Orphan drug legislation in the European Union has promoted the development of treatments for very rare diseases by bestowing 10 year marketing exclusivity. This market monopoly is one of the benefits that comes under the European Union’s Regulation EC No 141/2000 (see webtable 1) and has enabled manufacturers to charge what many consider “exorbitant” prices for orphan drugs1 (table 1⇓).

These necessary drugs, which are very costly, profoundly affect the wellbeing of patients who2 struggle to persuade national health systems, operating under significant constraints,3 to finance their treatment.4 5 Orphan drug costs already consume 5% of the Belgian national drug budget6 and are predicted soon to consume 6-8% of healthcare budgets of larger EU countries such as France5—an unsustainable position.

Current high pricing thus hinders access to treatment7 and contravenes the aim of the Orphan Regulation: “Patients suffering from rare conditions should be entitled to the same quality of treatment as other patients”.8