Precision medicine—tailoring treatments to the biochemistry of individual patients—has the potential to cure countless diseases. Molecular biomarkers are the foundation of this approach. Oncologists and many other doctors routinely prescribe drugs in ways that best fit patients’ biomarker profiles. The Food and Drug Administration (FDA), however, has been very cautious to incorporate biomarkers into the regulatory procedures for drug approval and, as a result, has significantly slowed the development of safe, effective treatments for many diseases.
This has left the FDA as a bystander to much of the ongoing revolution in molecular medicine. Molecular biomarker science is now being used at every other stage of the drug-development process and in many areas of medical practice. Ironically, much of the expertise about biomarkers can be found in the federal government itself—specifically at the NIH, which long ago expressed eagerness to help the FDA incorporate biomarkers into its approval process. In a recent report, Paul Howard and Peter Huber of the Manhattan Institute outline a path for reform of the FDA. The goal: anchor the FDA-approval process in the best available molecular biology; speed up regulatory decision making; and ensure that the FDA's review of biomarker submissions is based on a transparent, predictable, and efficient approach.
MI hosted a panel featuring nationally renowned disease and policy experts to discuss the specific approach that the FDA can take to base its approvals on science—to the great benefit of patients.