FDA bid shows it’s hard to test psychedelic treatments
The first attempt to win government approval of a psychedelic drug for mental health treatment is generating skepticism about its clinical trials, a case that reveals the unique challenges of bringing mind-altering, illegal drugs into mainstream medical care.
A recent independent analysis questioned the integrity of patient studies that are being used to support the Food and Drug Administration application for MDMA — also known under the street name ecstasy — to treat post-traumatic stress disorder, citing a host of problems.
Among the most difficult to overcome: Subjects in the placebo arm of the trials — who got dummy MDMA — knew they didn’t get the real drug because they didn’t experience distortions in consciousness, according to the draft report by the nonprofit Institute for Clinical and Economic Review. In other words, the trial was not “blinded” for purposes of comparing the drug’s effect, which undermines the results.
ICER researchers also said trials may have been skewed by therapist participants who are boosters of MDMA as a mental health treatment, as well as large numbers of subjects who had previous experience taking MDMA. The report cited concerns that some participants that received MDMA felt “pressured to report good outcomes and suppress bad outcomes,” and flagged a documented case of therapist misconduct that raised safety risks.
Meanwhile, a separate group of researchers is petitioning the FDA, which put MDMA-assisted therapy on a fast track for approval, to slow down and convene an extended public hearing to consider the many unique issues at play.
The potential of psychedelics has bloomed in recent years as a treatment for mental health disorders and addiction, with technology luminaries and investors betting hundreds of millions of dollars on companies developing a range of treatments, from psilocybin to ibogaine. MDMA is the first of these to reach the doorstep of FDA approval, and leaders in the psychedelic medicine field say the agency’s decision could lay the groundwork for how the federal government regulates drugs that do not fit the mold of traditional pharmaceuticals.
“It is a huge decision with pretty sweeping consequences,” said Shane Pennington, a Washington D.C. attorney who specializes in FDA regulations involving controlled substances. “It’s going to be the first shot fired. It’s going to be precedential.”
ICER’s report highlights the complexity of the FDA’s decision as well as considerations for insurers, who use ICER’s assessments to inform decisions about coverage. While ICER staff acknowledged that some PTSD patients experienced “substantial benefit,” they found that the evidence in two positive, late-stage trials of therapy with MDMA was too murky to determine a net benefit or a fair price, according to the draft report last month.
“This is pretty unusual all the way through,” said David Rind, ICER’s chief medical officer. “If this is the best trial that can be done, it’s not that good.”
Lykos Therapeutics, the company seeking the drug approval, has said the FDA is expected to make a decision by August and said it supports the agency scheduling an open public hearing.