The evidence framework for precision cancer medicine
The evidence framework for precision cancer medicine
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DOI：
10.1038/nrclinonc.2017.186
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发表时间：
2018-03-01
影响因子：
78.8
通讯作者：
Schilsky, Richard L.
中科院分区：
文献类型：
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作者：
Moscow, Jeffrey A.;Fojo, Tito;Schilsky, Richard L.
精准癌症医学（PCM）是一个概念，肿瘤学家越来越努力地调整靶向治疗的使用，以匹配癌症基因组的复杂性。这种方法与用于支持肿瘤学实践的历史框架相矛盾，该框架需要来自随机对照试验的证据才能改变护理标准。这种对比证明了 PCM 的讽刺之处：尽管支持这些疗法益处的临床证据通常不太精确，但疗法本身比标准细胞毒性药物更精确。尽管如此，PCM的实施仍应基于支持临床决策的循证开发框架；这种方法不应该是对肿瘤活检样本进行测序后简单的标签外药物处方。在 PCM 成为临床意义不确定的基因组异常肿瘤患者的治疗标准之前，需要对日益复杂的诊断测试进行临床验证，开发新的疗效评估方法，并重新评估足以证明精准癌症治疗益处的证据标准。
Precision cancer medicine (PCM) is a concept in which oncologists increasingly strive to tailor the use of targeted therapies in order to match the complexity of the cancer genome. This approach contradicts the historical framework used to support oncology practice that requires evidence from randomized controlled trials in order to change standards of care. This contrast demonstrates the irony of PCM: the therapies themselves are more precise than standard cytotoxic agents, although the clinical evidence supporting the benefits of these therapies is often considerably less precise. Nevertheless, the implementation of PCM should still be based on a framework of evidence-based development that supports clinical decision-making; this approach should not be simple off-label prescription of drugs following sequencing of a tumour biopsy sample. The clinical validation of increasingly complex diagnostic tests, the development of novel methods of evaluating efficacy, and the re-assessment of the standards of evidence sufficient to demonstrate the benefit of precision cancer therapies are all needed before PCM becomes the standard of care for patients with tumours harbouring genomic abnormalities of uncertain clinical significance.