MDMA and the FDA approval process

There is great excitement over the status of MDMA, also known as ecstasy. And for a good reason. As noted in the New York Times last month:

… those who received MDMA during therapy [in clinical trials] experienced a significantly greater reduction in the severity of their symptoms compared to those who received therapy and an inactive placebo. Two months after treatment, 67 percent of participants in the MDMA group no longer qualified for a PTSD diagnosis, compared with 32 percent in the placebo group.

This is very significant clinical evidence of the effectiveness of MDMA.

Photo by portkalis / Getty Images

Once relegated to the party scene, sponsors, scientists and doctors are now testing MDMA for the treatment of certain diseases. As many know, MDMA is currently in clinical trials as part of an Investigational New Drug (IND) filing with the U.S. Food & Drug Administration (FDA). The sponsor of this clinical trial is testing the effectiveness of MDMA in the treatment of post-traumatic stress disorder. MDMA is in Phase 3 clinical trials and approval as a new drug is no longer hypothetical. Once it has been proven that MDMA is effective and safe for people with PTSD, the FDA should approve MDMA.

RELATED: MDMA On The Cusp of FDA Approval

The pre-market FDA approval process is rigorous, demanding, and expensive. Very few IND applications ultimately receive FDA approval. To understand the requirements for a sponsor of an IND, an overview of the preclinical and clinical processes is instructive.

Before clinical trials begin, an IND sponsor often conducts preclinical animal studies to assess the drug’s toxicity. Such tests can be performed either in vivo or in vitro. The studies are usually small and test the dosage regimen and toxicity levels. If these preclinical tests show promise, the sponsors will switch to human clinical trials.

According to the FDA (click here for more information from the FDA), the clinical trial process typically includes the following four phases:

Phase 1 – This phase usually lasts a few months and deals with safety and dosage issues. The typical study size is 20 to 100 participants who meet certain criteria set by the sponsor. Approximately 70% of drugs move from phase 1 to phase 2.

As a phase 1 study continues, sponsors and researchers will answer research questions about how the drug works in the body, the side effects associated with an increased dose, and early information about how effective the drug is in order to determine how the drug works Best administered can take risks and maximize potential benefits. This is important for the design of phase 2 studies.

Phase 2 – Phase 2 studies can last anywhere from a few months to a few years. During this phase, the sponsor studies the drug’s effectiveness and side effects. The study sample is larger in phase 2 studies, usually several hundred participants are involved. Approximately 33% of drugs go from phase 2 to phase 3. Thus, this phase of clinical trials is more demanding than phase 1.

Phase 3 – Phase 3 studies are the larger clinical studies that involve 300 to 3,000 study participants. This phase is the final phase in the FDA’s pre-approval process. Phase 3 studies continue to test effectiveness and monitor side effects. Approximately 25 to 33% of drugs are approved in phase 3. Thus, at this point in time, only 6 to 8% of the drugs from phase 1 make it to FDA approval.

Phase 3 studies provide most of the safety data. In the earlier stages of clinical testing, it is possible that less common side effects may go undetected. As phase 3 studies are larger and longer, the results show more long-term or rare side effects.

Phase 4 occurs after a drug has received FDA approval. During this phase, the safety and effectiveness of thousands of patients are monitored.

The foregoing is a simplified summary of the FDA’s clinical trial process – the actual process is extremely detailed and demanding. But not every new drug follows the overview above. For example, some new drugs even have significantly smaller clinical trials in phase 3. As long as the study results are statistically significant (that is, the results can be extrapolated to the entirety of a population), the sample size is less important.

RELATED: Legalizing MDMA to Treat PTSD: How Close Are We?

In order for MDMA to reach Phase 3, significant clinical evidence regarding safety, dosage and effectiveness has been collected. It is no small matter. Every clinical study requires significant expenditure. According to the Johns Hopkins Bloomberg School of Public Health, “[c]linear trials that support FDA approvals of new drugs cost an average of $ 19 million … “However, adequate budget is only one of many variables required to successfully complete clinical trials. Ultimately, efficacy and safety are the two most difficult variables to control.

Will MDMA Get FDA Approval? Time will tell, but the first evidence is very promising. In the meantime, check out the following MDMA blog posts:

Ethan Minkin is an attorney with Harris Bricken. This article was originally published on the Canna Law Blog and is republished with permission.