1. Nov 30, 2020

    Broad methodological approaches needed for rare disease randomized controlled trials

    Due to the small patient populations found in rare diseases, researching these disease states can be difficult. Although it is feasible to complete randomized controlled trials (RCTs) in rare diseases, a more varied methodological approach is needed, including the use of adaptive trials, cross-over trials, and early escape designs, according to a new study.

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  1. Nov 30, 2020

Broad methodological approaches needed for rare disease randomized controlled trials

Due to the small patient populations found in rare diseases, researching these disease states can be difficult. Although it is feasible to complete randomized controlled trials (RCTs) in rare diseases, a more varied methodological approach is needed, including the use of adaptive…