Affiliation:
1. Department of Psychology The City College of New York New York New York USA
2. Department of Biostatistics Florida International University Miami Florida USA
3. Department of Psychiatry Columbia University Irving Medical Center New York New York USA
4. The Information School University of Wisconsin‐Madison Madison Wisconsin USA
5. Division of Biostatistics, Department of Public Health Sciences The University of Miami Miami Florida USA
Abstract
AbstractBackground and aimsA lack of consensus on the optimal outcome measures to assess opioid use disorder (OUD) treatment efficacy and their precise definition and computation has hampered the pooling of research data for evidence synthesis and meta‐analyses. This study aimed to empirically contrast multiple clinical trial definitions of treatment success by applying them to the same dataset.MethodsData analysis used a suite of functions, developed as a software package for the R language, to operationalize 61 treatment outcome definitions based on urine drug screening (UDS) results. Outcome definitions were derived from clinical trials that are among the most influential in the OUD treatment field. Outcome functions were applied to a harmonized dataset from three large‐scale National Drug Abuse Treatment Clinical Trials Network (CTN) studies, which tested various medication for OUD (MOUD) options (n = 2492). Hierarchical clustering was employed to empirically contrast outcome definitions.ResultsThe optimal number of clusters identified was three. Cluster 1, comprising eight definitions focused on detecting opioid‐positive UDS, did not include missing UDS in outcome calculations, potentially resulting in inflated rates of treatment success. Cluster 2, with the highest variability, included 10 definitions characterized by strict criteria for treatment success, relying heavily on UDS results from either a brief period or a single study visit. The 43 definitions in Cluster 3 represented a diverse range of outcomes, conceptualized as measuring abstinence, use reduction and relapse. These definitions potentially offer more balanced measures of treatment success or failure, as they avoid the extreme methodologies characteristic of Clusters 1 and 2.ConclusionsClinical trials using urine drug screening (UDS) for objective substance use assessment in outcome definitions should consider (1) incorporating missing UDS data in outcome computation and (2) avoiding over‐reliance on UDS data confined to a short time frame or the occurrence of a single positive urine test following a period of abstinence.
Funder
National Institute on Drug Abuse
National Institute on Minority Health and Health Disparities
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