Statistical Challenges in the Evaluation of Treatments for Small Patient Populations
作者： E. L. Korn , L. M. McShane , B. Freidlin
DOI: 10.1126/SCITRANSLMED.3004018
关键词: Randomized controlled trial 、 Common disease 、 Clinical study 、 Alternative medicine 、 Standard of care 、 Context (language use) 、 Intensive care medicine 、 Patient subgroups 、 Clinical trial 、 Medicine
摘要: The development of a new treatment typically involves evaluation its efficacy in large clinical trial which patients are randomly assigned either the or standard care. Results from these randomized trials allow for definitive and unbiased assessment benefit over one. For rare diseases small patient subgroups identified within context common disease, it may not be possible to conduct trial. In this Review, we discuss alternative study designs statistical challenges that arise when attempting assure results yield robust conclusions about safety effectiveness medical product.
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