Characteristics of Drugs from Non-Global Companies for Hematologic Malignancies and Impact on Global Regulatory Approval.

The number of drugs developed by non-global companies, including biotech start-ups, has increased; however, their characteristics and impact on global regulatory approval are not well understood. Using a public database, we identified new molecular entities (NMEs) approved for hematologic malignancies in the US from January 2011 to December 2022. They were divided into those submitted by non-global companies (non-global group) and those by global companies (global group). We identified 48 NMEs, of which 19 (40%) were classified as non-global. Of these, 13 (68%) were from US-based companies. In the non-global group, 63% (12/19) of the NMEs had received accelerated approval in the US, of which only 50% (6/12) had a post-approval confirmatory trial by September 2023. Regarding the impact on the approval in the European Union (EU) and Japan, the unapproval rate of 2 years after US approval was higher in the non-global group than in the global group in the EU (56% vs. 21%) and Japan (94% vs. 64%). In conclusion, many NMEs from non-global companies had received accelerated approval in the US based on phase I/II trials. NMEs from non-global companies had a higher unapproval rate at 2 years in both the EU and Japan.