Department of Clinical Medicine, Pharmaceutical Medicine, Graduate School of Pharmaceutical Sciences, Kitasato University, 5-9-1, Shirokane, Minato-Ku, Tokyo, Japan.
Daiichi Sankyo Co., Ltd., Tokyo, Japan.
Ther Innov Regul Sci. 2024 Jan;58(1):136-142. doi: 10.1007/s43441-023-00577-3. Epub 2023 Oct 3.
The accelerated approval (AA) program in the USA has succeeded in expediting the regulatory approval of new cancer drugs based on surrogate endpoint data. It is unclear whether the AA program promotes overall drug development, including verification of the clinical benefit, as the verification of drugs granted AA often takes long time. To determine the impact of the AA program on overall drug development, the time required for verification of clinical benefits was compared between anticancer drugs that initially received AA and those that received regular approval (RA). It was found that anticancer drugs that were approved under the AA program took longer time for verification, suggesting that the program may delay the start of a confirmatory study, and there may be room for speeding up the process. In addition, discordance was found in the pivotal study between the USA and the EU and the USA and Japan for obtaining the indication for which AA was granted in the USA and a delay in the start of the confirmatory study for the AA indication was considered to lead to a delay in approval in the EU and Japan. Early initiation of confirmatory studies for AA indications is recommended to reduce the time that patients receive drugs with unproven benefit in the USA, as well as to deliver innovative new drugs to patients earlier in the EU and Japan.
美国的加速审批(AA)计划成功地基于替代终点数据加快了新癌症药物的监管审批。尚不清楚 AA 计划是否促进了整体药物开发,包括对临床获益的验证,因为对获得 AA 的药物的验证往往需要很长时间。为了确定 AA 计划对整体药物开发的影响,比较了最初获得 AA 和常规批准(RA)的抗癌药物进行临床获益验证所需的时间。结果发现,根据 AA 计划获得批准的抗癌药物进行验证所需的时间更长,这表明该计划可能会延迟确证性研究的开始,并且可能有加快该过程的空间。此外,在美国、欧盟和日本之间,获得在美国获得 AA 批准的适应证的关键性研究存在差异,并且 AA 适应证的确证性研究的启动延迟被认为会导致在欧盟和日本的批准延迟。建议尽早启动 AA 适应证的确证性研究,以减少在美国接受未经证实获益的药物的患者的时间,并在欧盟和日本更早地为患者提供创新的新药。
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