The U.S. Food and Drug Administration (FDA) has released a guidance document titled “Clinical Considerations for Studies of Devices Intended to Treat Opioid Use Disorder,” aimed at assisting industry and FDA staff in designing pivotal clinical studies for medical devices intended to treat Opioid Use Disorder (OUD). U.S. Food and Drug Administration+1U.S. Food and Drug Administration+1
Background
The opioid crisis remains a significant public health emergency in the United States, with OUD contributing to substantial morbidity and mortality. Developing innovative medical devices to treat OUD is a priority, and the FDA’s guidance provides a framework for generating scientific evidence to support the safety and effectiveness of such devices. U.S. Food and Drug Administration
Scope of the Guidance
This guidance focuses on pivotal clinical studies—those intended to provide valid scientific evidence for evaluating the safety and effectiveness of devices designed to treat OUD. It addresses challenges unique to OUD device studies, including issues related to study design, data collection, and outcome measurement. U.S. Food and Drug Administration
Key Recommendations
- Patient Population and Study Design: Sponsors are encouraged to carefully define the patient population and utilize well-controlled study designs. Randomized controlled trials are recommended to minimize bias and establish a clear relationship between the device and observed outcomes. U.S. Food and Drug Administration
- Recording Medication Use: Accurate documentation of participants’ use of prescribed and non-prescribed medications is crucial. This information helps in understanding the device’s impact and potential interactions with other treatments. U.S. Food and Drug Administration+1FDA Access Data+1
- Monitoring Drug Use: Implementing strategies to monitor both prescribed and illicit drug use is essential. This may involve regular drug screenings and self-reported measures to ensure comprehensive data collection. U.S. Food and Drug Administration
- Study Duration and Evaluation: The guidance emphasizes the importance of an appropriate study length to assess both the immediate and long-term effects of the device. Continuous evaluation throughout the study period is necessary to capture the durability of treatment effects. U.S. Food and Drug Administration
- Participant Retention and Data Integrity: High rates of missing data can compromise study validity. Sponsors should employ strategies to enhance participant retention and address missing data effectively. U.S. Food and Drug Administration
- Clinical Outcome Measures: The guidance outlines several outcome measures to assess device effectiveness, including:FDA Access Data+2U.S. Food and Drug Administration+2FDA Access Data+2
- Change in Drug Use Patterns: Evaluating reductions in drug use or periods of abstinence.U.S. Food and Drug Administration
- OUD Disease Status: Assessing changes based on DSM-5-TR diagnostic criteria.
- Reduction in Adverse Outcomes: Monitoring decreases in OUD-related adverse events, such as overdoses.U.S. Food and Drug Administration
- Patient-Reported Outcomes: Utilizing instruments that capture the patient’s perspective on their health and treatment.
- Other Relevant Measures: Considering additional outcomes pertinent to the device’s intended effects. U.S. Food and Drug Administration
Conclusion
By adhering to the FDA’s guidance, sponsors can design robust clinical studies that generate the necessary evidence to demonstrate the safety and effectiveness of medical devices intended to treat OUD. This approach aims to facilitate the development of innovative treatments to address the ongoing opioid crisis. U.S. Food and Drug Administration
