The Food and Drug Administration has promised that after May 31, 2021, the agency will generally apply the requirements that developers of regenerative medicine products (therapies intended to repair or replace damaged cells, tissues and organs) comply with the relevant approval rules for new drugs, medical devices and biological products. The FDA’s increased enforcement doesn’t come too soon: hundreds of companies continue to sell without authorization– and unproven – regenerative therapies direct to consumers, resulting in injury and death in patients.
New research from The Pew Charitable Trusts underscores the need for aggressive FDA enforcement against these companies and for the agency to monitor consumer reviews online for signs of potentially dangerous products that need investigation. Pew found that between 2004 and September 2020, at least 360 people were reportedly injured by regenerative therapies not approved by the FDA or offered in clinical trials. The products involved were often derived from cells taken from patients or from given sources, such as umbilical cord blood and placental tissue. People suffered injuries, including life-threatening infections, blindness, cardiac arrest, and the growth of tumors and lesions. In at least 21 cases, the patients died.
To identify such incidents, Pew analyzed the medical literature; submissions from patients, companies and others to the FDA adverse event reporting system; government publications; Press articles; and online consumer reviews of stem cell companies. The 360 cases are probably only a fraction of the actual total. Even for legal medical products, many adverse events are not reported to the FDA. Companies selling unapproved products are particularly unlikely to notify the agency or other authorities of patient injuries.
Our results suggest that recent efforts by the FDA to reduce the marketing of unapproved therapies have been helpful but not sufficient to protect patients. In 2017, the agency provided a comprehensive framework and guidance to help regenerative medicine companies meet product approval requirements and gave them three years to become compliant. Authorities subsequently extended the deadline to May 31, citing the coronavirus pandemic. During this period, the FDA sent letters to more than 350 companies and individual suppliers warning them that their products could violate the law, and requested legal injunctions against a handful of clinics.
Despite these measures, patient injuries and troubling business practices remain all too common. Pew found reports of 59 people experiencing adverse events (adverse patient experiences associated with the use of medical treatments) since 2017. Clinics continue to market unapproved therapies, including exosomes derived from stem cells and platelet-rich plasma, which is marketed as a regenerative intervention and considered a blood product by the FDA. Companies also added COVID-19[female[feminine to the long list of diseases and health problems that they claim their regenerating products can treat. Such marketing often attracts very sick and desperate patients to try expensive therapies that have not been proven to work, but which can have serious and even fatal consequences.
FDA oversight of online reviews could aid oversight
The FDA should also consider looking for adverse event reports in online journals for regenerative medicine companies and use this data to inform its application priorities. The agency has recognized that despite the variability in the quality of these reviews, social media monitoring could speed the detection of potential medical product safety issues.
Pew’s research tested this hypothesis, examining consumer reviews on Facebook, Google, and Yelp for stem cell companies in a database compiled by Leigh Turner, a bioethicist who studies the market for direct-to-consumer stem cells. . We identified 21 probable cases of patient harm related to unapproved regenerative products. Most of the reviews (18) were published from 2018 to 2020, the first published in 2014. In contrast, we found only five probable cases submitted to the FDA’s MedWatch reporting system, with the most recent arriving in September. 2018.
The 21 reviews online described harms such as serious infections, pain, loss of mobility and severe allergic reactions. A consumer wrote that he had contracted “an infection which required 6 days of hospitalization, 2 emergency surgeries, 6 weeks of IV antibiotics and 6 months of oral antibiotics”. Another reviewer recalled that on the “5th day after my stem cell in my lumbar spine, I woke up in the most severe pain of my life. I couldn’t walk and was bedridden for 8 weeks. All patients reported receiving plasma products rich in stem cells or platelets, and 14 reported that the therapies were given by direct injection, often to the knees, elbows or other joints.
High rate of adverse events for stem cell injections
The majority of adverse events reported and identified by Pew were from a to study published in January 2021 which reviewed the medical records of an insurance company for 2,964 clients who received stem cell injections to treat osteoarthritis, a condition for which there is no approved stem cell product. FDA. Records revealed that 242, or 8%, of these patients had problems such as tumor formation, infection, and pain; 82 people were hospitalized. The author also found that adverse events were four times less likely in patients with osteoarthritis who received injections of steroids or fluids instead of stem cells.
Many consumers have expressed interest in the potential of regenerative medicine to treat disease and heal injuries, but widespread sales of unproven products threaten patient safety. The FDA must enforce the law to eliminate the marketing of unapproved products and ensure that developers of cell and gene therapy conduct clinical trials to demonstrate that their interventions are safe and effective. To support its enforcement work, the agency should encourage consumers and clinicians to report adverse events and watch for potential cases of harm described in online notices. These measures will help the FDA to more effectively eliminate risky products and promote a safer regenerative medicine market for patients.
Liz Richardson leads The Pew Charitable Trusts Health Products Project.