Taguchi Ayumi, Matsumaru Naoki, Tsukamoto Katsura
Global Regulatory Science, Gifu Pharmaceutical University, 1-25-4 Daigakunishi, Gifu, 501-1196, Japan.
Corporate Strategy & Business Portfolio, Daiichi Sankyo Co., Ltd., 3-5-1 Nihonbashi-Honcho, Chuo-ku, Tokyo, 103-8426, Japan.
Invest New Drugs. 2025 Jun 4. doi: 10.1007/s10637-025-01549-0.
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.
全球都在寻求能比现有治疗方法更有效且有前景的新药的早期使用机会。为促成这种机会，各国监管机构已实施加速药物审批的特殊监管措施。本研究考察了美国食品药品监督管理局（FDA）给予加速批准（AA）的抗癌药物的审批延迟情况及其在日本相应的审批时间线。此外，我们评估了日本加速监管途径的使用情况。我们的分析纳入了2012年至2021年间从FDA获得AA的55种抗癌药物，并评估了它们截至2022年在日本的审批情况。中位审批延迟为649天，主要归因于提交延迟。日本参与FDA关键研究的药物以及日本加速监管途径的使用使审批延迟显著缩短（分别为P < 0.001和P = 0.0172）。然而，最近实施的有条件早期批准制度的利用率有限。尽管AA抗癌药物通常基于早期临床试验数据获批，但我们的研究结果表明，参与关键研究对于最大限度减少日本的审批延迟至关重要。尽管药物审批时间有所改善，但仍存在大幅延迟，这凸显了更有效利用加速监管途径的必要性。加强早期临床试验中的合作并优化监管流程可能有助于在日本更快地获得高收益抗癌疗法。
