Chapter Eight - Designing and testing treatments for alcohol use disorder

Developing new treatments for AUD or personalizing existing treatments for AUD requires extensive consideration of research design, measurement, data analysis, and implementation. Without careful research design, valid measurement, appropriate analyses, and considerations of implementation, any attempts to draw conclusions from the study findings may be at higher risk of bias, and the results may not generalize to the effectiveness of the treatment in real world settings. In the study of treatments for AUD, biased conclusions from research can have implications that are widespread, including a perception among providers and patients that AUD treatments are not effective or that providers do not have the resources to provide the treatments (Williams et al., 2018, Young et al., 2018). To address the research to practice gap and inform the field, this chapter provides a broad overview of best practices in research design, measurement, analysis, and implementation to inform future AUD treatment research. As an update to a recent paper, Witkiewitz, Finney, Harris, Kivlahan, and Kranzler (2015), we make recommendations specific to AUD treatment studies in the following 10 areas: (1) regulatory guidance, (2) trial registration, (3) eligibility criteria, (4) recruitment, (5) randomization, (6) retention, (7) measurement selection, (8) measuring behavior change across time, (9) statistical methods, and (10) implementation science. Throughout this chapter, we may use the terms intervention and treatment interchangeably, whereby intervention is an umbrella term for any activity that has the “objective of improving human health” (Smith, Morrow, & Ross, 2015) and treatment is one specific therapeutic intervention. We have also geared this chapter toward AUD treatment trials, more generally, but with a specific focus on recommendations for phase 3 randomized clinical trials, which are fully powered clinical trials that are testing the efficacy of a medication or behavioral intervention in comparison to placebo medication or current standard of care (i.e., treatment as usual). Much of the guidance in this chapter is also relevant for phase 2 trials, which are often smaller studies that are designed to pilot test the treatment and evaluate possible benefits. The current chapter is less relevant for phase 1 studies that are designed to evaluate the safety of a medication or behavioral intervention and, oftentimes, the pharmacokinetics of a medication.

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