This NEHI white paper outlines recommendations for benefit-risk assessment of multi-cancer early detection tests (MCEDs).
MCED tests are an emerging class of innovative devices and analytical services that will detect many types of cancer based on the initial detection of blood-borne markers of cancer, such as cancer tumor DNA circulating in a patient’s blood. Many of these biomarkers are common to more than one type of cancer, including cancers that are currently targets of routine screening (such as breast and colorectal cancers) and cancers not subject to routine screening, such as pancreatic cancer. (Only four of the thirteen most common cancers, as reported by the National Cancer Institute, are subject to routine screening.) MCED tests now in development are intended for use as a complement to existing, single-cancer tests such as the mammogram and colonoscopy. Widespread use of MCED tests will depend in part on FDA approval based on favorable evaluation of MCED benefits and risks.
The MCED test could profoundly shift practices in cancer screening and diagnosis. The potential benefits from MCED testing are multi-faceted. For example: MCED screening will detect types of cancer that often go undetected until the disease progresses to a later stage. The potential impact will be felt at both the level of individual patients and their health, and on public health and cancer detection in the overall adult population. As with all medical testing, there is the possibility of incidental adverse risks to patients from the tests. A thoughtful approach is needed to evaluate benefits and risks, particularly due to the intended use of MCED tests as part of cancer screening among adults with no apparent symptoms of cancer.
The NEHI white paper follows several months of research and consultation that began with an expert panel convened in the Fall of 2020 and continued dialogue into 2021. The panel included recognized authorities in oncology, biostatistics, clinical trial design, and medical device regulation, as well as representatives from two MCED test developers. The white paper released by NEHI today calls on stakeholders to acknowledge the need for an MCED-specific approach to the evaluation of MCED benefits and risks and work towards a consensus approach. Since MCED testing will generate test results for many types of cancer, some of which are not screened today and to which no pre-existing standards of accuracy apply, a fit-for-purpose approach is needed that can yield one overall assessment of each MCED test seeking FDA approval. The effort should start with agreement on identifying major categories of benefit and risk that can be weighted in this overall assessment. The NEHI white paper offers a suggestive categorization of MCED benefits and risks as measured in the impact of MCED use on cancer detection and diagnosis, and the impact of MCED screening on persistent patient adherence to recommended modes of cancer screening.
This project was supported by Grail, LLC and Thrive, an Exact Sciences Company.
