The U.S. Food and Drug Administration (FDA) recently released draft guidance regarding clinical considerations for studies of medical devices intended to treat opioid use disorder (OUD). According to the FDA’s press release, the draft guidance aligns with the FDA’s Overdoes Prevention Framework and aims to advance evidence-based treatment for individuals with substance use disorders.
The press release identifies several challenges associated with these clinical 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 consideration of these challenges:
[T]he draft guidance recommends that pivotal OUD device studies to support marketing submissions should have a well-defined study population, appropriately monitor drug use,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The draft guidance relates specifically to devices intended to treat OUD and does not include diagnostic tests, devices for diagnosing or determining the risk of developing OUD, devices for treatment of pain, or combination products. Further, the draft guidance offers a non-exhaustive list of primary and secondary outcomes that are intended to provide valid scientific evidence of clinically significant benefits for OUD treatment devices. These outcomes include changes in drug use patterns, changes in OUD status using DSM-5-TR diagnostic criteria, reduction in adverse outcomes of OUD, and patient-reported outcome instruments. Additionally, the draft guidance encourages sponsors to submit other outcome measurements that demonstrate clinical benefits of OUD treatment devices.