Availability of medicines for rare diseases in EU Countries

The European Medicines Agency has expressed 50 positive opinions recommending the granting of a marketing authorisation for an orphan medicinal product since the Regulation on orphan medicinal products (OMPs) entered into force in 2000. However, OMPs authorised at EU level are not always available at Member States (MS) level. We developed and distributed a questionnaire to collect information on the availability of 20 OMPs authorised before October 2006. The questionnaire included questions on the date of national market availability; the possibility of pre-marketing access programme; the distribution channel, the availability of a reimbursement policy. Twelve MS provided information: Austria, Belgium, Czech Republic, Denmark, Estonia, Finland, Hungary, Ireland, Italy, Latvia, Slovakia and UK. Results demonstrate that the availability of OMPs varies greatly among the 12 MS considered and market availability delays are highly variable. OMPs are often expensive drugs and the different MS reimbursement policies are hindering access to OMPs. Data show that since 2000 the number of OMPs has increased however issues including costs and reimbursement policies at MS level represent major barriers to real OMPs accessibility. This is a critical situation that deserves attention because of the evident inequalities that do exist with regards to OMPs accessibility among MS.