FDA Issues First Draft Guidance on Clinical Trials with Psychedelic Drugs

Agency Recommendations Aim to Inform Psychedelic Drug Development

WEBWIRE – Monday, June 26, 2023

The U.S. Food and Drug Administration published a newdraft guidanceto 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 This is the first FDA draft guidance that presents considerations to industry for designing clinical trials for psychedelic drugs.

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.

Psychedelic drugs show initial promise as potential treatments for mood, anxiety and substance use disorders. However, these are still investigational products. Sponsors evaluating the therapeutic potential of these drugs should consider their unique characteristics when designing clinical studies, said Tiffany Farchione, M.D., director of the Division of Psychiatry in the FDAs Center for Drug Evaluation and Research. By publishing this draft guidance, the FDA hopes to outline the challenges inherent in designing psychedelic drug development programs and provide information on how to address these challenges. The goal is to help researchers design studies that will yield interpretable results that will be capable of supporting future drug applications.

The purpose of the draft guidance is to advise researchers on study design and other considerations as they develop medications that contain psychedelics. Within the draft guidance, the term psychedelics refers to classic psychedelics, typically understood to be drugs such as psilocybin and lysergic acid diethylamide (LSD) that act on the brains serotonin system, as well as entactogens or empathogens such as methylenedioxymethamphetamine (MDMA).

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 thepotential for abuseof 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.

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The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.