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基于多准则决策分析方法的诺西那生临床综合评价

Comprehensive Clinical Evaluation of Nusinersen Based on Multi-criteria Decision Analysis Method

    摘要
  • 摘要:
    目的 本研究对诺西那生进行临床综合评价, 旨在为不同药品决策场景提供理论依据。
    方法 基于多准则决策分析的方法, 建立诺西那生临床综合评价指标体系(包括核心准则模型和情境化准则模型), 该体系涵盖安全性、有效性、经济性、社会属性等多个评价维度。通过系统检索和评价汇总, 整合诺西那生对应各评价准则的证据, 不同利益相关者为核心准则赋予相应权重并进行客观评分, 经过标准化加权处理后形成诺西那生的综合价值估计, 结合情境化准则模型探讨诺西那生治疗脊髓性肌萎缩症的临床价值。
    结果 在核心准则模型中, 标准化权重占比较高的准则为药物带来的治疗获益(0.08±0.02)、疾病严重程度(0.08±0.01)、药物使用相关的证据质量(0.08±0.01)、药物有效性(0.08±0.01)、药物安全性(0.08±0.02)。诺西那生评分较高的评价准则分别为疾病严重程度(4.8±0.4)、药物创新性(4.7±0.6)、专家共识/临床指南(4.5±0.6)、药物有效性(4.0±1.0)和药物使用相关的证据质量(4.0±0.8)。加权后得出诺西那生的综合价值得分为0.41。在情境化模型中, 诺西那生的综合价值得分为0.26, 表明可能被高估。
    结论 多准则决策分析的方法肯定了诺西那生治疗脊髓性肌萎缩症的积极作用, 为临床药品决策提供了理论依据。

     

    Abstract:
    Objective To provide a theoretical basis for different drug decision-making scenarios by conducting a comprehensive clinical evaluation of nusinersen.
    Methods Based on the method of multi-criteria decision analysis, a comprehensive clinical evaluation index system of nusinersen was established. This system, including core criterion model and contextualized criterion model, covered multiple evaluation dimensions such as safety, effectiveness, economy, and social attributes. The evidence of nusinersen in various criteria was summarized and integrated through systematic reviews. Then, different stakeholders were assigned weights and scores in various criteria of the core criterion model. Finally, a holistic value of nusinersen was estimatedthrough standardizing and combining the results in contextualized criterion model.
    Results The criteria, type of therapeutic benefit disease severity (0.08±0.02), unmet needs (0.08±0.01), quality of evidence (0.08±0.01), effectiveness (0.08±0.01) and safety (0.08±0.02) received the greatest weights after standardization. The higher mean scores for nusinersen versus placebo for the treatment of spinal muscular atrophy were disease severity (4.8±0.4), innovation of the drug (4.7±0.6), expert consensus or clinical practice guidelines (4.5±0.6), effectiveness (4.0±1.0) and quality of evidence (4.0±0.8).The holistic value of nusinersen was 0.41. The overall impact of the contextualized model on the evaluation of the drug was 0.26, indicating that it may overestimate the comprehensive value of nusinersen.
    Conclusions The method of multi-criteria decision analysis proves the positive role that nusinersen plays in spinal muscular atrophy treatment.

     

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