Michael J Duffy1, Catharine M Sturgeon2, György Sölétormos3, Vivian Barak4, Rafael Molina5, Daniel F Hayes6, Eleftherios P Diamandis7, Patrick M M Bossuyt8. 1. Clinical Research Centre, St Vincent's University Hospital and UCD School of Medicine and Medical Science, Conway Institute of Bimolecular and Biomedical Research, University College Dublin, Dublin, Ireland; michael.j.duffy@ucd.ie. 2. Department of Clinical Biochemistry, Royal Infirmary of Edinburgh, Edinburgh, United Kingdom; 3. Department of Clinical Biochemistry, University of Copenhagen North Zealand Hospital, Hillerød, Denmark; 4. Oncology Department, Hadassah University Hospital, Jerusalem, Israel; 5. Hospital Clinic, University of Barcelona, Barcelona, Spain; 6. University of Michigan Comprehensive Cancer Center, Ann Arbor, MI; 7. Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario, Canada; 8. Department of Clinical Epidemiology, Biostatistics and Bioinformatics, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands.
Abstract
BACKGROUND: Biomarkers are playing increasingly important roles in the detection and management of patients with cancer. Despite an enormous number of publications on cancer biomarkers, few of these biomarkers are in widespread clinical use. CONTENT: In this review, we discuss the key steps in advancing a newly discovered cancer candidate biomarker from pilot studies to clinical application. Four main steps are necessary for a biomarker to reach the clinic: analytical validation of the biomarker assay, clinical validation of the biomarker test, demonstration of clinical value from performance of the biomarker test, and regulatory approval. In addition to these 4 steps, all biomarker studies should be reported in a detailed and transparent manner, using previously published checklists and guidelines. Finally, all biomarker studies relating to demonstration of clinical value should be registered before initiation of the study. SUMMARY: Application of the methodology outlined above should result in a more efficient and effective approach to the development of cancer biomarkers as well as the reporting of cancer biomarker studies. With rigorous application, all stakeholders, and especially patients, would be expected to benefit.
BACKGROUND: Biomarkers are playing increasingly important roles in the detection and management of patients with cancer. Despite an enormous number of publications on cancer biomarkers, few of these biomarkers are in widespread clinical use. CONTENT: In this review, we discuss the key steps in advancing a newly discovered cancer candidate biomarker from pilot studies to clinical application. Four main steps are necessary for a biomarker to reach the clinic: analytical validation of the biomarker assay, clinical validation of the biomarker test, demonstration of clinical value from performance of the biomarker test, and regulatory approval. In addition to these 4 steps, all biomarker studies should be reported in a detailed and transparent manner, using previously published checklists and guidelines. Finally, all biomarker studies relating to demonstration of clinical value should be registered before initiation of the study. SUMMARY: Application of the methodology outlined above should result in a more efficient and effective approach to the development of cancer biomarkers as well as the reporting of cancer biomarker studies. With rigorous application, all stakeholders, and especially patients, would be expected to benefit.
Authors: Andrea K Miyahira; Joshua M Lang; Robert B Den; Isla P Garraway; Tamara L Lotan; Ashley E Ross; Tanya Stoyanova; Steve Y Cho; Jonathan W Simons; Kenneth J Pienta; Howard R Soule Journal: Prostate Date: 2015-10-19 Impact factor: 4.104
Authors: Nathan G Lambert; Hanan ElShelmani; Malkit K Singh; Fiona C Mansergh; Michael A Wride; Maximilian Padilla; David Keegan; Ruth E Hogg; Balamurali K Ambati Journal: Prog Retin Eye Res Date: 2016-05-06 Impact factor: 21.198
Authors: Ann H Partridge; Karen Sepucha; Anne O'Neill; Kathy D Miller; Emily Baker; Chau T Dang; Donald W Northfelt; George W Sledge; Bryan P Schneider Journal: Breast Cancer Res Treat Date: 2017-06-23 Impact factor: 4.872
Authors: Ainhoa Arana Echarri; Mark Beresford; John P Campbell; Robert H Jones; Rachel Butler; Kenneth J Gollob; Patricia C Brum; Dylan Thompson; James E Turner Journal: Front Immunol Date: 2021-02-01 Impact factor: 7.561
Authors: Joseph A Sparano; Robert J Gray; Della F Makower; Kathleen I Pritchard; Kathy S Albain; Daniel F Hayes; Charles E Geyer; Elizabeth C Dees; Edith A Perez; John A Olson; JoAnne Zujewski; Tracy Lively; Sunil S Badve; Thomas J Saphner; Lynne I Wagner; Timothy J Whelan; Matthew J Ellis; Soonmyung Paik; William C Wood; Peter Ravdin; Maccon M Keane; Henry L Gomez Moreno; Pavan S Reddy; Timothy F Goggins; Ingrid A Mayer; Adam M Brufsky; Deborah L Toppmeyer; Virginia G Kaklamani; James N Atkins; Jeffrey L Berenberg; George W Sledge Journal: N Engl J Med Date: 2015-09-27 Impact factor: 91.245
Authors: Peter J Mazzone; Catherine Rufatto Sears; Doug A Arenberg; Mina Gaga; Michael K Gould; Pierre P Massion; Vish S Nair; Charles A Powell; Gerard A Silvestri; Anil Vachani; Renda Soylemez Wiener Journal: Am J Respir Crit Care Med Date: 2017-10-01 Impact factor: 21.405