Leveraging real-world data to conduct externally controlled trial for rare diseases with count-type endpoints: utilizing multiple entries - a simulation study.

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Title: Leveraging real-world data to conduct externally controlled trial for rare diseases with count-type endpoints: utilizing multiple entries - a simulation study.
Authors: Sun T; Biometrics, Moderna, Inc., Cambridge, Massachusetts, USA., Liao E; Biometrics, Moderna, Inc., Cambridge, Massachusetts, USA., Shao N; Biometrics, Moderna, Inc., Cambridge, Massachusetts, USA., Luo J; Biometrics, Moderna, Inc., Cambridge, Massachusetts, USA.
Source: Journal of biopharmaceutical statistics [J Biopharm Stat] 2026 May; Vol. 36 (3), pp. 337-349. Date of Electronic Publication: 2024 Oct 27.
Publication Type: Journal Article
Journal Info: Publisher: Taylor & Francis Country of Publication: England NLM ID: 9200436 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1520-5711 (Electronic) Linking ISSN: 10543406 NLM ISO Abbreviation: J Biopharm Stat Subsets: MEDLINE
Database: MEDLINE Ultimate
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ISSN:1520-5711
DOI:10.1080/10543406.2024.2420644