Speaker
Description
Fast-track procedures play an important role in the registration of health products, such as registration processes for digital health applications. These procedures offer the potential for patients to access innovative products earlier. The procedures involve two registration steps. Applicants can first apply for conditional registration. A successful conditional registration provides a limited funding or approval period and time to prepare the application for permanent registration. Products typically only have to fulfil weaker requirements for conditional registration than for permanent registration. The motivating example of the talk is the German two-stage fast-track registration process for digital health applications (DiGA) for reimbursement by statutory health insurances. This procedure has a pioneering role in other countries such as France, Belgium, Austria, Korea, and the UK, where similar procedures exist or are planned to be implemented (see also Chapman, 2025).
The talk addresses valid and efficient study designs for fast-track procedures. The current standard is to conduct two separate studies. Instead, we suggest using two-stage adaptive designs that permit to use the data from both stages for the application for permanent registration. They also allow to learn from the first-stage data (which we suggest to use for the application for conditional registration) by performing design modifications after the first stage while controlling the Type I error rate. We consider designs where the second-stage sample size is recalculated such that a specific conditional power is reached to ensure that the second stage will likely be successful. We also assume that a sufficient overall success probability for achieving permanent registration is targeted. Having sufficient overall and conditional power requires a minimum and maximum second-stage sample size, respectively. By investigating these parameters and the expected sample sizes, we will demonstrate that in most cases, adaptive designs bring a clear advantage over the current standard of two separate studies. A discussion of the registration requirements and their consequences will also be addressed. The results presented are based on numerical calculations supported by mathematical arguments. The talk will be based on our arXiv paper (Kluge & Brannath, 2025).
References:
Suzannah Chapman. Towards identifying good practices in the assessment of digital medical devices. OECD Health Working Papers, 2025.
Liane Kluge and Werner Brannath. Adaptive Designs in Fast-Track Registration Processes for Digital Health Applications. arXiv preprint arXiv:2507.04092v3, 2025.
53573509947