This report, which contains 10 key recommendations (please see below), is the product of an independent committee of 15 experts – including one from Fred Hutchinson Cancer Research Center – appointed by the National Academies of Sciences, Engineering and Medicine to examine regulatory, reimbursement, and clinical practice policy issues that currently influence the adoption of biomarker tests for molecularly targeted therapies into routine clinical practice. Such tests identify molecular variations specific to an individual patient, which can help determine the most effective therapy for a patient’s disease or avoid treatments that could be ineffective or harmful.
In a nutshell, they were tasked to answer this key question: How do we ensure patients have timely access to appropriate tests that may accurately direct targeted therapies, while at the same time protecting them from potential harm due to the adoption of poorly validated tests or inappropriately used tests?
For more information: Please see this Fred Hutch News Service article.
Fred Hutch committee member: Breast cancer oncologist and health economist Dr. Gary Lyman, a member of the Public Health Sciences and Clinical Research divisions and co-director of the Hutchinson Institute for Cancer Outcomes Research at Fred Hutchinson Cancer Research Center, Seattle, served on the Committee on Policy Issues in the Clinical Development and Use of Biomarkers for Molecularly Targeted Therapies. Lyman is also a professor of medicine, public health and pharmacy at the University of Washington.
Media availability: Due to travel, Lyman’s availability for interviews is limited to email and phone today and through the weekend.Lyman quotes: “We clearly believe as a panel that this is the future of medicine and particularly the future of oncology. If this field develops in a rigorous, evidence-based scientific way with appropriate studies, it will totally revolutionize the care of patients with cancer. Over a generation or two, we believe that precision medicine will totally change medical care and improve patient outcomes and quality of life.”
“There is great excitement and enthusiasm for the future (and for some patients, the present since we’ve already made substantive inroads and progress) and at the same time, there are challenges. It is essential that patients are having the right test performed and the right treatment is identified that matches the test results. And then there’s the financial impact for which we must find viable solutions so that properly evaluated tests and treatments are available and accessible. The development and validation of these biomarker tests and molecularly targeted therapies is complicated and costly and, of course, extremely expensive.”
“We’ve just scratched the surface as far as science goes. But the rate of progress is incredible and it’s accelerating almost daily which is exciting but represents a problem in its own right. It’s virtually impossible for any individual or even any group to stay totally on top of developments in this rapidly evolving area. The ideal solution is what we call a rapid learning system. We need to develop strategies that include experts in informatics as well as the clinicians and laboratory scientists to find a way to identify, catalog and annotate (learn what each mutation does). This has to be automated if we’re ever going to keep up with the rate of expansion of knowledge in this area. This isn’t something that a single institution or even a group of institutions is likely to keep on top of – this has to be a concerted national effort, just like the proposed ‘moonshot’. We’re calling for this to be a national priority.”
Goals for advancing appropriate use of biomarker tests for molecularly targeted therapies: 1 Establish common evidentiary standards of clinical utility – using evidence generated both within and outside the context of clinical trials – across all stakeholders.2 Establish a more coordinated and transparent federal process for regulatory and reimbursement decisions.3 Enhance communication to patients and providers about the performance characteristics and evidence for use of specific tests.4 Update and strengthen oversight and accreditation of laboratories providing these tests.5 Ensure ongoing assessment of the clinical utility of the tests.6 Ensure development and use of electronic health records (EHRs) and related biomedical informatics tools and assessment that support the effective clinical use of biomarker tests for molecularly targeted therapies.7 Develop and maintain a sustainable national database for these tests through biomedical informatics technology to promote rapid learning for the improvement of patient care.8 Promote equity in access to these tests and the expertise for effective use of test results in clinical decision making.9 Enhance specimen handling and documentation to ensure patient safety and the accuracy of biomarker test results.10 Improve the processes for developing and updating clinical practice guidelines for the effective use of these tests.
Funding and disclosures: Funding for this study was provided by the American Society for Radiation Oncology, American Society of Clinical Oncology, Breast Cancer Research Foundation, Centers for Disease Control and Prevention, College of American Pathologists, Gilead Sciences, Janssen Diagnostics, National Cancer Institute, Novartis, Pfizer, Quest Diagnostics, and Susan G. Komen.
About Fred Hutch
At Fred Hutchinson Cancer Research Center, home to three Nobel laureates, interdisciplinary teams of world-renowned scientists seek new and innovative ways to prevent, diagnose and treat cancer, HIV/AIDS and other life-threatening diseases. Fred Hutch’s pioneering work in bone marrow transplantation led to the development of immunotherapy, which harnesses the power of the immune system to treat cancer with minimal side effects. An independent, nonprofit research institute based in Seattle, Fred Hutch houses the nation’s first and largest cancer prevention research program, as well as the clinical coordinating center of the Women’s Health Initiative and the international headquarters of the HIV Vaccine Trials Network. Private contributions are essential for enabling Fred Hutch scientists to explore novel research opportunities that lead to important medical breakthroughs. For more information visit www.fredhutch.org or follow Fred Hutch on Facebook, Twitter or YouTube.
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