证据到决策（E2D）：为早期肿瘤学开发构建行动特异性证据
Evidence-to-Decision (E2D): Structuring Action-Specific Evidence for Early-Phase Oncology Development
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中文总结 AI 辅助
E2D框架将早期肿瘤学证据组织为围绕具体开发行动的结构化评估，通过预测概率量化行动特异性支持，从而将决策问题从治疗是否有前景转变为当前证据支持哪些后续研究。
中文摘要 AI 辅助
早期肿瘤学证据通常通过询问治疗是否有前景来总结，但有希望的信号可以支持几种不同的后续研究。我们提出证据到决策（E2D）框架，该框架围绕具体的开发行动组织证据。对于每个候选行动，E2D定义了预期的未来研究、什么算作成功、采取该步骤前需要什么证据、当前可获得的证据是什么，以及连接当前和未来环境的假设。统计方法随后可以量化行动特异性的支持度及对这些假设的敏感性，而最终的开发选择仍然是多学科的。我们在罕见的儿科肿瘤学环境中通过使用预测概率来说明E2D，包括同环境单臂扩展、同环境随机II期试验、向前线或一线随机研究的推进，以及一个独立的降级示例。校准后的支持规则产生了临床上不同的证据状态：中等证据可以支持同环境开发而不支持前线推进，安全问题可能缩小支持范围尽管有强大的活性，更强的证据可以同时支持多条路径。因此，E2D将问题从治疗是否普遍有前景重新定义为：当前可获得的证据支持哪些后续研究，哪些仍不支持，以及哪些额外证据可能改变该评估。
英文摘要
Early-phase oncology evidence is often summarized by asking whether a treatment is promising, but an encouraging signal can support several different next studies. We propose Evidence-to-Decision (E2D), a framework that organizes evidence around specific development actions. For each candidate action, E2D defines the contemplated future study, what would count as success, what evidence is needed before taking that step, what evidence is available now, and what assumptions connect the current and future settings. Statistical methods can then quantify action-specific support and sensitivity to those assumptions, while the final development choice remains multidisciplinary. We illustrate E2D in a rare pediatric oncology setting using predictive probabilities for same-setting single-arm expansion, same-setting randomized Phase II, movement to an earlier-line or frontline randomized study, and a separate de-escalation example. Calibrated support rules produced clinically distinct evidence states: intermediate evidence could support same-setting development without supporting frontline movement, safety concerns could narrow support despite strong activity, and stronger evidence could support several paths simultaneously. E2D therefore reframes the question from whether a treatment is generally promising to which next studies are supported by the evidence available now, what remains unsupported, and what additional evidence could change that assessment.
发表机构
- St. Jude Children's Research Hospital（圣裘德儿童研究医院）
机构由 AI 辅助整理，请以论文原文为准。
