October 1, 2020
The following quote is attributed to FDA Commissioner Stephen M. Hahn, M.D.:
“As we combat the COVID-19 crisis, we are also focused on addressing the other health crises facing our country, including the ongoing substance use disorder and overdose crisis that our country is battling. Drug overdose deaths continue to rise in our country, and opioids, especially synthetic opioids, continue to be a driver of that trend.
“One way we are doing so is through exploring new ways to measure effectiveness of treatments, as we seek to help individuals with opioid use disorder (OUD). We recognize that there’s great interest in developing new treatment options that result in meaningful outcomes. We know from research that treatment for OUD with both prescription drugs – including buprenorphine, methadone and naltrexone – and relevant social, medical and psychological services is a highly effective treatment.
“The FDA is committed to assisting in the development of high-quality, effective medications and encouraging health care professionals to ensure patients with OUD are offered an adequate chance to benefit from these therapies."
- Today, the U.S. Food and Drug Administration issued a final guidance, “Opioid Use Disorder: Endpoints for Demonstrating Effectiveness of Drugs for Treatment” which is intended to help companies develop drugs to treat OUD and addresses the clinical endpoints acceptable for demonstrating effectiveness of drugs to treat OUD. The guidance includes minor changes to the draft document. It also builds on another final guidance issued by the FDA in February 2019 that outlines the agency’s current thinking about drug development and trial design issues relevant to the study of depot buprenorphine products. Sponsors are encouraged to discuss their plans with the FDA early in the drug development process.
- Clinical trials to evaluate the effectiveness of medications for OUD for the purposes of FDA approval have generally used changes in drug-taking behavior (drug use patterns) as an endpoint. This final guidance identifies several additional potential clinical endpoints and other outcome measures that drug developers may consider.
- The final guidance encourages drug sponsors to consider a variety of ways to evaluate the effect and clinical benefit of medications to treat OUD. For example, using input from patients and family members to determine the most concerning symptoms and experiences associated with OUD, drug sponsors could develop a patient-reported outcome instrument to evaluate a direct effect on how patients feel or function, such as an improvement in sleep or mood. If sponsors plan to use a patient-reported outcome instrument as a secondary endpoint, drug sponsors should first determine the magnitude of the change that represents a clinical benefit and how long such change should be maintained in a clinical trial to predict a sustained clinical benefit.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.