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Does using biomarker to select patients for Phase 1 trials improve efficacy?

Precision or personalized medicine is about selecting patients with a particular protein or genomic biomarker who might benefit from a specific therapy.

So does using a biomarker to select patients for phase 1 trials improve efficacy outcomes?

Maria Schwaederle, Pharm. D., of the University of California, San Diego, and coauthors explored that question in a meta-analysis of 346 trials and 13,203 patients.

The authors examined response rate and progression-free survival but not overall survival because of insufficient data. They compared trials that used a personalized approach with a biomarker selection with those that did not.

The authors report their analysis suggests a personalized medicine approach was associated with improved outcomes, with a higher median response rate and longer progression-free survival. Additionally, the biomarker that was used appeared to matter, with a genomic DNA biomarker associated with a higher median response rate than a protein biomarker, according to the results. Trial arms that did not use a biomarker had median response rates comparable to those that tested a cytotoxic chemotherapy agent, the authors report.

The authors note study limitations in their meta-analysis, such as including only trial arms that reported single agents, not analyzing combination therapy, and not analyzing survival as an end point.

“These results argue strongly for the enrichment of phase 1 clinical trials with biomarker selection for targeted therapies. However, rigid exclusion based on biomarkers that have not been proven clinically could prove counterproductive in some cases,” the authors conclude.

Article: Association of Biomarker-Based Treatment Strategies With Response Rates and Progression-Free Survival in Refractory Malignant Neoplasms: A Meta-analysis, Maria Schwaederle, PharmD; Melissa Zhao, BS; J. Jack Lee, PhD; Vladimir Lazar, PhD; Brian Leyland-Jones, MD; Richard L. Schilsky, MD; John Mendelsohn, MD; Razelle Kurzrock, MD, JAMA Oncology, doi: 10.1001/jamaoncol.2016.2129, published 6 June 2016.