PW02-027 - CAPS and cost-effectiveness analysis project

Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally very expensive on a per patient basis. European Union (EU) regulations promote the development of orphan drugs; but to contain costs, EU healthcare systems will increasily need the cost-effectiveness analysis (CEA) of therapies when deciding if they should be funded. Conventional methods for CEA of drugs for common conditions do not apply to ultra-orphan drugs; therefore, additional factors need to be considered.