背景:全基因组测序(WGS)对血癌管理具有转化潜力,但报销受到相对于额外成本的不确定收益的阻碍。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,告知未来健康技术评估(HTA)提交的WGS在血癌中的应用。
方法:通过文献检索确定了影响血液癌症WGS报销的关键因素。使用形态学方法开发了描述HTA的WGS各种证据特征的假设方案。网上调查,结合MCDA重量,旨在收集利益相关者的偏好(消费者/患者,临床医生/卫生专业人员,行业代表,健康经济学家,和HTA委员会成员)对于这些情况。调查评估了参与者对每种情况下WGS报销的批准,场景偏好是使用几何平均方法确定的,应用算法通过解决不一致的响应来提高可靠性和精度。
结果:19名参与者提供了完整的调查答复,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后报销治疗的可用性”(0.16),和“基于可操作的WGS结果的报销治疗资格标准”和“WGS成本比较”(均为0.09)。参与者更喜欢有大量临床证据的场景,获得报销的有针对性的治疗,成本效益低于每质量调整生命年(QALY)50,000美元,和相对于标准分子测试的可负担性。最初反对补偿,直到达到标准测试的同等成本和更好的治疗可及性等标准。
结论:付款人通常强调可接受的成本效益,但许多变种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的积极报销决定.
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.
CONCLUSIONS: 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.