Lee Myrto, Larose Hugo, Gräbeldinger Martin, Williams Jon, Baird Anne-Marie, Brown Susan, Bruns Johannes, Clark Russell, Cortes Javier, Curigliano Giuseppe, Ferris Andrea, Garrison Louis P, Gupta Y K, Kanesvaran Ravindran, Lyman Gary, Pani Luca, Pemberton-Whiteley Zack, Salmonson Tomas, Sawicki Peter, Stein Barry, Suh Dong-Churl, Velikova Galina, Grueger Jens
Boston Consulting Group, London, UK.
Boston Consulting Group, Paris, France.
Health Policy Open. 2024 Mar 1;6:100116. doi: 10.1016/j.hpopen.2024.100116. eCollection 2024 Dec.
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).
向癌症早期检测和治疗的转变给使用传统终点进行价值评估带来了挑战。当前的癌症管理很少考虑治疗方法的全部经济和社会效益。我们的研究采用了改良的德尔菲法来制定定义和评估癌症治疗价值的原则，这些原则与肿瘤学研究的当前轨迹相一致，并反映了更广泛的价值观念。24名专家在5个月内参与了共识建立活动（16人参与了结构化互动，包括一项调查、全体会议、访谈和线下讨论，而8人参与了访谈）。讨论集中在：1）哪些与肿瘤学相关的终点应用于评估早期癌症的治疗以及早期治疗的准入决策，以及2）其他价值组成部分的重要性以及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（考虑总生存期以外的与肿瘤学相关的终点；为能更早显示疗效的终点建立证据；为下一代预测措施建立证据；使用由持续证据收集支持的有管理的准入协议来满足决策者的证据需求）和第二个领域的3项原则（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）达成了共识。
