To analyze the approval for marketing, drug characteristics, and differences of novel hematological antineoplastic drugs between China and the United States, and provide reference for pharmaceutical management, drug research and development, drug review and approval in China.

The websites of the U.S. Food and Drug Administration (FDA) and the National Medical Products Administration of China and package insert of drug were searched, and the novel hematological antineoplastic drugs approved for marketing in the United States from 1997 to 2021 and China from 1999 to 2021 were collected. The drug names, approval types, approval dates, accelerated drug marketing registration procedures, drug action mechanism and indications were extracted, and the approval status and timeliness of new drugs, as well as the number of approvals for different types of drugs were analyzed.

From 1997 to 2021, a total of 69 novel hematological antineoplastic drugs approved in the United States, which included 42 new molecular entities and 27 new therapeutic biologics. Sixty-five drugs were approved through expedited development and review pathways. From 1999 to 2021, China approved a total of 36 novel hematological antineoplastic drugs, including 24, 11, and 1 for chemical drugs, biologics products, and traditional Chinese medicine, respectively. Twenty-five drugs were approved to use accelerated drug marketing registration procedures. The number of approved drugs in both countries has significantly increased from 2017 to 2021, with 26 (87%) out of 30 novel hematological antineoplastic drugs in the United States being the first global approval drugs, and 8 (32%) out of 25 novel hematological antineoplastic drugs in China being the first global approval drugs, and the average approval time for the other 17 drugs was 6 years later than that of the United States. The novel hematological antineoplastic drugs approved in China covered multiple drug targets, which were closer to the targets of new molecular entities approved in the United States and could be used to treat multiple types of hematology neoplasms. However, the biological targets were still limited compared with the United States, which mainly used for lymphoid tissue tumors. There were no drugs used for myeloid tumors, and the types were relatively single. From 2017 to 2021, among the 17 novel hematological antineoplastic drugs approved for marketing in China had been approved in the United States, only 6 drugs had the same indications as the United States, and one drug had more indications than the United States.

In recent years, the number of novel hematological antineoplastic drugs approved for marketing in China has significantly increased. Diverse accelerated drug marketing registration procedures has been used for the review and approval of novel hematological antineoplastic drugs. The target types of approved novel hematological antineoplastic drugs have increased, but the timeliness of new drug review and approval and the international influence of domestic drugs still need to be enhanced. The research and development of domestic novel hematological antineoplastic drugs should avoid single target aggregation, and pay attention to the exploration of new indications of drugs and new combined treatment schemes.