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评估乐伐替尼对放射性碘难治性分化型甲状腺癌的整体价值:一项应用实用多标准决策分析的多国研究

Appraising the holistic value of Lenvatinib for radio-iodine refractory differentiated thyroid cancer: A multi-country study applying pragmatic MCDA.

作者信息

Wagner Monika, Khoury Hanane, Bennetts Liga, Berto Patrizia, Ehreth Jenifer, Badia Xavier, Goetghebeur Mireille

机构信息

LASER Analytica, Montreal, Quebec, Canada.

LASER Analytica, Milan, Italy.

出版信息

BMC Cancer. 2017 Apr 17;17(1):272. doi: 10.1186/s12885-017-3258-9.

Abstract

BACKGROUND

The objective of the study was to reveal through pragmatic MCDA (EVIDEM) the contribution of a broad range of criteria to the value of the orphan drug lenvatinib for radioiodine refractory differentiated thyroid cancer (RR-DTC) in country-specific contexts.

METHODS

The study was designed to enable comprehensive appraisal (12 quantitative, 7 qualitative criteria) in the current disease context (watchful waiting, sorafenib) of France, Italy and Spain. Data on the value of lenvatinib was collected from diverse stakeholders during country-specific panels and included: criteria weights (individual and social values); performance scores (judgments on evidence-collected through MCDA systematic review); qualitative impacts of contextual criteria; and verbal and written insights structured by criteria. The value contribution of each criterion was calculated and uncertainty explored.

RESULTS

Comparative effectiveness, Quality of evidence (Spain and Italy) and Disease severity (France) received the greatest weights. Four criteria contributed most to the value of lenvatinib, reflecting its superior Comparative effectiveness (16-22% of value), the severity of RR-DTC (16-22%), significant unmet needs (14-21%) and robust evidence (14-20%). Contributions varied by comparator, country and individuals, highlighting the importance of context and consultation. Results were reproducible at the group level. Impacts of contextual criteria varied across countries reflecting different health systems and cultural backgrounds. The MCDA process promoted sharing stakeholders' knowledge on lenvatinib and insights on context.

CONCLUSIONS

The value of lenvatinib was consistently positive across diverse therapeutic contexts. MCDA identified the aspects contributing most to value, revealed rich contextual insights, and helped participants express and explicitly tackle ethical trade-offs inherent to balanced appraisal and decisionmaking.

摘要

背景

本研究的目的是通过务实的多标准决策分析(EVIDEM),揭示在特定国家背景下,一系列广泛标准对治疗放射性碘难治性分化型甲状腺癌(RR-DTC)的孤儿药乐伐替尼价值的贡献。

方法

本研究旨在对法国、意大利和西班牙当前疾病背景(观察等待、索拉非尼)下的乐伐替尼进行全面评估(12项定量标准、7项定性标准)。在各国特定的研讨会上,从不同利益相关者处收集了乐伐替尼价值的数据,包括:标准权重(个人和社会价值);绩效得分(通过多标准决策分析系统评价对证据的判断);背景标准的定性影响;以及按标准构建的口头和书面见解。计算了每个标准的价值贡献并探讨了不确定性。

结果

比较有效性、证据质量(西班牙和意大利)和疾病严重程度(法国)的权重最大。四个标准对乐伐替尼的价值贡献最大,反映出其卓越的比较有效性(价值的16%-22%)、RR-DTC的严重程度(16%-22%)、重大未满足需求(14%-21%)和有力证据(14%-20%)。贡献因对照物、国家和个人而异,突出了背景和咨询的重要性。结果在群体层面具有可重复性。背景标准的影响因国家而异,反映了不同的卫生系统和文化背景。多标准决策分析过程促进了利益相关者对乐伐替尼知识的共享以及对背景的见解。

结论

在不同治疗背景下,乐伐替尼的价值始终为正。多标准决策分析确定了对价值贡献最大的方面,揭示了丰富的背景见解,并帮助参与者表达并明确应对平衡评估和决策中固有的伦理权衡。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/61b5/5393009/a76369c28cc8/12885_2017_3258_Fig1_HTML.jpg

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