FDA proposes broader outcomes for OUD treatment drug approvals


Manufacturers developing the next generation of drugs to help combat opioid use disorder could have a broader set of outcome measures to target when bringing their products before the Food and Drug Administration for approval.

Dr. Scott Gottlieb, commissioner of the Food and Drug Administration

Dr. Scott Gottlieb

Traditionally, the FDA has used a reduction in drug-taking behavior as the endpoint for approving a medication-assisted treatment to combat opioid use disorder. But a draft guidance issued Aug. 6 could change that.

The guidance, “Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment,” proposes numerous clinical endpoints, including reduction in adverse outcomes of opioid use disorder, (for example, mortality, the need for emergency medical interventions, or hepatitis C seroconversion); change in the disease status using diagnostic criteria for opioid use disorder; development of patient-reported outcome measures; or changes in drug use patterns other than the commonly used endpoint of abstinence.

The FDA also is asking for comment on other measures that might be useful in helping to understand the efficacy and effectiveness of medication-assisted treatments, such as the ability to resume work or school.

“The evidence is clear. Medication-assisted treatment works, and it is a key piece of defeating the drug crisis facing our country,” Department of Health and Human Services Secretary Alex Azar said in a statement. He added that the new guidance has “the potential to bring new medications to market that are more closely tailored to patient needs and help give Americans facing addiction a better change at recovery.”

FDA Commissioner Scott Gottlieb, MD, added in the statement: “We must consider new ways to gauge success beyond simply whether a patient in recovery has stopped using opioids, such as reducing relapse overdoses and infectious disease transmission. Treatments that can impact these aspects of addiction can be important parts of a comprehensive approach to the treatment of opioid use disorder.”

Guidance comments are due Oct. 9 and can be submitted online here.

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