The FDA announced that they have issued a draft proposal of new scientific recommendations to encourage the development of new drugs for the treatment of opioid-use disorder (OUD).
Previously, for a drug to be FDA-approved for the treatment of OUD, clinical trials had to demonstrate a reduction in drug-taking behavior, but the FDA’s new proposed guidance, Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment, has outlined different ways that clinical trials could potentially demonstrate drug effectiveness.
One new potential clinical endpoint involves reducing adverse outcomes that result from OUD, including, but not limited to; reduced mortality, fewer emergency medical interventions, and a decrease in Hepatitis C seroconversion. Drug developers can study several of these endpoints in the same trial, with one selected as the primary endpoint, and one or more selected as secondary endpoints.
Drug effectiveness can also be measured by changes in disease status using diagnostic criteria for OUD. The proportion of patients that transition from meeting criteria for moderate-to-severe OUD to being in remission by the end of the clinical trial could serve as a primary or secondary efficacy endpoint.
Patient-reported outcomes may also be used to demonstrate clinical benefit. With input from patients and family members, the most concerning symptoms/experiences associated with OUD can be identified and then later evaluated and monitored, serving as a measure for the intensity of the urge to use opioids. Patient-reported outcomes would include, but are not limited to, improvements in the ability to resume work, school, or other productive activities, and improvement in sleep or mood.
The FDA explicitly stated that retention in treatment of OUD is not a significant clinical endpoint to demonstrate drug effectiveness, but that they are interested in receiving other outcome measures that could be used to demonstrate clinical endpoints for OUD medication trials.
To effectively pursue these new clinical endpoints, drug developers would be required to communicate their plans with the FDA early on in the development cycle.
Comments and suggestions regarding this draft document should be submitted to the Federal Register by October 10th.