Raphael J, Helou J, Pritchard K I, Naimark D M
University of Toronto, Institute of Health Policy, Management and Evaluation, Toronto, ON, Canada; Department of Oncology, Western University, London Regional Cancer Program, London, ON, Canada.
University of Toronto, Institute of Health Policy, Management and Evaluation, Toronto, ON, Canada; Princess Margaret Cancer Centre, Radiation Medicine Program, Toronto, ON, Canada.
Eur J Cancer. 2017 Nov;85:146-154. doi: 10.1016/j.ejca.2017.08.018. Epub 2017 Sep 18.
The addition of palbociclib to letrozole improves progression-free survival in the first-line treatment of hormone receptor positive advanced breast cancer (ABC). This study assesses the cost-utility of palbociclib from the Canadian healthcare payer perspective.
A probabilistic discrete event simulation (DES) model was developed and parameterised with data from the PALOMA 1 and 2 trials and other sources. The incremental cost per quality-adjusted life-month (QALM) gained for palbociclib was calculated. A time horizon of 15 years was used in the base case with costs and effectiveness discounted at 5% annually. Time-to- progression and time-to-death were derived from a Weibull and exponential distribution. Expected costs were based on Ontario fees and other sources. Probabilistic sensitivity analyses were conducted to account for parameter uncertainty.
Compared to letrozole, the addition of palbociclib provided an additional 14.7 QALM at an incremental cost of $161,508. The resulting incremental cost-effectiveness ratio was $10,999/QALM gained. Assuming a willingness-to-pay (WTP) of $4167/QALM, the probability of palbociclib to be cost-effective was 0%. Cost-effectiveness acceptability curves derived from a probabilistic sensitivity analysis showed that at a WTP of $11,000/QALM gained, the probability of palbociclib to be cost-effective was 50%.
The addition of palbociclib to letrozole is unlikely to be cost-effective for the treatment of ABC from a Canadian healthcare perspective with its current price. While ABC patients derive a meaningful clinical benefit from palbociclib, considerations should be given to increase the WTP threshold and reduce the drug pricing, to render this strategy more affordable.
在来曲唑基础上加用帕博西尼可改善激素受体阳性晚期乳腺癌（ABC）一线治疗的无进展生存期。本研究从加拿大医疗保健支付方的角度评估帕博西尼的成本效益。
开发了一个概率性离散事件模拟（DES）模型，并用来自PALOMA 1和2试验及其他来源的数据进行参数化。计算了帕博西尼每获得一个质量调整生命月（QALM）的增量成本。基础病例采用15年的时间范围，成本和效果按每年5%进行贴现。疾病进展时间和死亡时间分别来自威布尔分布和指数分布。预期成本基于安大略省的费用及其他来源。进行概率敏感性分析以考虑参数不确定性。
与来曲唑相比，加用帕博西尼可额外获得14.7个QALM，增量成本为161,508美元。由此得出的增量成本效益比为每获得一个QALM需10,999美元。假设支付意愿（WTP）为每QALM 4167美元，帕博西尼具有成本效益的概率为0%。概率敏感性分析得出的成本效益可接受性曲线显示，当WTP为每获得一个QALM 11,000美元时，帕博西尼具有成本效益的概率为50%。
从加拿大医疗保健角度来看，以其当前价格，在来曲唑基础上加用帕博西尼治疗ABC不太可能具有成本效益。虽然ABC患者从帕博西尼中获得了有意义的临床益处，但应考虑提高WTP阈值并降低药物价格，以使该治疗策略更具可承受性。
