Clinical Study Design Recommendations for Devices to Treat Opioid Use Disorder
This guidance applies to pivotal clinical studies evaluating safety and effectiveness of devices intended to treat Opioid Use Disorder (OUD) for marketing submissions. It excludes diagnostic tests for opioid use detection, devices for OUD diagnosis/risk assessment, pain treatment devices, and combination products.
Recommended Actions
- Develop detailed protocol with well-defined study population and inclusion/exclusion criteria
- Implement robust drug monitoring system combining:
- Self-reporting
- Objective measures (urine, blood, or saliva tests)
- Validated toxicology assays
- Establish comprehensive data collection strategy:
- Baseline medication documentation
- Regular monitoring of medication changes
- Tracking of prescribed and non-prescribed drug use
- Design participant retention strategy to minimize missing data:
- Risk-based ongoing data management
- Procedures for retrieving missing primary outcome information
- Early detection system for missing critical data
- Select appropriate outcome measures:
- Define clear primary and secondary outcomes
- Include objective verification measures
- Incorporate patient-reported outcomes when appropriate
- Consider using Q-Submission process to discuss study design with FDA before initiation
- Develop statistical analysis plan addressing missing data before study starts
- Plan for minimum 6-month study duration with appropriate follow-up
- Include diverse population representative of US OUD population
- Implement effective blinding procedures for treatment assignment
Key Considerations
Clinical testing
- Minimum treatment duration of 6 months recommended
- Well-controlled studies with sham control recommended
- Need to demonstrate durability of treatment effect
- Need to record baseline medications and changes during study
- Need to monitor both prescribed and non-prescribed drug use through objective measures
- Need strategies for participant retention and minimizing missing data
Safety
- Need to monitor and record adverse events
- Access to clinic, hospital, and pharmacy records recommended
- Need to assess and record adverse life changes
Other considerations
- Study population should represent diversity prevalent among individuals with OUD
- Primary outcomes should demonstrate clinically significant benefits
- Recommended outcome measures include:
- Requests for Feedback and Meetings for Medical Device Submissions: The Q-Submission Program
- Collection and Reporting of Race and Ethnicity Data in Clinical Trials
- Patient-Reported Outcome Measures: Development, Implementation and Evaluation in Clinical Trials
- Design Controls for Medical Device Manufacturers
- Statistical Guidance for Reporting Diagnostic Test Results with Qualitative Outcomes
Related references and norms
- DSM-5-TR: Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition
Original guidance
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