An efficient strategy for evaluating new non-invasive screening tests for colorectal cancer: the guiding principles
Journal
Gut
ISSN
0017-5749
1468-3288
Date Issued
2023
Author(s)
Robert S Bresalier
Carlo Senore
Graeme P Young
James Allison
Robert Benamouzig
Sally Benton
Patrick M M Bossuyt
Luis Caro
Beatriz Carvalho
Han-Mo Chiu
Veerle M H Coupé
Willemijn de Klaver
Clasine Maria de Klerk
Evelien Dekker
Sunil Dolwani
Callum G Fraser
William Grady
Lydia Guittet
Samir Gupta
Stephen P Halloran
Ulrike Haug
Geir Hoff
Steven Itzkowitz
Tim Kortlever
Anastasios Koulaouzidis
Uri Ladabaum
Beatrice Lauby-Secretan
Mārcis Leja
Bernard Levin
Theodore Robert Levin
Finlay Macrae
Gerrit A Meijer
Joshua Melson
Colm O'Morain
Susan Parry
Linda Rabeneck
David F Ransohoff
Hiroshi Saito
Silvia Sanduleanu-Dascalescu
Robert E Schoen
Kevin Selby
Harminder Singh
Robert J C Steele
Joseph J Y Sung
Erin Leigh Symonds
Sidney J Winawer
Type
Resource Types::text::journal::journal article
URL Institutional Repository
Abstract
<jats:sec><jats:title>Objective</jats:title><jats:p>New screening tests for colorectal cancer (CRC) are rapidly emerging. Conducting trials with mortality reduction as the end point supporting their adoption is challenging. We re-examined the principles underlying evaluation of new non-invasive tests in view of technological developments and identification of new biomarkers.</jats:p></jats:sec><jats:sec><jats:title>Design</jats:title><jats:p>A formal consensus approach involving a multidisciplinary expert panel revised eight previously established principles.</jats:p></jats:sec><jats:sec><jats:title>Results</jats:title><jats:p>Twelve newly stated principles emerged. Effectiveness of a new test can be evaluated by comparison with a proven comparator non-invasive test. The faecal immunochemical test is now considered the appropriate comparator, while colonoscopy remains the diagnostic standard. For a new test to be able to meet differing screening goals and regulatory requirements, flexibility to adjust its positivity threshold is desirable. A rigorous and efficient four-phased approach is proposed, commencing with small studies assessing the test’s ability to discriminate between CRC and non-cancer states (<jats:italic>phase I</jats:italic>), followed by prospective estimation of accuracy across the continuum of neoplastic lesions in neoplasia-enriched populations (<jats:italic>phase II</jats:italic>). If these show promise, a provisional test positivity threshold is set before evaluation in typical screening populations.<jats:italic>Phase III</jats:italic>prospective studies determine single round intention-to-screen programme outcomes and confirm the test positivity threshold.<jats:italic>Phase IV</jats:italic>studies involve evaluation over repeated screening rounds with monitoring for missed lesions. Phases III and IV findings will provide the real-world data required to model test impact on CRC mortality and incidence.</jats:p></jats:sec><jats:sec><jats:title>Conclusion</jats:title><jats:p>New non-invasive tests can be efficiently evaluated by a rigorous phased comparative approach, generating data from unbiased populations that inform predictions of their health impact.</jats:p></jats:sec>
Subjects
colorectal adenomas
;
colorectal cancer
;
colorectal cancer screening
;
colonoscopy
;
colorectal neoplasms
;
early detection of cancer
;
feces
;
humans
;
mass screening
;
occult blood
;
prospective studies
;
biological marker
;
article
;
cancer incidence
;
cancer mortality
;
cancer screening
;
cancer staging
;
colonoscopy
;
colorectal cancer
;
consensus development
;
diagnostic accuracy
;
false positive result
;
human
;
intermethod comparison
;
occult blood test
;
predictive value
;
prospective study
;
colorectal tumor
;
early cancer diagnosis
;
feces
;
mass screening
;
occult blood