Sanofi, Cambridge, MA, USA.
AstraZeneca, Cambridge, MA, USA.
Ther Innov Regul Sci. 2024 Sep;58(5):978-986. doi: 10.1007/s43441-024-00676-9. Epub 2024 Jun 22.
Seamless study designs have the potential to accelerate clinical development. The use of innovative seamless designs has been increasing in the oncology area; however, while the concept of seamless designs becomes more popular and accepted, many challenges remain in both the design and conduct of these trials. This may be especially true when seamless designs are used in late phase development supporting regulatory decision-making. The Innovative Design Scientific Working Group (IDSWG) Oncology team conducted a survey to understand the current use of seamless study designs for registration purposes in oncology clinical development. The survey was designed to provide insights into the benefits and to identify the roadblocks. A total of 16 questions were included in the survey that was distributed using the ASA Biopharmaceutical Section and IDSWG email listings from August to September 2022. A total of 51 responses were received, with 39 (76%) respondents indicating that their organizations had seamless oncology studies in planning or implementation for registration purposes. Detailed survey results are presented in the manuscript. Overall, while seamless designs offer advantages in terms of timeline reduction and cost saving, they also present challenges related to additional complexity and the need for efficient surrogate clinical endpoints in oncology drug development.
无缝研究设计有可能加速临床开发。创新的无缝设计在肿瘤学领域的应用正在增加;然而,尽管无缝设计的概念变得越来越流行和被接受,但在这些试验的设计和实施中仍然存在许多挑战。当无缝设计用于支持监管决策的后期开发时,情况可能尤其如此。创新设计科学工作组 (IDSWG) 肿瘤学团队进行了一项调查,以了解当前在肿瘤学临床开发中为注册目的使用无缝研究设计的情况。该调查旨在了解其优势,并确定障碍。调查共包含 16 个问题,于 2022 年 8 月至 9 月通过 ASA 生物制药分会和 IDSWG 电子邮件列表进行分发。共收到 51 份回复,其中 39 份(76%)回复者表示其组织有用于注册目的的肿瘤学无缝研究正在计划或实施中。详细的调查结果在本文中呈现。总体而言,尽管无缝设计在缩短时间和节省成本方面具有优势,但它们也存在与额外复杂性和在肿瘤药物开发中需要高效替代临床终点相关的挑战。