标题： 快速通道注册过程中的自适应设计 显示英文标题

标题： Adaptive Designs in Fast-Track Registration Processes

摘要： 快速通道程序在有条件注册健康产品的情境中起着重要作用，例如创新产品的有条件批准流程和数字健康应用的上市流程。快速通道程序为患者提供了更早获得创新产品的机会。它们涉及两个注册步骤。申请人可以首先申请有条件注册。成功的有条件注册提供有限的资金或批准期限，并有时间准备永久注册的申请，这是注册过程的第二步。对于有条件注册，通常产品只需满足永久注册所需要求的一部分。人们对适用于快速通道程序的有效且高效的研究设计感兴趣。本文将解决这一问题。本文的一个激励性例子是德国数字健康应用（DiGA）通过法定健康保险报销的快速通道注册流程。本文的主要重点是在快速通道注册流程背景下系统地研究自适应设计的效用。本文还详细讨论了指南中发现的不同要求及其后果。我们证明，在像DiGA注册流程这样的快速通道流程中使用自适应设计，通常比目前的两项独立研究的标准要高效得多。本文 presented 的结果基于数值计算，并由数学论证支持。 显示英文摘要

摘要： Fast-track procedures play an important role in the context of conditional registration of health products, such as conditional approval processes and listing processes for digital health applications. Fast-track procedures offer the potential for earlier patient access to innovative products. They involve two registration steps. The applicants can apply first for conditional registration. A successful conditional registration provides a limited funding or approval period and time to prepare the application for permanent registration, which is the second step of the registration process. For conditional registration, typically, products have to fulfil only a part of the requirements necessary for permanent registration. There is interest in valid and efficient study designs for fast-track procedures. This will be addressed in this paper. A motivating example of the paper is the German fast-track registration process of digital health applications (DiGA) for reimbursement by statutory health insurances. The main focus of the paper is the systematic investigation of the utility of adaptive designs in the context of fast-track registration processes. The paper also covers a careful discussion of the different requirements found in the guidelines and their consequences. We demonstrate that the use of adaptive designs in the context of fast-track processes like the DiGA registration process is, in most cases, much more efficient than the current standard of two separate studies. The results presented in this paper are based on numerical calculations supported by mathematical arguments.