OBJECTIVES: Based on regulatory interest in utilizing patient-centric outcomes in clinical trials for treatments of opioid use disorder (OUD), a review of the published literature was conducted to understand current use of patient-reported outcomes (PROs) in clinical trials and non-interventional studies. Additionally, patient barriers to providing transparent PRO data were investigated.

METHODS: A targeted literature search was conducted to explore (1) current endpoints being utilized to assess buprenorphine and methadone treatment efficacy in both clinical trials and non-interventional clinical research studies, and (2) barriers to reporting transparent data in the target patient population (TPP). Potential articles were identified by title, and abstracts were reviewed before selecting full-text articles for inclusion. Additionally, relevant Food and Drug Administration regulatory documents were reviewed.

RESULTS: Eighty-five abstracts and two regulatory documents were reviewed. Seven full-text articles and two regulatory documents were selected for inclusion. One randomized controlled trial utilized a PRO for its primary endpoint and two trials utilized a range of PROs as secondary endpoints. In one longitudinal, non-interventional study, PROs were used at various timepoints. Non-PRO outcomes used in clinical trials and clinical studies largely relied on opioid-related overdose, acute care use, treatment adherence, and opioid-negative urine specimens as efficacy endpoints. After review of OUD-specific regulatory guidance documents, both suggested including patient-centric outcomes to measure how patients feel and function. Barriers to patient reporting identified in the literature included low rates of retention in clinical trials (except in populations that risk significant negative consequence for relapse), stigmatization of receiving medication-assisted therapy, and insufficient information regarding potential consequences of disclosing substance use to healthcare providers.

CONCLUSIONS: Patient-centric endpoints, including PROs, should be included at the forefront of OUD-related clinical trials. Full transparency regarding the use of PRO data and ensuring that data is safeguarded whenever possible may mitigate inaccuracies in PRO data reported by the TPP.