Régnier Stephane A, Malcolm William, Haig Jennifer, Xue Weiguang
Novartis Pharma AG, Basel, Switzerland.
Novartis Pharmaceuticals UK Ltd, Frimley Business Park, UK.
Clinicoecon Outcomes Res. 2015 May 6;7:235-47. doi: 10.2147/CEOR.S82556. eCollection 2015.
Ranibizumab and aflibercept are alternative anti-vascular endothelial growth factor agents approved for the treatment of visual impairment (VI) due to diabetic macular edema (DME).
To estimate, from a UK healthcare perspective, the cost-effectiveness of ranibizumab 0.5 mg pro re nata (PRN) and ranibizumab 0.5 mg treat and extend (T&E) compared with aflibercept 2 mg every 8 weeks after five initial monthly doses (2q8) in the treatment of VI due to DME.
A Markov model previously reviewed by the National Institute for Health and Care Excellence was used to simulate the long-term outcomes and costs of treating DME. Health states were defined by increments of ten letters in best-corrected visual acuity (BCVA), with a 3-month cycle length. Patients could gain (or lose) a maximum of two health states between cycles. A 3-year treatment time frame and a lifetime horizon were used. Future costs and health outcomes were discounted at 3.5% per annum. Patient baseline characteristics and the efficacy of ranibizumab PRN were derived using data from the RESTORE study. The relative efficacies of ranibizumab PRN, ranibizumab T&E, and aflibercept were assessed with a network meta-analysis. Different utilities were assigned based on BCVA and whether the treated eye was the better- or the worse-seeing eye. Sensitivity analyses tested the robustness of the model.
Lifetime costs per patient of treating DME were £20,019 for ranibizumab PRN, £22,930 for ranibizumab T&E, and £25,859 for aflibercept 2q8. Ranibizumab was dominant over aflibercept, with an incremental gain of 0.05 quality-adjusted life-years (QALYs) and cost savings of £5,841 (PRN) and £2,930 (T&E) compared with aflibercept. Ranibizumab PRN and ranibizumab T&E had 79% and 67% probability, respectively, of being cost-effective relative to aflibercept at a willingness-to-pay threshold of £20,000/QALY. When assuming the higher end of PRN injection frequency (15.9 over 3 years), the cost savings associated with ranibizumab were £3,969.
From a UK healthcare perspective, ranibizumab provides greater health gains with lower overall costs than aflibercept in patients with VI due to DME.
雷珠单抗和阿柏西普是已获批用于治疗糖尿病性黄斑水肿（DME）所致视力损害（VI）的替代性抗血管内皮生长因子药物。
从英国医疗保健的角度，评估与初始五个月剂量（2q8）后每8周注射2 mg阿柏西普相比，按需（PRN）使用0.5 mg雷珠单抗和治疗并延长（T&E）使用0.5 mg雷珠单抗治疗DME所致VI的成本效益。
使用先前经英国国家卫生与临床优化研究所审核的马尔可夫模型来模拟治疗DME的长期结局和成本。健康状态根据最佳矫正视力（BCVA）每提高10个字母来定义，周期长度为3个月。患者在两个周期之间最多可改善（或恶化）两个健康状态。采用3年的治疗时间范围和终身期限。未来成本和健康结局按每年3.5%进行贴现。雷珠单抗PRN的患者基线特征和疗效数据来自RESTORE研究。通过网络荟萃分析评估雷珠单抗PRN、雷珠单抗T&E和阿柏西普的相对疗效。根据BCVA以及治疗的眼睛是视力较好还是较差的眼睛来分配不同的效用值。敏感性分析检验了模型的稳健性。
治疗DME的每位患者终身成本，雷珠单抗PRN为20,019英镑，雷珠单抗T&E为22,930英镑，阿柏西普2q8为25,859英镑。与阿柏西普相比，雷珠单抗具有优势，质量调整生命年（QALY）增量为0.05，成本节省分别为5,841英镑（PRN）和2,930英镑（T&E）。在支付意愿阈值为20,000英镑/QALY时，雷珠单抗PRN和雷珠单抗T&E相对于阿柏西普具有成本效益的概率分别为79%和67%。假设PRN注射频率处于较高水平（3年内为15.9次），与雷珠单抗相关的成本节省为3,969英镑。
从英国医疗保健的角度来看，对于DME所致VI患者，雷珠单抗比阿柏西普能带来更大的健康收益且总体成本更低。
