A comparison of methods for designing hybrid type 2 cluster-randomized trials with continuous effectiveness and implementation endpoints
具有连续效果与实施终点的二型混合整群随机试验设计方法比较AI 解读 ↓
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Hybrid type 2 studies are gaining popularity for their ability to assess both implementation and health outcomes as co-primary endpoints. Often conducted as cluster-randomized trials (CRTs), five design methods can validly power these studies: p-value adjustment methods, combined outcomes approach, single weighted 1-DF test, disjunctive 2-DF test, and conjunctive test. We compared these methods theoretically and numerically. Theoretical comparisons of power equations allowed us to identify when one method had more or less power than another globally. We showed that p-value adjustment methods are always less powerful than both the combined outcomes approach and the single 1-DF test, and identified conditions where the disjunctive 2-DF test is less powerful than the single 1-DF test. To further investigate when power advantages shift, we conducted a large-scale numerical study using our novel crt2power R package, which calculates power or sample size for CRTs with two continuous co-primary endpoints using these methods. Across 45,000 input scenarios, we found specific patterns: when treatment effects are unequal, the disjunctive 2-DF test tends to be most powerful; when treatment effects are equal, the single 1-DF test tends to dominate. Together, these comparisons offer practical guidance for powering hybrid type 2 studies.
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Background: Generalized Odds (Gen Odds) provide summaries for ordinal outcomes without requiring the proportional odds assumption. However, conventional Gen Odds compares independently drawn treatment and control outcomes and targets a population-level contrast rather than an individual-level contrast. We developed an individual-level causal formulation of Gen Odds and examined its identification. Methods: We defined the Causal Gen Odds using the joint distribution of…
We develop a semiparametric continuous time inferential framework for testing cumulative treatment effects, including both immediate and carryover effects. The latent state process is modeled by an ordinary differential equation (ODE) whose drift depends on state and treatment, while the observed data are noisy measurements of this latent state at irregular, multi resolution time points. We define the average treatment effect (ATE) as the difference of expected cumulative…
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