How Global Regulators Handle Cancer Drugs After FDA ‘First’ Approval: Study

A new analysis published in the journal Frontiers in Pharmacology found substantial differences in how major international regulators evaluate and approve oncology drugs that first receive expedited approval from FDA, despite often reviewing largely the same clinical evidence.

Researchers from Ewha Womans University in South Korea and Keio University in Japan examined 36 oncology drug .indications that received FDA accelerated or other expedited approvals between 2019 and 2023 and compared subsequent regulatory decisions by the European Medicines Agency (EMA), Australia's Therapeutic Goods Administration (TGA), and Japan's Pharmaceuticals and Medical Devices Agency (PMDA).

The study found that Australia's TGA most closely aligned with FDA decisions, maintaining expedited review pathways in 72% of cases and showing the highest degree of concordance in its interpretation of pivotal clinical trial data. The EMA used expedited pathways at a similar rate, but frequently relied on more mature datasets and broader patient populations than those reviewed by the FDA. In contrast, Japan's PMDA relied primarily on standard approval pathways, using expedited mechanisms in only one of 15 cases examined.

Across all agencies, regulators generally agreed on primary efficacy endpoints and often relied on the same pivotal clinical trials that supported FDA approvals. However, differences emerged in the selection of patient populations, data cut-off dates, and approval pathways, leading to divergent regulatory outcomes.

The authors concluded that greater international harmonization of regulatory standards and evidentiary requirements could improve consistency in oncology drug approvals and potentially accelerate patient access to innovative cancer therapies worldwide.