The Green Park Collaborative (GPC-USA), sponsored by the Center for Medical Technology Policy (CMTP), today launched a new effort to develop methodological guidance on the kinds of evidence needed to evaluate promising tests in the rapidly developing field of Next Generation Sequencing (NGS) for cancer diagnosis and treatment.

NGS streamlines the technical aspects of molecular testing for a variety of diseases while producing higher volumes and breadth of genomic information, at increasingly competitive costs. When applied in oncology, NGS promises to accelerate our growing understanding of cancer, helping to define tumors' biological pathways and genetic characteristics. It is yielding a much more accurate picture of diseases and making possible a new generation of targeted therapies.  Assessing the clinical utility of these innovations, however, remains challenging.

"The rapid development of NGS-based tests for use in oncology has highlighted this technology's tremendous potential to improve clinical care and patient outcomes," said Sean Tunis, MD, MSc, President and CEO of CMTP. "At the same time, it underscores the need for a clearly defined, consistent and broadly accepted framework for evaluating the evidence of the clinical utility of tests in well-designed, feasible studies."

Providing needed methodological guidance
This 2014 initiative, organized within the GPC's existing Oncology Consortium (see below), will produce an Effectiveness Guidance Document (EGD) designed to inform evidence-based decision making regarding coverage and reimbursement for NGS-based tests. The work will be directed by Elisabeth Houtsmuller, PhD, senior program director for GPC, and driven by a technical working group that will include expert representatives from health plans, CMS, NGS test developers, FDA, patient groups, oncology guideline organizations, clinical oncologists, drug/biologic companies, and other groups with a direct interest in NGS in oncology.

The project's EGD will focus on NGS-based tests used to manage patients with a known diagnosis of cancer (as opposed to risk prediction testing). Its recommendations will be aligned with existing and emerging regulatory guidance where relevant. This effort will be informed by CMTP's recent work on evidentiary standards for studies of the clinical validity and clinical utility of molecular diagnostics in oncology.

"The kind of guidance this process is developing will be invaluable," said Elaine Jeter, MD, Medical Director of Palmetto, GBA, a Medicare Administrative Contractor and the developer of the MolDx program. "There is tremendous promise surrounding Next Generation Sequencing technologies, but we need guidance to help us evaluate the quality of the evidence supporting these tests and in turn guide test makers in their efforts to demonstrate these products' clinical utility."

The GPC's Oncology Consortium is also developing an EGD and associated methodologic recommendations and data strategies to help shape research on the best ways to sequence cancer care treatments.  This work is led by Donna Messner, PhD, Research Director at CMTP, and Anne Schott, MD, who specializes in medical oncology at the University of Michigan.  Dr. Schott is chairing the Technical Working Group responsible for developing initial recommendations for the guidance document.

About the Green Park Collaborative-USA (GPC-USA)
GPC-USA is a multi-stakeholder forum that is developing condition-specific study design recommendations to guide the generation of evidence needed to inform coverage and payment decisions in the United States. The GPC-USA serves as a trusted forum where payers, life sciences companies, patient groups, clinicians, researchers, regulators and other stakeholders can engage in dialogue regarding methodological standards for studies intended to demonstrate real world effectiveness and value. For more information click here.

About CMTP
The Center for Medical Technology Policy (CMTP) is an independent, non-profit 501(c)(3) organization that aims to make health care more effective and affordable by improving the quality, relevance, and efficiency of health care research. We focus on the design and implementation of comparative effectiveness research to produce information that helps patients, clinicians, and payers make informed treatment and policy decisions. CMTP provides a trusted forum in which a broad range of stakeholders can collaborate to identify important research questions, design appropriate studies, and develop innovative partnerships to implement these studies.