University of Bordeaux, Inserm, Bordeaux Population Health Research Center, team EMOS, Bordeaux, France.
University of Bordeaux, Inserm, Bordeaux Population Health Research Center, team EMOS, Bordeaux, France; CHU de Bordeaux, Pôle de santé publique, Service d'information médicale, Bordeaux, France.
Value Health. 2021 May;24(5):683-690. doi: 10.1016/j.jval.2020.11.010. Epub 2021 Mar 7.
Tyrosine kinase inhibitors (TKIs) account for the vast majority of healthcare expenditure on patients with chronic myeloid leukemia (CML), and it has been demonstrated that TKI discontinuation in patients in long-term deep molecular remission (DMR) is safe and improves quality of life. Our objective was to estimate the budget impact of TKI discontinuation in CML patients in long-term DMR from the perspective of the French healthcare system.
This analysis was conducted over a 5-year time horizon using a Markov model with cycles of 6 months. Transition probabilities were estimated through systematic reviews and meta-analyses. Costs were estimated from the French National Claims Database. Monte Carlo simulations were performed to take into account the uncertainty surrounding model parameters. Sensitivity analyses were carried out by varying the size of the target population and the cost of TKIs.
Over a 5-year period and for a target population of 100 patients each year eligible and agreeing to stop TKI, the TKI discontinuation strategy would save €25.5 million (95% confidence interval -39.3 to 70.0). In this model, the probability that TKI discontinuation would be more expensive than TKI continuation was 12.0%. In sensitivity analyses, mean savings ranged from €14.9 million to €62.9 million.
This study provides transparent, reproducible, and interpretable results for healthcare professionals and policy makers. Our results clearly show that innovative healthcare strategies can benefit both the healthcare system and patients. Savings from generalizing TKI discontinuation in CML patients in sustained DMR should yield health gains for other patients.
酪氨酸激酶抑制剂(TKI)在慢性髓性白血病(CML)患者的医疗保健支出中占绝大多数,并且已经证明,在长期深度分子缓解(DMR)的患者中停止 TKI 是安全的,并且可以提高生活质量。我们的目的是从法国医疗保健系统的角度来估算长期 DMR 的 CML 患者停止 TKI 的预算影响。
本分析在 5 年的时间内使用具有 6 个月周期的 Markov 模型进行。通过系统评价和荟萃分析来估计转移概率。从法国国家索赔数据库中估算成本。进行蒙特卡罗模拟以考虑模型参数的不确定性。通过改变目标人群的规模和 TKI 的成本来进行敏感性分析。
在 5 年内,对于每年有 100 名符合条件且同意停止 TKI 的目标人群,TKI 停药策略将节省 2550 万欧元(95%置信区间为-3930 万至 7000 万欧元)。在该模型中,TKI 停药比 TKI 继续治疗更昂贵的概率为 12.0%。在敏感性分析中,平均节省额在 1490 万欧元至 6290 万欧元之间。
这项研究为医疗保健专业人员和政策制定者提供了透明,可重现和可解释的结果。我们的研究结果清楚地表明,创新的医疗保健策略可以使医疗保健系统和患者受益。在持续 DMR 的 CML 患者中普遍停止 TKI 可以为其他患者带来健康收益,从而节省资金。