LAUSR

Different from less developed countries, 80% of children with cancers in the US 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 October 31, 2020 in China and the US. 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 October 31, 2020, 31 and 39 cancer drugs available in China and the US 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 US) were listed on the WHO EMLc. The correlation between indications and burden of disease was higher in the US (r=0.68) than China (r=0.59). More traditional chemotherapy drugs were approved in China (n=27) than the US (n=19). Of 20 targeted childhood anticancer medicines approved in the US, mainly on the basis of single arm trials (27/32 indications, 84.4%), only 4 were approved for paediatric indications in China, at a median of 2.8 years after FDA approval. A harmonized, evidence-based regulatory framework is needed to ensure approvals of needed, safe, and efficacious childhood cancer drugs across the world.