Primary Outcome Indices in Illicit Drug Dependence Treatment Research: Systematic Approach to Selection and Measurement of Drug Use End-Points in Clinical Trials

Publication Type
Journal Article
Year of Publication
Donovan, DM; Bigelow, GE; Brigham, GS; Carroll, KM; Cohen, AJ; Gardin, JG; Hamilton, JA; Huestis, MA; Hughes, JR; Lindblad, R; Marlatt, GA; Preston, KL; Selzer, JA; Somoza, EC; Wakim, PG; Wells, EA
Start Page
Date Published
Alcoholism; Biomedical Research; Clinical Trials as Topic; Consensus; Endpoint Determination; Humans; Self Report; Street Drugs; Substance Abuse Detection; Substance Withdrawal Syndrome; Substance-Related Disorders; Tobacco Use Disorder; Treatment Outcome

AIMS: Clinical trials test the safety and efficacy of behavioral and pharmacological interventions in drug-dependent individuals. However, there is no consensus about the most appropriate outcome(s) to consider in determining treatment efficacy or on the most appropriate methods for assessing selected outcome(s). We summarize the discussion and recommendations of treatment and research experts, convened by the US National Institute on Drug Abuse, to select appropriate primary outcomes for drug dependence treatment clinical trials, and in particular the feasibility of selecting a common outcome to be included in all or most trials.

METHODS: A brief history of outcomes employed in prior drug dependence treatment research, incorporating perspectives from tobacco and alcohol research, is included. The relative merits and limitations of focusing on drug-taking behavior, as measured by self-report and qualitative or quantitative biological markers, are evaluated.

RESULTS: Drug-taking behavior, measured ideally by a combination of self-report and biological indicators, is seen as the most appropriate proximal primary outcome in drug dependence treatment clinical trials.

CONCLUSIONS: We conclude that the most appropriate outcome will vary as a function of salient variables inherent in the clinical trial, such as the type of intervention, its target, treatment goals (e.g. abstinence or reduction of use) and the perspective being taken (e.g. researcher, clinical program, patient, society). It is recommended that a decision process, based on such trial variables, be developed to guide the selection of primary and secondary outcomes as well as the methods to assess them.

Comment in

The mountain roared and brought forth a mouse*: comments on the results of an expert panel on standardization of drug dependence treatment trial outcome variables. [Addiction. 2012]

How to measure outcome in clinical trials of substance abuse treatment. [Addiction. 2012]

'Nothing is more practical than a good theory': outcome measures in addictions treatment research. [Addiction. 2012]