FDA Releases First-Ever Draft Guidance on Psychedelic Drug Research

by Liz Neporent
June 26, 2023 at 10:05 AM UTC

FDA releases draft guidance for researchers studying psychedelic drugs in medicine, emphasizing the need for unique considerations in clinical trials

Clinical Relevance: Psychedelic drugs may have therapeutic potential in psychiatric and substance use disorders

  • FDA released draft guidance for researching psychedelic drugs in medicine.
  • Guidance emphasizes unique considerations for clinical trials and potential for abuse.
  • Inclusion of diverse representation and safety measures were highlighted for effective evaluation and development of psychedelics.

Last week, the U.S. Food and Drug Administration (FDA) published its first-ever draft guidance for researchers investigating the use of psychedelic drugs in medicine. The agency’s recommendations encompass clinical trial design for evaluating psychedelic drugs, including their application in treating psychiatric or substance use disorders.

“Psychedelic drugs show initial promise as potential treatments for mood, anxiety, and substance use disorders. However, these are still investigational products,” said Tiffany Farchione, MD, director of the Division of Psychiatry in the FDA’s Center for Drug Evaluation and Research. “Sponsors evaluating the therapeutic potential of these drugs should consider their unique characteristics when designing clinical studies.”

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Therapeutic Potential of Psychedelics

Classic Psychedelics and Self-Harm

According to the FDA, the term psychedelics refers to “classic psychedelics.” These typically include 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). Nine drugmakers have psychedelics in development. 

The guidance highlights the psychoactive properties of psychedelic drugs. The agency emphasizes safety measures throughout clinical development, highlighting the potential for mood-altering effects that can contribute to abuse. For psychedelics classified as Schedule I controlled substances, researchers must comply with DEA regulations for investigational activities under a New Drug Application.

Psychedelic Paradigm Shift

“This is huge,” said Ayana Jordan, MD, the Barbara Wilson Associate Professor of Psychiatry at NYU Langone Health’s Department of Psychiatry of the guideline publication. “The tide has turned for psychedelics and put them in a much more favorable place not only with the public but in medicine.” 

In a conversation with Psychiatrist.com, Jordan pointed out that society has historically villainized psychedelic drugs. Their negative reputation stems largely from cultural, political, and societal norms, not scientific reasoning, she noted.

“I think what has really happened is that there is a more welcoming milieu of using substances that have already existed naturally for discovery. We’ve realized, for instance, substances that were heavily criminalized can have a lot of medicinal benefits and actually it hurts people within the United States by keeping them illegal.”

Diverse Trial Representation

Jordan welcomed guidance inclusion of fair representation of races and sexes in trials as the best way to detect clinically significant variance in response.

“If a study is focused primarily on one race and sex, then I don’t know literally how that’s going to impact somebody else,” she said.

And, while there are some genetic differences among groups, Jordan said that’s not the whole story. 

“For example, we know that the way males and females engage in care is very different,” she said.

According to Jordan, these differences go beyond how different genders and cultures respond to a dosage of a medication. Jordan posed a question: If we haven’t studied diverse populations, is the ineffectiveness of a drug in certain groups the result of inadequate representation? How, when, and where they receive it may change the effect.

Psychedelic Momentum Builds

Psychedelics have been gaining acceptance. Speaking at Psychedelic Science 2023 in Denver last week, New York Jets quarterback Aaron Rodgers spoke about how psychedelics, like psilocybin and ayahuasca, have improved his mental health. (Taking ayahuasca doesn’t violate the NFL’s drug policy.)

In Oregon, physicians can now legally prescribe psilocybin “magic mushrooms.” Additionally, Colorado recently passed legislation that legalized magic mushrooms for recreational purposes. Several large cities have also decriminalized magic mushrooms, while at least seven states are actively considering similar measures.

The FDA encouraged the public to provide comments on the draft guidance. The agency will consider comments submitted within the next 60 days before it issues its final statement.

Watch as Scott T. Aaronson, MD, Chief Science Officer for the Institute for Advanced Diagnostics and Therapeutics at Sheppard Pratt, shares clinically relevant insights regarding psychiatry and psychedelics in this JCP 5 Minute Pearls.

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