QualityMetric’s rare disease experts provide knowledgeable insight into conditions that are often poorly characterized and researched. We help you document a disease’s etiology, natural history, progression, symptoms and impact.
Rare diseases tend to put patients, providers and caregivers in difficult positions, due to their little-known natural histories and the scarcity of approved treatment options. Patients typically face a life-long treatment journey, plagued by high costs and an endless consumption of medical resources. QualityMetric experts can help identify unmet patient needs through the use of our proprietary databases, analyses of existing data and literature, and by conducting prospective data collection studies.
For pharma companies, study design and replication potential are often restricted by a variety of hurdles – from small patient populations to a lack of validated measures and tools, ethical considerations, and the tendency for rare diseases to be extremely aggressive and life-threatening.
Having spent decades in the rare disease space, we equip you with the tools and tactics to navigate the difficulties of rare disease research. Our research experts have helped advance knowledge and conducted studies in a wide array of rare diseases, such as AL amyloidosis, TTR amyloidosis, Duchenne muscular dystrophy, hemophilia, and X-linked hypophosphatemia. Using retrospective patient-reported data, we have also performed exploratory analysis to better understand and communicate the humanistic burden of rare diseases.