On September 6, 2024, the Reagan-Udall Foundation for the U.S. Food and Drug Administration (FDA)1 held a hybrid public meeting, comprising both in-person and virtual attendance, on advancing treatments for post-traumatic stress disorder (PTSD), including the potential role of psychedelics.2 The meeting included a panel of representatives from several key federal agencies, including representatives from the FDA, Substance Abuse and Mental Health Services Administration (SAMHSA), U.S. Department of Defense (DoD) and U.S. Department of Veterans Affairs (VA).
This meeting took place nearly a month after the FDA declined the approval of Lykos Therapeutics’ New Drug Application (NDA) for midomafetamine-assisted therapy (MDMA-AT) as a treatment for PTSD. The decision, which surprised and disappointed many researchers, health care providers and patients, delayed what many hoped would be a breakthrough in FDA-approved PTSD treatments.
In the first portion of the public meeting, representatives from federal agencies discussed their various efforts and projects to advance PTSD research and treatment, including psychedelic research. The second portion of the meeting consisted of public comment, with many of the commenters expressing a need for additional PTSD treatment options and disappointment over the FDA’s decision to decline approval of MDMA-AT.
This public meeting underscored the urgent need for more effective mental health treatments. The initiatives discussed by federal agency representatives offer promising opportunities for drug developers, researchers, health care providers and patients.
Bernard Fischer, Deputy Director of the Division of Psychiatry in the Office of New Drugs at the FDA, acknowledged that the two currently available FDA-approved drugs for PTSD do not work for everyone and emphasized that the FDA and the federal government at large understand the existence of an unmet need for safe and effective PTSD treatments.
Dr. Fischer provided advice for PTSD drug developers. He noted that the FDA wants to see studies with a demographic mix representative of the U.S. as well as studies representing the different aspects of PTSD, including various kinds of traumatic events, various ranges of time since traumatic event, and participants with single and repeated traumatic event(s).
He also mentioned the Breakthrough Therapy designation programs that exist for drugs and devices. These programs allow for additional communication and guidance along the drug approval process for drugs or devices which the FDA considers to potentially hold a meaningful advantage over existing therapies. The FDA has already granted this designation to several psychedelics in development as well as for devices to treat PTSD.
Neeraj Gandotra, Chief Medical Officer for SAMHSA, described SAMHSA’s focus on service grants and technical assistance. He highlighted SAMHSA’s mental health block grants and their community behavioral health clinics, where SAMHSA has invested over a billion dollars for mental health services, including crisis services. SAMHSA’s ReCAST program has provided technical assistance to over 950 partner organizations, training more than 100,000 mental health professionals and community members in trauma-informed care. In addition, SAMHSA’s National Child Traumatic Stress Network has assisted over a million individuals.
Elyse Katz, a DoD contractor, discussed the design of the Military and Veterans PTSD Adaptive Platform Clinical Trial (M-PACT). This large trial is the first platform-type trial in psychiatry, allowing for testing of multiple drugs simultaneously and sequentially with the sharing of placebo data across treatment arms. Four drugs have been chosen for study at this time, including an intranasal racemic ketamine formulation. The trial incorporates the collection of biomarker and clinical assessment data, aiming to adopt a precision medicine approach. This approach tailors treatment to individual patients, ensuring they receive the therapy most likely to be effective for their unique condition. The trial is accepting requests from industry stakeholders interested in having their product considered for study, organizations interested in serving as clinical trial sites and patients interested in enrolling in the trial.
Paula Schnurr, the Executive Director of the VA National Center for PTSD, emphasized the VA’s commitment for potential future implementation of psychedelics by supporting and conducting psychedelic research, including evaluation of MDMA and psilocybin for PTSD and depression.
Miriam Smyth, the Acting Director of the Brain, Behavioral and Mental Health Broad Portfolio (BBMH), discussed the VA’s funding for psychedelic research where, since January 2024, VA researchers could submit proposals and requests for funding on psychedelic research through the VA’s intramural research program, with funding decisions coming this fall. Prior to this, psychedelic research involving VA researchers was funded by external organizations and included fewer patients.
Miriam Smyth also discussed the rollout of a mental health survey, the Million Veterans Program Measures Investigating Neuropsychiatric Disorders (MVP MIND), which is in its early stages with five VA facilities participating and further expansion planned.
While some public comments supported the FDA’s decision to decline MDMA-AT approval, many speakers shared personal accounts of positive, healing experiences with MDMA-assisted therapy, highlighting its potential benefits for treating PTSD. The public comment period included numerous stories from veterans who shared the hardships they endured from ineffectively treated PTSD and who highlighted the significant suicide risk associated with PTSD. Many of the comments emphasized the need for effective and safe PTSD treatment options.
Following the public meeting, Rick Doblin, MAPS Founder and President, and Betty Aldworth, MAPS Director of Education, submitted comments on behalf of MAPS to the Reagan-Udall Foundation, offering insights on the potential of psychedelic therapies for PTSD.3,4 MAPS praised the foundation for holding the meeting and discussed the potential for psychedelic-assisted therapies to help treat PTSD.
Regarding separating the role of therapy versus drug in clinical trials, MAPS noted that treating PTSD patients with MDMA but without therapy is an “ethically questionable approach” and postulated that the “MDMA-assisted therapy placebo group in the Lykos trials would likely demonstrate that the inner-directed therapy alone generates better responses than current front-line treatments (with significantly lower drop-out rates).”5
The commentary provided four strategies to increase research and FDA approval of psychedelic-assisted therapies:
The Reagan-Udall Foundation for the FDA public meeting highlighted that, notwithstanding the FDA’s decision to decline approval of MDMA-AT, federal agencies understand the crucial need for safe and effective PTSD treatment options and have developed programs to further research, therapist training and patient access for mental health therapies, including psychedelics. Psychedelic stakeholders can learn about and take advantage of these programs, including submitting psychedelics in development for research, accessing research funding, participating as study trial sites, enrolling patients in psychedelic trials, therapist training or patient-access advocacy.
1 The Reagan-Udall Foundation for the FDA is a nonprofit organization created by Congress to assist the FDA in protecting and promoting public health.
2 Advancing Treatments for Post-Traumatic Stress Disorder, Hybrid Public Meeting, Reagan-Udall Found. for the Food & Drug Admin. (Sept. 6, 2024, 1-3:30pm ET), https://reaganudall.org/news-and-events/events/advancing-treatments-post-traumatic-stress-disorder.
3 MAPS is a 501(c)(3) nonprofit research and educational organization. In 2014, MAPS founded and incubated Lykos Therapeutics, a drug-development public benefits company (formerly known as MAPS PBC until it changed names to Lykos Therapeutics in January 2024) to develop investigational MDMA-AT for PTSD. Following FDA’s decision to decline approval of MDMA-AT, Rick Doblin resigned from the Lykos Board, but remains president of MAPS.
4 MAPS Comment to Reagan Udall on Advancing Psychedelic Therapies, Multidisciplinary Ass’n for Psychedelic Studies (MAPS), https://maps.org/news/update/maps-comment-to-reagan-udall-on-advancing-psychedelic-therapies/ (last visited Oct. 7, 2024).
5 Id.
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