Abstract
OBJECTIVE: The affordability of rare disease drugs has become a social issue that cannot be ignored. This study aims to evaluate the current price and affordability of rare disease drugs in China, with evidence from Shandong province. METHODS: Data on prices and affordability of 50 drugs for 22 rare diseases were collected from secondary and tertiary public hospitals in Shandong Province, using an adaptation of the World Health Organization/Health Action International (WHO/HAI) methodology. Prices were measured as Median Price Ratios (MPRs). Affordability was measured as days of daily per capita disposable income required for the cost of one month's treatment. RESULTS: Out of the 50 rare disease drugs, 11 drugs had MSH reference prices and 34 had PBS reference prices. Median prices of 11 drugs were higher than MSH reference prices (median 1.33), and median prices of 34 drugs were higher that Australian PBS prices (median 1.97). Thirty-six (72.00%) and forty-four (88.00%) drugs were unaffordable for urban and rural residents, respectively. Thirty-four (68.00%) and thirty-eight (76.00%) drugs were unaffordable for urban and rural residents even after reimbursement by the health insurance schemes of China, respectively. CONCLUSIONS: The affordability of some rare disease drugs remained poor with their relatively high prices in Shandong Province. Sustainable mechanisms are needed to reduce the price of rare disease drugs and to improve the affordability of rare disease patients.