Nerich Virginie, Fleck Camille, Chaigneau Loïc, Isambert Nicolas, Borg Christophe, Kalbacher Elsa, Jary Marine, Simon Pauline, Pivot Xavier, Blay Jean-Yves, Limat Samuel
Department of Pharmacy, University Hospital, Boulevard Fleming, 25030, Besançon Cedex, France.
University Bourgogne Franche-Comté, INSERM, EFS BFC, UMR1098, Interactions hôte-greffon-tumeur - Ingénierie Cellulaire et Génique, Besançon, France.
Clin Drug Investig. 2017 Jan;37(1):85-94. doi: 10.1007/s40261-016-0463-2.
The management of advanced gastrointestinal stromal tumors (GISTs) has been modified considerably by the availability of costly tyrosine kinase inhibitors (TKIs); however, the best therapeutic sequence in terms of cost and effectiveness remains unknown.
The aim of this study was to compare four potential strategies (reflecting the potential daily practice), each including imatinib 400 mg/day, as first-line treatment: S1 (imatinib/best supportive care [BSC]); S2 (imatinib/imatinib/BSC); S3 (imatinib/sunitinib/BSC); and S4 (imatinib/imatinib/sunitinib/BSC).
A Markov model was developed with a hypothetical cohort of patients and a lifetime horizon. Transition probabilities were estimated from the results of clinical trials. The analysis was performed from the French payer perspective, and only direct medical costs were included. Clinical and economic parameters were discounted, and the robustness of results was assessed.
The least costly and effective strategy was S1, at a cost of €65,744 for 32.9 life months (reference). S3 was the most cost-effective strategy, with an incremental cost-effectiveness ratio (ICER) of €48,277/life-year saved (LYS). S2 was dominated, and S4 yielded an ICER of €363,320/LYS compared with S3. Sensitivity analyses confirmed the robustness of these results; however, when taking into account a price reduction of 80 % for imatinib, S2 and S4 become the most cost-effective strategies.
Our approach is innovative to the extent that our analysis takes into account the sequential application of TKIs. The results suggest that the S1 strategy is the best cost-effective strategy, but a price reduction of imatinib impacts on the results. This approach must continue, including new drugs and their impact on the quality of life of patients with advanced GISTs.
昂贵的酪氨酸激酶抑制剂(TKIs)的出现极大地改变了晚期胃肠道间质瘤(GISTs)的治疗方式;然而,在成本和疗效方面最佳的治疗顺序仍不明确。
本研究旨在比较四种潜在策略(反映潜在的日常实践),每种策略均包含每日400毫克伊马替尼作为一线治疗:S1(伊马替尼/最佳支持治疗[BSC]);S2(伊马替尼/伊马替尼/BSC);S3(伊马替尼/舒尼替尼/BSC);以及S4(伊马替尼/伊马替尼/舒尼替尼/BSC)。
采用马尔可夫模型,以一组假设的患者为队列,时间跨度为终身。转移概率根据临床试验结果估算。分析从法国医保支付方的角度进行,仅纳入直接医疗成本。对临床和经济参数进行贴现,并评估结果的稳健性。
成本最低且疗效最佳的策略是S1,32.9个生命月的成本为65,744欧元(参考值)。S3是最具成本效益的策略,每挽救一个生命年(LYS)的增量成本效益比(ICER)为48,277欧元。S2被其他策略主导,与S3相比,S4的ICER为363,320欧元/LYS。敏感性分析证实了这些结果的稳健性;然而,当考虑伊马替尼降价80%时,S2和S4成为最具成本效益的策略。
我们的方法具有创新性,因为我们的分析考虑了TKIs的序贯应用。结果表明,S1策略是最佳的成本效益策略,但伊马替尼的降价会影响结果。这种方法必须持续进行,包括新药及其对晚期GISTs患者生活质量的影响。