The US Food and Drug Administration (FDA) on Monday released new draft guidance outlining the clinical endpoints acceptable to demonstrate effectiveness for medication-assisted treatment of opioid use disorder (OUD).
The 8-page guidance, following draft guidance from April on the development and clinical trial design
issues relevant to the study of sustained-release “depot” buprenorphine products, discusses clinical endpoints, including adverse outcomes of OUD, change in disease status using diagnostic criteria for OUD, patient-reported outcomes, change in drug use pattern and other outcome measures.
“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,” said FDA Commissioner Scott Gottlieb.
FDA encourages sponsors to consider a variety of ways to evaluate the effect and clinical benefit of MAT, including the impact on mortality (overall mortality or overdose mortality), emergency medical interventions and hepatitis C seroconversion (the period during which antibodies develop and become detectable).
“Efficacy may also be measured by studying the proportion of patients that transition from meeting criteria for being diagnosed with moderate to severe OUD – based on both drug use and its impact on patient wellbeing – at baseline to being considered in remission at the end of the study. Improvements in the ability to resume work, school, or other productive activity may also demonstrate clinical benefit,” the agency added.
In addition to the draft, FDA also recently published a paper
with the National Institute on Drug Abuse that describes efforts to overcome some of the barriers to new drug development and the issues with determining effectiveness.
In an effort to adequately incorporate patient experience, the agency also held a meeting
in April to hear directly from those with OUD on a wide range of topics. Back in November, FDA also finalized guidance detailing its expectations
for drugmakers looking to develop generic versions of abuse-deterrent opioids
Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment: Draft Guidance for Industry