Real-world Evidence Requirements with Respect to External Control Arms of Single-arm Clinical Trials by Major Regulatory and HTA Agencies – Comparative Review of Guidelines and Identification of Trends Based on Seven Recent Case Studies

Volker Schuster and Anna Tena Marco
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Anna Tena Marco: SAI MedPartners, Neuhofstrasse 12, 6341 Baar, Switzerland

Biomedical Journal of Scientific & Technical Research, 2024, vol. 58, issue 1, 49952-49966

Abstract: Shifting R&D focus towards the (ultra-)rare diseases and precision medicine leads to a rising need to assess single-arm clinical trials for regulatory and HTA purposes. While numerous time-tested methods exist to utilize real-world evidence as external control groups while limiting the confounding risk, there is still limited international consensus on how to generate, curate and use real-world-data in order to serve as acceptable external control.

Keywords: Journals on Medical Drug and Therapeutics; Journals on Emergency Medicine; Physical Medicine and Rehabilitation; Journals on Infectious Diseases Addiction Science and Clinical Pathology; Open Access Clinical and Medical Journal; Journals on Biomedical Science; List of Open Access Medical Journal; Journals on Biomedical Engineering; Open Access Medical Journal; Biomedical Science Articles; Journal of Scientific and Technical Research (search for similar items in EconPapers)
Date: 2024
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Persistent link: https://EconPapers.repec.org/RePEc:abf:journl:v:58:y:2024:i:1:p:49952-49966

DOI: 10.26717/BJSTR.2024.58.009096

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