Biomarker Tests for Molecularly Targeted Therapies Key to Unlocking Precision Medicine (2016) / Chapter Skim
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1 Introduction
1 Introduction
Pages 23-46
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Known as biomarker tests for molecularly targeted therapies, these complex tests have the potential to enable selection of the most beneficial treatment for the molecular underpinnings of an individual patient's disease. Such tests are key to unlocking the promise of precision medicine (see Box 1-1)
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. The defini tion, which was developed by the President's Council of Advisors on Science and Technology, specifies precision medicine as: The tailoring of medical treatment to the individual characteristics of each patient .
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Definitions and terminology are critical to a complex, rapidly evolving field such as biomarker tests for molecularly targeted therapies (see Box 1-2)
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. Clinical Utility: "Evidence of improved measurable clinical outcomes, and [a test's]
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In oncology, for example, such a test result could suggest treatment with a variety of drugs, each with varying levels of evidence supporting their efficacy. The implications of this transition, specifically related to tests guiding the use of molecularly targeted therapies, are one of the central topics addressed throughout the subsequent chapters of this report.
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PRECISION MEDICINE Biomarker tests for molecularly targeted therapies are used to select the therapy most likely to result in a favorable response in a given patient (IOM, 2012a)
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or predicted to have an adverse reaction to treatment. Moreover, biomarker tests for molecularly targeted therapies may have the potential to "bend the health care cost curve" (Armstrong, 2012)
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is being expanded to include the use of genomic BOX 1-3 Precision Medicine Initiative This study was launched against the backdrop of the Precision Medicine Initia tive, the White House announcement of which coincided with the first meeting of the Committee on Policy Issues in the Clinical Development and Use of Biomark ers for Molecularly Targeted Therapies on January 30, 2015. Characterized as "a bold new research effort to revolutionize how we improve health and treat disease," the Precision Medicine Initiative calls for significant investments (totaling $215 million in fiscal year 2016)
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. Advanced biomarker tests to direct molecularly targeted therapy can be used to characterize a patient's dis
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Perhaps most importantly, this evolution is a continuous process: current NSCLC treatments targeting EGFR are not effective for every patient whose cancer has an EGFR-activating mutation. Future research will continue to characterize response and resistance to treatment, and this will further contribute to the evolving under standing of molecular profiles of disease and the impact of molecularly targeted therapies.
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These data on biomarker test use and treatment selection, as well as patient outcomes, need to be systematically captured, analyzed, and shared for continuous learning. In this respect the use of molecularly targeted therapies, particularly in patients with an unmet need for effective treatment, represents a blurring of the line between clinical research and clinical care.
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. Moreover, there is concern that payer decisions about coverage and reimbursement for biomarker tests for molecularly targeted therapies are not reflective of their clinical value, leading to reluctance on the part of test developers to invest resources to demonstrate clinical utility to support reimbursement decisions -- what Hayes and colleagues characterize as "a vicious cycle" (Hayes et al., 2013)
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INTRODUCTION 35 FIGURE 1-2 Recommended development and evaluation process for omics-based tests. NOTE: FDA = Food and Drug Administration; IDE = investigational device exemption; IRB = institutional review board; LDT = laboratory-developed test.
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In light of the 2012 report's thorough treatment of the clinical development of complex omics-based biomarker tests, the committee did not believe it could improve upon or expand on the report's comprehensive treatment of the topic. This committee's statement of task was to examine the interconnected regulatory, reimbursement, and clinical practice policy issues related to the use of biomarker tests for molecularly targeted therapies (see Box 1-5)
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Brief biographies of the 15 committee members are available in Appendix A CONTEXT OF THE STUDY Biomarker tests for molecularly targeted therapies do not exist in a vacuum, and must be viewed within the context of the broader health care system.
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In July 2015, the House Energy and Commerce Subcommittee voted in a bipartisan fashion to move HR 6, known as the 21st Century Cures Act,2 out of committee. The bill aims to accelerate the availability of safe and effective treatments and contains a number of provisions related to biomedical research generally and precision medicine and biomarkers in particular.
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.3 METHODS OF THE STUDY The committee sought to expand its understanding of the full range of challenges and opportunities facing biomarker tests for molecularly targeted therapies. A diverse range of sources informed the committee's work, including published literature and expert testimony.
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Finally, in addition to benefiting from a range of expert oral and written input, the committee reviewed an extensive body of literature on biomarker tests for molecularly targeted therapies to inform its deliberations. Framework for the Study The successful adoption of biomarker tests for molecularly targeted therapies into routine clinical practice to improve patient outcomes depends on a number of interrelated factors: ongoing research and development of targeted therapies and associated biomarker tests with a changing body of evidence over time, a responsive regulatory and reimbursement process capable of keeping pace with rapid technological developments, health care providers trained in and knowledgeable about which test(s)
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Chapter 4 discusses the challenges involved in the development and implementation of a supportive data infrastructure. Chapter 5 focuses on the final component of the committee's vision of a rapid learning health system for biomarker tests for molecularly targeted therapies: processes to improve patient care.
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2015. Pragmatic issues in biomarker evaluation for targeted therapies in cancer.
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2015c. Policy issues in the development and adoption of biomarkers for molecularly targeted cancer therapies: Workshop summary.
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2015. Fact sheet: President Obama's Precision Medicine Initia tive.
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2015. Precision medicine: The future or simply politics?
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