Reflection on clinical and methodological issues in rare disease clinical trials

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Experts provide a comprehensive reflection on the unique clinical and methodological challenges inherent in designing and executing trials for rare diseases.

This paper discusses the specific hurdles faced in rare disease research, including small patient populations and recruitment difficulties. It reflects on innovative trial designs and statistical methods that can enhance the robustness of clinical evidence in these settings.

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Orphanet Journal of Rare Diseases

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