Evaluating the FDA Regenerative Medicine Framework: Opportunities for Stakeholders

Though stem cell products, gene therapies and other regenerative techniques have significant potential to 1-day treat traumatic injuries or serious disease, relatively few regenerative medicine products have been approved by the US FDA. However, this has not stopped hundreds of US businesses from selling unproven – and, in many cases, harmful – stem cell and regenerative interventions (SCRIs). Clinics around the world offer unproven SCRIs for a host of conditions to patients with few to no clinical options. As of early 2017, there were estimates of 716 clinics offering SCRIs in the USA alone – a number that has likely grown since then. Using aggressive marketing primarily through patient testimonials and online information that is often exaggerated and incorrect, many clinics offer scientifically under- or untested interventions that have been shown to result in physical, emotional and financial harm. Within the USA, there have been significant efforts made at the national, state and institutional level to regulate the market, educate physicians and inform patients. Most notable are the efforts made by the FDA in developing guidance to delineate more precisely which products would be required to go through the full drug development process, enforcing regulatory standards to curb the practices of riskier clinics and informing the scientific community and the public about responsible practices. Yet the smaller size of clinics, online nature of the marketing, limited resources of regulatory and oversight organizations and lack of overall coordination by multiple stakeholders has resulted in a patchwork of policy and regulatory efforts making it difficult for the FDA and other organizations to curtail the market.