FDA’s Breakthrough Therapy Designation

Many of you may have heard of the FDA’s Fast Track Development program. For drugs that treat a serious medical condition and address an unmet medical need, the FDA will provide expedited review. In fact, the FDA has 4 different accelerated review processes:

  • Breakthrough Therapy
  • Priority Review
  • Accelerated Approval
  • Fast Track

These other 3 programs, however, have been in effect for 15 years already, the Breakthrough Therapy Designation (BTD) is the new kid on the block. A breakthrough therapy is one where the treatment of interest shows significant improvement over existing therapy for a serious or life-threatening disease.  Brookings summarizes the BTD program as:

BTD was established in 2012 under the Food and Drug Administration Safety and Innovation Act, and is intended to expedite the development and review of drugs that show signs of extraordinary benefit at early stages of the clinical development process. However, BTD is not an automatic approval. The drug still has to undergo clinical testing and review by the FDA. Rather, BTD is designed to facilitate and shorten the clinical development process, which can otherwise take many years to complete.

The FDA considers the following criteria when considering a treatment for BTD:

  1. the quantity and quality of the clinical evidence being submitted;
  2. the available therapies that the drug is being compared to; and
  3. the magnitude of treatment effect shown on the outcome being studied.

Of the 293 requsts for a BTD designation to date, 82 have successfully received the designation, and 23 of these have subsequently been approved for marketing. Note that 10 of the 23 approvals were actually for new indications for previously-approved drugs.

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