Evidenzbasierte Arzneimittelversorgung bei Seltenen Erkrankungen: die Rolle der Digitalisierung

Knowledge generation in the field of drug development for people with rare diseases (RDs) faces particular difficulties. This paper will show what improvements are expected from increasing digitalisation from the perspective of three healthcare institutions: the Federal Institute for Drugs and Medical Devices, the Institute for Quality and Efficiency in Health Care and the Federal Joint Committee. First, the potential of digitalisation to increase the efficiency of clinical development and regulatory decision-making through earlier collaboration of all stakeholders is proposed. Subsequently, it is argued that digitalisation should be used to reduce barriers to the implementation of care-associated randomised controlled trials, including those based on registries. High-quality registry studies should not only be started after approval but during the approval process, so that the evidence necessary for therapy decisions is available promptly after approval. Finally, it is stated that improving the evidence base through qualitative improvement of the data sources and their linkages directly benefits patients. Usable evidence that can be generated over a longer period of time – also beyond approval – and contribute to decisions within healthcare system ensures effective drug provision. The institutions agree that high-quality indication registries should be developed as product-independent, standing infrastructures so that high-quality data can be accessed early in the development of medicines for RD.