FDA offers guidance for new drug development for opioid use disorder

The Food and Drug Administration (FDA) believes it can facilitate the development of new medication options for treating opioid use disorder by encouraging clinical trials that use measures of success beyond reduction in drug use alone.

New scientific recommendations issued in draft form today by the federal agency state that drug sponsors can evaluate the benefit of medication-assisted treatment options by examining their effects on factors such as mortality, emergency medical care and transmission of hepatitis C. Past medication trials conducted for FDA approval of a drug for treating opioid use disorder have tended to focus only on the outcome of reduced drug-taking behavior.

FDA Commissioner Scott Gottlieb, MD, said in a news release that “we mut 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.”

The draft guidance from the FDA adds that quality-of-life outcomes in areas such as work and education also can be used to demonstrate the clinical benefit of a drug treatment.

The news release adds, “While understanding that many of these outcomes could be highly valuable, the agency recognizes that evaluating them could require larger trials than those usually conducted for marketing approval. To that end, the FDA is encouraging sponsors to discuss their plans with the agency early in the drug development process.”

Methadone, buprenorphine and naltrexone are the three available MAT options for opioid use disorder, and the FDA is seeking to encourage development of and access to additional and possibly lower-cost medication options.