31 May 2021 - Conventional appraisal and reimbursement processes are being challenged by the increasing number of rare disease treatments with a small evidence base and often a high price. 

Processes to appraise rare disease treatments vary across countries; some use standard processes, others have separate processes or adapted processes that explicitly deal with rare disease specificities. 

The objective of this study was to examine the impacts of different appraisal processes for two rare disease treatments.

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