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Beyond Breakthrough: Use of Innovative Drug Pathways at FDA

Beyond Breakthrough: Use of Innovative Drug Pathways at FDA

This is the first of two blog posts highlighting Friends’ upcoming September 20th event discussing the Breakthrough Therapy designation (BTD). In this post, we will summarize the recent history of this innovative drug approval pathway and also address potential areas of improvement.

BTD is a process that expedites the development and review of drugs intended to treat serious or life-threatening diseases with preliminary clinical evidence  demonstrating substantial improvement over existing therapies. Once an indication receives BTD, the U.S. Food and Drug Administration (FDA) and drug sponsor communicate more frequently with the intent to accelerate the approval of new drugs that address unmet needs for patients. BTD can be granted at any point during development but ideally should be requested before the end-of-phase-2 meetings. Friends reviewed documents from Drugs@FDA for novel oncology products approved between 2018 and 2021 and determined that sponsors with BTD drugs met more frequently with FDA once they received the designation (post-BTD) than before the designation (pre-BTD) (87 meetings post-BTD vs. 58 meetings total pre-BTD).

Friends used publicly available review documents on Drugs@FDA to develop a dashboard displaying the use of Expedited Programs in oncology. Since its inception in 2013, FDA granted 405 BTD requests and subsequently approved more than 200 of these applications. This includes 61 new oncology approvals. BTD has shortened the time to approval for new oncology products that received BTD by a median of 2.3 years compared to new oncology products without BTD. 

While this program has been invaluable in advancing oncology research, like all scientific programs, there are opportunities for improvement. Last year Friends published two white papers released during our 2020 Annual Meeting, titled “Modernizing Expedited Development Programs” and “Optimizing the Use of Accelerated Approval” that laid out recommendations for how expedited programs like BTD can be improved to better serve patients, the FDA, and drug sponsors.

Building on this previous work, Friends will host a virtual meeting on September 20, 2021, Beyond Breakthrough: Optimizing the BTD, to further discuss improvements to BTD.

Register today for our Sept. 20th meeting to find out more about these recommendations and hear from experts in the field. To read more about Friends’ work with breakthrough therapies, take a look at our webpage on BTD. 


Data Driven FDA