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Record Percentage of New Therapies FDA Approved in 2018 Includes Label Specifying Which Patients Will Likely Realize Outsized Benefits or Experience Fewer Side Effects, Analysis Shows

Report Suggests Personalized Medicine Will Play Increasingly Prominent Role in Improving Efficiency, Effectiveness of Health Care

WASHINGTON (Feb. 13, 2019) 


In a development that demonstrates the emergence of a new set of tools and therapies designed to help health systems improve patient care and make better use of scarce resources by targeting treatments to only those patients who will benefit from them, the Personalized Medicine Coalition (PMC) today released a report documenting that more than one of every three therapies the U.S. Food and Drug Administration (FDA) has approved in the last two years includes information on its label that identifies patients who are most likely to realize outsized benefits or experience fewer side effects from the treatment.

Personalized Medicine at FDA: A Progress & Outlook Report suggests that U.S. policymakers could help address rising health care costs by putting policies in place that encourage personalized medicine, which is defined by the use of diagnostic tests to inform which medical treatments will work best for each patient.

The report notes that personalized medicines have accounted for more than 20 percent of FDA approvals for the last five years and set a record in 2018 by topping 40 percent of approvals. The report credits policies recently advanced by FDA for fostering a favorable environment for innovation that has accelerated this trend toward personalized medicine. But as the report also notes, some emerging public policies still assume that providers and payers will continue to make decisions based on data from population averages, instead of embracing personalized medicine.

The Trump administration's recent decision to encourage commercial insurers administering Medicare benefits through the Medicare Advantage program to implement "step therapy" policies that require patients to try cheaper treatments before gaining access to more expensive options under Medicare Part B, for example, ignores the documented trend toward therapies that have information in their labels about the populations they will likely benefit. The report notes that by the time a patient who will likely respond to a personalized therapy first completes a less expensive, one-size-fits-all course of treatment for a disease like cancer, the disease may have progressed so far that the personalized treatment can no longer help.

"Driving treatment decisions by cost considerations rather than determining what treatment would work best for an individual patient fundamentally conflicts with personalized medicine, and in many cases will increase downstream costs brought on by continued progression of disease and more adverse side effects," the report reads.

PMC President Edward Abrahams noted that "it falls on Congress, the Centers for Medicare and Medicaid Services (CMS), and FDA to continue implementing public policies that recognize the principles of the field, which are responsible for the burst of innovation that the report documents.”


Christopher J. Wells
Phone: 202-589-1755

About Personalized Medicine at FDA: A Progress & Outlook Report:
PMC's Personalized Medicine at FDA: A Progress & Outlook Report, which was developed under the direction of Senior Vice President for Science Policy Daryl Pritchard, Ph.D., summarizes the U.S. Food and Drug Administration (FDA)'s progress in 2018 in reviewing diagnostics and treatments that can guide personalized health care strategies. The report defines personalized medicines as "those therapeutic products for which the label includes reference to specific biological markers, identified by diagnostic tools, that help guide decisions and/or procedures for their use in individual patients."

About the Personalized Medicine Coalition:
The Personalized Medicine Coalition, representing innovators, scientists, patients, providers and payers, promotes the understanding and adoption of personalized medicine concepts, services and products to benefit patients and the health system. For more information about PMC, please visit

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