Orphan drugs in EU: Early generation of evidence urgently needed

A team of German Institute for Quality and Efficiency in Health Care (IQWiG) authors, together with Rita Banzi from the Italian Mario Negri Institute, analyzed the effects of the lack of evidence on orphan drugs at the European and national level. The conclusion is that the need for reform is great.

Since the introduction of the European Union (EU) Regulation on drugs for rare diseases (orphan drugs) in the year 2000, the number of newly approved orphan drugs has markedly increased. While 63 orphan drugs were approved in the first decade after the EU Regulation, the number rose to 133 in the second decade. In 2022 alone, 22 orphan drugs were approved.

The orphan drug status granted by the European Medicines Agency (EMA) implies a superior therapeutic benefit (called “significant benefit”) over existing treatment options. This has knock-on effects on national policies: In Germany, for example, a superior therapeutic benefit (called “added benefit”) is automatically assumed for orphan drugs at market entry regardless of the actual evidence available, meaning that this benefit is fictitious