Study design considerations for trials to evaluate multicancer early detection assays for clinical utility.

Autor:	Minasian LM; Division of Cancer Prevention, National Cancer Institute, Bethesda, MD, USA., Pinsky P; Division of Cancer Prevention, National Cancer Institute, Bethesda, MD, USA., Katki HA; Division of Cancer Epidemiology and Genetics, Biostatistics Branch, National Cancer Institute, Bethesda, MD, USA., Dickherber T; Center for Scientific Strategic Initiatives, National Cancer Institute, Bethesda, MD, USA., Han PKJ; Division of Cancer Control and Population Sciences, National Cancer Institute, Bethesda, MD, USA., Harris L; Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, MD, USA., Patriotis C; Division of Cancer Prevention, National Cancer Institute, Bethesda, MD, USA., Srivastava S; Division of Cancer Prevention, National Cancer Institute, Bethesda, MD, USA., Weil CJ; Division of Cancer Prevention, National Cancer Institute, Bethesda, MD, USA., Prorok PC; Division of Cancer Prevention, National Cancer Institute, Bethesda, MD, USA., Castle PE; Division of Cancer Prevention, National Cancer Institute, Bethesda, MD, USA.
Jazyk:	angličtina
Zdroj:	Journal of the National Cancer Institute [J Natl Cancer Inst] 2023 Mar 09; Vol. 115 (3), pp. 250-257.
DOI:	10.1093/jnci/djac218
Abstrakt:	Blood-based assays using various technologies and biomarkers are in commercial development for the purpose of detecting multiple cancer types concurrently at an early stage of disease. These multicancer early detection (MCED) assays have the potential to improve the detection of cancers, particularly those for which no current screening modality exists. However, the unknown clinical benefits and harms of using MCED assays for cancer screening necessitate the development and implementation of a randomized controlled trial (RCT) to ascertain their clinical effectiveness. This was the consensus of experts at a National Cancer Institute-hosted workshop to discuss initial design concepts for such a trial. Using these assays to screen simultaneously for multiple cancers poses novel uncertainties for patient care compared with conventional screening tests for single cancers, such as establishing the diagnostic workup to confirm the presence of cancer at any organ site; clarifying appropriate follow-up for a positive assay for which there is no definitive diagnosis; identifying potential harms such as overdiagnosis of indolent disease; determining clinically effective and efficient strategies for disseminating MCED screening in real-world practice; and understanding the ethical implications, such as potentially alleviating or exacerbating existing health disparities. These assays present new and complex challenges for designing an RCT. Issues that emerged from the meeting centered around the need for a flexibly designed, clinical utility RCT to rigorously capture the evidence required to fully understand the net benefit of this promising technology. Specific topic areas were endpoints, screening protocols, recruitment, diagnostic pathway, pilot phase, data elements, specimen collection, and ethical considerations. (Published by Oxford University Press 2022.)
Databáze:	MEDLINE
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