The Advancing Breakthrough Therapies for Patients Act was first introduced in the Senate by Senators Bennet ( D-CO), Hatch (R-UT) and Burr (R-NC). Two months later, Congresswoman DeGette (D-CO) and Congressman Bilbray (R-CA) introduced the act in the House of Representatives. The bills, which received bipartisan support, were included as an amendment to the Food and Drug Administration’s Safety and Innovation Act (FDASIA), the latest iteration of the Prescription Drug User Fee bill, and in July of 2012, the breakthrough therapy designation was signed into law.
As a result of this law, a new drug may be designated as a breakthrough therapy if it is intended to treat a serious or life-threatening disease, and if preliminary clinical evidence suggests it provides a substantial improvement over existing therapies. Once a breakthrough therapy designation is granted, the FDA and drug sponsor work together to determine the most efficient path forward. In just two years, 178 requests for breakthrough designation have been submitted, 44 designations have been granted, and 6 drugs have been approved within the program.
On May 6 of this year, Mary Hesdorffer, the executive director of the Meso Foundation, and Melinda Kotzian, its chief executive officer, attended the congressional briefing titled Friends of Cancer Research, which focused on evaluating the progress of this program.
The panel agreed that the next task will be to streamline the process. This will eliminate applications that absolutely do not meet the criteria for breakthrough designation.
Janet Woodcock, MD, director, of the Center for Drug Evaluation and Research within Food and Drug Administration, stated that they “are not planning on moving drugs through this process that offer only incremental progress, but those that demonstrate a substantial benefit.”
Two representatives of the pharmaceutical industry shared their companies’ experience in gaining the first designation under this act. They noted that the time and commitment of all parties involved made this process work.
Dr. Woodcock cautioned that sequestration had an impact on the financing of this program. It will take more funding to continue operating the Breakthrough Therapies program at the present level, but she hopes that streamlining the process will allow for a more drugs to reach the patients in need.