The U.S. Food and Drug Administration today 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).

The new draft guidance outlines new ways for drug developers to consider measuring and demonstrating the effectiveness and benefits of new or existing MAT products. This new draft guidance is part of the FDA's ongoing commitment to promote more widespread development, access to and adoption of MAT.

"The Trump Administration is pursuing every opportunity to address our country's opioid epidemic and support patients struggling with opioid use disorder. This work at HHS includes placing a special priority on ensuring access to a full range of safe and effective options for medication-assisted treatment," said HHS Secretary Alex Azar.

"The evidence is clear: medication-assisted treatment works, and it is a key piece of defeating the drug crisis facing our country. The FDA's new guidances have the potential to bring new medications to market that are more closely tailored to patient needs and help give Americans facing addiction a better chance at recovery."

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.

Social, medical and psychological services

MAT, coupled with relevant social, medical and psychological services, is a highly effective treatment for OUD. Additionally, patients receiving MAT cut their risk of death from all causes in half, according to the Substance Abuse and Mental Health Services Administration.

Treatments that can impact these aspects of addiction can be important parts of a comprehensive approach to the treatment of opioid use disorder. 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.

Clinical trials

Clinical trials to evaluate the effectiveness of MAT for the purposes of FDA approval have generally used reduction in drug-taking behavior (drug use patterns) as an endpoint. The new draft guidance, "Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Medication-Assisted Treatment," identifies several additional potential clinical endpoints and other outcome measures that drug developers may consider.

While understanding that many of these outcomes could be highly valuable, the agency recognizes that evaluating them could require larger trials than those usually conducted for marketing approval. To that end, the FDA is encouraging 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. Patient-reported experiences could be used to complement other endpoints and help determine how a new treatment's effects on such experiences support the goal of sustained clinical response.

In an effort to adequately incorporate patient experience into the drug development and review paradigm, the agency also held a meeting in April to hear from directly from those with OUD on a wide range of topics, including the effects on their health and well-being that have the greatest impact on daily life, their experience using prescription medical treatments and other treatments or therapies for OUD, and challenges or barriers to accessing or using medical treatments for OUD.