Orphan medicines: Impact of draft pharma legislation proposals

Plans to overhaul the European Union (EU) regulation on rare disease therapies risk undermining two decades of progress, a new report has found.

The assessment, commissioned by the European Federation of Pharmaceutical Industries and Associations (EFPIA), was conducted by Dolon, a strategic consultancy specialising in rare diseases.

The European Orphan Medicinal Product Regulation was introduced in 2000 to incentivise the development of new medicines for people living with rare disease. It worked: the number of EU-approved medicines for rare diseases was in single digits two decades ago. To date, over 205 new treatments for orphan diseases have been approved.