American regulators get tough
A strongly worded article in JAMA gives a heads-up to stem cell clinics operating outside of the regulatory framework that they may not be granted more time to reach compliance with Food and Drug Administration (FDA) regulations.
The article, written by the FDA during a pandemic when it is busy with other concerns, underscores the agency’s duty to promote evidence-based regenerative medicine.
Authors Stephen Hahn, the FDA’s new commissioner, and Peter Marks, the director of the Center for Biologics and Evaluation and Research, write that “these products, whether autologous or allogeneic, are not inherently safe and may be associated with serious adverse consequences”.
There has been an increasing number of unscrupulous clinics in US advertising treatments for diseases and conditions ranging from autism to ageing. But the safety and efficacy of these products are yet to be proven. For instance, three women became blind after receiving untested stem cell treatment on their eyes in a Florida clinic.
This article also puts the unapproved stem cell industry issue in the context of Covid-19.
It also warns that “the increasing number of adverse events being reported following the widespread use of unapproved regenerative medicine therapies at hundreds of clinics across the country make it necessary for the FDA to act to prevent harm to individuals receiving them.”
To evaluate whether a particular cellular product complies with FDA regulations, the authors make practical recommendations for patients. For people considering therapies involving a cellular product, the following steps are suggested.
First, the patient should verify whether the product is FDA-approved or whether an investigational new drug (IND) application has been filed with the regulatory body.
Next, the patient must provide written informed consent to participate in a clinical trial under that IND and compliance with institutional review board (IRB) requirements.
It is critical that the patient not be charged for the product or for participation in the clinical trial. However, if there is evidence of benefit to the patient, the amount charged must not be more than the direct costs to make the product – which is known as cost recovery. A certified accountant must document this.
Moreover, patients (and their family, friends and doctors) are strongly encouraged to report adverse events. The medical provider should explain the precise steps for doing so like using patient-friendly forms to report the matter. Such reports help the FDA to get a whole picture of the full spectrum of adverse events.
Finally, patients enrolled in the clinical trial should receive a summary of results after the clinical trial is completed.
The authors opine that medical professionals are in the best position to help patients to identify whether a particular regenerative medicine treatment is appropriate. As there may be a wide range of knowledge and awareness of stem cell interventions among doctors, this may not always be true.
There are clinics which may attempt to fly under the FDA’s radar. The authors request engagement “to help to ensure that instead of remaining unintentionally or intentionally hidden, potentially harmful unapproved regenerative medicine therapies are identified and then removed from the market.”
In fact, there are credible studies about the emergence of numerous such clinics but the FDA needs information from the patients themselves.
As the FDA’s enforcement discretion period finishes in November 2020, the next few months may provide some inkling of what the enforcement landscape will be like.
Earlier, the FDA announced regulatory criteria for marketing with the publication of guidance documents. These documents, which build upon the agency’s risk-based, flexible regulatory regime, enable stakeholders to comprehend better how the criteria apply to their products by clarifying technical terms including “minimal manipulation” and “homologous use”.
This approach allows manufacturers sufficient time to engage with the FDA to comply with the requirements. For instance, the clinic may need to determine whether they must submit a marketing authorisation application to the FDA for their approval (premarket review). In the recent article, there is no indication of a deadline extension.
Taken together, the recent FDA article and the rise in the warning letters sent by the FDA to clinics offering unapproved stem cell treatments signal that these establishments may not get more time to comply with stringent FDA requirements.
Dr Patrick Foong is a law lecturer at Western Sydney University. His research interest lies in bioethics and health law.
stem cell therapies