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Notes on odds ratio estimation for a randomized clinical trial with noncompliance and missing outcomes

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  • Kung-Jong Lui
  • Kuang-Chao Chang

Abstract

The odds ratio (OR) has been recommended elsewhere to measure the relative treatment efficacy in a randomized clinical trial (RCT), because it possesses a few desirable statistical properties. In practice, it is not uncommon to come across an RCT in which there are patients who do not comply with their assigned treatments and patients whose outcomes are missing. Under the compound exclusion restriction, latent ignorable and monotonicity assumptions, we derive the maximum likelihood estimator (MLE) of the OR and apply Monte Carlo simulation to compare its performance with those of the other two commonly used estimators for missing completely at random (MCAR) and for the intention-to-treat (ITT) analysis based on patients with known outcomes, respectively. We note that both estimators for MCAR and the ITT analysis may produce a misleading inference of the OR even when the relative treatment effect is equal. We further derive three asymptotic interval estimators for the OR, including the interval estimator using Wald's statistic, the interval estimator using the logarithmic transformation, and the interval estimator using an ad hoc procedure of combining the above two interval estimators. On the basis of a Monte Carlo simulation, we evaluate the finite-sample performance of these interval estimators in a variety of situations. Finally, we use the data taken from a randomized encouragement design studying the effect of flu shots on the flu-related hospitalization rate to illustrate the use of the MLE and the asymptotic interval estimators for the OR developed here.

Suggested Citation

  • Kung-Jong Lui & Kuang-Chao Chang, 2010. "Notes on odds ratio estimation for a randomized clinical trial with noncompliance and missing outcomes," Journal of Applied Statistics, Taylor & Francis Journals, vol. 37(12), pages 2057-2071.
    • Handle: RePEc:taf:japsta:v:37:y:2010:i:12:p:2057-2071
    DOI: 10.1080/02664760903214411
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    References listed on IDEAS

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    1. Lui, Kung-Jong & Cumberland, William G., 2008. "Notes on estimation of proportion ratio under a non-compliance randomized trial with missing outcomes," Computational Statistics & Data Analysis, Elsevier, vol. 52(9), pages 4325-4345, May.
    2. Shigeyuki Matsui, 2005. "Stratified Analysis in Randomized Trials with Noncompliance," Biometrics, The International Biometric Society, vol. 61(3), pages 816-823, September.
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