23 January 2017 - The success of the regulation on orphan medicinal products in the European Union is evidenced by the 127 orphan drugs that have had market authorisation since 2000. 

However the incentives aimed to stimulate research and development have had the unintended consequence of increasing drug cost, resulting in many orphan drugs not being cost-effective. Orphan drugs command an increasing share of the pharmaceutical market and account for a disproportionate amount of healthcare expenditure. Orphan drug ownership by socially motivated, not-for-profit organisations may facilitate access to more affordable orphan drugs, for the benefit of patients and health care systems alike. 

Using repurposed drugs as a paradigm, this review navigates the regulatory hurdles, describes the legal context and identifies funding opportunities, in a bid to facilitate and encourage not-for-profit organisations to lead on the development of affordable orphan drugs.

Read British Journal of Clinical Pharmacology article