Statistical controversies in clinical research: basket trials, umbrella trials, and other master protocols: a review and examples
作者： L.A. Renfro , D.J. Sargent
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摘要: In recent years, cancers once viewed as relatively homogeneous in terms of organ location and treatment strategy are now better understood to be increasingly heterogeneous across biomarker genetically defined patient subgroups. This has produced a shift toward development biomarker-targeted agents during time when funding for cancer research been limited; result, the need improved operational efficiency studying many agent-and-target combinations parallel emerged. Platform trials, basket umbrella trials new approaches clinical driven by this enhanced modern era specific subpopulations decreased resources study treatments individual subtypes traditional way. review, we provide an overview these types trial designs, including discussions motivation their use, recommended terminology, examples, challenges encountered application.
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