Gadbury G L, Iyer H K
Department of Mathematical Sciences, University of North Carolina-Greensboro 27402, USA.
Biometrics. 2000 Sep;56(3):882-5. doi: 10.1111/j.0006-341x.2000.00882.x.
Most statistical characterizations of a treatment effect focus on the average effect of the treatment over an entire population. However, average effects may provide inadequate information, sometimes misleading information, when a substantial unit-treatment interaction is present in the population. It is even possible that a nonnegligible proportion of the individuals in the population experience an unfavorable treatment effect even though the treatment might appear to be beneficial when considering population averages. This paper examines the extent to which information about unit-treatment interaction can be extracted using observed data from a two-treatment completely randomized experiment. A method for utilizing the information from an available covariate is proposed. Although unit-treatment interaction is a nonidentifiable quantity, we show that mathematical bounds for it can be estimated from observed data. These bounds lead to estimated bounds for the probability of an unfavorable treatment effect. Maximum likelihood estimators of the bounds and their corresponding large-sample distributions are given. The use of the estimated bounds is illustrated in a clinical trials data example.
大多数对治疗效果的统计描述都集中在治疗对整个人口的平均效果上。然而,当人群中存在显著的个体-治疗相互作用时,平均效果可能提供的信息不足,有时甚至会产生误导。即使在考虑总体平均值时治疗似乎是有益的,但人群中仍可能有不可忽略比例的个体经历不利的治疗效果。本文研究了在双治疗完全随机实验中,能从观测数据中提取关于个体-治疗相互作用信息的程度。提出了一种利用可用协变量信息的方法。虽然个体-治疗相互作用是一个不可识别的量,但我们表明可以从观测数据中估计其数学界限。这些界限导致了对不利治疗效果概率的估计界限。给出了界限的最大似然估计量及其相应的大样本分布。在一个临床试验数据示例中说明了估计界限的使用。