There are substantial uncertainties around the clinical trial results; given the inability to assess net benefit, ICER will not recommend a health-benefit price benchmark —

BOSTON, May 14, 2024 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of 3,4-Methylenedioxymethamphetamine-assisted psychotherapy (MDMA-AP; Lykos Therapeutics) for the treatment of post-traumatic stress disorder (PTSD).

“PTSD can be a severe condition affecting nearly all aspects of an individual’s life,” said ICER’s Chief Medical Officer David Rind, MD. “Current therapeutic options are insufficient for many people with PTSD. While MDMA-AP may be a promising therapy for PTSD, functional unblinding in the clinical trials and additional concerns around trial design and conduct led to ICER concluding that the publicly available evidence is insufficient to assess the balance of benefits and harms. It will be incumbent on regulators with complete access to primary data to carefully evaluate whether MDMA-AP has been proven safe and effective.”

This Evidence Report will be reviewed at a virtual public meeting of the New England CEPAC (New England CEPAC) on May 30, 2024. The New England CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.

A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from individuals living with PTSD and patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.

Key Clinical Findings

Although ICER attempted to explore the concerns raised about MDMA-AP and the MAPP trials, ICER was not able to assess the extent that bias influenced reporting of benefits or the frequency with which there may have been misreporting of harms. As such, ICER concluded that the current publicly available evidence is insufficient (“I”) to assess the overall net benefit of MDMA-AP.

Key Cost-Effectiveness Findings

Given the “I” rating, the economic analyses of MDMA-AP in this Evidence Report are only exploratory analyses that provide insights into costs and benefits if it is assumed that the results of the MAPP trials are accurate. ICER did not calculate a health-benefit price benchmark for MDMA-AP.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.