Methodology

A comparison of missing data methods for hypothesis tests of the treatment effect in substance abuse clinical trials: a Monte-Carlo simulation study

Sarra L Hedden^1,2,3 , Robert F Woolson¹ and Robert J Malcolm²

¹  Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, 135 Cannon Place, Charleston, SC 29425, USA

²  Department of Psychiatry and Behavioral Research, Medical University of South Carolina, 67 President Street, Charleston, SC 29425, USA

³  Department of Mental Health, Johns Hopkins Bloomberg School of Public Health, 2213 McElderry St., Suite 400, Baltimore, MD 21205, USA

author email corresponding author email

Substance Abuse Treatment, Prevention, and Policy 2008, 3:13doi:10.1186/1747-597X-3-13

Published:	3 June 2008

Abstract

Background

Missing data due to attrition are rampant in substance abuse clinical trials. However, missing data are often ignored in the presentation of substance abuse clinical trials. This paper demonstrates missing data methods which may be used for hypothesis testing.

Methods

Methods involving stratifying and weighting individuals based on missing data pattern are shown to produce tests that are robust to missing data mechanisms in terms of Type I error and power. In this article, we describe several methods of combining data that may be used for testing hypotheses of the treatment effect. Furthermore, illustrations of each test's Type I error and power under different missing data percentages and mechanisms are quantified using a Monte-Carlo simulation study.

Results

Type I error rates were similar for each method, while powers depended on missing data assumptions. Specifically, power was greatest for the weighted, compared to un-weighted methods, especially for greater missing data percentages.

Conclusion

Results of this study as well as extant literature demonstrate the need for standards of design and analysis specific to substance abuse clinical trials. Given the known substantial attrition rates and concern for the missing data mechanism in substance abuse clinical trials, investigators need to incorporate missing data methods a priori. That is, missing data methods should be specified at the outset of the study and not after the data have been collected.