Ethical and Economic Issues in the Appraisal of Medicines for Ultra-Rare Conditions

Henderson N., Errea M., Skedgel C. and Jofre-Bonet, M.

Consulting Report
January 2020

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In light of concerns that not all medicines for ultra-rare (also known as ultra-orphan) conditions are appraised under the same NICE process, a new OHE Consulting Report discusses the distinct ethical and economic challenges faced by medicines for ultra-rare conditions, with particular reference to the challenges of HTA in the UKA failure to consistently consider all ultra-rare disease medicines under the HST process could lead to inequalities in access and health outcomes for patients with ultra-rare conditions.