Analysis of treatment drugs for 121 rare diseases in China and their medical coverage status
OBJECTIVE To identify domestic and foreign therapeutic drugs for 121 rare diseases and explore the current sta-tus of medical coverage of these drugs in China.METHODS The list of therapeutic drugs for 121 rare diseases was established through the databases of National Medical Products Administration of China,US Food and Drug Administration,Orphanet of European Commission,package inserts and the relevant literatures.Drug prices and medical insurance coverage data were retrieved through the Yaozhi database and the official website of National Healthcare Security Administration of China.RESULTS As of October 2022,China had marketed 194 drugs with a total of 219 dosage forms for treating 109 rare diseases.However,12 rare diseases lacked therapeutic drugs in China and 93 foreign therapeutic drugs for 55 rare diseases have not been marketed domestically.Bidding price information was available for 205 dosage forms of therapeutic drugs with an average unit packaging price of 1363.58 RMB.Among them,132 drugs(64.39%)had an average unit package price under 50 RMB and 165 drugs(80.49%)were covered by National Basic Medical Insurance Catalog(NBMIC).The coverage proportion of drugs in NBMIC with an average unit packaging price under 50 RMB was 89.39%.And the coverage proportion of drugs in NBMIC with an average unit packaging price of above 200 RMB was 54.72%.CONCLUSION Most therapeutic drugs of 121 rare diseases in China are currently covered by NBMIC.However,the capacity for sustained supply of drugs for rare diseases remains quite frag-ile.Therefore it is imperative to establish a categorical guarantee system for supply,utilization and insurance payment according to the characteristics of these drugs for rare diseases in China.
rare diseasestherapeutic drugsmarketing statusNational Basic Medical Insurance Cataloguecoverage proportion