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Updated: Jul 4, 2025

Inverse Probability of Treatment Weighting Propensity Score using the Military Health System Data Repository and National Death Index
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现实世界的证据用于为罕见疾病治疗的覆盖范围的确定

Victoria W Dayer1, Michael F Drummond2, Omar Dabbous3

  • 1CHOICE Institute, School of Pharmacy, University of Washington, 1959 NE Pacific Street, Seattle, WA, 98195, USA. vwinslow@uw.edu.

Orphanet journal of rare diseases
|February 7, 2024
PubMed
概括

现实世界的证据 (RWE) 帮助卫生技术评估 (HTA) 治疗罕见疾病,但面临着重大的方法挑战. 解决这些问题对于明智的HTA决策和有效的市场准入至关重要.

关键词:
细胞和基因疗法细胞和基因疗法.覆盖范围的确定方法覆盖范围包括证据开发.卫生技术评估 卫生技术评估罕见疾病是一种罕见的疾病.现实世界的证据 现实世界的证据

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