e18270 Background: With the importance of speed-to-market and addressing unmet needs, pharmaceutical companies have sought accelerated approvals through the Food and Drug Administration (FDA). Introduced with the FDA Safety and Innovation Act (FDASIA) of 2012, Breakthrough Therapy Designation (BTD) has become an important mechanism for approval of serious and life-threatening conditions that do not have adequate therapies. Notably, these pathways have been ill-understood by both pharmaceutical companies and health care providers. This study assessed how BTD and other FDA designations have played a role in the approval of oncology drug marketing applications and evaluated trends in the use of these regulatory pathways. Methods: We analyzed publicly available data on novel oncology drug approvals by the FDA from 2012-2016, including the 4 expedited programs for serious conditions (BTD, Accelerated Approval, Fast Track, and Priority Review). Results: Of the 43 novel oncology drugs approved by the FDA between 2012-2016, 42 used at least 1 of the expedited approval programs, including 65% that used ≥2 programs and 35% that used ≥3 programs. The BTD has been used by 15 of the 43 (35%) approved novel oncology drugs since 2012. The use of the BTD, Accelerated Approval pathway, and Priority Review designation among approved oncology drugs has generally increased each year from 2012-2016, while the use of the Fast Track designation has decreased over the same time period. Conclusions: Companies seeking oncology approvals often use more than one expediting strategy. Alone or in combinations, the BTD, Accelerated Approval, and Priority Review have been shown to play an increasingly important role in oncology drug development. Data collected between 2012-2016 suggest that use of BTD is growing more common, while use of the Fast Track designation has decreased among approved oncology drugs. Additional expedited approval programs have remained steady or increased since the FDA introduced BTD. Based on these observations, we anticipate use of the BTD, Accelerated Approval, and Priority Review designation will grow in future oncology drug applications.
e13578 Background: Following approval of the Food and Drug Administration (FDA) Safety and Innovation Act in 2012, the FDA launched a 3-year plan to promote innovation, increase stakeholder involvement, secure the drug supply chain, and fund these endeavors by collecting user fees. The FDA created its fourth expedited pathway, Breakthrough Therapy Designation (BTD) to focus on the development and approval of therapies to treat serious and life-threatening conditions that lack adequate therapies. Our 2017 study (ASCO abstract #e18270) identified a trend of increased use of these approaches with oncology drugs, particularly the BTD, which was a nascent pathway at the time. We aimed to reexamine FDA pathway and approval trends in oncology interventions. Methods: We analyzed publicly available data on novel drug approvals by the FDA’s Center for Drug Evaluation and Research (CDER) from 2012-2020 and the 4 expedited pathways (BTD, Accelerated Approval, Fast Track Designation [FTD], and Priority Review). Results: Between 2012 and 2020, CDER approved 380 new chemical entities, including 101 oncology drugs. Due to data limitations, 3 novel oncology biologics (approved by the Center for Biologics Evaluation and Research) and 4 diagnostics were excluded from the analyses. Oncology drugs comprised a mean 27% (range 14-34%) of all approvals from 2012-2020, including 25% from 2012–2016 and 28% from 2017-2020. Of all approved oncology drugs from 2012-2020, 94% (range 82-100%) utilized at least one expedited pathway. Oncology approvals were more likely than non-oncology approvals to have used one or more expedited pathways. Use of these pathways for oncology approvals increased from 2012-2016 to 2017-2020: ≥2 (65% vs 78%) and ≥3 (35% vs 50%) pathways. BTD usage for oncology drugs increased from 35% in 2012-2016 to 57% in 2017-2020, though for 2012-2016, the time interval between awarding the (new) designation and the remainder of development activities must be considered. Presumably because BTD grants additional benefits over FTD, generally the use of FTD for oncology drugs has declined over time. The use of Priority Review and Accelerated Approval has remained the same. Despite the pandemic, for oncology and non-oncology drugs alike, approvals using pathways remained consistent between 2019 and 2020. Conclusions: Efficient FDA review plays an important role in oncology drug development. Since its inception in 2012, BTD has been adopted and expeditiously used, comprising more than half of all novel oncology drug approvals from 2017-2020. Our data show a gradual increase in approvals of drugs granted BTD, which, given the duration of drug development, likely reflects the time between the acceptance of the BTD request and remainder of the pre-submission development activities. As BTD was expressly developed to increase stakeholder interaction and prioritize innovation, the speed-to-adoption and use of this pathway among oncology interventions is promising.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.