Calculating sample size for studies with expected all-or-none nonadherence and selection bias.

Abstract:

SUMMARY:We develop sample size formulas for studies aiming to test mean differences between a treatment and control group when all-or-none nonadherence (noncompliance) and selection bias are expected. Recent work by Fay, Halloran, and Follmann (2007, Biometrics 63, 465-474) addressed the increased variances within groups defined by treatment assignment when nonadherence occurs, compared to the scenario of full adherence, under the assumption of no selection bias. In this article, we extend the authors' approach to allow selection bias in the form of systematic differences in means and variances among latent adherence subgroups. We illustrate the approach by performing sample size calculations to plan clinical trials with and without pilot adherence data. Sample size formulas and tests for normally distributed outcomes are also developed in a Web Appendix that account for uncertainty of estimates from external or internal pilot data.

journal_name

Biometrics

journal_title

Biometrics

authors

Shardell MD,El-Kamary SS

doi

10.1111/j.1541-0420.2008.01114.x

subject

Has Abstract

pub_date

2009-06-01 00:00:00

pages

635-9

issue

2

eissn

0006-341X

issn

1541-0420

pii

BIOM1114

journal_volume

65

pub_type

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