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Updated: Aug 19, 2025
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Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
Published on: February 6, 2015
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Study design considerations for trials to evaluate multicancer early detection assays for clinical utility
Lori M Minasian1, Paul Pinsky1, Hormuzd A Katki2
1Division of Cancer Prevention, National Cancer Institute, Bethesda, MD, USA.
Journal of the National Cancer Institute
|December 2, 2022
Summary
Multicancer early detection (MCED) blood tests show promise for improving cancer detection, especially for rare cancers. A randomized controlled trial (RCT) is essential to determine the clinical benefits and harms of these novel screening assays.
Area of Science:
- Oncology
- Genomics
- Clinical Trials
Background:
- Multicancer early detection (MCED) blood-based assays are emerging technologies for concurrent detection of multiple cancer types.
- These assays hold potential for improving early cancer diagnosis, particularly for cancers lacking current screening methods.
Purpose of the Study:
- To establish the need for a randomized controlled trial (RCT) to evaluate the clinical effectiveness of MCED assays.
- To identify key design considerations for an RCT investigating MCED assay utility.
Main Methods:
- Expert consensus from a National Cancer Institute-hosted workshop informed the discussion on RCT design.
- Key challenges and uncertainties associated with MCED screening were identified.
Main Results:
- Experts agreed on the necessity of a flexibly designed, clinical utility RCT to assess MCED assays.
- Novel uncertainties include diagnostic workup, follow-up for positive results, overdiagnosis, dissemination strategies, and ethical implications.
Conclusions:
- Designing an RCT for MCED assays presents unique challenges compared to single-cancer screening.
- Rigorous evidence generation through a clinical utility RCT is crucial to understand the net benefit of MCED technology.
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