Challenges in Implementing Revocation of Oncology Drugs Under Conditional Early Approval in Japan: Regulatory Insights From Comparisons With FDA / EMA

ABSTRACT

Expedited pathways in the United States (U.S.), European Union (EU), and Japan (JP) enable earlier access to innovative therapies for rare and serious diseases. In 2025, Japan explicitly introduced revocation provisions to its Conditional Early Approval (CEA). Considering the challenges in a related prior scheme—conditional and time‐limited approval for regenerative medical products—particularly withdrawals prior to full approval, this study examined oncology cases under the U.S. Accelerated Approval (AA) and EU Conditional Marketing Authorization (CMA) pathways to identify operational challenges for revocation under Japan's CEA. We conducted a descriptive comparative analysis of oncology drugs granted AA or CMA between January 2015 and December 2024 using publicly available data. Post‐authorization outcomes and JP approval/development status were summarized. AA encompassed 97 products (138 indications), CMA included 27 products (30 indications), and CEA covered 4 products (4 indications). Approximately half of AA/CMA indications were approved in Japan, while some remained undeveloped. The mean time from AA to full approval was 2.7 years and to withdrawal was 3.6 years. Overall, about 15% of AA indications were withdrawn, primarily due to failed confirmatory trials; 8 products/10 indications underwent Oncologic Drugs Advisory Committee deliberations prior to withdrawal or continuation. With recent policy changes aligning CEA eligibility with AA/CMA, broader implementation of CEA is anticipated. For revocation operations, three priorities emerge: (1) timely confirmatory trials and alternative approaches to reduce uncertainty; (2) transparent public evaluation and decision‐making; and (3) flexible patient‐access systems following withdrawal. Continued discussion is required to support effective use of CEA.