Analysis of differences in orphan drug indications and pediatric use between domestic and international approvals
Objective To summarize the differences in the approved indications and pediatric use of orphan drugs between the US Food and Drug Administration(FDA)/European Medicines Agency(EMA)and China's National Medical Products Administration(NMPA),providing reference for drug treatment,clinical trials and off-label drug use management of rare diseases.Methods Orphan drugs approved by the FDA or EMA and granted marketing authorization in China were analyzed to compare differences in indications and pediatric use.The anatomical,therapeutic,and chemical(ATC)classifications of drugs approved abroad but not in China were also reviewed.Results As of December 31,2023,319 and 123 orphan drugs approved by the FDA and EMA,respectively,had marketing authorizations in China,of which 134 and 69 were approved for the treatment of diseases listed in the first and second batches of China's Rare Disease Catalog.There were 117 orphan drugs of FDA and 38 orphan drugs of EMA that were not approved in domestic,of which 47 and 13 were related to the treatment of diseases in the Rare Disease Catalog,respectively.Additionally,22 drugs approved by both the FDA and EMA had unapproved pediatric use in China,with 12 and 14 targeting diseases in the Rare Disease Catalog.Compared to the FDA and EMA,China exhibited a higher proportion of unapproved indications for drugs used in non-Rare Disease Catalog conditions and unapproved pediatric use for drugs in the Rare Disease Catalog.Anti-cancer and immunomodulatory drugs were the most frequently proportion of orphan drugs of approved and unapproved categories in terms of indications and pediatric use.Conclusions:Compared with the FDA and EMA,there is a certain lag in the approval of orphan drug indications and pediatric use in China.The publication of the Rare Disease Catalog has played a role in promoting the approval of drug indications,but further attention should be paid to the phenomenon of off-label use of rare disease drugs for children.
NETL
NSTL
万方数据
