Understanding patient preferences and willingness to pay for hemophilia therapies
Understanding patient preferences and willingness to pay for hemophilia therapies
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DOI：
10.2147/ppa.s92985
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发表时间：
2015-01-01
影响因子：
2.2
通讯作者：
Young, Guy
中科院分区：
文献类型：
--
作者：
Chaugule, Shraddha S.;Hay, Joel W.;Young, Guy
背景：尽管与按需治疗相比，预防性治疗的临床结果明显改善，但在美国，平均只有56%被诊断为严重血友病的患者接受了预防性因子替代治疗。随着患者从童年过渡到成年，预防性使用率通常会下降，部分原因是患者成年后变得不那么粘连。评估患者的喜好很重要，因为这可能会转化为更高的治疗满意度和依从性。在这项研究中，我们评估了美国血友病患者样本中按需、预防和长效预防治疗的偏好和支付意愿(WTP)。方法：成年美国血友病患者和照顾者(N=79)完成了一项离散选择调查，提出了一系列权衡问题，每个问题包括一对假设的治疗概况。使用混合Logit模型进行分析，我们比较了五个治疗特征的相对重要性：1)自付治疗费用(由患者支付)，2)因子剂量调整，3)治疗副作用，4)预混合因子的可用性，5)治疗有效性和剂量频率。结果：患者自付治疗费用(P
Background: Despite clearly improved clinical outcomes for prophylaxis compared to on-demand therapy, on average only 56% of patients diagnosed with severe hemophilia receive prophylactic factor replacement therapy in the US. Prophylaxis rates generally drop as patients transition from childhood to adulthood, partly due to patients becoming less adherent when they reach adulthood. Assessment of patient preferences is important because these are likely to translate into increased treatment satisfaction and adherence. In this study, we assessed preferences and willingness to pay (WTP) for on-demand, prophylaxis, and longer acting prophylaxis therapies in a sample of US hemophilia patients.Methods: Adult US hemophilia patients and caregivers (N=79) completed a discrete-choice survey that presented a series of trade-off questions, each including a pair of hypothetical treatment profiles. Using a mixed logit model for analysis, we compared the relative importance of five treatment characteristics: 1) out-of-pocket treatment costs (paid by patients), 2) factor dose adjustment, 3) treatment side effects, 4) availability of premixed factor, and 5) treatment effectiveness and dosing frequency. Based on these attribute estimates, we calculated patients' WTP.Results: Out-of-pocket treatment costs (P