Advancing Evidence for Opioid Use Disorder Treatments through Real-World Data and Novel Statistical Methods

Music City Center 

The opioid epidemic remains a major public health crisis. Although evidence-based treatments for opioid use disorder (OUD) exist, most people with OUD do not receive treatment. Pragmatic trial designs have therefore been proposed to evaluate interventions designed to increase OUD treatment within entire clinics or health systems by leveraging health records (EHR) and other real-world data sources. In this talk, we present case studies that illustrate key challenges of using real-world data for evaluating intervention effects, including post-randomization selection bias that arises due to the intervention impacting diagnosis of OUD in the EHR, and observational outcome assessment processes, in which follow-up times from EHRs are irregularly spaced and may be intervention or outcome dependent. We clarify which estimands are being estimated in these settings, present simulation studies to evaluate the performance of methods addressing these challenges, and highlight novel statistical methods that have been developed and which are being implemented in these case studies to provide robust evidence on intervention effects to improve outcomes of people with OUD.

pragmatic trials

opioid use disorder

real-world data

electronic health records