FDA’s Framework for Regulating Regenerative Medicine Will Improve OversightOctober 17, 2019
Over the last two decades, cell therapies (which involve the transplantation of whole cells into a patient), gene therapies (which use genetic material to manipulate a patient’s cells), and other medical treatments intended to repair or replace damaged, diseased, or dysfunctional cells, tissues, and organs have generated increasing public interest. Such treatments, which together make up the field of regenerative medicine, may have the potential to treat a range of problems such as organ failure, traumatic injuries, and serious diseases.
The increase in public interest has been accompanied by substantial private-sector financial investments in the development of regenerative treatments. Relatively few such treatments have been approved by the Food and Drug Administration (FDA) for use in the United States, but many more are in clinical development, and the number of approved products is expected to grow over the next several years.
But interventions that are marketed as regenerative therapies but have not been reviewed or approved by FDA are widely available, sometimes through clinics that provide these procedures exclusively, though some hospitals or other providers offer them as part of their broader medical services. Most of these businesses or providers offer stem cell therapies derived from a variety of sources—often the patient’s own body—and maintain that these products can be used for a broad array of applications, from cosmetic procedures to treating multiple sclerosis and other serious conditions. In many cases, there is little or no reliable evidence to support the claims behind these potentially unsafe and usually expensive treatments, which are normally not covered by the patient’s health insurance
These businesses have emerged against the backdrop of limited regulatory oversight and enforcement from either state or federal authorities. In the rapidly evolving field of regenerative medicine, it has not always been clear where the responsibility for regulation lies. This lack of clarity has created opportunities for some unscrupulous businesses to market products that have not been fully evaluated for safety and effectiveness.
FDA has recognized the promise of the field of regenerative medicine and the growing risks posed by the proliferation of clinics offering unapproved therapies. In November 2017, the agency released four guidance documents that together constitute its regulatory framework for regenerative medicine, which aims to:
- Clarify the distinctions between products that are subject to the agency’s full drug approval requirements and those that are not.
- Streamline the review process for new therapies and reduce some of the regulatory requirements on product developers.
The agency also pledged to increase its enforcement efforts against providers offering high-risk, unapproved interventions.
This report is drawn from a commissioned legal analysis; interviews with experts from the…