A Review of the Data-Driven Policy Making of Medical Financial Assistance for Rare Diseases: Current Situation, Trends and Opportunities

The inherent clinical uncertainties, substantial costs, and small patient cohorts of orphan drugs limit the applicability of randomized controlled trial (RCT)-based health technology assessments (HTAs) in guiding coverage criteria, sustainable financing models, and equitable reimbursement frameworks...

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Bibliographic Details

Published in:	罕见病研究
Main Authors:	DUAN Yuehan, FAN Zhiyu, LI Qianhui, PENG Zhaiwen
Format:	Article
Language:	Chinese
Published:	Editorial Office of Journal of Rare Diseases 2025-01-01
Subjects:	rare diseases orphan drugs medical financial assistance data-driven decision-making
Online Access:	https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2025.01.006