FDA Issues Guidance on Psychedelic Drug Use in Clinical Trials

On June 23, 2023, the United States Food and Drug Administration issued its first draft guidance on using psychedelic drugs in clinical trials. Marking a pivot in the clinical trial landscape, these guidelines hope to inform drug development for this class of drugs.

The 14-page draft offers insight into chemistry, manufacturing, and controls for clinical trials. It also provides recommendations for nonclinical use, information on clinical pharmacology, assessments for abuse potential, and clinical use.

Although many researchers and clinicians have theorized that psychedelics could have critical benefits in healthcare, a lack of guidance and fearful rhetoric surrounding these drugs may have deterred or delayed research.

The FDA acknowledges that, despite the potential uses, designing clinical trials to assess psychedelics’ safety and efficacy can be a challenging approach.

“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 the FDA press release.

“By publishing this draft guidance, the FDA hopes to outline the challenges inherent in designing psychedelic drug development programs and provide information on how to address these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 guidance is focused on classic psychedelics, which are 5-HT2 agonists, entactogens, or empathogens, including psilocybin, lysergic acid diethylamide (LSD), and methylenedioxymethamphetamine (MDMA).

The FDA maintains that all drugs developed, including psychedelic drugs for clinical trials, must follow current good manufacturing practices (CGMP) under the Federal Food, Drug, and Cosmetics Act. Sponsors and manufacturers will be responsible for providing information on the drug’s identification, purity, and strength.

Other recommendations suggest that sponsors evaluate how diet and food consumption may impact the pharmacokinetics of oral psychedelic drug formulations. Additionally, researchers should conduct in-depth assessments of the potential drug–drug and drug–disease interactions.

The FDA will continue accepting commentary on the guidance until August 23, 2023, before finalizing any industry standards.