Approval of high-benefit oncology drugs in Japan: utilization of expedited regulatory pathways for the accelerated approved anticancer drugs.

Abstract

Early access to promising new drugs with superior efficacy compared to existing treatments is globally sought after. To facilitate such access, regulatory authorities in each country have implemented special regulatory measures that expedite drug approval. This study examined the approval lag of anticancer drugs granted accelerated approval (AA) by the U.S. Food and Drug Administration (FDA) and their corresponding approval timelines in Japan. Additionally, we assessed the use of expedited regulatory pathways in Japan. Our analysis included 55 anticancer drugs that received AA from the FDA between 2012 and 2021 and evaluated their approval in Japan through 2022. The median approval lag was 649 days, primarily attributable to submission delays. Drugs for which Japan participated in the pivotal FDA study and the use of expedited regulatory pathways in Japan had significantly shorter approval lags (P < 0.001 and P = 0.0172, respectively). However, the utilization of the recently implemented conditional early approval system was limited. Although AA anticancer drugs are often approved based on early clinical trial data, our findings highlight that participation in pivotal studies is crucial for minimizing approval delays in Japan. Despite improvements in drug approval timelines, substantial delays persist, underscoring the need for more effective utilization of expedited regulatory pathways. Enhancing collaboration in early-stage clinical trials and optimizing regulatory processes may facilitate faster access to high-benefit anticancer therapies in Japan.