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

IF 1.8 4区医学 Q3 HEALTH CARE SCIENCES & SERVICES

Expert Review of Pharmacoeconomics & Outcomes Research Pub Date : 2025-05-21 DOI:10.1080/14737167.2025.2507426

Joab Williamson, Syed Shahzad Hasan, Vijay S Gc

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引用次数: 0

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.

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FDA指定的经济和发展影响：系统回顾和荟萃分析。

简介：美国FDA指定：突破性治疗（BTD），快速通道（FTD），孤儿药（ODD）和再生医学高级治疗（RMAT）旨在加快药物开发，但其综合经济和发展效应尚未得到广泛研究。本系统综述评估了它们对第1天累积平均异常回报（CAAR）的影响以及从研究新药提交到批准的时间表。方法：系统检索PubMed、Scopus、ScienceDirect、GreyNet、OpenGrey、ProQuest和Cochrane Library，检索1997年1月至2024年9月间发表的文章。随机效应模型产生95%置信区间（CI）的汇总估计，并使用Cochrane-Q和I2统计量评估异质性。使用Drummond的检查表和Barker的改进检查表评估研究质量。结果：纳入25项研究。各指定的合并第1天CAAR为5.94% （95% CI: 3.69-8.18）。亚组分析显示，FTD具有最强的即时市场影响（7.72%,95% CI: 4.73-10.67）， BTD具有最短的平均批准时间（69.96个月，95% CI: 60.25-79.67）。结论：FDA的指定提供了经济优势，特别是对小公司来说，并且可以加快高优先级治疗的批准。值得注意的异质性，特别是RMAT，需要进一步研究以阐明疾病区域和公司规模如何影响现实世界的结果。注册号：ResearchRegistry ID11080。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Expert Review of Pharmacoeconomics & Outcomes Research HEALTH CARE SCIENCES & SERVICES-PHARMACOLOGY & PHARMACY

CiteScore

4.00

自引率

4.30%

发文量

审稿时长

6-12 weeks

期刊介绍： Expert Review of Pharmacoeconomics & Outcomes Research (ISSN 1473-7167) provides expert reviews on cost-benefit and pharmacoeconomic issues relating to the clinical use of drugs and therapeutic approaches. Coverage includes pharmacoeconomics and quality-of-life research, therapeutic outcomes, evidence-based medicine and cost-benefit research. All articles are subject to rigorous peer-review. The journal adopts the unique Expert Review article format, offering a complete overview of current thinking in a key technology area, research or clinical practice, augmented by the following sections: Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results Article Highlights – an executive summary of the author’s most critical points.