Biomarker tests are not being adequately used in clinical practice due to a lack of common evidentiary standards for regulatory, reimbursement, and clinical decisions regarding their use, according to a report by the National Academies of Sciences, Engineering, and Medicine.
The report calls for the creation of a rapid learning system that would integrate research on the tests and their associated treatments. Appropriate oversight will also be needed to ensure that tests and targeted therapies are accurate, reliable, and properly validated and implemented.
Biomarker tests can be extremely useful for the treatment of diseases such as cancer, where they can help physicians identify specific molecular variants unique to each patient. With that knowledge, physicians can more easily choose the drug that would most benefit the patient by understanding which therapies would target his or her mutation. Today, numerous tests are available to physicians for disease states such as breast and lung cancer, but progress has been inhibited by regulatory and reimbursement uncertainties, clinical practice challenges, and limitations in data collection and analysis.
To address these problems and ensure that patients have better access to effective tests and treatments, a committee led by Harold L Moses, MD, Vanderbilt-Ingram Cancer Center (Nashville, TN) authored a report outlining 10 specific goals to advance the development and appropriate clinical use of biomarker tests for molecularly targeted therapies:
- Establish common evidentiary standards of clinical utility—using evidence generated both within and outside the context of clinical trials—across all stakeholders.
- Establish a more coordinated and transparent federal process for regulatory and reimbursement decisions for biomarker tests for molecularly targeted therapies.
- Enhance communication to patients and providers about the performance characteristics and evidence for use of specific biomarker tests for molecularly targeted agents.
- Update and strengthen oversight and accreditation of laboratories providing biomarker tests for molecularly targeted therapies.
- Ensure ongoing assessment of the clinical utility of biomarker tests for molecularly targeted therapies.
- Ensure development and use of electronic health records and related biomedical informatics tools and assessments that support the effective clinical use of biomarker tests for molecularly targeted therapies.
- Develop and maintain a sustainable national database for biomarker tests for molecularly targeted therapies through biomedical informatics technology to promote rapid learning for the improvement of patient care.
- Promote equity in access to biomarker tests for molecularly targeted therapies and the expertise for effective use of the results in clinical decision-making.
- Enhance specimen handling and documentation to ensure patient safety and the accuracy of biomarker test results.
- Improve the processes for developing and updating clinical practice guidelines for the effective use of biomarker tests for molecularly targeted therapies.
These goals, wrote the authors, are intended to be interrelated components of a rapid learning system for biomarkers for molecularly targeted agents, which would accelerate the translation of lessons learned into better patient care and improved clinical outcomes. Through these goals, the committee believes the efficiency of care, policy environment, and data infrastructure can all be improved without sacrificing the patient as the primary focal point.
