<p>Randomized clinical trials are usually designed to test whether a new treatment is equivalent to a reference treatment. To establish assay sensitivity and constancy, if ethically justifiable and practically feasible, the equivalence may be assessed through a three-arm trial, consisting of a new treatment, an active reference treatment and a placebo. In this article, we consider three-arm trials with multinomial outcome variables. Based on a constrained bootstrap approach, we develop a novel test for assessing the equivalence of reference and new treatments. We show that the proposed test is asymptotically of level <InlineEquation ID="IEq1"> <EquationSource Format="TEX">\(\alpha\)</EquationSource> </InlineEquation> and is consistent. Simulation studies demonstrate that the proposed test can control type I errors well and detect the equivalence effectively with moderate sample sizes.</p>

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Testing equivalence for three-arm trials with multinomial outcomes

  • Pao-sheng Shen

摘要

Randomized clinical trials are usually designed to test whether a new treatment is equivalent to a reference treatment. To establish assay sensitivity and constancy, if ethically justifiable and practically feasible, the equivalence may be assessed through a three-arm trial, consisting of a new treatment, an active reference treatment and a placebo. In this article, we consider three-arm trials with multinomial outcome variables. Based on a constrained bootstrap approach, we develop a novel test for assessing the equivalence of reference and new treatments. We show that the proposed test is asymptotically of level \(\alpha\) and is consistent. Simulation studies demonstrate that the proposed test can control type I errors well and detect the equivalence effectively with moderate sample sizes.