New Report: How To Make Precision Medicine A Reality

NEW YORK, NY – Ever since President Obama mentioned it in his State of the Union Address, personalized (or precision) medicine—the idea of tailoring treatments to the unique biochemistry of individual patients—has been the hot topic in health care. Before customized treatments can become full reality, though, the FDA needs to play catchup with rapid advances in science outside the agency. In a new report out today, Manhattan Institute Senior Fellows Peter Huber and Paul Howard explain how to bridge the gap between precision medicine and FDA regulation.

Precision medicine has the potential to save countless lives and find valuable uses for treatments currently relegated to industry dustbins. For instance, a bladder cancer drug failed to elicit any response from more than 90 percent of patients in a trial at New York’s Memorial Sloan Kettering Cancer Center. Standard trial protocols, which date back to 1938, would have deemed the drug a failure. However, one patient’s cancer went into remission – becoming undetectable in just months. A genetic analysis of her tumor revealed that a particular mutation—a “biomarker” in regulatory jargon—made her tumor highly responsive to the treatment. Researchers found similar mutations in about 8% of bladder-cancer patients—corresponding with the tumor’s sensitivity to the drug.

Cases like this one raise the question of how many drugs that failed in clinical trials could be helping patients if we used the right tools to match patients to promising treatments. Huber and Howard’s report explains how the FDA can update its outdated clinical trial protocols to reflect the rapidly-advancing science of precision medicine, thereby bringing custom-tailored cures to patients – slashing years from clinical trials, improving patient outcomes, at lower cost than the currently regulatory framework allows.

The full report is available here.