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Perspectives on Enquiry & Evidence

Cytel's blog featuring the latest industry insights.

Strategies to Overcome Limited Patient Population Challenges in Rare Disease Studies

Rare disease studies come with their own unique challenges, particularly limited patient populations. However, a number of adaptive clinical trial design strategies can be used to mitigate these challenges, including sample size optimization, sample size re-estimation, phase II/III seamless designs, and Exact Statistical methodologies.

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Perspectives on Enquiry and Evidence
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