Current Situation and Prospect of Pharmacoeconomic Evaluation of Hemophilia in China / 罕见病研究

ABSTRACT

In 2018, hemophilia, one of the first ones, entered into the catalogue of Rare Disease in China. However, drug accessibility and affordability have been the challenges for hemophilia patients. Therefore, a comprehensive evaluation of the cost and efficacy of hemophilia drugs is essential to push forward the appropriate application of hemophilia drugs. In the article, we have teased out the existing process of the review and approval of hemophilia drugs; have examined the procedures for the drug entry into the medical insurance policy; have systematically searched the pharmacoeconomic evaluation studies of hemophilia in published literature in both China and overseas; and have comprehensively analyzed the quality, the content of evaluation, and challenges of pharmacoeconomic studies of hemophilia in China. At last, we discuss the prospect for the future based on the following three aspects: developing a special model of hemophilia, obtaining the health utility from various sources, and establishing the threshold of willingness to pay for rare diseases, hoping to provide reference and examples to the research into pharmacoeconomic evaluation of hemophilia in China.