About Breakthrough Therapies

A new drug may be designated as a breakthrough therapy by the Food and Drug Administration (FDA) if it is intended to treat a serious or life-threatening disease and preliminary clinical evidence suggests it provides a substantial improvement over existing therapies. Once the breakthrough therapy designation is requested by the drug sponsor, the FDA and sponsor work together to determine the most efficient path forward.

As of June 30, 2024, the FDA has approved 317* breakthrough therapy designated products and lists that there have been 1516 total requests for the designation with 587 designations granted.

History of Breakthrough Therapy Designation

Working with our partners in all sectors, Friends took ‘Breakthrough’ from concept, to scientific whitepaper, to bipartisan legislative solution, to a tool in full use by FDA to expedite the approval of multiple drugs in 13 months. On November 1, 2013, only a year after being signed into law, and 29 designations being announced, the first Breakthrough designation received full FDA approval. Here is a list of 2013’s breakthrough designations and approvals. Of the year’s 28 publicly announced designations, 12 were for cancer drugs, as well as 2 of the FDA’s 3 breakthrough approvals.

The Issue

As knowledge about complex diseases such as cancer and HIV/AIDS has expanded, researchers have been able to develop increasingly precise methods of treatment. Targeted therapies, a variety of drugs that target specific molecular pathways, allow physicians and researchers to identify patients highly likely to respond to treatment. Frequently, these new drugs show major clinical activity and significant improvement over currently available treatment early in their development. In cases where new drugs show so much promise, and particularly in cases where existing treatment options are limited, the traditional multi-phase, sequential drug development process may not be appropriate.

Our Work Toward a Solution for Patients

On November 10, 2011, Friends of Cancer Research and the Engelberg Center for Healthcare Reform at Brookings co-hosted the fourth-annual Conference on Clinical Cancer Research, an event which brings together experts in cancer drug development from academia, industry, federal health and regulatory agencies, and patient advocacy. A panel from this conference discussed potential approaches to speed the FDA approval process for drugs that show large treatment effects early in development while still ensuring drug safety and efficacy.

Creating bipartisan legislative action

The “Advancing Breakthrough Therapies for Patients Act” was introduced in the Senate by Senators Bennet (D-CO), Hatch (R-UT), and Burr (R-NC) on March 26, 2012. Two months later, Congresswoman DeGette (D-CO) and Congressman Bilbray (R-CA) introduced the “Breakthrough Therapy Act” in the House of Representatives. The bills received bipartisan support, and were included as an amendment to the Food and Drug Administration Safety and Innovation Act, the latest iteration of the Prescription Drug User Fee bill. On July 9, 2012, the breakthrough therapy designation was signed into law.

A new drug may be designated as a breakthrough therapy if it is intended to treat a serious or life-threatening disease and preliminary clinical evidence suggests it provides a substantial improvement over existing therapies. Once the breakthrough therapy designation is requested by the drug sponsor, the FDA and sponsor work together to determine the most efficient path forward. The legislation mandates FDA guidance be developed outlining the criteria and process for the breakthrough therapy designation.

At the 2012 conference, Friends developed a white paper and panel discussion in the pursuit of encouraging use of the new pathway and informing FDA guidance.