Application of observational research methods to real-world studies for rare disease drugs: A scoping review protocol

Yuti P Patel, Lea Ghaddar, Yuqi Lin, Nuzat Karim, Kelvin Chan, Lee Dupuis and Mina Tadrous

PLOS ONE, 2025, vol. 20, issue 3, 1-6

Abstract: The primary objective is to identify which observational research methods have been used in the last 5 years in rare disease drug evaluation and how they are applied to generate adequate evidence regarding the real-world effectiveness or safety of rare disease drugs. Rare disease is an umbrella term for a condition which affects

Date: 2025
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Persistent link: https://EconPapers.repec.org/RePEc:plo:pone00:0304540

DOI: 10.1371/journal.pone.0304540

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