The U.S. Food and Drug Administration (FDA) approved the most novel oncology drugs in 2015 compared with the past 19 years. Researchers evaluated factors associated with the significant increase in FDA-approved oncology agents over the past five years. The researchers presented the study at the ASCO Annual Meeting. 

From 2011 to 2015, 47 novel oncology drugs were approved, of which 72.3% (n = 34) were new drug applications and 27.7% (n = 13) were biologic license applications. Most were orphan drugs (64.5%; n = 35), whereas 40.4% (n = 19) were first-in-class. 

The drugs received the following review status.

  • Priority review (76.6%; n = 36)
  • Fast track (57.5%; n = 27)
  • Accelerated (36.2%; n = 17)
  • Breakthrough designation (23.4%; n = 11)

Most approvals (66%; n = 31) were for solid tumors, whereas 34% were for liquid cancers (n = 16). 

Drug approvals were based on the following primary endpoints: clinical response (42.6%; n = 20), progression-free survival (34%; n=16), and overall survival (23.4%; n = 11). The median time from initiation of clinical trial to FDA approval was 42 months (range = 17–78), and trials completed within a median of less 42 months were more likely to receive fast track designation (36.2%) compared with those requiring a longer time (21.3%; P=0.03). 

The researchers divided the study period into three time points. 

  1. January 2011–August 2012 (27.7% of approvals; n = 13)
  2. September 2012–April 2014 (31.9%; n = 15)
  3. May 2014–December 2015 (40.4%; n = 19)

Over time, the data showed an increased trend in orphan designations: 14.9%, 25.5%, and 34%, respectively (p = 0.13), as well as an increase (although not significant) in accelerated approvals: 15.4%, 33.3%, and 52.6%, respectively (p = 0.09). Among those three time points, no changes were indicated with regard to application type, phase of clinical trial, and type of trial endpoint.

The researchers concluded, “The majority (65%) of new cancer drugs were approved under the orphan drug designation, which has increased over the past five years. The median time from clinical trial initiation to FDA approval was 42 months. The increasing trend for novel drug approvals through these expedited programs based on shorter and smaller clinical trials warrants further investigation.”

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