Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment (Draft Guidance for Industry)

Policy Details

Policy Details

Last Action
Public Comment Period Closed
Date of Last Action
Oct 9 2018
Date Introduced
Aug 7 2018
Publication Date
Oct 29 2018

SciPol Summary

The Food and Drug Administration (FDA) released a draft guidance for industry, “Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment,” to determine acceptable clinical markers for proving the effectiveness of treatments combating opioid use disorder. Furthermore, this guidance will address whether placebo or active controls are most appropriate in clinical trials for such treatments.

During the last decade, the number of opioid related deaths and overdoses in the US has increased dramatically. This rise has precipitated increased engagement by policy makers hoping to address the issue. As part of this response, the FDA is taking steps to identify treatments that can help prevent overdoses from prescription and illicit opioids and manage treatment outcomes once patients have stopped using prescription and illicit opioids. Currently, clinical trials for opioid use disorder look for three endpoints when analyzing success: changes in disease status using diagnostic criteria for opioid use disorder, patient-reported outcomes, and changes in drug-use patterns. The proposed guidance aims to identify other potential primary and secondary endpoints that are important to users, their families, clinicians, and the public.

The guidance makes clear that FDA would like to see new clinical endpoints proposed: “There is great societal interest in assessing additional, clinically meaningful endpoints such as reduction in hospitalizations, emergency department visits, overdose, and death as well as improvements in the ability to resume work, school, or other productive activity.” If such endpoints can be effectively demonstrated, the guidance suggests such FDA-approved labeling of drugs for opioid use disorder would be forthcoming.

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