Department of Epidemiology, Gillings School of Global Public Health, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, United States.
Unit of Epidemiology, Institute of Environmental Medicine, Karolinska Institutet, SE-171 77 Stockholm, Sweden.
Am J Epidemiol. 2024 Nov 4;193(11):1503-1506. doi: 10.1093/aje/kwae129.
Randomized trials estimate the average treatment effect within individuals who are eligible, invited, and agree to enroll. However, decision-makers often require evidence that extends beyond the trial's enrolled population to inform policy or actions for their specific target population. Each decision-maker has distinct target populations, the composition of which may not often align with that of the trial population. As researchers, we should identify a decision-maker for whom we aim to generate evidence early in the research process. We can then specify a target population of their interest and determine if a policy or action can be informed using results from a trial alone, or if additional complementary real-world data and analysis are required. In this commentary, we outline 5 key groupings of decision-makers: policymakers, payers, purchasers, providers, and patients. We then specify relevant target populations for decision-makers interested in the effectiveness of beta-blockers after a myocardial infarction with preserved ejection fraction. Finally, we summarize the scenarios in which results from a randomized trial may or may not apply to these target populations and suggest relevant analytic approaches that can generate evidence to better align with a decision-maker's needs. This article is part of a Special Collection on Pharmacoepidemiology.
随机试验估计了符合条件、被邀请并同意入组的个体的平均治疗效果。然而，决策者通常需要超出试验入组人群的证据，以便为其特定目标人群提供政策或行动的信息。每个决策者都有不同的目标人群，其构成通常与试验人群不一致。作为研究人员，我们应该在研究过程的早期确定我们希望为其生成证据的决策者。然后，我们可以指定他们感兴趣的目标人群，并确定是否可以仅使用试验结果来为政策或行动提供信息，或者是否需要额外的补充真实世界数据和分析。在这篇评论中，我们概述了决策者的 5 个关键群体：政策制定者、支付者、购买者、提供者和患者。然后，我们为对射血分数保留型心肌梗死后β受体阻滞剂有效性感兴趣的决策者指定了相关的目标人群。最后，我们总结了随机试验结果可能适用于或不适用于这些目标人群的情况，并提出了相关的分析方法，可以生成更符合决策者需求的证据。本文是药物流行病学特刊的一部分。
