||5R01CA236468-03 Interpret this number
||University Of Pennsylvania
||Data and Information Integration for Risk Prediction in the Era of Big Data
Toward precision medicine and precision disease prevention, the overarching goal of this proposal is to
develop innovative statistical methods for accurate risk prediction. We address three challenges that
plague studies on the value of candidate risk predictors that adds to established predictors for improved
predictive accuracy: there is often a lack of independent validation data, the source population for the
study sample and the target population of prediction are often different, no statistical methods are
currently available for developing risk prediction models using individually-matched case-control data,
and there is a lack of statistical methods for helping assess study feasibility beyond standard power
calculation for testing predictor-outcome association. On the other hand, data and information that are
external to the study may well exist and can be exploited to alleviate these challenges. For example, a
model with only standard predictors often exists and has been validated, and the distribution of standard
risk predictors in the target population of prediction is often available. We propose that external data and
information can be exploited to address the above-mentioned challenges for candidate predictor
evaluation, and develop innovative statistical methods to bring this idea to fruition. Considering
prediction of a binary outcome, we propose a novel method to building logistic prediction models that are
guaranteed to calibrate well in the target population, an innovative method for risk prediction with
individually matched case-control data, and a method to project the added value of candidate predictors to
help assess study feasibility. Our methods, accompanied by user-friendly software, will facilitate cost
effective and timely predictor evaluation for predicting binary outcomes. Our methods were motivated by
and will be applied to several PI Chen's collaborative studies.
Phenotyping issues for exploring electronic health records to design clinical trials.
, Zhang L.
, Chen J.
Clinical trials (London, England), 2020 08; 17(4), p. 402-404.
Novel two-phase sampling designs for studying binary outcomes.
, Williams M.L.
, Chen Y.
, Chen J.
Biometrics, 2020 03; 76(1), p. 210-223.
Phenotype validation in electronic health records based genetic association studies.
, Damrauer S.M.
, Zhang H.
, Zhang A.X.
, Xiao R.
, Moore J.H.
, Chen J.
Genetic epidemiology, 2017 12; 41(8), p. 790-800.