FDA Drafts First Guidance on Psychedelic Drug Trials

Credit: US Food and Drug Administration

The US Food and Drug Administration (FDA) has released draft guidelines for future research involving psychedelic drugs for the treatment of psychiatric or substance use disorders, including depression and post-traumatic stress disorder.¹

The move represents the first draft guidance that presents considerations to industry for designing clinical trials for psychedelic drugs.

The guidance are aimed at directing study designers as they develop medications that contain psychedelics, including psilocybin and lysergic acid diethylamide (LSD), as well as “entactogens” or “empathogens” such as methylenedioxymethamphetamine (MDMA).

“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, MD, director of the Division of Psychiatry in the FDA’s Center for Drug Evaluation and Research, in a statement. "By publishing this draft guidance, the FDA hopes to outline the challenges inherent in designing psychedelic drug development programs and provide information on how to address these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."

In the guidelines, drugs should be manufactured in compliance with current good manufacturing practice.

In addition, clinical studies with certain psychedelic drugs could be initiated under an IND in the absence of the typical animal toxicology testing when extensive human exposure and information are available from previously conducted clinical studies 116 and no serious safety concerns were identified.

The guidelines also contain safety warnings because psychedelic drugs could produce psychoactive effects such as mood, cognitive changes, and hallucinations. These drugs do have the potential for abuse.

For psychedelics that are currently Schedule I controlled substances, the draft guidance notes that activities associated with investigations under an Investigational New Drug Application must comply with applicable Drug Enforcement Administration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The draft guidance also looks at the role of psychotherapy in psychedelic drug development, considerations for safety monitoring, and characterizing dose-response and the durability of any treatment effect.

The public will have 60 days to submit comments on the guidelines.