TY - JOUR
T1 - Economic and developmental impacts of FDA designations
T2 - a systematic review and meta-analysis
AU - Williamson, Joab
AU - Hasan, Syed Shahzad
AU - Gc, Vijay S.
N1 - Publisher Copyright:
© 2025 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
PY - 2025/5/21
Y1 - 2025/5/21
N2 - Introduction: U.S. FDA designations: Breakthrough Therapy (BTD), Fast Track (FTD), Orphan Drug (ODD), and Regenerative Medicine Advanced Therapy (RMAT) aim to expedite drug development, yet their combined economic and developmental effects have not been extensively studied. This systematic review evaluates their impacts on Day 1 cumulative average abnormal returns (CAAR) and timelines from Investigational New Drug submission to approval. Methods: A systematic search of PubMed, Scopus, ScienceDirect, GreyNet, OpenGrey, ProQuest, and Cochrane Library was conducted for articles published between January 1997 and September 2024. A random-effects model generated pooled estimates with 95% confidence intervals (CI), and heterogeneity was assessed using the Cochrane-Q and I2 statistic. Study quality was evaluated using Drummond’s checklist and an adapted Barker checklist. Results: Twenty-five studies were included. The pooled Day 1 CAAR across designations was 6.12% (95% CI: 3.64–8.61). Subgroup analysis revealed FTD with the strongest immediate market impact (8.20%%, 95% CI: 4.38–12.03) and BTD with the shortest mean approval timeline (69.96 months, 95% CI: 60.25–79.67). Conclusions: FDA designations provide economic advantages, especially for smaller companies, and can expedite approvals for high-priority therapies. Notable heterogeneity, particularly with RMAT, warrants further research to clarify how disease area and company size shape real-world outcomes. Registration: ResearchRegistry ID11080.
AB - Introduction: U.S. FDA designations: Breakthrough Therapy (BTD), Fast Track (FTD), Orphan Drug (ODD), and Regenerative Medicine Advanced Therapy (RMAT) aim to expedite drug development, yet their combined economic and developmental effects have not been extensively studied. This systematic review evaluates their impacts on Day 1 cumulative average abnormal returns (CAAR) and timelines from Investigational New Drug submission to approval. Methods: A systematic search of PubMed, Scopus, ScienceDirect, GreyNet, OpenGrey, ProQuest, and Cochrane Library was conducted for articles published between January 1997 and September 2024. A random-effects model generated pooled estimates with 95% confidence intervals (CI), and heterogeneity was assessed using the Cochrane-Q and I2 statistic. Study quality was evaluated using Drummond’s checklist and an adapted Barker checklist. Results: Twenty-five studies were included. The pooled Day 1 CAAR across designations was 6.12% (95% CI: 3.64–8.61). Subgroup analysis revealed FTD with the strongest immediate market impact (8.20%%, 95% CI: 4.38–12.03) and BTD with the shortest mean approval timeline (69.96 months, 95% CI: 60.25–79.67). Conclusions: FDA designations provide economic advantages, especially for smaller companies, and can expedite approvals for high-priority therapies. Notable heterogeneity, particularly with RMAT, warrants further research to clarify how disease area and company size shape real-world outcomes. Registration: ResearchRegistry ID11080.
KW - breakthrough therapy designation
KW - drug development timelines
KW - fast track designation
KW - FDA designations
KW - orphan drug designation
KW - pharmacoeconomics
KW - regenerative medicine advanced therapy
UR - http://www.scopus.com/inward/record.url?scp=105005838500&partnerID=8YFLogxK
U2 - 10.1080/14737167.2025.2507426
DO - 10.1080/14737167.2025.2507426
M3 - Review article
C2 - 40387898
AN - SCOPUS:105005838500
JO - Expert Review of Pharmacoeconomics and Outcomes Research
JF - Expert Review of Pharmacoeconomics and Outcomes Research
SN - 1473-7167
ER -
