February 13, 2015
In an article published in Health Policy and Technology, Bellgard and colleagues outline ten components necessary in translational research for rare diseases. According to the authors, attention to these ten components provides a comprehensive approach to further the field of translational research in rare disease. These ten components include client-practitioner partnerships, disease registries, biobanks, -omics platforms, community-based and population wide studies, bioinformatics and high performance computing, interactions with pharma, personalised treatments, eHealth, and regulatory frameworks. The authors provide an overview of these components of the “rare disease research roadmap”, the challenges faced while navigating the components as well as the “way forward”.