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CMTP Issues Guidance for Clinical Utility Studies of Molecular Diagnostics in Oncology

May 2, 2013

CMTP has issued an Effectiveness Guidance Document (EGD) for molecular diagnostics tests in oncology that provides specific study design recommendations for researchers and test developers to evaluate the impact of their tests on patient health outcomes.

Several molecular diagnostic tests currently have strong evidence that their clinical use leads to improved health outcomes. However, there is a large and growing group of promising tests that lack adequate evidence of clinical utility. There is also considerable uncertainty over the types and levels of evidence needed to demonstrate clinical utility. This EGD, which is the culmination of a multi-year project involving key stakeholders, sets out evidence targets and guidance that will improve the quality, relevance and efficiency of these studies.

“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,” said Sean Tunis, president and CEO of CMTP. “This guidance provides test developers and those designing clinical studies with concrete recommendations on what types of studies are needed.”

“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,” said Pat Deverka, senior research director at CMTP and lead author of the document. “Our overarching goal is to bring greater clarity and predictability regarding the evidence requirements of all interested stakeholders.”

“These standards for clinical utility, which were developed with diverse stakeholder input, should help to ensure that the quality of future studies is sufficient for clinical decision making and the development of professional society guidelines, such as those produced by the American Society of Clinical Oncology. We will then be more confident that we are providing clinicians with the evidence they need to provide high quality care for their patients.” said Gary Lyman, Professor and Director of Comparative Effectiveness and Outcomes Research at Duke University and a member of the Technical Working Group involved with developing EGD recommendations on molecular diagnostic tests in oncology.

CMTP consulted with a wide range of experts and stakeholders to develop these EGD study design recommendations, which are analogous to regulatory guidance documents (such as those issued by the Food and Drug Administration). However, EGDs are focused on evidence requirements that are relevant to reimbursement policy and clinical decisions. This EGD was created through an iterative process that incorporated the perspectives of major stakeholder groups, including researchers, clinicians, payers, industry, guideline developers and patients.