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.
What You Need to Know? 👇
What are the key clinical study design requirements for devices treating opioid use disorder?
Pivotal OUD device studies should be well-controlled with minimum 6-month treatment duration, include sham controls, monitor both prescribed and non-prescribed drug use objectively, and have detailed participant retention plans to minimize missing data.
How should drug use be monitored in clinical trials for OUD treatment devices?
Studies must combine participant self-reports with objective verification measures like random/scheduled urine tests using validated toxicology assays. Positive toxicology results confirmed by quantitative reference methods take precedence over self-reported abstinence.
What clinical outcomes are recommended for demonstrating device effectiveness in OUD treatment?
Primary outcomes include change in drug use patterns, change in OUD disease status using DSM-5-TR criteria, and reduction in adverse OUD outcomes. Secondary outcomes may include patient-reported outcomes and improvements in daily functioning.
What patient population considerations are important for OUD device studies?
Studies should define specific inclusion/exclusion criteria that translate to clinical practice, include diverse populations representative of US OUD demographics, and ensure prescribers can confidently apply study results to their patients.
How should missing data be handled in OUD device clinical trials?
Protocols must anticipate high missing data rates with prespecified conservative analysis methods. Proactive data management should detect missing data early when still retrievable, and retention strategies should be detailed and well-resourced.
What regulatory pathway should sponsors use for discussing alternative study approaches?
FDA recommends using the Q-Submission process to discuss alternative methodologies before study initiation, especially when designing effective sham controls or overcoming unique OUD study challenges while maintaining valid scientific evidence.
What You Need to Do 👇
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:
- Change in drug use pattern
- Change in OUD disease status using DSM-5-TR criteria
- Reduction in adverse outcomes of OUD
- Patient-reported outcomes
- Treatment retention and participant satisfaction
Relevant Guidances 🔗
- Q-Submission Program: Strategic Framework for FDA Interactions in Medical Device Development
- 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