Abstract: Methodological Challenges in the Estimation of the Incidence Rate of Rare Diseases from Specialized Centers: Lessons Learned from a Study of Multicentric Castleman's Disease

 

Abstract: Methodological Challenges in the Estimation of the Incidence Rate of Rare Diseases from Specialized Centers: Lessons Learned from a Study of Multicentric Castleman's Disease

Studies that estimate incidence of very rare diseases (less than 1 in 100,000 of the general population) often use cases seen at specialized centers. However, multiple potential sources of both systematic error and random error complicate this estimation. We calculated the incidence rate of Multicentric Castleman’s Disease (MCD) based on data from two specialized centers. Our objective is to describe the main challenges of incidence estimation of rare diseases in general, and specifically of MCD, and to suggest how to improve the assessment accuracy.