Rare disease patients, advocacy groups, philanthropists, and researchers have overcome significant challenges in their efforts to improve treatment and care. Over the past decade, clinicians have made remarkable progress by adopting innovative techniques, including holistic patient assessments, using genomics and other data-driven approaches, and targeting the root causes of these conditions rather than just symptoms. These methods, though initially applied to rare diseases, could also enhance diagnosis and reduce costs for common conditions. The rare disease sector exemplifies effective cross-stakeholder collaboration, potentially offering a model for transforming medical research and creating a more efficient R&D process for all diseases.