More rigor, insists the National Academy of Sciences (NAS), must be shown by practically everyone who is working on biomarker tests for molecularly targeted therapies. Through a report announced on March 4, the NAS is calling for “common evidentiary standards” and better coordination of regulatory and reimbursement activities.
The NAS report is entitled “Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine.” It argues that biomarker tests need to be integrated into clinical practice more quickly. It also cautions that these tests need to be accurate, reliable, and properly validated. “Getting biomarker tests right is imperative,” the report insists, “because a bad biomarker test is as problematic and potentially harmful as a bad drug.”
Biomarker tests for molecularly targeted therapies 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. Advances in research over the past 15 years have led to hundreds of molecularly targeted agents entering the drug development pipeline; numerous biomarker tests and associated therapies have been approved for clinical use in treating cancer and other diseases. However, progress has been hampered by regulatory and reimbursement uncertainties, clinical practice challenges, and limitations in data collection and analysis.
“The timely development of biomarker tests and associated therapies is critical to realizing the full potential of 'precision medicine,'” said Harold L. Moses, M.D., a cancer researcher at Vanderbilt University and chair of the committee that wrote the report. “Our report lays out a strategy to ensure that patients have access to effective tests and treatments that are based on solid evidence of their ability to improve health outcomes.”
The committee identified 10 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 therapies.
- 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 EHRs and related biomedical informatics tools and assessment 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.
Overall, the report calls for the creation of a “rapid learning system” that would integrate research on biomarker tests and associated treatments with clinical practice. Also, the report recommends that generating evidence of the overall clinical benefit of any biomarker test should be viewed as a continuous process.
Currently, data on biomarker tests and patient outcomes are not collected or shared in a way that can inform clinical practice. This problem, however, could be addressed by some of the report’s recommendations, particularly those with respect to electronic health records and laboratory information systems. In addition, Centers for Medicare and Medicaid Service and private insurance companies could develop payment models to support ongoing data collection on tests and associated therapies.
Some patients may face challenges in obtaining access to biomarker tests and therapies because of economic, ethnic, cultural, or geographic barriers. The report calls for research on how to provide equitable access to biomarker tests and targeted treatments. And to empower patients and their health care providers to make informed decisions, the FDA should develop patient- and provider-friendly standardized labels and additional information about tests' performance, evidence, and uses.
Oversight and accreditation of laboratories providing biomarker tests for molecularly targeted therapies should also be updated and strengthened, the report says. Current regulatory oversight of these labs is widely viewed as insufficient for increasingly complex biomarker tests. And professional organizations and health care institutions should develop and implement standards for obtaining patient specimens to ensure patient safety as well as the accuracy of biomarker test results.