Research performance of centers of expertise for rare diseases—The influence of network integration, internal resource access and operational experience

Abstract

ObjectivesCenters of expertise (CoE) are vital to the care and treatment of patients with rare diseases and play a central role in generating medical and scientific knowledge. This study explores the relationship between a CoEs internal resource availability, operational experience and functional network integration and its research performance. MethodHypotheses were tested based on a sample of 64 CoEs in Europe and North America that specialize in 6 rare diseases. We collected questionnaire data related to strategic and structural antecedents and subsequently examined bibliometric data in the ISI Web of Science database to determine (1) research output by publication counts and (2) research impact by the number of citations. ResultsA CoEs research performance is greatly determined by available experiences and internal resources as well as by the nature of networks. While research output increases with the number of treated patients, the amount of resources and access to R&D networks, the impact of the identified publications is not related to a CoEs availability of internal resources or to the size of its R&D network. Nevertheless, a CoEs focus on care-oriented networks increases its research impact. ConclusionUnder the guidance of different national efforts, this study aims to support a nationwide establishment of specialized CoEs for rare diseases. It emphasizes the relevance of CoEs for the generation of medical knowledge for rare diseases as well as their need for essential support through internal and external resources. The findings further show the necessity for CoEs not only to concentrate on R&D collaborations to generate completely new knowledge and opportunities but also to establish networks with operational care partners such as general practitioners, local therapists, rehabilitation centers or other hospitals.