Innovative Clinical Trial Approach for Evaluating Digital Medical Devices Under European Fast‐Track Regulatory Frameworks

To address patient demand for rapid access to innovative digital medical devices (DMDs), several health technology assessment (HTA) authorities in European Union countries provide transitional or provisional access and reimbursement pathways. These pathways are available when only incomplete clinical trial data are accessible, and significant uncertainty remains regarding the clinical benefits, even after CE (European conformity) marking has been obtained. Once manufacturers complete the clinical studies, additional real-world data (RWD) may become available as a result of the device's use in the target population. Consequently, regulators can draw on both sources of information to support their final decision-making processes. For a statistically principled evaluation of such settings, we propose a statistical framework suitable for DMD evaluation under European HTA fast-track requirements, integrating both clinical trial data and RWD. The framework consists of three key steps: (1) an interim analysis of clinical trial data, which can support temporary regulatory authorization and enable the collection of RWD; (2) a final analysis of the clinical trial data; and (3) a meta-analysis combining the clinical trial data and RWD, contingent upon obtaining temporary authorization. To optimize the timing of the interim analysis and the application for temporary authorization, we introduce several metrics. The proposed framework was assessed by means of an extensive simulation study. This framework should be complemented by a post-market evaluation of the DMD once it has been widely adopted, aligning with the principles of phase IV studies.