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Adapting preference-based utility measures to capture the impact of cancer treatment-related symptoms
The European Journal of Health Economics ( IF 5.271 ) Pub Date : 2021-06-17 , DOI: 10.1007/s10198-021-01337-6
Koonal K Shah 1, 2 , Bryan Bennett 3 , Andrew Lenny 1 , Louise Longworth 1 , John E Brazier 2 , Mark Oppe 4 , A Simon Pickard 5 , James W Shaw 6
Affiliation  

It is important that patient-reported outcome (PRO) measures used to assess cancer therapies adequately capture the benefits and risks experienced by patients, particularly when adverse event profiles differ across therapies. This study explores the case for augmenting preference-based utility measures to capture the impact of cancer treatment-related symptoms. Additional cancer treatment-related items could be specific (e.g., rash) or global. While specific items are easier to describe and understand, their use may miss rarer symptoms and those that are currently unknown but will arise from future medical advancements. The appropriate number of additional items, the independence of those items, and their impact on the psychometric properties of the core instrument require consideration. Alternatively, a global item could encompass all potential treatment-related symptoms, of any treatments for any disease. However, such an item may not be well understood by general public respondents in valuation exercises. Further challenges include the decision about whether to generate de novo value sets for the modified instrument or to map to existing tariffs. The fluctuating and transient nature of treatment-related symptoms may be inconsistent with the methods used in conventional valuation exercises. Fluctuating symptoms could be missed by sub-optimal measure administration timing. The addition of items also poses double-counting risks. In summary, the addition of treatment-related symptom items could increase the sensitivity of existing utility measures to capture known and unknown treatment effects in oncology, while retaining the core domains. However, more research is needed to investigate the challenges, particularly regarding valuation.



中文翻译:

调整基于偏好的效用措施以捕捉癌症治疗相关症状的影响

重要的是,用于评估癌症治疗的患者报告结果 (PRO) 措施充分捕捉患者所经历的益处和风险,特别是当不同疗法的不良事件概况不同时。本研究探讨了增加基于偏好的效用措施以捕捉癌症治疗相关症状的影响的案例。其他与癌症治疗相关的项目可能是特定的(例如皮疹)或全局性的。虽然特定项目更容易描述和理解,但它们的使用可能会遗漏更罕见的症状以及那些目前未知但将来自未来医学进步的症状。需要考虑适当数量的附加项目、这些项目的独立性以及它们对核心工具的心理测量特性的影响。或者,一个全局项目可以涵盖任何疾病的任何治疗的所有潜在治疗相关症状。然而,一般公众受访者在估值工作中可能不太了解此类项目。进一步的挑战包括决定是否为修改后的工具生成从头价值集或映射到现有关税。治疗相关症状的波动性和短暂性可能与传统评估活动中使用的方法不一致。次优的措施给药时间可能会错过波动的症状。增加项目也会带来重复计算的风险。总之,添加与治疗相关的症状项目可以提高现有实用措施的敏感性,以捕捉肿瘤学中已知和未知的治疗效果,同时保留核心领域。

更新日期:2021-06-18
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