美国癌症药物加速审批及转为常规审批所需的临床证据

IF 2.7 4区 医学 Q2 PHARMACOLOGY & PHARMACY Journal of Pharmaceutical Innovation Pub Date : 2024-09-04 DOI:10.1007/s12247-024-09851-9
Satoshi Kato, Shunsuke Ono
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引用次数: 0

摘要

目的肿瘤产品经常利用加速审批计划来争取更早在美国上市。人们对美国食品药品管理局(FDA)如何平衡证据与准入之间的关系,以及可能影响加速审批后要求的因素知之甚少。本研究旨在通过调查加速审批前与未来常规审批所需的补充临床证据之间的平衡,深入了解加速审批是如何确定的。方法根据公共数据库总结了肿瘤加速审批的产品特性、加速审批前的证据、理由、加速审批后的要求以及之前的监管指定。结果1992年至2021年间共批准了175项加速审批。加速审批前试验增加一项,补充试验就减少 20%。当产品用于治疗常见癌症时,加速审批后试验的终点往往更可靠,而当产品用于治疗的受试者较少、产品用于治疗呼吸系统癌症和皮肤癌或针对的恶性程度较低的癌症时,终点则不那么可靠。然而,在加速审批后的要求中也发现了相当大的差异。在确定加速审批和加速审批后的要求时,可能会考虑加速审批前证据的质量和数量、监管指定和操作可行性等因素。
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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Accelerated Approval for Cancer Drugs in the United States and the Clinical Evidence Required for Conversion to Regular Approval

Purpose

Oncology products often leverage accelerated approval program to seek earlier launches in the United States. Little is known about how the Food and Drug Administration (FDA) has been balancing evidence and access, and the factors that may characterize post-accelerated approval requirements. The present study aimed to obtain insights on how accelerated approval is determined by investigating the balance between pre-accelerated approval and supplemental clinical evidence required for future regular approval.

Methods

The product properties, pre-accelerated approval evidence, rationale, post-accelerated approval requirements, and prior regulatory designations for oncology accelerated approvals were summarized based on public databases. Regression analyses were performed to investigate factors that may have been associated with the post-accelerated approval requirements.

Results

Hundred fifty-seven accelerated approvals were granted between 1992 and 2021. An increase in the number of pre-accelerated approval trials by one trial resulted in a 20% decrease in supplemental trials. The endpoint for the post-accelerated approval trial tended to be more robust when products were indicated for common cancers, while less robust when products had been used to treat fewer subjects, when products were indicated for respiratory and skin cancers, or when less malignant cancers were targeted.

Conclusions

Accelerated approvals for oncology products were often based on the response rate of a noncomparative trial. However, considerable variation was observed in the post-accelerated approval requirements. Factors such as the quality and quantity of pre-accelerated approval evidence, regulatory designations, and operational feasibility may have been considered when determining the accelerated approvals and post-accelerated approval requirements.

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来源期刊
Journal of Pharmaceutical Innovation
Journal of Pharmaceutical Innovation PHARMACOLOGY & PHARMACY-
CiteScore
3.70
自引率
3.80%
发文量
90
审稿时长
>12 weeks
期刊介绍: The Journal of Pharmaceutical Innovation (JPI), is an international, multidisciplinary peer-reviewed scientific journal dedicated to publishing high quality papers emphasizing innovative research and applied technologies within the pharmaceutical and biotechnology industries. JPI''s goal is to be the premier communication vehicle for the critical body of knowledge that is needed for scientific evolution and technical innovation, from R&D to market. Topics will fall under the following categories: Materials science, Product design, Process design, optimization, automation and control, Facilities; Information management, Regulatory policy and strategy, Supply chain developments , Education and professional development, Journal of Pharmaceutical Innovation publishes four issues a year.
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