BOX 6-3Recommendations for the Clinical Development Stage

Recommendation 6-2. Require additional studies and the collection and analysis of data needed for a thorough assessment of broad public health considerations. To utilize a systems approach that adequately assesses the public health benefits and risks described in Recommendation 6-1, the U.S. Food and Drug Administration (FDA) should continue to require safety and efficacy evidence from well-designed clinical trials while also seeking data from less traditional data sources, including nonhealth data, that pertain to real-world impacts of the availability and use of the approved drug on all relevant outcomes. The FDA should develop guidelines for the collection of these less traditional data sources and their integration in a systems approach.

Recommendation 6-3. Ensure that public health considerations are adequately incorporated into clinical development. The U.S. Food and Drug Administration (FDA) should create an internal system to scrutinize all Investigational New Drug (IND) applications for opioids. This review should examine whether public health considerations are adequately incorporated into clinical development (e.g., satisfactory trial design; see Recommendation 6-2). In implementing this recommendation, the FDA should rarely, if ever, use expedited development or review pathways or designations for opioid drugs and should review each application in its entirety.