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Watch Now: “Beyond Breakthrough: Optimizing the Breakthrough Therapy Designation” 

Watch Now: “Beyond Breakthrough: Optimizing the Breakthrough Therapy Designation” 

On Monday, September 20th, 2021, Friends of Cancer Research (Friends) hosted a virtual event that brought together industry experts, the U.S. FDA, and patient advocates to discuss improving the Breakthrough Therapy designation (BTD) program. The panelists outlined opportunities to enhance the existing expedited drug development programs, including specific recommendations for the BTD, as outlined in the meeting white paper.

The live event was broadcast through YouTube and is available to watch here.

Ellen Sigal, Chair and Founder of Friends, opened the meeting by emphasizing how important BTD is for effectively and efficiently giving patients access to new and innovative treatments. Senator Michael Bennet (D-CO) then provided the opening keynote, praising the work conducted by Friends and members of Congress to establish BTD. The Senator also highlighted the designation’s important impact by helping patients get access to new, life-saving treatments for deadly diseases quickly and safely.

Jeff Allen, President and CEO of Friends, thanked Sen. Bennet and introduced Bea Lavery from Genentech, a Member of the Roche Group, the moderator of the first panel, made up of project working group members. Bea Lavery teed up the panel’s discussion of the work building the meeting white paper. Other panelists included Martha Donoghue and Atik Rahman from the FDA, Tina Kim Hafken from Seagen, and Giuseppe Randazzo from Novartis. The discussion focused on topics such as improving communication between FDA and drug sponsors, clarifying the type of evidence needed to receive BTD, supporting interdisciplinary coordination, and addressing rate-limiting factors in the BTD process.

All panelists underscored that BTD could be optimized through better communication. Martha Donoghue highlighted ways FDA could work to improve the BTD process, emphasizing the importance of proper timing for BTD requests and granting BTD, as well as dose optimization within the BTD program and. Atik Rahman also called attention to the white paper’s recommendation of pre-BTD meetings to discuss strategies for dose optimization and noted how the use of a dosing “snapshot” could help identify problems earlier. Guiseppe Randazzo emphasized the importance of “early, frequent, and timely communication” between FDA and sponsors as well as from FDA’s centers, including the Oncology Center of Excellence.

In the second panel, moderator Nicholas Florko from STAT News, spoke with Katherine Couvillon, a patient advocate, Sandra Horning, an Emerita Professor of Medicine at Stanford University, Michelle McMurry-Heath, the President and CEO of BIO, and Richard Pazdur, the director ofFDA’s Oncology Center of Excellence. The panel focused on expedited drug development programs as a whole and how the FDA can continue to best use them and ensure sustainability. The panel also discussed various aspects of the accelerated approval process, including the importance of confirmatory trials.

Katherine Couvillon spoke about her personal experience with the expedited pathways, noting how the pathways had given her and other patients more hope for future development of needed treatments. Sandra Horning and Michelle McMurry-Heath mentioned improvements that can be made to these expedited pathways, while noting they are still extremely useful for getting life-saving new treatments to patients. Richard Pazdur described how these expedited pathways could be simplified to reduce the amount of bureaucratic speedbumps. He also mentioned there should be an early discussion on “what constitutes the entire package for accelerated approval” when sponsors are preparing an application, such as determination of unmet medical need, agreement on endpoints and study design for the confirmatory study, and specifying key milestones

Read more about Friends work with the Breakthrough Therapy designation.

Watch a recording of the event.


Conference FDA Research Video