Article

Variation in the Accuracy of Endpoint Selection in Clinical Studies for Rare Diseases in Respect of Increasing Knowledge on Disease-Severity Measurement

2021-12-30

Abstract excerpt

<h4>Background: </h4> Previous research assessed the accuracy of disease-severity measurement in clinical studies as a mathematical relationship between the set of endpoints selected and the disease-severity scale (DSS), a surrogate for the theoretical Neutral list of indicators representing the disease phenotype. New DSSs are continually developed, so clinical studies’ operationalisation of the Neutral list and r...

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Literature Corpus work: 0b351faa-7bd0-5c62-9c65-f6f1731f8f51
DOI: 10.21203/rs.3.rs-1167543/v1

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