4/11/2023 We developed a unique strategy for advancing biomedical research that prioritizes high-impact, patient-centered studies by crowdsourcing research ideas from the entire community of stakeholders, including patients, loved ones, physicians, and researchers.
Through our approach, we successfully created a patient-centered research agenda for Castleman disease and generated a list of best practices that can be used as a model for other rare diseases. We hope that by sharing our insights and approach, other rare disease organizations will pursue a patient-centric approach and prioritize high-impact research that truly addresses the needs of patients.
There are approximately 10,000 rare diseases that affect around 30,000,000 individuals in the U.S.A., most of which do not have an FDA-approved treatment. This fact highlights the failure of traditional research approaches to overcome the unique challenges of developing rare disease treatments. The Castleman Disease Collaborative Network was founded in 2012 to advance research and treatments for Castleman disease, a rare and deadly disease that involves the immune system attacking the body’s vital organs for an unknown cause. It has spearheaded a novel strategy for advancing biomedical research, the Collaborative Network Approach. This approach consists of eight steps, one of which is to identify and prioritize high-impact research questions through crowdsourcing ideas from the entire community of stakeholders: patients, loved ones, physicians, and researchers. Rather than hoping that the right researcher will apply for the right research project at the right time, crowdsourcing high-priority research projects into a research strategy ensures that the most high-impact, patient-centered studies are prioritized. The Castleman Disease Collaborative Network launched an initiative in 2021 to systematically generate this list of community-directed studies to focus Castleman disease research efforts.