Economic and developmental impacts of FDA designations: a systematic review and meta-analysis

Joab Williamson; Syed Shahzad Hasan; Vijay S. Gc

doi:10.1080/14737167.2025.2507426

Economic and developmental impacts of FDA designations: a systematic review and meta-analysis

Joab Williamson, Syed Shahzad Hasan, Vijay S. Gc

Research output: Contribution to journal › Review article › peer-review

6 Citations (Scopus)

Abstract

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.

Original language	English
Pages (from-to)	1051-1061
Number of pages	11
Journal	Expert Review of Pharmacoeconomics and Outcomes Research
Volume	25
Issue number	7
Early online date	21 May 2025
DOIs	https://doi.org/10.1080/14737167.2025.2507426
Publication status	Published - 1 Sept 2025

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SDG 3 Good Health and Well-being

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Cite this

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title = "Economic and developmental impacts of FDA designations: a systematic review and meta-analysis",

abstract = "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{\textquoteright}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.",

keywords = "breakthrough therapy designation, drug development timelines, fast track designation, FDA designations, orphan drug designation, pharmacoeconomics, regenerative medicine advanced therapy",

author = "Joab Williamson and Hasan, \{Syed Shahzad\} and Gc, \{Vijay S.\}",

note = "Publisher Copyright: {\textcopyright} 2025 The Author(s). Published by Informa UK Limited, trading as Taylor \& Francis Group.",

year = "2025",

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doi = "10.1080/14737167.2025.2507426",

language = "English",

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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.

PY - 2025/9/1

Y1 - 2025/9/1

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 - https://www.scopus.com/pages/publications/105005838500

U2 - 10.1080/14737167.2025.2507426

DO - 10.1080/14737167.2025.2507426

M3 - Review article

C2 - 40387898

AN - SCOPUS:105005838500

SN - 1473-7167

VL - 25

SP - 1051

EP - 1061

JO - Expert Review of Pharmacoeconomics and Outcomes Research

JF - Expert Review of Pharmacoeconomics and Outcomes Research

IS - 7

ER -

Economic and developmental impacts of FDA designations: a systematic review and meta-analysis

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