Li Sheyu, Litvin Valentyn, Manski Charles F
From the Department of Endocrinology and Metabolism, MAGIC China Centre, Cochrane China Centre, Chinese Evidence-based Medicine Centre, West China Hospital, Sichuan University, Chengdu, 610041, China.
Department of Economics, Northwestern University, Evanston, IL 60208-2600, USA.
Epidemiology. 2023 May 1;34(3):319-324. doi: 10.1097/EDE.0000000000001593. Epub 2023 Jan 30.
Medical journals have adhered to a reporting practice that seriously limits the usefulness of published trial findings. Medical decision makers commonly observe many patient covariates and seek to use this information to personalize treatment choices. Yet standard summaries of trial findings only partition subjects into broad subgroups, typically binary categories. Given this reporting practice, we study the problem of inference on long mean treatment outcomes E[y(t)|x], where t is a treatment, y(t) is a treatment outcome, and the covariate vector x has length K, each component being a binary variable. The available data are estimates of {E[y(t)|x k = 0], E[y(t)|x k = 1], P(x k )}, k = 1,..., K reported in journal articles. We show that reported trial findings partially identify {E[y(t)|x], P(x)}. Illustrative computations demonstrate that the summaries of trial findings in journal articles may imply only wide bounds on long mean outcomes. One can realistically tighten inferences if one can combine reported trial findings with credible assumptions having identifying power, such as bounded-variation assumptions.
医学期刊一直遵循一种报告方式，这种方式严重限制了已发表试验结果的实用性。医学决策者通常会观察许多患者协变量，并试图利用这些信息来个性化治疗选择。然而，试验结果的标准总结仅将受试者分为宽泛的亚组，通常是二元类别。鉴于这种报告方式，我们研究了关于长期平均治疗结果E[y(t)|x]的推断问题，其中t是一种治疗方法，y(t)是治疗结果，协变量向量x的长度为K，每个分量都是一个二元变量。可用数据是期刊文章中报告的{E[y(t)|x k = 0], E[y(t)|x k = 1], P(x k )}的估计值，k = 1,..., K。我们表明，报告的试验结果部分识别了{E[y(t)|x], P(x)}。示例计算表明，期刊文章中试验结果的总结可能仅意味着长期平均结果的宽泛界限。如果能够将报告的试验结果与具有识别能力的可信假设（如有界变差假设）相结合，就可以切实收紧推断。
