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CMTP Publishes Guidance for Assessing Utility and Validity of Cancer Molecular Dx

NEW YORK (GenomeWeb News) – Seeking to set a standard for how molecular diagnostic cancer tests are evaluated, the Center for Medical Technology Policy has issued a set of recommendations covering how to best assess the clinical validity and utility of these tests.

While molecular diagnostic tests offer great promise for oncology, evidence supporting the use and safety of these tests is lacking, and there is "considerable debate" about how much evidence is required and how it should be acquired, CMTP said yesterday.

Although several molecular diagnostic tests for cancer do have strong data that they improve patient outcomes, a "large and growing" group of tests are lacking in evidence, particularly regarding their clinical utility, the nonprofit center said.

The effectiveness guidance document, or EGD, that CMTP published yesterday — which the group likened to a Food and Drug Administration Guidance document — was developed through a multi-year process with major stakeholders and was created to improve the quality, relevance, and efficiency of studies of the utility and validity of such oncology tests for adults.

"Determining what constitutes a reasonable level of evidence of clinical utility for molecular diagnostics could significantly accelerate access to useful tests, while preventing widespread adoption of tests that don't improve patient outcomes," CMTP President and CEO Sean Tunis said in a statement. "This guidance provides test developers and those designing clinical studies with concrete recommendations on what types of studies are needed."

The goal was to create a framework of recommendations that test developers and researchers could use to plan studies that anticipate the evidence that will be required for the tests to be reimbursed. Additionally, payers and technology assessors would use the same standards to assess study results.

"The purpose of this initiative was to close the gap between the presumed benefits of tests undergoing technology assessments and the information needs of payers, clinicians and patients," added Pat Deverka, a senior research director at CMTP and the EGD's lead author. "Our overarching goal is to bring greater clarity and predictability regarding the evidence requirements of all interested stakeholders."

The project was funded by a number of life sciences industry and healthcare firms and foundations, including Becton Dickinson; Cepheid; Myriad Genetics; Genomic Health; Amgen; Daiichi Sankyo; AstraZeneca; Sanofi- Aventis; and others.

The document includes recommendations for reporting analytic validity; selecting patient populations for assessing validity; the metrics required for clinical validation; anticipating clinical pathways; measuring outcomes for clinical utility; and using randomized controlled trials for clinical utility, prospective-retrospective clinical trials, single-arm studies, longitudinal observational studies, and decision-making techniques.

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