Alternative strategies in orphan drug development

Abstract

Charging high prices for orphan drugs is unsustainable due to limited healthcare budgets. New models of orphan drug development are needed to help find treatments for the majority of rare diseases which have no treatment. This article discusses the challenges of value-based pricing and whether this would be a viable solution to the pricing problem. It explores how the search for cheaper models of orphan drug development could be through non-profit initiatives and drug repurposing. It ends with a case study of DevelopAKUre, an EC-funded consortium developing a repurposed drug for AKU, a rare genetic disease.

Keywords::

• not-for-profit
• orphan drug
• rare disease
• repurposing