We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
Evaluating biomarkers for prognostic enrichment of clinical trials.
- Authors
Kerr, Kathleen F.; Roth, Jeremy; Kehao Zhu; Thiessen-Philbrook, Heather; Meisner, Allison; Wilson, Francis Perry; Coca, Steven; Parikh, Chirag R.
- Abstract
Background/Aims: A potential use of biomarkers is to assist in prognostic enrichment of clinical trials, where only patients at relatively higher risk for an outcome of interest are eligible for the trial. We investigated methods for evaluating biomarkers for prognostic enrichment. Methods: We identified five key considerations when considering a biomarker and a screening threshold for prognostic enrichment: (1) clinical trial sample size, (2) calendar time to enroll the trial, (3) total patient screening costs and the total per-patient trial costs, (4) generalizability of trial results, and (5) ethical evaluation of trial eligibility criteria. Items (1)-(3) are amenable to quantitative analysis. We developed the Biomarker Prognostic Enrichment Tool for evaluating biomarkers for prognostic enrichment at varying levels of screening stringency. Results: We demonstrate that both modestly prognostic and strongly prognostic biomarkers can improve trial metrics using Biomarker Prognostic Enrichment Tool. Biomarker Prognostic Enrichment Tool is available as a webtool at http://prognosticenrichment.com and as a package for the R statistical computing platform. Conclusion: In some clinical settings, even biomarkers with modest prognostic performance can be useful for prognostic enrichment. In addition to the quantitative analysis provided by Biomarker Prognostic Enrichment Tool, investigators must consider the generalizability of trial results and evaluate the ethics of trial eligibility criteria.
- Subjects
EVALUATION of clinical trials; ATTITUDE (Psychology); BIOMARKERS; MEDICAL needs assessment; PROGNOSIS; RISK perception; SAMPLE size (Statistics); HUMAN research subjects; PATIENT selection
- Publication
Clinical Trials, 2017, Vol 14, Issue 6, p629
- ISSN
1740-7745
- Publication type
Article
- DOI
10.1177/1740774517723588