FDA’s Expanded Access or Compassionate Use Program

This is the third in a series of three blogs examining the use of MDMA as a psychedelic medicine. In this installment, we will discuss the FDA’s Expanded Access or Compassionate Use program and how The Pearl Psychedelic Institute is the first site in the United States to begin this program and how it will be conducted.


MDMA-assisted therapy is not exactly a new idea. Although it was first synthesized in 1912 by a German chemist, MDMA had no known utility until an American chemist, Sasha Shulgin, re-synthesized it in his home lab in the Bay Area around 1976 and gave it to a therapist friend of his, Leo Zeff, who helped realize and instruct others in some of the potential uses of MDMA in the context of psychotherapy. MDMA was used as an adjunct to psychotherapy legally by, at one time, as many as 4,000 psychiatric and mental health professionals until increasing recreational use alarmed the authorities to the point that the DEA responded by classifying MDMA as a Schedule 1 substance which is the most restrictive scheduling possible which implies that MDMA has no recognized medical use and it has a high possibility for abuse. Thanks to the diligent efforts of Rick Doblin and his staff at the Multidisciplinary Association for Psychedelic Studies (MAPS), MDMA is currently in Phase 3 studies for its use in the treatment of PTSD which is the last phase of investigative research before it can be considered for approval by the FDA.


A promising event along this road for MDMA occurred on August 16, 2017 when the FDA granted Breakthrough Therapy status to MDMA-assisted therapy for the treatment of PTSD. Many mistakenly assumed that this designation would put MDMA-assisted therapy on a fast track to approval but that was not to be the case. However, it did allow for the FDA to publicly recognize that at that time in the research, the evidence was demonstrating that this treatment is not only possibly effective in mitigating the symptoms of a life-threatening condition (PTSD) but that it also appears to demonstrate substantial improvement over current accepted therapies for PTSD.


After an investigational drug or therapy has demonstrated sufficient safety and efficacy during clinical trials, the FDA may allow certain sites with properly trained providers to begin treating patients with the drug or therapy even though it has not been approved yet by the FDA. This special program is known as Expanded Access or “Compassionate Use” and the FDA stipulates that the patients being treated have a “serious or immediate life-threatening condition” which qualifies those suffering with treatment-resistant PTSD. On December 20, 2019, the FDA gave MAPS permission to treat a total of 50 patients with treatment-resistant PTSD with MDMA-assisted therapy through the Expanded Access program. MAPS selected 10 sites around the United States to participate in this program. While this is a very small number of patients that can currently be treated nationwide, the goal is to be able to complete the specialized training of several additional therapist teams around the United States and to continue to gather safety and efficacy data pertaining to MDMA-assisted therapy.


“After an investigational drug or therapy has demonstrated sufficient safety and efficacy during clinical trials, the FDA may allow certain sites with properly trained providers to begin treating patients with the drug or therapy even though it has not been approved yet by the FDA. This special program is known as Expanded Access or “Compassionate Use” and the FDA stipulates that the patients being treated have a “serious or immediate life-threatening condition” which qualifies those suffering with treatment-resistant PTSD.”

The Pearl Selected

On July 27, 2020 The Pearl MDMA Project, based in Waynesville, NC, was notified by MAPS that we had been selected as one of the 10 sites to be able to conduct MDMA-assisted therapy within the Expanded Access program. Each site will generally be allowed to treat up to 5 patients but this number could be fluid although the cap at 50 patients nationwide is not. Each site is permitted to have up to two treatment teams (pairs) and each pair must be led by a physician or a doctoral level psychologist. Expanded Access will be conducted under strict protocols and supervision in order to ensure safety and treatment integrity. Each treatment team will be mandated to treat their first patient under direct supervision from an experienced MAPS researcher and sessions will be recorded with frequent meetings with said supervisor. Once the first patient has completed the treatment protocol and supervisor gives their approval, treatment teams are then certified to provide MDMA-assisted therapy and recording sessions is no longer required although MAPS, as the Expanded Access program sponsor, will continue to maintain close contact with treatment teams with both the DEA and the FDA likely to provide further oversight.


The Pearl MDMA Project became the catalyst for the forming of The Pearl Psychedelic Institute which is a non-profit organization that was founded in Waynesville, NC in 2021. The Pearl provides a non-profit host for the MDMA-assisted therapy Expanded Access program and it has a three-pronged mission to conduct research and treatment using psychedelic medicines, to provide training and community education and to eventually have a reduced-cost clinic in WNC where patients can have access to affordable psychedelic-assisted therapy in a safe environment with well-trained and skilled professionals. The MDMA-assisted therapy Expanded Access program was formally activated by MAPS in early 2022 and the screening process for the first two patients is currently underway.


“The Pearl MDMA Project became the catalyst for the forming of The Pearl Psychedelic Institute which is a non-profit organization that was founded in Waynesville, NC in 2021.”

How a Patient Can Be Considered for the Expanded Access Program

To be considered for this program, one must visit the website at www.pearlpsychedelicinstitute.org and under the Contact tab there is an online form titled “EAMP1 Inquiry Form” and this is where one would go to register their interest in being considered for MDMA-assisted therapy. Another route to registering interest in participating is to send an email to program1@pearlpsychedelicinstitute.org and an auto-reply email will then be sent to you with a link to the appropriate page on our website.


