A Breakthrough Therapy, in addition to treating a serious or life-threatening condition, demonstrates early clinical improvement over existing therapies. Sponsors of Breakthrough drugs meet with FDA officials early and often to ensure the most efficient possible path to approval.


  • A Breakthrough Therapy drug must address a serious or life-threatening condition and
  • The sponsor must show a high magnitude of clinical activity indicating that the drug may demonstrate substantial improvements over existing therapies.

When to Submit a Request:


  • Drug sponsors get extra opportunities to meet with the FDA and discuss study design, safety and efficacy requirements, dose-response concerns, use of biomarkers, and other critical development issues.
  • FDA may consider rolling review, allowing early review of portions of a drug sponsor’s marketing application before the complete application has been submitted.
  • Due to their large early clinical effect, Breakthrough drugs can sometimes skip portions of the standard FDA review process without compromising safety and efficacy standards.
  • Drug sponsors get increased exposure to FDA senior managers, experienced review staff, and cross-disciplinary experts to help coordinate internally and aid in efficient development and review of clinical and non-clinical components of the application.
  • Because Breakthrough drugs have early ability to benefit patients, the FDA aims to collaboratively examine a Breakthrough drug’s entire development program and take scientifically appropriate steps to minimize the number of patients receiving placebos or less efficacious treatment as part of the testing process.

Breakthrough Therapy vs. Fast Track:

  • Breakthrough has stricter requirements than Fast Track—Breakthrough drugs must show clinical evidence of improvement over existing therapies
  • Breakthrough has greater benefits—the FDA grants additional early attention and personnel to ensure that they avoid any redundancies or unnecessary steps in the review process

The Perfect Candidate: The ideal Breakthrough Therapy drug would employ a newly-understood mechanism to achieve obvious benefits over more dated therapies, e.g. a cancer drug addressing a new molecular target with overwhelming early clinical success.