FDA Approved a Record Number of Personalized Medicines in 2017

One in every 4 drugs approved by the FDA over the past 4 years was a personalized medicine, and the agency approved a record-breaking 16 personalized therapies in the past year, according to a new report from the multi-stakeholder group the Personalized Medicine Coalition (PMC).

In the 2017 progress report on regulatory advances in personalized medicine (defined in the report as inclusive of all “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”), PMC hailed the approval of 3 new gene therapies: tisagenlecleucel (Kymriah) for the treatment of acute lymphoblastic leukemia; axicabtagene ciloleucel (Yescarta) for the treatment of large B-cell lymphoma; and voretigene neparvovecrzyl (Luxturna) for the treatment of retinal dystrophy.

The FDA also approved the first biosimilar of a personalized medicine in 2017 when it gave its authorization to trastuzumab-dkst (Ogivri) for the treatment of HER2-positive breast cancer. The drug was developed with Herceptin as its reference.

Also highlighted in the report as approval milestones are emicizumab-kxwh (Helimbra) for the treatment of hemophilia A; sofosbuvir, velpatasvir, and voxilaprevir (Vosevi) for the treatment of heptatitis C; and ribociclib (Kisquali) for the treatment of advanced breast cancer, among others.

Additionally, existing personalized therapies received expanded indications. Pembrolizumab (Keytruda), for example, received an expanded approval for all solid tumor types in advanced cancers with microsatellite instability-high or mismatch repair deficiency, marking the first occasion on which an oncology drug has been approved based on a biomarker without respect to the location of the tumor.

Yet it was not only drug approvals that made 2017 a banner year for personalized medicine, according to the report. The FDA’s authorization of direct-to-consumer health-related genetic tests, such as the 23andMe Personal Genome Service Genetic Health Risk test, which screens for biomarkers for diseases and conditions including as late-onset Alzheimer disease and Parkinson disease, “brought clarity to companies seeking to market genetic tests that provide information on an individual’s genetic predisposition to certain medical diseases or conditions,” said PMC.

The report also praised the FDA and CMS for issuing a joint approval and coverage decision for Foundation Medicine’s FoundationOne CDx, a broad companion diagnostic test clinically and analytically validated for solid tumors. PMC said that while the joint approval and clearance of the test has led to questions regarding the setting in which CMS will pay for next-generation sequencing tests, the approval and coverage decision “demonstrates the viability of the Parallel Review pathway.”

The successful approval and coverage decision for FoundationOne CDx was welcome news to PMC, which, earlier this year, voiced concern over the lack of a clear regulatory pathway for such companion diagnostics. While the FDA has released 2 draft guidance documents that describe the use of public human genetic variant databases to support clinical validity for next-generation sequencing—based in vitro diagnostics and the use of standards in the FDA’s oversight of these diagnostics for diagnosing germline diseases, these documents have yet to be finalized.

Despite a robust regulatory year in 2017, some hurdles to personalized medicine remain. In another recent report, PMC identified the reluctance of health systems to adopt personalized medicine in clinical practice as a significant factor slowing the use of personal data in clinical practice. Education—of physicians, patients, and payers—says PMC, may be crucial to paving the way for greater use of these tools and therapies.