Sponsors of opioid use disorder (OUD) treatments should expand their trials beyond collecting evidence for an NDA submission and gather data on additional clinically meaningful outcomes the FDA considers “highly valuable,” according to a final guidance the agency released last week.

A change in drug-use pattern, particularly abstinence, is typically used as a primary endpoint for OUD treatment trials, according to the guidance, but the FDA is interested in expanding primary and secondary endpoints to include outcome measures “important to patients and their families, clinicians and the public.”

Additional endpoints could include reduction in such adverse outcomes as mortality, need for emergency medical intervention, and hepatitis C infection or reinfection. The guidance also recommends endpoints focusing on change in disease status as measured by standard diagnostic criteria, patient-reported effects on feeling or function (e.g., reduction in intensity of the urge to use opioids) or reduction of opioid use below a clinically significant level.

Sponsors can propose other endpoints, the guidance says, and can study several endpoints or composite endpoints in one trial.

The guidance is virtually unchanged from the August 2018 draft save for a new section on benefit-risk considerations that suggests trials of products associated with a risk of serious adverse events present “compelling demonstrations of clinical benefit” that outweigh the risk. If the investigational drug itself has abuse potential, the guidance says the FDA will consider its positive and negative public health effects, including its effect on nonpatients, such as members of the patient’s household.

Drugs intended to provide relief of withdrawal symptoms are not covered by the guidance, the FDA says, because they usually are not sufficient to reduce the risk of returning to opioid abuse.

To read the guidance, click here: https://bit.ly/3lcHLWf.