Rare disease clinical trials in the European Union: navigating regulatory and clinical challenges.

ABSTRACT: Clinical development for orphan drugs presents significant difficulties and challenges. There is no unique or standard design, conduct, and outcome assessment methodology and it is sometimes impractical to fit design models of rare disease trials in any practiced and well-known framework. In the European Union (EU) these challenges encompass a broad array of subjects, including trial design, study outcomes, patient recruitment, trial conduct ethics, trial cost, and chances of success. This literature-based review study aims to provide a thorough overview of the critical aspects of rare disease trials in the EU by analyzing the current landscape of rare disease trials, highlighting key challenges, delving into regulatory and research initiatives and innovation in trial designs, and proposin

SUBMITTER: Mishra S 

PROVIDER: S-EPMC11292868 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature