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Perspectives on Enquiry & Evidence

Cytel's blog featuring the latest industry insights.

Quantitative Strategies for Rare Disease Clinical Trials

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.

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Perspectives on Enquiry and Evidence
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