美国食品和药物管理局批准的实体瘤癌症单药和联合疗法的复杂性趋势。

IF 4.8 2区 医学 Q1 ONCOLOGY Oncologist Pub Date : 2024-11-15 DOI:10.1093/oncolo/oyae302
Emerson Y Chen, Manoj Rai, Yash Tadikonda, Preeyam Roy, Dakota W Nollner, Akshit Chitkara, Julia Hamilton, Rajat Thawani
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引用次数: 0

摘要

背景:美国食品及药物管理局(FDA)批准的许多癌症疗法,无论是作为多药联合疗法还是作为单药疗法,都只取得了有限的临床疗效。为了调查药物开发情况,本分析主要关注新批准的药物是作为联合疗法加入现有标准,还是作为单一疗法取代以前的药物:方法:我们对 2011 年 1 月至 2023 年 12 月期间治疗非血液学实体瘤恶性肿瘤的包装说明书和相应试验进行了回顾性分析,将批准的药物分为单一疗法和联合疗法。对两组药物的特征、治疗适应症、研究设计、批准历史和疗效结果进行了比较:结果:在 292 项审批条目和 110 种药物中,193 种(66.1%)为单一疗法,99 种(33.9%)为联合疗法。与单一疗法相比,联合疗法获得常规批准的频率高于加速批准(85 [85.9%] vs 132 [68.4%],P 讨论):鉴于与单一疗法相比,联合疗法的临床获益似乎有限,因此药物开发的重点应放在简化癌症疗法上,形成以患者为中心的范例。
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Trends in complexity of single-agent and combination therapies for solid tumor cancers approved by the US Food and Drug Administration.

Background: Many FDA-approved cancer therapies, whether as a multiagent combination or as a single agent, have demonstrated only modest clinical benefit. To investigate the drug development landscape, this analysis focuses on whether newly approved drugs are added to existing standards as combination therapy or replace a former drug as monotherapy.

Methods: A retrospective analysis of package inserts and corresponding trials for the treatment of nonhematology solid tumor malignancies from January 2011 to December 2023 was conducted to categorize an approval as monotherapy or combination therapy. Drug characteristics, treatment indications, study design, approval history, and efficacy results were compared between the 2 cohorts.

Results: Among the 292 approval entries and 110 drugs, 193 (66.1%) were monotherapies and 99 (33.9%) were combinations. Combinations, when compared with monotherapies, were more frequently approved as regular than accelerated approval (85 [85.9%] vs 132 [68.4%], P <.01), in the first-line setting (66 [66.7%] vs 69 [35.8%], P <.01), and with overall survival as the criteria (49 [49.5%] vs 40 [20.7%], P <.01). Monotherapies were more likely to be novel drugs compared with combinations (80 [41.5%] vs 14 [14.1%] P <.01). Monotherapies were more likely to be small molecule targeted agents, while combinations were more likely to be immunotherapies (P <.02). There was no difference comparing the time-to-event endpoints and validated clinical benefit scale, but the median response rate of combinations (46%) was higher than monotherapies (34%, P <.01).

Discussion: Given that clinical benefit appears limited in combination therapy compared with monotherapy, drug development could focus on simplifying cancer therapies toward patient-centered paradigms.

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来源期刊
Oncologist
Oncologist 医学-肿瘤学
CiteScore
10.40
自引率
3.40%
发文量
309
审稿时长
3-8 weeks
期刊介绍: The Oncologist® is dedicated to translating the latest research developments into the best multidimensional care for cancer patients. Thus, The Oncologist is committed to helping physicians excel in this ever-expanding environment through the publication of timely reviews, original studies, and commentaries on important developments. We believe that the practice of oncology requires both an understanding of a range of disciplines encompassing basic science related to cancer, translational research, and clinical practice, but also the socioeconomic and psychosocial factors that determine access to care and quality of life and function following cancer treatment.
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