ABSTRACT
The European Orphan Medicines Regulation, which came into effect in 2000, stimulates the development of medicines for rare diseases. The number of registered orphan medicines has increased from eight in 2000 to 169 in 2019; 38% of these are available in the Netherlands. The costs for orphan medicines in the Netherlands are increasing by 9% every year, and now amount to 272 million annually. There is, however, still no effective medication for thousands of rare diseases. The Orphan Regulation was intended for new orphan medicines, but some companies also use it for 'repurposing' of old agents, for which they also get 10 years exclusive market rights in the EU. The improper use of legislation has led to concern among patients, doctors, and medical insurance companies. The Netherlands Council for Public Health and Society and MPs in the lower house of the Netherlands parliament have proposed measures for tackling the high prices of medicines and Mr. Bruins, the former Minister of Health, wanted to propose legislative change in the EU. The EU 'pharmaceutical incentive review' offers the possibility to revise the current rules.