Since MDMA-assisted therapy for PTSD is still considered an experimental treatment, the Expanded Access program will generally follow much of the protocol of the formal Phase 2 and Phase 3 research. There are numerous Inclusion and Exclusion Criteria that concern psychological characteristics as well as medical issues which, due to the experimental nature of the Expanded Access program, we are unable to discuss but are in place to help protect the safety and welfare of potential patients. Although the Pearl Expanded Access program is only going to be able to initially treat a small number of patients, those interested in being considered for this treatment should register any way so that we know who you are and we know how to contact you when there may be the opportunity to treat more patients. Since this MDMA-assisted therapy program is targeted for treatment-resistant PTSD, one required qualification is that the person wanting consideration for the treatment must have a documented diagnosis of PTSD from a medical or mental health professional.


The process begins with an initial phone screen where our Research Assistant will ask a set of questions designed to learn more about the potential patient. If there are no initial concerns, the next step is a therapist call-back which can last up to about an hour where more detailed information is gleaned. If the results of the therapist call-back are conducive to advancing, the next step is the Informed Consent Visit where the treatment team will carefully review the extensive Informed Consent form with the potential patient so that they are aware of the demands of the treatment protocol and they are fully informed of their rights as a patient in the program (including the right that they can choose to withdraw from the program at any time). After this, the formal screening process begins which is quite extensive and can take up to 8 weeks. If, at that point, the potential patient has met all the Inclusion Criteria and avoided all of the Exclusion Criteria, the patient is invited to officially enroll in the program. However, it is important to note that the next few weeks have continuing measures for monitoring patient qualifications and safety and there are no guarantees that the patient will continue to qualify up until the first MDMA-assisted therapy session takes place.

“To be considered for this program, one must visit the website at www.pearlpsychedelicinstitute.org and under the Contact tab there is an online form titled “EAMP1 Inquiry Form” and this is where one would go to register their interest in being considered for MDMA-assisted therapy.”


Once Enrolled

Once a patient is enrolled in the MDMA-assisted therapy program, they will meet three times with their treatment team for 90-minute non-drug sessions where the goals are to build rapport and trust with their team, to allow the treatment team to learn more about the patient’s trauma history, and to help the patient prepare for navigating the effects of the MDMA. Particular attention is paid to helping the patient understand the comfort and importance of their breath during the session and how focused breathing can not only help the patient work with the effects of the MDMA but can also help them confront and work through the anxiety and the emotions that are often elicited when working with traumatic material. The first dosing session takes place on a Saturday morning and the session is expected to last between 6-8 hours. During this time, the treatment team is with the patient constantly providing a stable presence and a safe space so that the patient can focus on their internal processes, but the team is also there to provide support should the patient need or want support. The patient is encouraged to wear eye shades while listening to pre-selected music through headphones as the instrumental music is a factor in that it can help encourage the movement of the trauma processing while also likely providing additional emotional context to the work that is being done by the patient. Following the completion of the dosing session, the patient remains at the clinic overnight with a night attendant present nearby should the patient require any kind of assistance. The next morning, the treatment team returns and the patient has their first 90-minute integration session where the experiences of the previous day are discussed before the patient is allowed to leave the clinic with their ride home.


Following this and the next two dosing sessions in the protocol, there are frequent phone check-ins from the treatment team and the patient knows they can access their team 24/7 should they have questions or need support. One of the Pearl’s site physicians is an important part of this team providing medical supervision throughout the protocol. The rest of the protocol follows this same rhythm with there being three 90-minute non-drug integration sessions following each of the remaining two dosing sessions with the first occurring the morning after. These non-drug integration sessions are a crucial part of MDMA-assisted therapy in that these are vital periods where the patient works with their treatment team to understand the experiences from their sessions and, just as importantly, how to take what has been learned and changed and bring it back to the patient’s everyday life.


“The first dosing session takes place on a Saturday morning and the session is expected to last between 6-8 hours. During this time, the treatment team is with the patient constantly providing a stable presence and a safe space so that the patient can focus on their internal processes, but the team is also there to provide support should the patient need or want support.”

Conclusion

MDMA-assisted therapy for treatment-resistant PTSD has been a long time coming and there is still much work to be done before this treatment can be considered for approval by the FDA. Unlike the previous Phase 2 and Phase 3 studies, Expanded Access patients will be responsible for paying for their MDMA-assisted therapy and this cost is still being determined but MAPS has generously made some funding available from their Health Equity Fund to assist with treatment costs. Looking to the future, the Pearl will pursue grants and donations to continue to assist with lowering the cost of treatment should MDMA be approved. Although the number of patients that can currently be treated in Expanded Access with MDMA-assisted therapy is small, we want to encourage anyone suffering from PTSD that is interested in being considered for the program to visit our website and submit your contact information. One of the main purposes of this MDMA-assisted therapy program is to allow The Pearl Psychedelic Institute to be able to introduce this cutting-edge treatment to Asheville and Western North Carolina so that we can help psychedelic-assisted therapists get training, continue researching safe and effective methods for providing these treatments, provide our community with accurate, useful information and education and to eventually make psychedelic-assisted therapies accessible to the many people who could possibly benefit from their unique healing properties.


“Although the number of patients that can currently be treated in Expanded Access with MDMA-assisted therapy is small, we want to encourage anyone suffering from PTSD that is interested in being considered for the program to visit our website and submit your contact information.”

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