"The FDA continues to seek new ways to advance science to address the public health challenges that families and communities across the country face," said FDA Commissioner Robert M. Califf, M.D. "We are committed to helping industry develop solutions that are proven to be effective to address the devastation caused by the overdose crisis. This draft guidance for device studies should prompt industry to collect more comprehensive, timely and diverse data to support device submissions that may help improve the lives of those with opioid use disorder. These and similar efforts will continue to advance within the agency, with the input from industry, stakeholders and, most importantly, those managing opioid use disorder."

The draft guidance, Clinical Considerations for Studies of Devices Intended to Treat Opioid Use Disorder, outlines key considerations intended to aid sponsors in designing clinical studies for devices intended to treat OUD that address some of the complex challenges associated with these studies, including inaccurate participant reports of drug use, high rates of missing data, the confounding effects of concomitant drug treatments and the need to demonstrate the durability of the treatment effect of the device, which can necessitate prolonged observation. In particular, the draft guidance recommends that pivotal OUD device studies to support marketing submissions should have a well-defined study population, appropriately monitor drug use, control for bias and include an appropriate follow-up period, study participant retention plans and data analysis plans. By addressing these challenges early in the development of their clinical studies, sponsors should be well-positioned to obtain the most useful data to provide a reasonable assurance of safety and effectiveness for a device intended to treat OUD.  

The draft guidance provides industry critical recommendations toward efforts to bring safe and effective devices intended to treat OUD to market and provide more tools to reduce the harms that opioid use disorder causes patients, families and communities. It also provides recommendations that may be applicable to other types of studies intended to generate valid scientific evidence that may be used in providing a reasonable assurance of safety and effectiveness. Stakeholders can share feedback on the draft guidance, which the FDA will review before it is finalized.

Certain products are outside the scope of the draft guidance, including diagnostic tests for the detection of opioid use, devices intended to diagnose or to help determine the risk of developing OUD, devices intended to treat pain and combination products.

"Promoting device innovation to assist in the treatment of individuals with opioid use disorder is vital to our ongoing public health response," said Jeff Shuren, M.D., J.D., director of the FDA's Center for Devices and Radiological Health. "This guidance supports innovative approaches for evaluating medical devices that can help combat this national crisis and give patients new tools to treat opioid use disorder, with the assurance that the solutions we need will be driven by robust data."

In August 2022, the FDA introduced the Overdose Prevention Framework, a comprehensive, agency-wide approach to address the overdose crisis and sustain long-term recovery outcomes. The framework details the agency's priorities, including supporting primary prevention by eliminating unnecessary initial prescription drug exposure and inappropriate prolonged prescribing, encouraging harm reduction through innovation and education, advancing the development of evidence-based treatments for substance use disorders and protecting the public from unapproved, diverted or counterfeit drugs presenting overdose risks.

The agency will continue to work across its centers, with sponsors, the public and stakeholders to respond to all facets of substance use, misuse, substance use disorders, overdose and death in the U.S., relying on evidence-based treatments, to develop new options for OUD and other substance use disorders.