Personalized Medicine Approvals Continued to Surge in 2017

The FDA made significant strides to advance patient care in 2017, including the pioneering of developments in personalized medicine.

Remarkably, more than 1 in 4 drugs approved over the past 4 years have been a personalized medicine, according to a new report from the Personalized Medicine Coalition (PMC). The FDA approved 16 new personalized treatments in 2017 alone compared with a decade ago when these drugs only accounted for less than 10% of approvals, the PMC noted.

Additionally, the FDA expanded the indication of several existing treatments to reach new patients. Last year also marked the first approvals of 3 gene therapies and a personalized biosimilar drug.

The report highlighted that of the 46 new drug approvals in 2017, nearly 35% were deemed personalized therapies, which was higher than any recent year, according to the PMC.

The FDA continues to make progress towards precision health care. Since 2014, personalized drugs have accounted for more than 20% of FDA approvals, with their prevalence reaching 28% in 2016, according to the study.

Perhaps the most notable advancement was the FDA issuing the first gene therapy approval to tisagenlecleucel (Kymriah) for refractory B-cell precursor acute lymphoblastic leukemia, followed by the subsequent approvals of axicabtagene ciloleucel (Yescarta) for diffuse large B-cell lymphoma and Luxturna (voretigene neparvovec-rzyl) for rare vision loss disease.

The FDA also expanded the approval of well-established personalized treatments in 2017, including pembrolizumab (Keytruda), nivolumab (Opdvio), sofosbuvir (Sovaldi), and nilotinib (Tasigna).

The PMC noted that the expanded approval of pembrolizumab to include solid tumor types in advanced cancers with microsatellite instability-high or mismatch repair deficiency was noteworthy because it was the first to be granted based on a biomarker regardless of tumor location.

The FDA approved the first personalized biosimilar, trastuzumab-dkst (Oigivir), in 2017. The treatment is a biosimilar to trastuzumab (Herceptin) and is approved to treat HER2-positive breast cancer.

Direct-to-consumer genetic tests were also authorized in 2017. The 23and Me Personal Genome Service Genetic Health Risk examines patients’ DNA to determine their risk for 10 diseases, according to the study. The agency, in collaboration with the Centers for Medicare and Medicaid Services, approved and covered the use of FoundationOne CDx, which is a next generation sequencing test.

These approvals and authorizations represent continuing efforts to bring new and innovative drugs to patients who may not otherwise have treatment options, according to the PMC.

“While ongoing challenges in the areas of scientific discovery, diagnostic regulatory policy, coverage and reimbursement, and implementation of new technologies into clinical practice are still outstanding, the science leading health care away from one-size-fits-all, trial-and-error medicine and toward the utilization of molecular information to improve outcomes and make the US health system more efficient is clear,” the authors concluded.