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Background: Well-designed phase II trials must have acceptable error ratesrelative to a pre-specified success criterion, usually a statisticallysignificant p-value. Such standard designs may not always suffice from aclinical perspective because clinical relevance may call for more. For example,proof-of-concept in phase II often requires not only statistical significancebut also a sufficiently large effect estimate.  Purpose: We propose dual-criterion designs to complement statisticalsignificance with clinical relevance, discuss their methodology, and illustratetheir implementation in phase II.  Methods: Clinical relevance requires the effect estimate to pass a clinicallymotivated threshold (the decision value). In contrast to standard designs, therequired effect estimate is an explicit design input whereas study power isimplicit. The sample size for a dual-criterion design needs carefulconsiderations of the study's operating characteristics (type-I error, power).  Results: Dual-criterion designs are discussed for a randomized controlled anda single-arm phase II trial, including decision criteria, sample sizecalculations, decisions under various data scenarios, and operatingcharacteristics. The designs facilitate GO/NO-GO decisions due to theircomplementary statistical-clinical criterion.  Conclusion: To improve evidence-based decision-making, a formal yettransparent quantitative framework is important. Dual-criterion designs offeran appealing statistical-clinical compromise, which may be preferable tostandard designs if evidence against the null hypothesis alone does not sufficefor an efficacy claim.

Beyond p-values: a phase II dual-criterion design with statistical significance and clinical relevance