FDA issues first draft guidance on clinical trials with psychedelic drugs

The U.S. Food and Drug Administration published a new draft guidance to highlight fundamental considerations to researchers investigating the use of psychedelic drugs for potential treatment of medical conditions, including psychiatric or substance use disorders. This is the first FDA draft guidance that presents considerations to industry for designing clinical trials for psychedelic drugs. The agency said, “There has been growing interest in the therapeutic potential of psychedelic drugs in recent years. They are being evaluated for use in the potential treatment of conditions such as depression, post-traumatic stress disorder, substance use disorders and other conditions. However, designing clinical studies to evaluate the safety and effectiveness of these compounds presents a number of unique challenges that require careful consideration…The purpose of the draft guidance is to advise researchers on study design and other considerations as they develop medications that contain psychedelics. Within the draft guidance, the term psychedelics refers to ‘classic psychedelics,’ typically understood to be drugs such as psilocybin and lysergic acid diethylamide that act on the brain’s serotonin system, as well as ‘entactogens’ or ’empathogens’ such as methylenedioxymethamphetamine. The document describes basic considerations throughout the drug development process including trial conduct, data collection, subject safety and new drug application requirements. For example, psychedelic drugs may produce psychoactive effects such as mood and cognitive changes, as well as hallucinations. As a result, there is the potential for abuse of these drugs, which is a drug safety issue that requires careful consideration and putting sufficient safety measures in place for preventing misuse throughout clinical development. For psychedelics that are currently Schedule I controlled substances, the draft guidance notes that activities associated with investigations under an Investigational New Drug Application must comply with applicable Drug Enforcement Administration regulatory requirements. The evidentiary standard for establishing effectiveness of psychedelic drugs is the same as for all other drugs. However, there are unique factors investigators may need to consider when designing their clinical trials if those trials are to be considered adequate and well-controlled. The draft guidance also addresses the role of psychotherapy in psychedelic drug development, considerations for safety monitoring and the importance of characterizing dose-response and the durability of any treatment effect. The FDA is encouraging the public to provide comments on the draft guidance. Comments should be submitted within 60 days to ensure the agency considers them.” Publicly-traded companies in the space include Compass Pathways (CMPS), Mind Medicine (MNMD), Atai Life Sciences (ATAI), Relmada Therapeutics (RLMD) and Clearmind Medicine (CMND).