Treatment effect estimates are often available from randomized controlled trials as a single average treatment effect for a certain patient population. Estimates of the conditional average treatment effect (CATE) are more useful for individualized treatment decision making, but randomized trials are often too small to estimate the CATE. There are several examples in medical literature where the assumption of a known constant relative treatment effect (e.g. an odds-ratio) is used to estimate CATE models from large observational datasets. One approach to estimating these CATE models is by using the relative treatment effect as an offset, while estimating the covariate-specific baseline risk. Whether this is a valid approach in the presence of unobserved confounding is unknown. We demonstrate for a simple example that offset models do not recover the true CATE in the presence of unobserved confounding. We then explore the magnitude of this bias in numerical experiments. For virtually all plausible confounding magnitudes, estimating the CATE using offset models is more accurate than assuming a single absolute treatment effect whenever there is sufficient variation in the baseline risk. Next, we observe that the odds-ratios reported in randomized controlled trials are not the odds-ratios that are needed in offset models because trials often report the marginal odds-ratio. We introduce a constraint to better use marginal odds-ratios from randomized controlled trials and find that the newly introduced constrained offset models have lower bias than standard offset models. Finally, we highlight directions for future research for exploiting the assumption of a constant relative treatment effect with offset models.
翻译:有条件平均治疗效果(CATE)的估计数对于个别治疗决策而言更有用,但随机试验往往太小,无法估计CATE。医学文献中有几个例子,用已知的经常相对治疗效应(如不测)的假设来估计CATE模型从大型观测数据集中得出。估计CATE模型的一种方法是,利用相对治疗效应作为抵消,同时估计具体COVI的基准风险。在出现未观察到的混凝土时,这种估计是否有效。我们用一个简单的例子来证明,当出现无法观察到的混凝土时,抵消模型并不能恢复真正的CAATE。然后我们探讨数字实验中的这种偏差程度。对于几乎所有令人信服的测深程度,使用CAATE模型估计的准确性比假设在基准风险出现充分差异时的单一绝对治疗效果更准确。我们发现,在随机研究效果中报告的概率比假设的数值要低。我们所报告,在随机控制的实验中,从随机分析模型中往往没有恢复真正的CATE 限制实验,因为我们所需要的的是,在最后的精确度试验中,我们所需要的是比较精确的精确的模型,我们没有抵消了对结果的精确的分析。