Childhood cancer drugs in China: An overview and comparison of regulatory approvals in China and the United States.

Different from less developed countries, 80% of children with cancers in the United States are cured. Traditional chemotherapy drugs are the mainstay of therapies; new targeted medications have become available recently. Using publicly available data, we created a database of cancer drugs with paediatric malignancy indications approved by 31 October 2020 in China and the United States. We compared numbers, type, indications and listing on the World Health Organization Model List of Essential Medicines for Children (WHO EMLc) between the two countries, assessed the correlation between paediatric indications and cancer incidences, and described evidence supporting approvals of targeted medications in the two settings. Our study showed that by 31 October 2020, 31 and 39 cancer drugs available in China and the United States were approved for use in children, corresponding to 137 and 102 paediatric cancer indications, respectively. About half of these drugs (17 in China and 18 in the United States) were listed on the WHO EMLc. The correlation between indications and burden of disease was higher in the United States (r = 0.68) than China (r = 0.59). More traditional chemotherapy drugs were approved in China (n = 27) than the United States (n = 19). Of 20 targeted childhood anticancer medicines approved in the United States, mainly on the basis of single arm trials (27/32 indications, 84.4%), only four were approved for paediatric indications in China, at a median of 2.8 years after US Food and Drug Administration approval. A harmonised, evidence‐based regulatory framework is needed to ensure approvals of needed, safe and efficacious childhood cancer drugs across the world. What's new? This comprehensive review of the childhood cancer drugs and their paediatric indications approved in China and the United States suggests that key therapeutics for the treatment of children with cancers are approved in China. But more traditional chemotherapy drugs are approved in China than in the United States, and only a few of the targeted childhood anticancer medications approved in the United States, mainly on the basis of single‐arm clinical trials, are also approved in China. A harmonised, evidence‐based regulatory framework is needed to ensure the development and approval of needed, safe, and efficacious childhood cancer drugs across the world. [ABSTRACT FROM AUTHOR]