Unit of Macula, Oftalvist Clinic, Valencia, Spain.
Department of Ophthalmology, Hospital dos de Maig, Barcelona, Spain.
PLoS One. 2021 Dec 31;16(12):e0261955. doi: 10.1371/journal.pone.0261955. eCollection 2021.
Neovascular age-related macular degeneration (nAMD) leads to severe and permanent visual impairment, significantly impacting patients' quality of life and functional independence. Although treatment with anti- vascular endothelial growth factor (VEGF) prevents and, in some cases, reverses visual damage, the need for frequent monitoring visits and intravitreal injections represents a significant burden on patients, caregivers and retina specialists.
To elicit preferences for nAMD treatment characteristics from the perspectives of patients and retina specialists.
A discrete choice experiment was conducted. Participants (patients > 50 years with nAMD receiving anti-VEGF drugs for at least 2 years and without previous experience with anti-VEGF and retina specialists working in the Spanish National Healthcare System) were asked to select one of two hypothetical treatments resulting from the combination of five attributes (effects on visual function, effects on retinal fluid, treatment regimen, monitoring frequency, and cost); their levels were identified by reviewing the literature and two focus groups. The relative importance (RI) given to each attribute was estimated using a mixed logit model. The marginal rates of substitution (MRS) were calculated taking cost as the risk attribute.
A total of 110 patients (P) [aged 79.0 (SD:7.4) years; 57.3% women; 2.3 (SD:0.7) years with nAMD; 2.1 years (SD:0.1) in treatment] and 66 retina specialists (RS) participated in the study. Participants gave greater RI to improvements in their visual function [60.0% (P); 52.7% (RS)], lower monitoring frequency [20.2% (P); 27.1% (RS)] and reduction in retinal fluid [9.8% (P); 13.0%(RS)]. Patients and retina specialists would agree to an increase in cost by 65.0% and 56.5%, respectively, in exchange for improvements of visual function; and 25.5% and 43.3% on delaying monitoring frequency by one month.
Efficacy of treatment, in terms of visual function improvements, is the main driver for treatment election for both patients and retina specialists. Treatment monitoring requirements are also considered, mainly from the retina specialist's perspective. These results suggest that the use of more efficacious anti-VEGF agents with a longer duration of action may contribute to aligning treatment characteristics with patients/specialists' preferences. A better alignment would facilitate better disease management, fulfilling the unmet needs of patients and retina specialists.
新生血管性年龄相关性黄斑变性（nAMD）可导致严重且永久性的视力损害，显著影响患者的生活质量和功能独立性。尽管抗血管内皮生长因子（VEGF）治疗可预防和在某些情况下逆转视觉损害，但频繁的监测就诊和玻璃体内注射给患者、照护者和视网膜专家带来了巨大负担。
从患者和视网膜专家的角度出发，探讨 nAMD 治疗特征的偏好。
采用离散选择实验。参与者（年龄＞50 岁，患有 nAMD，接受抗 VEGF 药物治疗至少 2 年，且无抗 VEGF 治疗史）和视网膜专家（在西班牙国家医疗保健系统工作）被要求从五种属性（对视觉功能的影响、对视网膜液的影响、治疗方案、监测频率和成本）的组合中选择两种假设治疗方法之一；通过回顾文献和两个焦点小组确定属性的水平。使用混合 logit 模型估计每个属性的相对重要性（RI）。以成本为风险属性，计算边际替代率（MRS）。
共有 110 名患者（P）[年龄 79.0（标准差：7.4）岁；57.3%为女性；nAMD 病程 2.3（标准差：0.7）年；治疗时间 2.1（标准差：0.1）年]和 66 名视网膜专家（RS）参与了研究。参与者更看重对视觉功能的改善[60.0%（P）；52.7%（RS）]、监测频率降低[20.2%（P）；27.1%（RS）]和视网膜液减少[9.8%（P）；13.0%（RS）]。患者和视网膜专家分别愿意为改善视觉功能而将成本增加 65.0%和 56.5%；分别愿意将监测频率延迟一个月而将成本增加 25.5%和 43.3%。
在患者和视网膜专家看来，治疗效果，即视觉功能的改善，是选择治疗方案的主要驱动因素。治疗监测需求也很重要，主要是从视网膜专家的角度来看。这些结果表明，使用更有效的、作用持续时间更长的抗 VEGF 药物可能有助于使治疗特征与患者/专家的偏好保持一致。更好的一致性将有助于更好地管理疾病，满足患者和视网膜专家的未满足需求。
