A Recent FDA-Sponsored Meeting Addressed Psychedelic Drug Development, Including the Limits of FDA’s Regulatory Authority

Dr. Farchione discussed two aspects — especially relevant to psychedelics — that the FDA does not regulate: psychotherapy and the practice of medicine.

Dr. Farchione noted that psychotherapy is not just a confounding factor but could potentially act as an integral component of a psychedelic medication’s safety and efficacy profile. However, within the context of a psychedelic intervention, there is no rigorous definition of what exactly the psychotherapeutic portion entails. This complicates the FDA’s assessments of the drug and future product labeling. She also pointed out that the assumption that psychotherapy is a necessary component in a psychedelic intervention has not been rigorously tested. According to Dr. Farchione, well-done factorial studies — studies that address more than one intervention and which are unfortunately large and costly — would be highly informative in helping to determine the contributions of psychotherapy to the safety and efficacy of a psychedelic medication and would assist in creating better product labels.

Dr. Farchione discussed that the Code of Federal Regulations allows the FDA to specify if a medication should be used only in conjunction with another mode of therapy and included a slide with a table (shown below) showcasing the somewhat abbreviated nature of the psychotherapy-type language in the product labeling for several FDA-approved medications.

She noted that if the FDA is going to specify that a psychedelic should only be used in conjunction with a primary mode of therapy, “we probably ought to know if that mode of therapy is necessary.”⁴

Regarding the practice of medicine, Dr. Farchione pointed out that many of the psychedelic therapy aspects of set (often defined as the patient’s mindset and expectations) and setting (often defined as the physical and social environment) are outside the FDA’s regulatory authority. She also discussed that, while a drug safety program called REMS (Risk Evaluation and Mitigation Strategy) — which the FDA can require for certain medications with serious safety concerns — might be able to address some of the safety components of the psychedelic sessions by notifying, educating or enforcing actions to decrease the frequency or severity of the risk(s), a REMS is not intended to address efficacy. As such, a REMS would not be able to address aspects of psychotherapy or treatment settings related to efficacy. However, given the novel and potent nature of psychedelic drugs, the FDA is likely to implement stringent post-market surveillance requirements to monitor the drugs’ real-world efficacy and safety.

In addition, while the FDA has authority to mandate the monitors’ credentials in clinical trials, the FDA would not have the same authority post-marketing; the regulation of the monitors’/therapists’ credentials would likely fall under state medical boards or other state-level authorities.

Although the FDA approves a medication for a specific indication, prescribers can use the medication to treat a different condition(s) because this falls under the practice of medicine, which the FDA does not regulate. As such, “off-label” prescribing is not regulated by the FDA. However, the FDA is able to take regulatory action against companies who use information not included in the product label to promote “off-label” use.