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FDA Issues Draft Guidance on Measuring Efficacy of Medication-Assisted OUD Treatment

August 7, 2018
Agency encourages developers to consider other ways to evaluate patient outcomes when treating for opioid addiction.

A PPM Brief

In late May, FDA announced a challenge1 to the healthcare and device community to develop innovative technologies that would help could provide solutions for detecting, treating, and preventing opioid addiction, or Opioid Use Disorder (OUD). Now the agency has issued a draft guidance2 aimed at encouraging more widespread innovation and development of novel medication-assisted treatment (MAT) drugs for the treatment of OUD. The scientific recommendations outline “new ways for drug developers to consider measuring and demonstrating the effectiveness and benefits of new or existing MAT products. This new draft guidance is part of the FDA’s ongoing commitment to promote more widespread development, access to and adoption of MAT,” according to the FDA release.3

According to the Substance Abuse and Mental Health Services Administration (SAMHSA), as stated in the release, MAT for opioid dependence involves prescription use of buprenorphine, methadone, or naltrexone “to stabilize brain chemistry; reduce or block the euphoric effects of opioids; relieve physiological cravings; and normalize body functions.” Regular adherence to the treatment may help patients “gain control over their use of opioids, without causing the cycle of highs and lows associated with opioid misuse or abuse.” In addition, SAMHSA points out that MAT should be inclusive of related social, medical and psychological services.

The new draft guidance identifies several potential clinical endpoints and outcome measures for MAT effectiveness that drug developers may consider—beyond the traditional endpoint of “reduction in drug-taking behavior (ie, drug use patterns).”

For example, the guidance states that efficacy may be measured by:

  • the impact of a new drug on adverse outcomes such as mortality (overall mortality or overdose mortality)
  • emergency medical interventions
  • Hepatitis C seroconversion
  • the proportion of patients that transition from meeting criteria for being diagnosed with moderate to severe OUD 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.

In its announcement, FDA acknowledges that these additional measures may require larger clinical trials and therefore encourages sponsors to discuss their plans with the agency early in the drug development process. “The agency is also committed to providing assistance to sponsors interested in developing a validated measurement of patient-reported experiences, such as ‘craving’ or ‘urge to use’ opioids, which make it difficult for patients with OUD to sustain recovery.”3

The new guidance is related to the agency’s March 2018 draft guidance, Opioid Dependence: Developing Depot Buprenorphine Products for Treatment.4

Last updated on: August 8, 2018
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FDA Calls for Innovative Technologies to Treat Opioid Use Disorder
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