The Food and Drug Administration released guidance in July outlining recommendations for sponsors developing psychedelic drugs for the treatment of medical conditions, including psychiatric disorders and substance use disorders. The guidance addresses chemistry, manufacturing, nonclinical testing, abuse potential and clinical trial design, while emphasizing that it reflects the agency’s current thinking and is not legally binding.
Here are eight things to know:
- The guidance covers multiple psychedelic drug classes. FDA said the recommendations apply to classic psychedelics, including psilocybin and lysergic acid diethylamide (LSD), as well as entactogens such as 3,4-methylenedioxymethamphetamine (MDMA).
- Sponsors should address challenges in trial design. FDA said psychedelic drugs present unique challenges because they can produce perceptual disturbances and alterations in consciousness that may lead to functional unblinding and bias in clinical trials.
- The agency encourages early engagement. FDA said sponsors should meet with the agency early in the drug development process to discuss approaches for addressing clinical development considerations.
- Treatment durability should be evaluated. For products intended to treat chronic illnesses, FDA recommends evaluating treatment effects at 12 weeks and continuing blinded follow-up, typically for 12 months, with prespecified retreatment criteria.
- Psychotherapy should be carefully evaluated. FDA said sponsors should describe whether psychological support or psychotherapy is paired with drug administration and consider study designs that help distinguish the contribution of each to treatment outcomes.
- Safety monitoring expectations are extensive. The agency recommends two monitors during treatment sessions, including a licensed mental healthcare professional as the lead monitor. If the lead monitor is not a physician, an on-call physician should be able to reach the clinical site within 15 minutes.
- Abuse potential remains a key focus. FDA said psychedelic drugs should undergo abuse potential assessment during development, with data supporting scheduling recommendations if a product is approved.
- The guidance applies beyond commercial drug developers. FDA said the recommendations also apply to clinical trials conducted under research investigational new drug applications, including studies led by academic researchers that are not intended to support marketing applications.
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