The FDA Breakthrough Therapy Designation: A Primer



By Peter Martin, M.D.

The Breakthrough Therapy Designation was introduced as part of the 2012 Food and Drug Administration Safety and Innovation Act and is designed to expedite the development of new treatments for serious conditions like lymphoma. If preliminary evidence from clinical trials demonstrates that a new drug represents a significant improvement over currently available therapies, the drug developer (also called the Sponsor) may request a Breakthrough Therapy Designation.

If the FDA agrees with the Sponsor and grants the designation, they will subsequently commit to providing additional resources to the development and review process.  Practically this means  more frequent meetings and communications with senior FDA officials, and aid in designing more efficient clinical trials. Although the Breakthrough Therapy Designation does not make a new drug available, early experience with the program suggests that it can shave years off of the typical development process.

Since 2012, the Sponsors of 342 treatments have applied for this status, with 111 receiving the designation. Of the treatments that have received the designation 42 have received full approval. In 2016 pembrolizumab received the designation for the treatment of patients with relapsed or refractory classical Hodgkin lymphoma and venetoclax received it in combination with rituximab to treat patients with relapsed/refractory chronic lymphocytic leukemia. Previously nivolumab, idelalisib, ofatumumab, obintuzumab, and ibrutinib all received the Breakthrough Therapy Designation for different lymphoma indications.

Despite the success of the program there are several points to keep in mind. The FDA’s definition of “breakthrough” is very different from its interpretation by lay persons and the media. The FDA’s definition refers to a drug that in the early stages of development has shown the potential for an improvement in patient care. It is not a guarantee of the approval or long-term success of the treatment.

As the FDA and Sponsors gain additional experience with the program we are likely to see some changes, including the number of applications and the cost of requesting a Breakthrough Therapy Designation. We are also likely to witness some unintended consequences, including investments by venture capitalists and opaque marketing strategies. When in doubt a patient should consult a physician about the usefulness of any treatment.

At the Weill Cornell Lymphoma Program, we are interested in any program that helps make promising new therapies available to patients as quickly as possible, and so we continue to follow this and other related programs closely. In the next post in the series we will discuss the Fast Track Designation.

Previous Entries in the Primer Series

The FDA Approval Process

Author: lymphomaprogram

Located on the Upper East Side of New York City, the Lymphoma Program at Weill Cornell Medical College/NewYork Presbyterian Hospital is internationally recognized for our efforts to enable patients with non-Hodgkin lymphoma, Hodgkin disease and related disorders to have the best possible clinical outcome, including cure when possible.

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