The FDA has issued new scientific recommendations aimed at encouraging more widespread innovation and development of novel medication-assisted treatment (MAT) drugs for the treatment of opioid use disorder (OUD). Draft guidance issued this week outlines new ways for drug developers to measure and demonstrate the effectiveness and benefits of new or existing MAT products. This new draft guidance is part of a larger effort to promote more widespread development, access to and adoption of MAT.
MAT for opioid dependence relies on prescription drugs, including buprenorphine, methadone and naltrexone, to stabilize brain chemistry; reduce or block the euphoric effects of opioids; relieve physiological cravings; and normalize body functions. Regular adherence to MAT helps patients gain control over their use of opioids, without causing the cycle of highs and lows associated with opioid misuse or abuse. MAT, coupled with relevant social, medical and psychological services, is a highly effective treatment for OUD. Patients receiving MAT cut their risk of death from all causes in half, according to the Substance Abuse and Mental Health Services Administration.
Clinical trials to evaluate effectiveness of MAT for potential FDA approval have generally used reduction in drug-taking behavior as an endpoint. The new draft guidance, “Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment,” identifies several additional clinical endpoints and other outcome measures that drug developers may consider.
In the guidance, the FDA encourages drug sponsors to consider a variety of ways to evaluate the effect and clinical benefit of MAT. These include the impact of a new drug on adverse outcomes like mortality (overall mortality or overdose mortality), emergency medical interventions and hepatitis C seroconversion. 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.
Today’s action builds on another draft guidance issued by the FDA in April that outlines the agency’s current thinking about drug development and trial design issues relevant to the study of depot buprenorphine products. The 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.
As part of HHS’ Five-Point Strategy to Combat the Opioid Crisis, the FDA is working to addressing the epidemic on all fronts, with a significant focus on decreasing exposure to opioids and preventing new addiction by taking new steps to encourage more appropriate prescribing; supporting the treatment of those with OUD and promoting the development of improved as well as lower cost forms of MAT; fostering the development of novel pain treatment therapies that may not be as addictive as opioids, and opioids more resistant to abuse and misuse; and taking action against those who contribute to the illegal importation and sale of opioid products. The FDA will also continue to evaluate how drugs currently on the market are used, in both medical and illicit settings.
Source: FDA.gov, August 6, 2018