Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses the importance of rare disease registries.
One of the fundamental challenges of rare diseases is the scarcity of data. While traditional medical research utilizes large sample sizes and datasets to observe disease patterns and efficacy of treatment, rare diseases are not afforded the same luxury. Registries address this challenge by acting as an aggregated global platform for rare disease data.
By consolidating information from patients, caregivers, healthcare providers, and researchers, registries offer a comprehensive understanding of disease prevalence and clinical outcomes. This is essential in guiding research efforts and informing clinical decision making. Registries also foster collaboration through exchange of insights, best practices, and research findings, accelerating discovery and innovation. They can also help patients feel empowered and assist in finding community.
Dr. Hamed also notes the difference between clinical data derived from controlled studies and real world data where observations take place under the many factors of everyday life. Both datasets are of great importance and interest when it comes to developing new treatment options for rare disease patients.
For more information on Sanofi and their work, visit https://www.sanofi.com/en
For more information on rare diseases, visit https://checkrare.com/