the FDA will be incorporating some new concepts for how small investigators and firms can seek and meet the approval standard for products through efficient, expedited pathways.”

For example, the FDA will assist and encourage individuals or small groups of physicians working in regenerative medicine. This could be followed by the approval of a “biologics license” for these physicians or groups.

“How might this work?” the authors asked. “The investigators who manufacture the product will need to agree on and follow a common manufacturing protocol and develop a common clinical trial protocol. Each site will then produce the product to treat the patients who are enrolled in the clinical trial at its own site. Subsequently, the pooled safety and efficacy data from the various sites that are participating in the trial will be submitted as part of a biologics license application for each.”

As part of its efforts to facilitate regenerative research, the FDA is encouraging investigators to reach out to the agency early in the process to open a dialogue before more formal discussions are required for an investigational new drug.


1. Marks P, Gottlieb S. Balancing Safety and Innovation for Cell-Based Regenerative Medicine. N Engl J Med 2018;378:954-959 DOI: 10.1056/NEJMsr1715626.