External Controls to Study Treatment Effects in Rare Diseases: Challenges and Future Directions

IF 6.3 2区 医学 Q1 PHARMACOLOGY & PHARMACY
Janick Weberpals, Shirley V. Wang
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引用次数: 0

Abstract

Regulators increasingly rely on real-world evidence generated from routine-care health data to evaluate novel therapies. Particularly, external control arms are increasingly used to supplement and contextualize efficacy and safety claims of single arm clinical trials for rare disease therapies. However, there are a number of methodological issues that may affect the validity of results derived from such comparisons. In this mini-review, we briefly summarize frequently used approaches and outline some of the most important criticisms and paths forward.

研究罕见病治疗效果的外部对照:挑战与未来方向
监管机构越来越依赖于从日常护理健康数据中生成的真实世界证据来评估新型疗法。特别是,外部对照臂越来越多地被用来补充罕见病疗法单臂临床试验的疗效和安全性要求,并使之符合实际情况。然而,有一些方法学问题可能会影响此类比较结果的有效性。在这篇小型综述中,我们简要总结了常用的方法,并概述了一些最重要的批评意见和前进方向。
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来源期刊
CiteScore
12.70
自引率
7.50%
发文量
290
审稿时长
2 months
期刊介绍: Clinical Pharmacology & Therapeutics (CPT) is the authoritative cross-disciplinary journal in experimental and clinical medicine devoted to publishing advances in the nature, action, efficacy, and evaluation of therapeutics. CPT welcomes original Articles in the emerging areas of translational, predictive and personalized medicine; new therapeutic modalities including gene and cell therapies; pharmacogenomics, proteomics and metabolomics; bioinformation and applied systems biology complementing areas of pharmacokinetics and pharmacodynamics, human investigation and clinical trials, pharmacovigilence, pharmacoepidemiology, pharmacometrics, and population pharmacology.
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