The evolving value assessment of cancer therapies: Results from a modified Delphi study

IF 1.7 Q3 HEALTH CARE SCIENCES & SERVICES Health Policy Open Pub Date : 2024-03-01 DOI:10.1016/j.hpopen.2024.100116
Myrto Lee , Hugo Larose , Martin Gräbeldinger , Jon Williams , Anne-Marie Baird , Susan Brown , Johannes Bruns , Russell Clark , Javier Cortes , Giuseppe Curigliano , Andrea Ferris , Louis P. Garrison , Y.K. Gupta , Ravindran Kanesvaran , Gary Lyman , Luca Pani , Zack Pemberton-Whiteley , Tomas Salmonson , Peter Sawicki , Barry Stein , Jens Grueger
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Abstract

The move toward early detection and treatment of cancer presents challenges for value assessment using traditional endpoints. Current cancer management rarely considers the full economic and societal benefits of therapies. Our study used a modified Delphi process to develop principles for defining and assessing value of cancer therapies that aligns with the current trajectory of oncology research and reflects broader notions of value. 24 experts participated in consensus-building activities across 5 months (16 took part in structured interactions, including a survey, plenary sessions, interviews, and off-line discussions, while 8 participated in interviews). Discussion focused on: 1) which oncology-relevant endpoints should be used for assessing treatments for early-stage cancer and access decisions for early-stage treatments, and 2) the importance of additional value components and how these can be integrated in value assessments. The expert group reached consensus on 4 principles in relation to the first area (consider oncology-relevant endpoints other than overall survival; build evidence for endpoints that provide earlier indication of efficacy; develop evidence for the next generation of predictive measures; use managed entry agreements supported by ongoing evidence collection to address decision-maker evidence needs) and 3 principles in relation to the second (routinely use patient reported outcomes in value assessments; assess broad economic impact of new medicines; consider other value aspects of relevance to patients and society).

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不断发展的癌症疗法价值评估:改良德尔菲研究的结果
癌症向早期检测和治疗发展,给使用传统终点进行价值评估带来了挑战。目前的癌症管理很少考虑疗法的全部经济和社会效益。我们的研究采用改良的德尔菲流程来制定癌症疗法价值的定义和评估原则,该原则符合当前肿瘤学研究的发展轨迹,并反映了更广泛的价值概念。24 位专家参加了历时 5 个月的建立共识活动(16 位参加了结构化互动,包括调查、全体会议、访谈和离线讨论,8 位参加了访谈)。讨论的重点是1) 评估早期癌症治疗和早期治疗准入决策时应使用哪些肿瘤相关终点,以及 2) 其他价值组成部分的重要性和如何将这些组成部分纳入价值评估。专家组就第一个领域的 4 项原则达成共识(考虑除总生存期以外的肿瘤相关终点;为能更早显示疗效的终点建立证据;为下一代预测性措施开发证据;使用由持续证据收集支持的管理下进入协议来满足决策者的证据需求),就第二个领域的 3 项原则达成共识(在价值评估中常规使用患者报告的结果;评估新药的广泛经济影响;考虑与患者和社会相关的其他价值方面)。
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来源期刊
Health Policy Open
Health Policy Open Medicine-Health Policy
CiteScore
3.80
自引率
0.00%
发文量
21
审稿时长
40 weeks
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