Biomarker tests for molecularly targeted therapies need better evidence, oversight

ScienceDaily

Potentially useful biomarker tests for molecularly targeted therapies are not being adopted appropriately into clinical practice because of a lack of common evidentiary standards necessary for regulatory, reimbursement, and treatment decisions, says a new report.

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Potentially useful biomarker tests for molecularly targeted therapies are not being adopted appropriately into clinical practice because of a lack of common evidentiary standards necessary for regulatory, reimbursement, and treatment decisions, says a new report by the National Academies of Sciences, Engineering, and Medicine. To enhance patient care and clinical outcomes, the report calls for the creation of a "rapid learning system" that would integrate research on these tests and associated treatments with clinical practice. Appropriate regulatory oversight is also needed to ensure that biomarker tests and targeted therapies are accurate, reliable, and properly validated and implemented.

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, chair of the committee that wrote the report, and Ingram Professor of Cancer Research, professor of medicine, pathology, microbiology, and immunology, and chair of the department of cancer biology at Vanderbilt University. "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."....