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

Abstract

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.