Biomarker tests for 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. Hundreds of molecularly targeted agents have entered the drug development pipeline in recent years, and numerous biomarker tests and associated therapies have been approved for clinical use in treating cancer and other diseases.
“The objective of this report is to call for and frame evidentiary standards that might be applied to biomarkers so that patients, health care providers, and payers can determine the clinical validity and utility of what is on offer,” said Garret FitzGerald, MD, FRS, director of the Institute for Translational Medicine and Therapeutics (ITMAT) and chair of the department of Systems Pharmacology and Translational Therapeutics at the University of Pennsylvania's Perelman School of Medicine. “Presently it is the Wild West.” FitzGerald is a member of the NAS advisory committee that wrote the report.
The broader implementation of these potentially useful tests into clinical practice is slowed by the lack of common standards of evidence of the clinical benefit of the tests, which are necessary for regulatory, coverage, and reimbursement decisions. The committee’s integrated set of recommendations, presented within the framework of a rapid learning system, aims to establish the evidence for initial clinical use of these tests, while enabling ongoing assessment of their benefit. The overarching goal of the committee’s recommended actions is to significantly enhance patients’ access to appropriate, accurate, and reliable biomarker tests to direct targeted therapy.
Considered essential to the advancement of precision medicine, new initiatives such as the White House Cancer Moonshot Initiative are likely to increase interest in these tests and treatments. However, progress has been uneven and hampered by regulatory and reimbursement uncertainties, clinical practice challenges, and other issues, note report authors.
To obtain a copy of the report, contact the Academies’ Office of News and Public Information; tel. 202-334-2138 or email [email protected].
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