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Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review.

Tobias Sydendal GrandShijie RenJames A HallDaniel Oudin ÅstrømStephane Alexandre RegnierPraveen Thokala
Published in: PharmacoEconomics (2024)
The results highlighted issues around a considerable paucity of evidence for economic evaluations and few cost-effectiveness analyses, supporting the notion that a paucity of evidence makes economic evaluations of rare diseases more challenging compared with more prevalent diseases. Furthermore, we provide recommendations for more sustainable approaches in economic evaluations of rare diseases.
Keyphrases
  • randomized controlled trial
  • systematic review