US Food and Drug Administration Approvals, Regulatory Pathways, and Review Time of New Drugs and Biologics, 1980–2024
Author(s)
Rosa Rodriguez-monguio, PhD, MS1, Enrique Seoane-Vazquez, PhD2, John H. Powers, III, MD3.
1School of Pharmacy, University of California San Francisco, San Francisco, CA, USA, 2Professor, Chapman University School of Pharmacy, Irvine, CA, USA, 3George Washington University School of Medicine, Washington, DC, USA.
1School of Pharmacy, University of California San Francisco, San Francisco, CA, USA, 2Professor, Chapman University School of Pharmacy, Irvine, CA, USA, 3George Washington University School of Medicine, Washington, DC, USA.
OBJECTIVES: U.S. legislation enacted since 1983 have aimed to enhance the development and marketing of new pharmaceutical products. We assessed trends in approvals, regulatory pathways and designations, and the review timed for new molecular entities (NMEs), therapeutic biologics (BLA), and gene and cell therapies approved by the FDA from 1980 to 2024.
METHODS: We collected regulatory information from the FDA databases drugs@FDA, FDA Label Search, Orange Book, and Purple Book, and DailyMeds. We assessed trends in the number of approvals, regulatory pathways and designations, and FDA review time. Descriptive analyses were performed.
RESULTS: RESULTS: Between 1980 and 2024, the FDA approved 1,475 new drugs, comprising 1,174 NMEs, 267 BLA, and 34 gene and cell therapies. The average annual drug approvals increased from 23.1 in the period 1980-Prescription Drug User Fee Act (PDUFA, 1992) to 47.4 after the enactment of the Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012. The FDA approved 51.5% of drugs using priority reivew, 14.5% accelerated approval, 29.5% fast-track designation, and 27.3% breakthrough therapy designation. Antineoplastic and immunomodulating agents led in approvals (24.4%) and in receiving expedited designations. The median FDA review time decreased from 26.6 (interquartile range 23.6) months in pre-PDUFA period to 10.0 (4.1) months in the FDASIA-2024 period.
CONCLUSIONS: CONCLUSIONS: From 1980 to 2024, the approval of new drugs increased significantly. A substantial proportion of these drugs were approved through expedited review pathways and designations, which do not require evidence of addressing unmet medical needs or offering superior patient benefits compared to existing alternatives. Further studies are needed to evaluate the therapeutic value of drugs approved through expedited pathways in meeting patient needs.
METHODS: We collected regulatory information from the FDA databases drugs@FDA, FDA Label Search, Orange Book, and Purple Book, and DailyMeds. We assessed trends in the number of approvals, regulatory pathways and designations, and FDA review time. Descriptive analyses were performed.
RESULTS: RESULTS: Between 1980 and 2024, the FDA approved 1,475 new drugs, comprising 1,174 NMEs, 267 BLA, and 34 gene and cell therapies. The average annual drug approvals increased from 23.1 in the period 1980-Prescription Drug User Fee Act (PDUFA, 1992) to 47.4 after the enactment of the Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012. The FDA approved 51.5% of drugs using priority reivew, 14.5% accelerated approval, 29.5% fast-track designation, and 27.3% breakthrough therapy designation. Antineoplastic and immunomodulating agents led in approvals (24.4%) and in receiving expedited designations. The median FDA review time decreased from 26.6 (interquartile range 23.6) months in pre-PDUFA period to 10.0 (4.1) months in the FDASIA-2024 period.
CONCLUSIONS: CONCLUSIONS: From 1980 to 2024, the approval of new drugs increased significantly. A substantial proportion of these drugs were approved through expedited review pathways and designations, which do not require evidence of addressing unmet medical needs or offering superior patient benefits compared to existing alternatives. Further studies are needed to evaluate the therapeutic value of drugs approved through expedited pathways in meeting patient needs.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR27
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas