As we enter year two of the current Rare Diseases Clinical Research Network, a number of important accomplishments should be emphasized. First, the inaugural issue of Spotlight on Rare Diseases under David Robertson's editorship is an opportunity to highlight our individual and collective progress, underscore the importance of rare diseases research, and emphasize the collaborative efforts of this Network of widely diverse disorders. This is our chance to show our federal sponsors, patient advocacy groups, and the American public that orphan diseases are being taken seriously and that our ultimate goal is engagement in clinical trials to provide safe and effective interventions. While we work in our respective silos, the Network is a critical key to promoting the notion that the sum of our efforts exceeds that of the individual consortia.
Second, at the outset, we committed to establishing committees to address issues and opportunities confronting each consortium based on the experiences of the so-called legacy consortia from the first funding cycle. These committees are the Strategic Planning Committee (Peter Merkel, Chair), the Registry Committee (Rachel Richesson, Chair), the Training Committee (Robert Steiner, Chair), and the Operations Committee (Michael Knowles, Chair).
Third, the September in-person Steering Committee meeting was preceded by the second Conference on Clinical Research for Rare Diseases. This was a terrific opportunity for trainees to present their work and to learn from the impressive faculty and for Network investigators to understand the activities of each consortium.
Alan Percy, MD
Professor, University of Alabama at Birmingham
PI of Angelman, Rett and Prader-Willi Syndromes Consortium
Chair, RDCRN Steering Committee