Presentation (CTI)

OBJECTIVES: Drug affordability is a critical issue in the US. Some observers point to increasing launch prices for new drugs as a barrier to patient access. This concern may have validity if new drugs are the same as existing drugs: treating the same conditions and patients in similar ways. This research aims to evaluate FDA’s review and approval designations for drugs approved 2012-2024 to assess trends in the innovativeness of new drugs.
METHODS: Data on initial approvals and review designations for novel drugs were abstracted from FDA-CDER’s New Drug Therapy Approvals annual reports and CBER’s Biological Approvals by Year website, with supplemental data from peer-reviewed literature from 2012-2024. FDA designations for expedited review, including Accelerated Review, Breakthrough Therapy, Fast Track, Orphan, and Priority Review, were identified. Designation of “First in Class” for drugs approved by CDER 2015-2024 were also recorded. Blood products, diagnostic agents, and human tissue products were excluded.
RESULTS: Of the 630 included drugs from 2012-2024, 70% received one or more expedited review designation with a rising trend since 2017, peaking at 82% in 2024. Highest rates of any expedited designation over the study period were in oncology (94%) and rare disease (88%). Use of Accelerated Approval was relatively stable over the study period at 17% for all drugs, with the greatest use in oncology (44%), with a peak of 69% in 2021. Use of Accelerated Approvals were less than 10% in other therapy areas. About 40% of included CDER-approved drugs were classified as First in Class with a rising trend since 2020; highest rates were in rare disease (62%) and cardiovascular disease (56%).
CONCLUSIONS: Trends in FDA review designations indicate that measures of innovativeness of new drugs are stable or increasing. Assessments of drug launch prices should account for the scientific advancements inherent in new products that reflect continuing innovation.