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Seventh Annual Edition of PMC’s Personalized Medicine at FDA Finds Field Expanding Across Disease States, Shows Personalized Medicines Accounting for More Than a Third of Newly Approved Drugs

The Personalized Medicine Coalition (PMC) today released the seventh edition of its annual report titled Personalized Medicine at FDA, documenting the U.S. Food and Drug Administration’s approval last year of 17 personalized therapies, two cell-based immunotherapies, and significant new diagnostic indications for nine testing platforms that will help physicians target treatments to only those patients who will benefit. With personalized medicines accounting for more than 25 percent of newly approved drugs for each of the last seven years and topping one-third of new drug approvals for four of the last five, PMC President Edward Abrahams said the report’s findings “leave no question” that “the era of personalized medicine is upon us.”

Authored by PMC Senior Vice President for Science Policy Daryl Pritchard, Ph.D., Personalized Medicine at FDA: The Scope & Significance of Progress in 2021 describes how newly approved tests and therapies will help transform care for molecularly selected subsets of patients. Although the report shows continued progress for personalized medicine in oncology, it also reveals an expanding footprint for the field across other disease states. More than half of the newly approved personalized treatments are indicated for rare, common, or infectious diseases.

Continued Progress in Oncology

For cancer patients, the report spotlights how seven newly approved drugs, two newly approved cell-based therapies, and 20 newly approved indications for existing drugs will extend the promise of personalized medicine to more patients, including those patients whose tumors harbor KRAS genetic mutations once considered “undruggable.” New diagnostic approvals and FDA’s recognition of the validity of a public tumor-mutation database, the report notes, will help pave the way for continued progress toward an era of “tumor-agnostic” treatment in which treatment decisions are based on the presence of key biomarkers as opposed to a tumor’s location in the body.

Expanding Footprint Across Rare, Common, and Infectious Diseases

Seven newly approved drugs will give patients with rare diseases new personalized treatment options designed to target the molecular causes of their conditions. The newly approved rare disease drugs include a therapy that provides patients with a rare severe form of primary familial hypercholesterolemia, a genetically inherited disorder, with a new treatment designed to address the root cause of their disease. A second pathbreaking siRNA-based familial hypercholesterolemia therapy will provide an important new option for patients with more common forms of the disease, which affects about 1.3 million people in the United States.

Other newly approved drugs are indicated for patients with HIV and for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis, a disorder that causes progressive liver disease.

“With findings showing that personalized medicines have accounted for more than a quarter of newly approved drugs for each of the last seven years and for more than a third of the drugs approved since 2017, not to mention the approval of new or expanded indications for a countless number of paradigm-shifting diagnostic tests, Personalized Medicine at FDA: The Scope & Significance of Progress in 2021 leaves no question that the era of personalized medicine is upon us, presenting both opportunities and challenges for patients and health systems,” said PMC President Edward Abrahams.