Opioid Epidemic: FDA Releases Draft Guidance on Endpoints for Medication-Assisted Treatment


On Monday, August 6th, FDA released a new draft guidance on clinical endpoints for demonstrating effectiveness of drugs for medication-assisted treatment for opioid use disorder (OUD), entitled “Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment.”

Clinical Endpoints

Currently, most clinical trials that evaluate the effectiveness of OUD treatments are using “reduction in drug-taking behavior (drug use patterns)” as a primary endpoint. However, there has recently been interest in adding additional endpoints to evaluate the effectiveness of such treatments.

According to a recent statement from Scott Gottlieb, M.D., FDA Commissioner, the Agency feels that it “must consider new ways to gauge success beyond simply whether a patient in recovery has stopped using opioids […] Treatments that can impact these aspects of addiction can be important parts of a comprehensive approach to the treatment of opioid use disorder. This new guidance is an important step in fostering the development of new treatment options that help patients achieve these and other real-world outcomes, by providing a pathway for how innovators can use these clinically relevant measures as part of new drug development programs”

This guidance discusses four main clinical endpoints for measuring the effectiveness of a treatment for OUD.  These include:

  1. Adverse outcomes of OUD
  2. Change in disease status using diagnostic criteria for OUD
  3. Patient-reported outcomes
  4. Change in drug use pattern

Although these are the four main endpoints that are mentioned in the document, the Agency is also interested in other outcome measures that demonstrate the benefits of an OUD treatment. As such, the draft guidance states that “there is great societal interest in assessing additional, clinically meaningful endpoints such as reduction in hospitalizations, emergency department visits, overdose, and death as well as improvements in the ability to resume work, school, or other productive activity […] If a sponsor plans to include novel endpoints in a drug development program for the treatment of OUD, FDA strongly encourages the sponsor to discuss such plans with the division early in the drug development process.”

Are you in the process of developing a drug for the treatment of opioid use disorder? We can help you achieve successful interactions with the Agency and get your product approved by the FDA. Contact us today to learn more about our services and how we can be of assistance to you.