The US Food and Drug Administration (FDA) has published new draft guidance to highlight fundamental considerations to researchers investigating the use of psychedelic drugs for the potential treatment of medical conditions, including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This is the first FDA draft guidance that presents considerations to industry for designing clinical trials for psychedelic drugs.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, Director of the Division of Psychiatry in the FDA’s Center for Drug Evaluation and Research.
“By publishing this draft guidance, the FDA hopes to outline the challenges inherent in designing psychedelic drug development programmes and provide information on how to address these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.”
Within the draft guidance, the term psychedelics refers to ‘classic psychedelics’, typically understood to be drugs such as psilocybin and lysergic acid diethylamide (LSD) that act on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 brain’s 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 the potential for abuse of these drugs, so safety measures are needed to prevent 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For psychedelics that are currently Schedule I controlled substances, the draft guidance notes that activities associated with investigations under an Investigational New Drug Application must comply with applicable Drug Enforcement Administration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 draft guidance also addresses the role of psychotherapy in psychedelic drug development, considerations for safety monitoring and the importance of characterising dose-response and the durability of any treatment effect.
The FDA is encouraging the public to provide comments on the guidance within 60 days.