Reducing selection bias in case-control studies from rare disease registries

  title={Reducing selection bias in case-control studies from rare disease registries},
  author={J. Alexander Cole and John S. Taylor and Thomas N. Hangartner and Neal J Weinreb and Pramod K Mistry and Aneal A Khan},
  booktitle={Orphanet journal of rare diseases},
BACKGROUND In clinical research of rare diseases, where small patient numbers and disease heterogeneity limit study design options, registries are a valuable resource for demographic and outcome information. However, in contrast to prospective, randomized clinical trials, the observational design of registries is prone to introduce selection bias and negatively impact the validity of data analyses. The objective of the study was to demonstrate the utility of case-control matching and the risk… CONTINUE READING
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