The Personalized Medicine Coalition (PMC) recently published its “Personalized Medicine at FDA: 2017 Progress Report” (Report) that highlights drug approval developments and milestones achieved in 2017. Last year was notable for a record number of personalized medicine approvals by the FDA, and 5 “firsts” for the industry:

  1. Three gene therapies were approved;
  2. A tissue agnostic indication for cancer therapy was approved;
  3. 23andMe received approval of its direct to consumer genetic test;
  4. A personalized medicine biosimilar was approved; and
  5. A test using next-generation sequencing technology was jointly approved by FDA and CMS.

Record of New Drug Approvals

FDA approved 16 new personalized medicine therapies in 2017. The PMC defines personalized medicine therapies as those treatments for which the label includes reference to specific biological markers, identified with the assistance of a diagnostic test, that guide the use of the medicine or procedure in individual patients. The therapies approved in 2017 target cancer (e.g., breast cancer, metastatic Merkel cell carcinoma, non-small cell lung cancer, and myeloid leukemia), orphan diseases (e.g., CLN2 Batten disease and Mucopolysaccharidosis type VII (Sly syndrome), and infectious disease such as hepatitis C.

Two CAR-T gene therapies were approved, one for the treatment of acute lymphoblastic leukemia and the other for the treatment of large B-cell lymphoma. The first ever fully integrated gene therapy for the treatment of retinal dystrophy also was approved last year. And, Ogivri (trastuzumab-dkst), a biosimilar for Herceptin (trastuzumab), was approved for the treatment of HER-2 positive breast cancer.

Revlimid (lenalidomide), Keytruda (Pembrolizumab), Tecentriq (atezolizumab) are among several on-the-market drugs that received expanded indications in 2017 to treat new patient populations.

The PMC Report also highlights that FDA brought clarity to direct-to-consumer testing by its approval of 23andMe’s Personal Genome Service Genetic Health Risk product. This diagnostic product tests an individual’s genetic predisposition to certain medical diseases or conditions.

A joint approval by FDA and CMS under the Parallel Review program was announced – the second only in the history of these agencies. The agencies jointly approved Foundation Medicine’s FoundationOne CDx – a next-generation sequencing based platform.

Momentum Building

Much was accomplished in 2017, and as PMC correctly notes in its Report, the “extraordinary pace of scientific innovation in personalized medicine, progress that is largely due to the pharmaceutical and diagnostic industries’ commitment to personalized medicine as well as the leadership at FDA.”

That is not to say that challenges do not remain. Reimbursement, the expense and delay in the approval of personalized therapies, clinical adoption of personalized medicine and adequate research dollars for breakthrough technologies are impediments to realizing the full promise of personalized medicine. Nonetheless, the trend as a whole is moving in the right direction and here’s to further progress in 2018.