2024
DOI: 10.1007/s40273-024-01370-2
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Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review

Tobias Sydendal Grand,
Shijie Ren,
James Hall
et al.

Abstract: Background and Objectives There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. Many of them have been highlighted in previous systematic reviews but they have not been summarised in a comprehensive manner. For all stakeholders working with rare diseases, it is important to be aware and understand these issues. The objective of this review is to identify the main challenges for the economic evaluation of orphan drugs in r… Show more

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References 67 publications
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