创新的监管激励：FDA的突破性疗法认定
Regulatory Incentives for Innovation: The FDA's Breakthrough Therapy Designation
Review of Economics and Statistics · 2024 · [{"name": "Amitabh Chandra", "affiliation": ["Dana-Farber/Harvard Cancer Center"]}, {"name": "Jennifer L. Kao", "affiliation": ["University of California, Los Angeles", "UCLA Health"]}, {"name": "Kathleen L. Miller", "affiliation": ["United States Department of Health and Human Services", "United States Food and Drug Administration"]}, {"name": "Ariel Dora Stern", "affiliation": ["Harvard–MIT Division of Health Sciences and Technology"]}]
中文摘要
摘要：新产品监管者在加快产品上市与收集额外产品质量信息之间面临权衡。美国食品药品监督管理局（FDA）的突破性疗法认定（BTD）为理解监管者能否利用新政策围绕这一权衡进行创新提供了机会。我们发现，BTD 项目将临床开发时间缩短了 23%，且并未影响获得该认定药物的事后安全性特征。BTD 项目对经验较少的企业影响最大，并降低了临床试验设计的复杂性。结果表明，有针对性的监管创新可以在不损害产品质量的情况下缩短研发周期。
Abstract
Abstract Regulators of new products confront a trade-off between speeding a product to market and collecting additional product quality information. The FDA's Breakthrough Therapy Designation (BTD) provides an opportunity to understand if regulators can use new policy to innovate around this trade-off. We find that the BTD program shortened clinical development times by 23% and did not affect the ex post safety profile of drugs with the designation. The BTD program had the greatest impact on less experienced firms and reduced clinical trial design complexity. The results suggest that targeted regulatory innovation can shorten R&D periods without compromising product quality.