Repositioning for Rare Diseases; Too Much, Too Little or Just Right?

Drug repositioning refers to the idea of expanding the use of an already authorised drug, for the treatment of new patient categories with the same diseases as well as the treatment of new diseases. Repositioning is considered an important innovation modality to increase treatments for patients with rare diseases. Yet, two recent EU studies have raised concerns that developers who reposition drugs are financially overcompensated to the detriment of national healthcare systems and patients and, therefore, suggest limiting the benefits provided by the orphan drug regulation. In this study, we show that the number of drugs repositioned for rare diseases (22%) is more modest than predicated and argue this indicates that developers may not be overcompensated, otherwise we would expect a much higher number. Furthermore, we argue that changing the incentives provided by the orphan drug regulation to address overcompensation is not the option if policymakers want to realise the benefits of repositioning. Instead, we suggest policymakers consider other legal and regulatory tools to address overcompensation issues, such as competition law and novel mechanisms emerging in Canada.