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情景分析和多准则决策分析探索血液癌症患者全基因组测序的替代报销途径。

Scenario analysis and multi-criteria decision analysis to explore alternative reimbursement pathways for whole genome sequencing for blood cancer patients.

机构信息

Cancer Health Services Research, Centre for Cancer Research, Faculty of Medicine, Dentistry and Health Sciences, The University of Melbourne, Melbourne, Australia; Cancer Health Services Research, Centre for Health Policy, Melbourne School of Population and Global Health, Faculty of Medicine, Dentistry and Health Sciences, The University of Melbourne, Melbourne, Australia.

Department of Pathology, Peter MacCallum Cancer Centre, Melbourne, Australia; Sir Peter MacCallum Department of Oncology, The University of Melbourne, Melbourne, Australia; Clinical Haematology, Peter MacCallum Cancer Centre/Royal Melbourne Hospital, Melbourne, Australia.

出版信息

J Cancer Policy. 2024 Sep;41:100501. doi: 10.1016/j.jcpo.2024.100501. Epub 2024 Aug 12.

Abstract

BACKGROUND

Whole genome sequencing (WGS) has transformative potential for blood cancer management, but reimbursement is hindered by uncertain benefits relative to added costs. This study employed scenario planning and multi-criteria decision analysis (MCDA) to evaluate stakeholders' preferences for alternative reimbursement pathways, informing future health technology assessment (HTA) submission of WGS in blood cancer.

METHODS

Key factors influencing WGS reimbursement in blood cancers were identified through a literature search. Hypothetical scenarios describing various evidential characteristics of WGS for HTA were developed using the morphological approach. An online survey, incorporating MCDA weights, was designed to gather stakeholder preferences (consumers/patients, clinicians/health professionals, industry representatives, health economists, and HTA committee members) for these scenarios. The survey assessed participants' approval of WGS reimbursement for each scenario, and scenario preferences were determined using the geometric mean method, applying an algorithm to improve reliability and precision by addressing inconsistent responses.

RESULTS

Nineteen participants provided complete survey responses, primarily clinicians or health professionals (n = 6; 32 %), consumers/patients and industry representatives (both at n = 5; 26 %). "Clinical impact of WGS results on patient care" was the most critical criterion (criteria weight of 0.25), followed by "diagnostic accuracy of WGS" (0.21), "cost-effectiveness of WGS" (0.19), "availability of reimbursed treatment after WGS" (0.16), and "eligibility criteria for reimbursed treatment based on actionable WGS results" and "cost comparison of WGS" (both at 0.09). Participants preferred a scenario with substantial clinical evidence, high access to reimbursed targeted treatment, cost-effectiveness below $50,000 per quality-adjusted life year (QALY) gained, and affordability relative to standard molecular tests. Reimbursement was initially opposed until criteria such as equal cost to standard tests and better treatment accessibility were met.

CONCLUSION

Payers commonly emphasize acceptable cost-effectiveness, but strong clinical evidence for many variants and comparable costs to standard tests are likely to drive positive reimbursement decisions for WGS.

摘要

背景

全基因组测序(WGS)在血液癌管理方面具有变革性的潜力,但由于相对于增加的成本,其收益不确定,因此报销受到阻碍。本研究采用情景规划和多准则决策分析(MCDA)来评估利益相关者对替代报销途径的偏好,为 WGS 在血液癌中的未来健康技术评估(HTA)提交提供信息。

方法

通过文献检索确定了影响血液癌 WGS 报销的关键因素。使用形态学方法开发了描述 WGS 用于 HTA 的各种证据特征的假设情景。设计了一个在线调查,结合 MCDA 权重,以收集利益相关者对这些情景的偏好(消费者/患者、临床医生/卫生专业人员、行业代表、卫生经济学家和 HTA 委员会成员)。该调查评估了参与者对每个情景的 WGS 报销的批准程度,并使用几何平均值方法确定了情景偏好,该方法通过解决不一致的响应来提高可靠性和精度的算法。

结果

19 名参与者提供了完整的调查回复,主要是临床医生或卫生专业人员(n = 6;32%)、消费者/患者和行业代表(均为 n = 5;26%)。“WGS 结果对患者护理的临床影响”是最关键的标准(标准权重为 0.25),其次是“WGS 的诊断准确性”(0.21)、“WGS 的成本效益”(0.19)、“WGS 后可报销治疗的可用性”(0.16)和“基于可操作的 WGS 结果的可报销治疗的资格标准”和“WGS 的成本比较”(均为 0.09)。参与者更喜欢具有大量临床证据、可获得大量报销靶向治疗、成本效益低于每获得一个质量调整生命年(QALY)50,000 美元、以及相对于标准分子测试具有可负担性的情景。在满足标准测试成本相等和更好的治疗可及性等标准之前,报销最初遭到反对。

结论

支付者通常强调可接受的成本效益,但对于许多变体的强有力的临床证据和与标准测试相当的成本可能会推动 WGS 的积极报销决策。

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