The authors presented arguments for using cost-based pricing models to develop pricing strategies for repurposing existing or previously authorized medicines in new rare disease indications.1 We believe that they unfortunately oversimplify the pricing of repurposed orphan medicinal products, given that a cost-based determination of price of medicine meeting the high unmet needs of orphan diseases rewards high-cost developments
rather than high-value products in an underserved patient population. On the contrary, basing prices on the overall value proposition of an orphan medicinal product represents the healthcare payers’ willingness to reimburse such products based on healthcare outcomes over and above efficacy, as well as the correlation between economic and clinical benefits to stakeholders involved.2

Below, we provide a perspective that supports the argument that the value of repurposing medicines in rare indications is not suitably captured using a cost-based pricing methodology to ensure the sustainability of this branch of drug development by building on the mexiletine example described by the authors.