Drug development for rare diseases: a case for patient-centricity, equity, and access to clinical trials

Abstract Purpose Clinical trials often represent the only viable treatment option for many rare disease patients. However, these patients face significant barriers to participation, including cognitive and physical limitations, geographic disparities, misdiagnosis, and socioeconomic challenges, with...

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Bibliographic Details
Main Authors: Allison E. Bowen, Satish Nargundkar
Format: Article
Language:English
Published: Springer 2025-05-01
Series:Journal of Rare Diseases
Subjects:
Online Access:https://doi.org/10.1007/s44162-025-00069-y
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