OBJECTIVES: Orphan medicinal product legislation was adopted in Europe in 2000 with the aim of promoting the development of treatments for rare diseases. This has resulted in 515 EU designations for orphan medicinal products in June 2008. Many orphan drugs (ODs) receive market authorisation, however there are problems regarding access and reimbursement. This study compares OD policies and examines the reimbursement of ODs across Europe. METHODS: Policies on ODs in European countries for which sufficient detail had been published were examined. HTA websites were searched for assessments on ODs and data extracted on the recommendations. RESULTS: In some countries, including Sweden and the Netherlands, ODs may be approved for reimbursement with minimal documentation of cost-effectiveness. However, many countries, including England and Scotland do not have specific polices for the appraisal and reimbursement of ODs. This results in many patients across Europe who do not see any benefit from the orphan product legislation. By April 2008, 45 drugs with designated OD status had been given European marketing authorisation in 42 different indications. Thirty-seven submissions had been assessed by the SMC by June 2008 according to the standard appraisal procedures, with 4 accepted for use, 13 accepted for restricted use and 20 not recommended in NHS Scotland (11%, 35% and 54%, respectively). This contrasts with previously published data on recommendations from the SMC; 30% approved, 35% restricted, 36% not recommended (Karia et al 2008). Furthermore, by June 2008, NICE had only assessed 2 OD indications, with 4 ODs currently undergoing assessment and 3 proposed as topics for assessment. CONCLUSIONS: The granting of market authorisation by the EMEA does not always lead to reimbursement of a drug; patient access to ODs varies across different European countries.