Neutral theory: applicability and neutrality of clinical study endpoints where a disease-specific instrument is available.

ABSTRACT:

Background

There is a pressing need to improve the accuracy of rare disease clinical study endpoints. Neutral theory, first described here, can be used to assess the accuracy of endpoints and improve their selection in rare disease clinical studies, reducing the risk of patient misclassification.

Methods

Neutral theory was used to assess the accuracy of rare disease clinical study endpoints and the resulting probability of false positive and false negative classifications at different disease prevalence rates. Search strings were extracted from the Orphanet Register of Rare Diseases using a proprietary algorithm to conduct a systematic review of studies published until January 2021. Overall, 11 rare diseases with one disease-specific disease severity scale (133 studies) and

SUBMITTER: Jandhyala R 

PROVIDER: S-EPMC10199426 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature