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

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Tác giả: Vijay S Gc, Syed Shahzad Hasan, Joab Williamson

Ngôn ngữ: eng

Ký hiệu phân loại: 355.6213 Military administration

Thông tin xuất bản: England : Expert review of pharmacoeconomics & outcomes research , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 750221

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 I RESULTS: Twenty-five studies were included. The pooled Day 1 CAAR across designations was 5.94% (95% CI: 3.69-8.18). Subgroup analysis revealed FTD with the strongest immediate market impact (7.72%, 95% CI: 4.73-10.67) 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.
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