Reed Shelby D, Sutphin Jessie, Gonzalez Juan Marcos, Wallace Matthew J, Stephenson Judith J, Electricwala Batul, Bosworth Hayden B, Pagidipati Neha
Duke Clinical Research Institute, Duke University, Durham, NC. (S.D.R., J.S., J.M.G., M.J.W., H.B.B., N.P.).
Department of Population Health Sciences, Duke University, Durham, NC. (S.D.R., J.M.G., H.B.B.).
Circ Cardiovasc Qual Outcomes. 2025 Jul 11;18(8):e011804. doi: 10.1161/CIRCOUTCOMES.124.011804.
Despite the growing number of nonstatin lipid-lowering treatments (NS-LLTs), data are lacking on how patients value their various features and outcomes. Study objectives were to quantify patients' preferences across levels of efficacy, treatment regimens, side effects, and out-of-pocket costs of NS-LLTs and compare approaches with framing treatment efficacy.
A discrete choice experiment survey was administered to US adults aged ≥40 years with medical claims indicating statin use and atherosclerotic cardiovascular disease. Each participant was administered 12 sets of experimentally designed pairs of add-on NS-LLT profiles that varied in efficacy, administration regimen, injection-site reaction, joint pain, out-of-pocket cost, and a no-additional treatment option. Random-parameter logit models were used to estimate preference weights, and tradeoffs across attributes were reported as willingness-to-pay estimates.
A total of 1193 participants completed the survey (36% female; 90% White; mean age, 68.2±9.7 years). Across treatment features assessed, out-of-pocket cost ranging from $0 to $200 per month was the most important factor. All else being equal, a daily oral dosing regimen was the most preferred regimen. Among injectable regimens, participants preferred dosing every 6 months versus every 2 weeks (<0.001) or every month (<0.001). Efficacy presented as 25% to 60% reductions in LDL-C (low-density lipoprotein-cholesterol) levels was valued greater than equivalent reductions in 5-year cardiovascular risks. Among those reporting annual household incomes <$150 000 (93.5%), the average maximum willingness to pay for an add-on NS-LLT as a daily, oral medication without side effects ranged from $131 to $175 per month with efficacy framed as a 25% reduction in LDL-C levels versus $89 to $124 with efficacy framed as corresponding reductions in 5-year cardiovascular risk.
Among treatment features assessed, out-of-pocket costs were the primary factor driving choices. Those opting for an add-on NS-LLT were willing to trade off additional efficacy for less frequent injections or a daily oral medication.
尽管非他汀类降脂治疗（NS-LLTs）的数量不断增加，但关于患者如何评估其各种特征和结果的数据却很缺乏。研究目的是量化患者对NS-LLTs不同疗效水平、治疗方案、副作用和自付费用的偏好，并比较不同的治疗疗效表述方式。
对年龄≥40岁、有他汀类药物使用记录且患有动脉粥样硬化性心血管疾病的美国成年人进行了一项离散选择实验调查。每位参与者被给予12组经过实验设计的附加NS-LLT方案对，这些方案在疗效、给药方案、注射部位反应、关节疼痛、自付费用以及无附加治疗选项方面存在差异。使用随机参数logit模型来估计偏好权重，并将各属性之间的权衡以支付意愿估计值的形式报告。
共有1193名参与者完成了调查（36%为女性；90%为白人；平均年龄68.2±9.7岁）。在所评估的治疗特征中，每月自付费用从0美元到200美元是最重要的因素。在其他条件相同的情况下，每日口服给药方案是最受欢迎的方案。在注射给药方案中，参与者更喜欢每6个月给药一次，而不是每2周（<0.001）或每月（<0.001）给药一次。将疗效表述为低密度脂蛋白胆固醇（LDL-C）水平降低25%至60%比将5年心血管风险同等降低更受重视。在报告家庭年收入<150,000美元的人群中（93.5%），作为无副作用的每日口服药物，附加NS-LLT的平均最大支付意愿为每月131美元至175美元，此时疗效表述为LDL-C水平降低25%；而当疗效表述为5年心血管风险相应降低时，平均最大支付意愿为每月89美元至124美元。
在所评估的治疗特征中，自付费用是驱动选择的主要因素。选择附加NS-LLT的患者愿意为减少注射频率或每日口服药物而牺牲额外的疗效。
