OBJECTIVES: To investigate whether rare disease patient perspectives align with quantitative reimbursement and healthcare expenditure indicators across the EU5 countries (UK, Spain, Italy, Germany and France).

METHODS: Patient-reported indicators of rare disease treatment access were classified as “patient-reported affordability” or “patient-reported wait for reimbursement” (2000–2014 European Organisation for Rare Diseases [EURORDIS] Access Campaign online survey), and compared to economic/reimbursement measures (World Bank [WB], 2000–2014 and the Office of Health Economics [OHE], 2000–2017). Economic/reimbursement measures included gross domestic product (GDP) per capita [WB], total health expenditure (THE)/GDP [WB], private health expenditure (PHE)/THE [WB], time to reimbursement [OHE] and number of orphan medicinal products (OMPs) reimbursed following EU marketing authorisation [OHE]. OHE UK values presented are the mean of individual values for England, Scotland and Wales.

RESULTS: GDP per capita ranged from US$30,365 (Spain) to US$41,008 (Germany) (constant-price 2010). France (10.9%) and Germany (10.7%) had the highest THE/GDP, with 8.1–8.9% for UK, Italy and Spain. PHE/THE ranged from 18.5% (UK) to 27.4% (Spain). Germany reimburses treatments immediately; remaining reimbursement times were 19 (Italy), 20 (France), 23 (Spain) and 27 months (UK). However, Germany and France had the most issues with patient-reported wait for reimbursement (8.1%). Of the 143 OMPs with marketing authorisation, the highest reimbursement rates were observed in Germany (133) and France (116), with lower rates seen in the UK (57), Spain (75) and Italy (84). Despite these differences, both Germany and UK had the fewest patient-reported affordability issues (12.8%), whilst Italy reported the most (25.3%).

CONCLUSIONS: Across the EU5, healthcare expenditure and reimbursement measures did not fully explain differences in rare disease patient-reported difficulties. Research into healthcare systems, patient engagement and cultural differences may identify why countries with more favourable economic/reimbursement indicators did not have consistently fewer patient-reported treatment access concerns.