Marking an impressive milestone in the development of MDMA-assisted psychotherapy for PTSD, the U.S. Food and Drug Administration (FDA) has approved the treatment for Expanded Access, allowing certain patients access to the therapy before full market approval is granted. Ten treatment clinics in the United States are reported as ready to commence administration of the therapy, while Phase 3 trials are ongoing and full approval is estimated for 2022.

It takes a long time for a new pharmaceutical treatment to move from initial discovery to market approval. The process of human clinical trials alone can take anywhere from five to seven years, and that is assuming everything goes perfectly. The non-profit Multidisciplinary Association for Psychedelic Studies (MAPS) has been working for decades to establish MDMA-assisted psychotherapy for PTSD.

The FDA’s Expanded Access program is designed to allow patients with serious illnesses access to treatments still in the processes of clinical investigation. Often informally referred to as the compassionate use program, Expanded Access offers individuals conditional access to specific treatments.

MDMA-assisted psychotherapy for PTSD is currently deep in Phase 3 clinical trials. While complete market approval is still most likely two to three years away, Expanded Access to the treatment has now been granted by the FDA.

MAPS has announced 10 treatment sites in the United States will initially begin Expanded Access administration of the therapy. The FDA Expanded Access approval is slightly more limited than the broader application being tested for, so pre-approval administration will only apply to “treatment-resistant patients with moderate to severe treatment-resistant PTSD”.

Moving forward, MAPS suggests more than 120 clinics have already applied to commence MDMA therapy under Expanded Access conditions. Once the program is up and running patients will apply for treatment directly to the individual clinic, however, the FDA still has ultimate approval over patient applications. Historically, the FDA has approved the vast majority of Expanded Access patient requests.

Fifty patients have been initially approved for Expanded Access treatment and MAPS will present patient data to the FDA from a number of these first Expanded Access treatments. The plan is to subsequently expand the Expanded Access program once its real-world efficacy has been established.

“We commend FDA for recognizing the great unmet medical need of PTSD by allowing access to MDMA-assisted psychotherapy on a compassionate basis for people with treatment-resistant PTSD,” says Rick Doblin, founder of MAPS. “We are delighted to begin generating real-world evidence about this potential new treatment.”

A view of a treatment room at the University of Wisconsin ahead of the Phase 3 trials MAPS

The patients being administered the treatment under the Expanded Access program will not formally be included in the Phase 3 trial data used to evaluate final market approval, but the FDA does review Expanded Access patient data annually and adverse events that occur as part of these Expanded Access treatments can be taken into account during final market approval decisions. A large review of Expanded Access requests over the past decade did reveal that it is very rare for the program to disrupt clinical trial results.

As the treatment has yet to be officially approved, the patient costs cannot be covered by health insurance. In the past MAPS has estimated the initial cost per patient of a program of MDMA-assisted psychotherapy could be around US$15,000.

“Combining the powerful effects of pharmacology with the potential depth of psychotherapy is a compelling model for harnessing advances in neuroscience and psychopharmacology without ignoring the complexity, richness and innate capacity of the human psyche,” explains Michael Mithoefer, Acting Medical Director for MAPS Public Benefit Corporation. “I’m delighted that the Expanded Access Program will now allow some patients to access to this modality as MAPS’ Phase 3 research continues.”

Full market approval for the treatment is still most likely over two years away, however, Expanded Access marks a significant milestone for a number of researchers who have been toiling for decades on this project. The program allows for the first psychedelic-assisted psychotherapy clinics to begin legally administering MDMA therapy to patients.

MAPS has previously hypothesized thousands of psychedelic-assisted psychotherapy clinics will open up across the United States. In the near future these clinics may not only administer MDMA for PTSD but also psilocybin, which is currently proving promising for a variety of conditions, including major depression.

The FDA approval for Expanded Access is yet another validation of MDMA-assisted psychotherapy’s safety and efficacy following extraordinarily positive early clinical trial results. Current Phase 3 trials are set to run until 2021 and complete market approval could come as early as 2022.

Source: MAPS