Personalized medicine in oncology is maturing and evolving rapidly, and the utilization of molecular biomarkers in clinical decision-making is growing. As the breadth of information advances, so too does the need to provide authoritative guidance regarding proper tests and their corresponding clinical utility, both from a clinical and coverage policy standpoint. Ideally, guidance regarding the utilization of molecular testing would be modeled after the current process for determining coverage of drugs and biologics.
Additionally, recent regulatory changes surrounding molecular and biomarker testing have the potential to affect oncology care. For example, the Food and Drug Administration (FDA) has recently announced plans for oversight of Laboratory Developed Tests (LDT). As recognized by the FDA, these types of diagnostic tests are used increasingly to directly inform treatment decisions, and this especially impacts patients with cancer, and their oncologists.
On Thursday, July 14, 2011, the National Comprehensive Cancer Network (NCCN) convened the NCCN Oncology Policy Summit: Molecular Testing – Effectiveness, Efficiency, and Reimbursement in Washington, DC at the National Press Club. This invite-only Policy Summit was attended by patient advocates, providers (e.g., oncologists, pathologists, other laboratory professionals, etc.), payors, laboratory accreditation organizations, government, and industry representatives within the oncology community. These key stakeholders gathered for a day-long session to discuss current policy issues surrounding molecular testing in oncology.
The Summit featured two expert roundtable discussions, moderated by William T. McGivney, PhD, Chief Executive Officer of NCCN, and several presentations by members of the NCCN Molecular Testing Work Group, comprised of thought leaders from NCCN Member Institutions and external organizations, representing providers (oncologists and pathologists), patients, administrators, industry, payors, and government, who worked over the past several months to identify challenges and reach consensus on molecular testing issues in oncology. The Work Group's efforts will culminate in the publication of a white paper on molecular testing in oncology later this year.
Marc Ladanyi, MD, Director of the Diagnostic Molecular Pathology Laboratory at Memorial Sloan-Kettering Cancer Center and Work Group member, provided an overview of the role of molecular testing in oncology, including a description of the various uses of molecular tests (risk assessment, diagnosis and disease classification, prognosis, response prediction, toxicity prediction, and dose determination), how clinical and analytic validity of these tests are assessed, and highlighted some important areas of stakeholder interest regarding molecular testing, including but not limited to safety, efficacy, efficiency, access to testing, and affordability.
During the second presentation, Paul Engstrom, MD, Senior Vice President, Extramural Research Programs at Fox Chase Cancer Center and Work Group Chair, discussed the challenges and recommendations compiled by the Work Group which will be explored further in the aforementioned white paper. It was noted that the Work Group agreed on a definition of molecular testing in oncology, which refers to the detection of somatic or germline mutations, as well as changes in gene or protein expression that could impact the diagnosis, prognosis, prediction, and evaluation of therapy for cancer patients. Dr. Engstrom reviewed challenges for molecular testing identified by the Work Group from the regulatory policy, clinical, and reimbursement/coverage policy perspectives. Recommendations addressed education for patients, clinicians, and policy-makers and the need to ensure patient access to safe, effective, and efficient molecular tests.
The first expert roundtable panel addressed clinician, patient, and scientific issues related to molecular testing. Panelists representing these areas discussed how molecular testing is currently being used in oncology practice, how the clinical utility of tests is being assessed, current reimbursement experiences, and what kinds of education regarding molecular testing would be most helpful for patients and providers.
The second panel was introduced by presentations providing FDA and Centers for Medicare and Medicaid Services (CMS) perspectives regarding molecular testing in oncology. The panel, moderator, and audience members discussed expectations for FDA's LDT policy, FDA's recently released In Vitro Companion Diagnostic Device guidance, as well as how CMS and private payors are addressing molecular testing as it related to coverage policy.
Through stakeholder engagement and dialogue, the consensus of the Summit was apparent. It is crucial that the oncology community continue to work collaboratively to ensure molecular testing is safe, effective, and efficient for patients with cancer.
Based on the NCCN Molecular Testing Work Group discussions and subsequent NCCN Molecular Testing Policy Summit, NCCN published a white paper entitled "NCCN Molecular Testing White Paper: Effectiveness, Efficiency, and Reimbursement" as a JNCCN supplement.
NCCN Molecular Testing Work Group
Mara Bloom, JD, MS, Executive Director of Administration, Massachusetts General Hospital Cancer Center
George Demetri, MD, Director, Center for Sarcoma and Bone Oncology, Senior Vice President for Experimental Therapeutics, Dana-Farber Cancer Institute
Paul Engstrom, MD (CHAIRPERSON), Senior Vice President, Extramural Research Programs, Fox Chase Cancer Center
Phillip Febbo, MD, Associate Professor, Department of Medicine (Hematology/Oncology), UCSF Helen Diller Family Comprehensive Cancer Center
William Goeckeler, PhD, Oncology Medical Affairs, Boehringer Ingelheim Pharmaceuticals, Inc.
Marc Ladanyi, MD, Director, Diagnostic Molecular Pathology Laboratory, Memorial Sloan-Kettering Cancer Center
Bryan Loy, MD, MBA, Market Medical Officer, Humana
Elizabeth Mansfield, PhD, Director, Personalized Medicine, U.S. Food and Drug Administration
Kate Murphy, Director of Research Communication, Fight Colorectal Cancer
Michael Nerenberg, MD, Chief Technical Officer and Senior Vice President, Medical Affairs, Genoptix
Paul Papagni, JD, Executive Director, Office of VP for Clinical Research, The University of Texas MD Anderson Cancer Center
Mark Robson, MD, Clinical Director, Clinical Genetics Service, Memorial Sloan-Kettering Cancer Center
Robert W. Sweetman, MD, Senior Director Medical Affairs, Oncology Business Unit, Pfizer, Inc
Sean Tunis, MD, MSc, Director, Center for Medical Technology Policy