An efficient strategy for evaluating new non-invasive screening tests for colorectal cancer: the guiding principles.
Abstract
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.
A formal consensus approach involving a multidisciplinary expert panel revised eight previously established principles.
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 (<i>phase I</i>), followed by prospective estimation of accuracy across the continuum of neoplastic lesions in neoplasia-enriched populations (<i>phase II</i>). If these show promise, a provisional test positivity threshold is set before evaluation in typical screening populations. <i>Phase III</i> prospective studies determine single round intention-to-screen programme outcomes and confirm the test positivity threshold. <i>Phase IV</i> 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.
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.
EDRN PI Authors
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Medline Author List
- Allison J
- Benamouzig R
- Benton S
- Bossuyt PMM
- Bresalier RS
- Caro L
- Carvalho B
- Chiu HM
- Coupé VMH
- Dekker E
- Dolwani S
- Fraser CG
- Grady W
- Guittet L
- Gupta S
- Halloran SP
- Haug U
- Hoff G
- Itzkowitz S
- Kortlever T
- Koulaouzidis A
- Ladabaum U
- Lauby-Secretan B
- Leja M
- Levin B
- Levin TR
- Macrae F
- Meijer GA
- Melson J
- O'Morain C
- Parry S
- Rabeneck L
- Ransohoff DF
- Saito H
- Sanduleanu-Dascalescu S
- Schoen RE
- Selby K
- Senore C
- Singh H
- Steele RJC
- Sung JJY
- Symonds EL
- Sáenz R
- Winawer SJ
- Young GP
- de Klaver W
- de Klerk CM