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Examining the impact of different country processes for appraising rare disease treatments: a case study analysis

Amanda Whittal, Elena Nicod, Mike Drummond, Karen Facey
March 2021

Conventional appraisal approaches are often unsuitable for rare disease treatments (RDTs) due to the nature of rare conditions, which makes it more difficult to generate evidence that meets the standards of HTA bodies. The objective of this case study analysis was to examine the impacts of various types of appraisal processes on two contrasting RDTs, nusinersen and vortigene neparvovec, by systematically comparing them. Appraisals from nine selected countries were divided into separate process and adapted process categories, and reviewed for their impact on the selected treatments. Both separate and adapted approaches were found to potentially facilitate more structured, consistent decision-making and better management of RDT specificities.

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