Choosing and Specifying End Points and Outcomes

Section 6



Lesley Curtis, PhD

Adrian F. Hernandez, MD, MHS

Kevin P. Weinfurt, PhD



Contributing Editor

Karen Staman, MS

Resource Description
Living Textbook Resource Chapters
Patient-Reported Outcomes This resource chapter defines patient-reported outcomes and describes how to use, measure, interpret, and implement PRO measures. PRO data are used to inform and guide patient-centered care as well as clinical and health policy decision-making.
Electronic Health Records-Based Phenotyping This chapter defines phenotypes; describes how to find, evaluate, and implement appropriate phenotypes; recommends phenotype definitions; and describes phenotype use in the NIH Collaboratory and the Patient-Centered Outcomes Research Network (PCORnet). In the context of EHRs, a computable phenotype or simply phenotype refers to a clinical condition or characteristic that can be ascertained via a computerized query to an EHR system or clinical data repository using a defined set of data elements and logical expressions. Standardized computable phenotypes can enable large-scale PCTs across multiple health systems while ensuring reliability and reproducibility.
White Papers
Resources from the Collaboratory Patient-Reported Outcomes (PRO) Core
Journal Articles
Electronic health records based phenotyping in next-generation clinical trials: a perspective from the NIH Health Care Systems Collaboratory

(Richesson et al. JAMIA 2013)

The Phenotypes, Data Standards, and Data Quality Core, present early observations from researchers implementing PCTs within large healthcare systems. The authors identify gaps in knowledge and present an informatics research agenda that includes identifying methods for the definition and appropriate application of phenotypes in diverse healthcare settings, and methods for validating both the definition and execution of EHR-based phenotypes.




Curtis L, Hernandez A, Weinfurt K. Choosing and Specifying End Points and Outcomes: Resources. In: Rethinking Clinical Trials: A Living Textbook of Pragmatic Clinical Trials. Bethesda, MD: NIH Health Care Systems Research Collaboratory. Available at: Updated September 14, 2017.