FDA Lays Out First Guidance for Testing Psychedelics as Treatments for Mental Health

The Food and Drug Administration (FDA) has issued its first draft guidance on protocols for research in psychedelic drugs, a sign that the Schedule I substances may soon begin use as medication for mood disorders such as depression and post traumatic stress disorder (PTSD).

"Psychedelic drugs show initial promise as potential treatments for mood, anxiety and substance use disorders. However, these are still investigational products,” Dr. Tiffany Farchione, who oversees the FDA’s regulation of psychiatric drugs, said 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"By publishing this draft guidance, the FDA hopes to outline the challenges inherent in designing psychedelic drug development programs and provide information on how to address these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."

Psychedelic therapy has grown in popularity in recent years, despite the fact that the substances typically involved — psilocybin, ayahuasca and MDMA — remain illegal in much of the world. 

The U.S. Drug Enforcement Administration (DEA) currently lists LSD, MDMA (ecstasy) and peyote as Schedule I substances, alongside other drugs like heroin and cannabis, meaning that the drugs have “no currently accepted medical use and a high potential for abuse.”

That may soon change. Experts suggest MDMA and other once-taboo drugs could receive approval for treatment of mood disorders as soon as next year. Ketamine, a psychedelic drug which has received FDA approval to be used as an anesthetic, is sometimes used off-label for these purposes in specialized clinics.

The National Institutes of Health (NIH) writes that these drugs may stimulate the growth of new neuron connections in the brain, allowing them to be a tool in treatment for mental health conditions.

Early trials have shown improvements among people who suffer from mood disorders when they use the psychedelic drugs alongside other treatments such as talk therapy.

These hopes have spurred a massive market. Some analysts believe the global market for psychedelic drugs will reach $11.82 billion by 2029. And the interest has drawn huge financial backers, too. On Thursday, hedge fund manager and billionaire owner of the New York Mets Steve Cohen announced a $5 million donation to study the benefits of ecstasy to treat PTSD.

However, the NIH notes that it is still unclear how these drugs promote the growth of neurons and what mechanism that serves in bettering a person’s mental health.

The FDA guidelines, revealed Friday and set to be discussed by regulators in August, lay out the requirements scientists must follow when performing trials using these drugs.

“In recent years, interest in the therapeutic potential of psychedelic drugs has been increasing,” the FDA writes.

“However, designing clinical studies to evaluate the safety and effectiveness of these compounds presents a number of unique challenges,” the agency continued.

Those challenges include managing to build trials around drugs that cause “intense perceptual disturbances and alterations in consciousness that can last for several hours”.

“These and other unusual characteristics should be considered when designing clinical studies so that the results of those studies can be interpretable,” the FDA continued.

The FDA says groups hoping to perform clinical trials using these drugs need to assure proper controls are used during their manufacturing to determine that they are pure and chemically sound.

Researchers should also have protocols in place that prevent abuse of the substances during clinical trials, the agency recommends.

Because psychedelic substances are almost always banned in the U.S. — with  rare exceptions like ketamine —  they are only legally allowed for use in FDA-approved clinical trials to study mood disorders.

Upwards of 100 clinical trials using these drugs as medicine are currently recruiting volunteers across the U.S. and Canada.