U.S. Food and Drug Administration, Silver Spring, MD, USA.
Clin Trials. 2021 Dec;18(6):706-710. doi: 10.1177/17407745211050580. Epub 2021 Oct 16.
The Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research of the U.S. Food and Drug Administration have been leaders in advancing science to protect and promote public health by ensuring that safe and effective drugs and biological products are available to those who need them. Recently, new therapeutic discoveries, increased understanding of disease mechanisms, the need for innovation to optimally use resources, and global public health crises have led to an evolving drug development landscape. As a result, the U.S. Food and Drug Administration and medical product developers are faced with unique challenges and opportunities. The U.S. Food and Drug Administration is proactively meeting the challenges of this evolving landscape through various efforts, including the Complex Innovative Trial Design Pilot Meeting Program. Our focus, here, will be on the pilot meeting program.
The U.S. Food and Drug Administration has defined a process to facilitate the implementation of the Complex Innovative Trial Design Pilot Meeting Program. The process is transparent and outlines the steps and timeline for submission, review, and meetings.
Five submitted meeting requests have been selected for participation in the Complex Innovative Trial Design Pilot Meeting Program.
The pilot meeting program has been successful in further educating stakeholders on the potential uses of complex innovative designs in trials intended to provide substantial evidence of effectiveness. The selected submissions, thus far, have all utilized a Bayesian framework. The reasons for the use of Bayesian approaches may be due to the flexibility provided, the ability to incorporate multiple sources of evidence, and a desire to better understand the U.S. Food and Drug Administration perspective on such approaches. We are confident the pilot meeting program will have continued success and impact the collective goal of bringing safe and effective medical products to patients.
美国食品和药物管理局的药品评估和研究中心和生物制品评估和研究中心一直致力于推进科学，通过确保安全有效的药物和生物制品能够提供给需要的人，保护和促进公众健康。最近，新的治疗发现、对疾病机制的深入了解、创新以最佳利用资源的需求以及全球公共卫生危机，导致了药物开发格局的不断发展。因此，美国食品和药物管理局和医疗产品开发商面临着独特的挑战和机遇。美国食品和药物管理局正在通过各种努力积极应对这一不断变化的格局，包括复杂创新试验设计试点会议计划。我们在这里关注的是试点会议计划。
美国食品和药物管理局已经定义了一个流程，以促进复杂创新试验设计试点会议计划的实施。该过程是透明的，并概述了提交、审查和会议的步骤和时间表。
已经选择了五个提交的会议请求参加复杂创新试验设计试点会议计划。
试点会议计划成功地进一步教育了利益相关者关于复杂创新设计在旨在提供有效性实质性证据的试验中的潜在用途。迄今为止，选定的提交材料都使用了贝叶斯框架。使用贝叶斯方法的原因可能是由于其提供的灵活性、能够结合多种来源的证据以及更深入了解美国食品和药物管理局对这些方法的看法的愿望。我们有信心试点会议计划将继续取得成功，并影响为患者带来安全有效的医疗产品的集体目标。
