Research funding for rare diseases in Germany

Abstract

There is high need for more research in the field of rare diseases. Not only must the causes and mechanisms of the numerous and often heterogeneous diseases be delineated, but criteria must also be defined for optimal stratification of patients for individualized therapies. In this context, research and innovative diagnostics are linked together more closely than in other fields of medicine. The early stages of disease-oriented research can be performed in individual institutions but, due to low numbers of patients, late translation and transfer into clinics requires multicentric and international collaboration. In Germany research on rare diseases takes place mostly in faculties of medicine at universities. Since the institutional financial support is very low, research grants have substantial significance. The German Research Foundation (DFG) and the Federal Ministry of Education and Research (BMBF) are the main grant agencies for national projects, but foundations and patient advocacy groups also finance research to acertain extent. The ERA-Net "E-Rare" and the programs of the EU target primarily international cross-border projects and patient trials. All of these programs need to be adapted more efficiently to the particular needs of rare disease research. For national and international research projects on rare diseases, sufficient funds are needed but also sustainable interdisciplinary platforms and centers must be established in order to share expert knowledge and to implement complex programs such as proof-of-concept studies in humans.