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Rethinking placebos: embracing synthetic control arms in clinical trials for rare tumors.

作者信息

Serrano César, Rothschild Sara, Villacampa Guillermo, Heinrich Michael C, George Suzanne, Blay Jean-Yves, Sicklick Jason K, Schwartz Gary K, Rastogi Sameer, Jones Robin L, Rutkowski Piotr, Somaiah Neeta, Navarro Víctor, Evans Denisse, Trent Jonathan C

机构信息

Department of Medical Oncology, Vall d'Hebron University Hospital, Barcelona, Spain.

Sarcoma Translational Research Program, Vall d'Hebron Institute of Oncology (VHIO), Barcelona, Spain.

出版信息

Nat Med. 2023 Nov;29(11):2689-2692. doi: 10.1038/s41591-023-02578-z.

DOI:10.1038/s41591-023-02578-z
PMID:37828359
Abstract
摘要

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The next generation of evidence-based medicine.循证医学的下一代。
Nat Med. 2023 Jan;29(1):49-58. doi: 10.1038/s41591-022-02160-z. Epub 2023 Jan 16.
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Audit of Data Sharing by Pharmaceutical Companies for Anticancer Medicines Approved by the US Food and Drug Administration.美国食品和药物管理局批准的抗癌药物的制药公司的数据共享审核。
JAMA Oncol. 2022 Sep 1;8(9):1310-1316. doi: 10.1001/jamaoncol.2022.2867.
3
Pimitespib in patients with advanced gastrointestinal stromal tumor (CHAPTER-GIST-301): a randomized, double-blind, placebo-controlled phase III trial.
医疗保健与药物研发中的合成数据:定义、监管框架、问题
CPT Pharmacometrics Syst Pharmacol. 2025 May;14(5):840-852. doi: 10.1002/psp4.70021. Epub 2025 Apr 7.
4
Patient-derived xenograft models of gastrointestinal stromal tumors provide a ready-to-use platform for translational research.胃肠道间质瘤的患者来源异种移植模型为转化研究提供了一个现成可用的平台。
Dis Model Mech. 2025 Feb 1;18(2). doi: 10.1242/dmm.052225. Epub 2025 Feb 21.
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Novel clinical trial designs emerging from the molecular reclassification of cancer.源于癌症分子重新分类的新型临床试验设计。
CA Cancer J Clin. 2025 May-Jun;75(3):243-267. doi: 10.3322/caac.21880. Epub 2025 Jan 22.
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AI meets real-world patients.人工智能与现实世界中的患者相遇。
Nat Biotechnol. 2024 Oct;42(10):1475. doi: 10.1038/s41587-024-02455-1.
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Cancers (Basel). 2024 Jun 29;16(13):2400. doi: 10.3390/cancers16132400.
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Cancers (Basel). 2024 Feb 28;16(5):985. doi: 10.3390/cancers16050985.
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