Purpose

Monoclonal antibodies (mAbs) are important active ingredients of molecularly targeted drugs, which are only effective for specific patient groups. Early assessment of their effectiveness is important for more efficient use of time and resources. Companion diagnostics (CDx) are medical devices or tests to identify groups of promising patients based on specific biomarkers. This work offers a systems evaluation model and a comprehensive assessment from multiple stakeholder perspectives.

Methods

This work introduces a new systems model for assessing available treatment options. Process system diagrams, consisting of independently defined unit structures, are applied to represent the expected decision points and outcomes. A sensitivity analysis is conducted to identify the critical requirements for achieving cost-effectiveness. The model was applied to a case of terminal colorectal cancer treatment to compare mAb drugs to standard therapy.

Results

The results showed that from the payers’ perspective, the cost and response rates of the mAb drug were critical parameters to improve for achieving the target cost-effectiveness. The results give quantitative guidance for the required improvement.

Conclusion

This work represents an important step towards a fair and systematic assessment of treatment alternatives and serves as a guideline for future CDx and therapy technology development efforts.

中文翻译：

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用于开发伴随诊断和相关分子靶向治疗的系统评估模型

目的

单克隆抗体（mAb）是分子靶向药物的重要活性成分，仅对特定患者群体有效。尽早评估其有效性对于更有效地利用时间和资源非常重要。伴随诊断 (CDx) 是一种医疗设备或测试，可根据特定的生物标志物识别有希望的患者群体。这项工作提供了一个系统评估模型，并从多个利益相关者的角度进行综合评估。

方法

这项工作引入了一种用于评估可用治疗方案的新系统模型。由独立定义的单元结构组成的过程系统图用于表示预期的决策点和结果。进行敏感性分析以确定实现成本效益的关键要求。该模型应用于晚期结直肠癌治疗病例，以将单克隆抗体药物与标准疗法进行比较。

结果

结果表明，从付款人的角度来看，单克隆抗体药物的成本和反应率是实现目标成本效益的关键参数。结果为所需的改进提供了定量指导。

结论

这项工作代表了对治疗方案进行公平和系统评估的重要一步，并可作为未来伴随诊断和治疗技术开发工作的指南。