NBER Working Papers · 2026 · Jason Abaluck、Leila Agha、David C. Chan Jr
中文摘要
医学指南将临床证据转化为治疗规则，但证据对治疗获益的识别可能比对治疗危害的识别更为清晰。我们结合随机试验与退伍军人健康管理局的数据，研究心房颤动的抗凝治疗。试验证据揭示，卒中预防获益存在较大的异质性，但无法可靠识别治疗所致的出血危害在患者之间如何变化。在目标人群中，预测的卒中预防获益较大的患者，未接受治疗时的出血风险也较高，因此，这种不明确性会带来实质性后果。我们评估现有指南，并制定对不同危害结构均稳健的极大极小和极小极大后悔治疗规则。在预防50%的可预防卒中这一基准下，稳健规则引发的出血比随机治疗少33%—41%，且表现优于现有指南。有限样本不确定性使患者的精细排序出现明显的不稳定性，但治疗分配或福利的不稳定性很小。医生的表现，以及指南依从性与医生技能之间的关系，也在很大程度上取决于有关治疗所致的出血危害在患者之间如何变化的假设。
Abstract
Medical guidelines translate clinical evidence into treatment rules, but the evidence may identify treatment benefits more clearly than harms. We study anticoagulation for atrial fibrillation, combining randomized trials with Veterans Health Administration data. Trial evidence reveals substantial heterogeneity in stroke-prevention benefits but does not reliably identify how treatment-induced bleeding harms vary across patients. In the target population, patients with larger predicted stroke benefits also have higher untreated bleeding risk, making this ambiguity consequential. We evaluate existing guidelines and develop maximin and minimax-regret treatment rules robust to alternative harm structures. At a benchmark preventing 50% of preventable strokes, robust rules induce 33-41% fewer bleeds than random treatment and outperform existing guidelines. Finite-sample uncertainty generates noticeable instability in fine patient rankings but little instability in treatment assignments or welfare. Physician performance and the relationship between guideline adherence and skill also depend substantially on assumptions about how treatment-induced bleeding harms vary across patients.