Priority Review and Rare Diseases Reign Supreme for 2019

Based on the FDA’s count, there were 48 novel drug approvals in 2019 with 60% receiving FDA fast track status, priority review, or accelerated approval.  This count does not include vaccines, plasma products, or gene therapy products.  When the majority of drugs are being reviewed in an expedited time frame, it is a strong indicator that the market is shifting towards the specialty drug space.  The high number of expedited approvals begs the question, “how much of an advantage is an expedited approved when everyone else is getting the same treatment?”

Many of the newly approved agents are first-in-class and indicated for rare disease states.  Oncology approvals continue to outpace approvals for other disease states, with approximately 20% of drugs approved for the oncology space, whether for diagnosis or treatment.

Three new infectious disease agents were approved in 2019, which is a positive step in developing new therapies to combat antibiotic resistance.  Because of the Generating Antibiotics Incentives Now Act (GAIN Act), three agents (Fetroja, Recarbrio, and Xenleta) received priority review as Qualified Infectious Disease Products (QIDPs).  With the rise of antibiotic resistance, the need for new antibiotic innovation is increasingly important, and the FDA’s Safety and Innovation Act (FDASIA) seeks to promote their development.

What does 2020 hold?  PHSL highlights five top trends to watch out for in this year’s drug approval space:

  1. Digital Drugs – expect to see an increase in the approval of digital therapeutics and digitally enabled drugs, as technology moves into the mainstream pharmacy space.
  2. NASH Drugs – Drugs indicated to treat nonalcoholic steatohepatitis (NASH), or a fatty liver, are expected to be approved in 2020. With a high price tag and potentially huge patient market, payers should monitor these approvals closely.
  3. Gene Therapy – targeted therapies based on patients’ genetic factors promises to be a key area. This high-dollar space may be a cash cow for those targeting disease states with a sizeable patient population.
  4. Biosimilars – Will 2020 be the first year that an “interchangeable” biosimilar is approved? If not, we expect the number of biosimilar approvals to steadily increase, creating a more commodity-like atmosphere in the biologic space in products with multiple biosimilar competitors.
  5. Expedited Reviews and Approvals – As seen in 2019, expect the FDA to continue approving many products via the fast track, breakthrough, priority review, or accelerated approval processes. With the high number of specialty drugs being approved, this may become the new norm of drug approval.

A full listing of 2019 approvals, by approval type, is shown in the chart below.

2019 FDA New Drugs


Posted February 2020