Perspectives on Enquiry & Evidence
Cytel's blog featuring the latest industry insights.
Featured post
Rare disease studies have specific challenges, such as small populations, limited prior knowledge on a disease, and difficulty in defining appropriate endpoints. Effective clinical development strategy for rare diseases requires agility to adapt to accumulating learning.
Here, we provide a brief overview on the construction of endpoints, adaptive and seamless clinical trial designs, Bayesian approaches, and the use of natural history studies in the development of rare disease studies.