Duke Clinical Research Institute, Duke University, Durham, NC, USA.
Erasmus Choice Modelling Centre, Erasmus University Rotterdam, Rotterdam, the Netherlands.
Med Decis Making. 2020 Feb;40(2):198-211. doi: 10.1177/0272989X19897944. Epub 2020 Feb 17.
Quantitatively summarize patient preferences for European licensed relapsing-remitting multiple sclerosis (RRMS) disease-modifying treatment (DMT) options. To identify and summarize the most important RRMS DMT characteristics, a literature review, exploratory physician interviews, patient focus groups, and confirmatory physician interviews were conducted in Germany, the United Kingdom, and the Netherlands. A discrete choice experiment (DCE) was developed and executed to measure patient preferences for the most important DMT characteristics. The resulting DCE data (=799 and =363 respondents in the United Kingdom and Germany, respectively) were analyzed using Bayesian mixed logit models. The estimated individual-level patient preferences were subsequently summarized using 3 additional analyses: the quality of the choice data was assessed using individual-level estimates, individual-level preferences for the available DMTs were aggregated into DMT-specific preference shares, and a principal component analysis was performed to explain the patients' choice process. DMT usage differed between RRMS patients in Germany and the United Kingdom but aggregate patient preferences were similar. Across countries, 42% of all patients preferred oral medications, 38% infusions, 16% injections, and 4% no DMT. The most often preferred DMT was natalizumab (26%) and oral DMT cladribine tablets (22%). The least often preferred were mitoxantrone and the beta-interferon injections (1%-3%). Patient preferences were strongly correlated with patients' MS disease duration and DMT experience, and differences in patient preferences could be summarized using 8 principle components that together explain 99% of the variation in patients' DMT preferences. This study summarizes patient preferences for the included DMTs, facilitates shared decision making along the dimensions that are relevant to RRMS patients, and introduces methods in the medical DCE literature that are ideally suited to summarize the impact of DMT introductions in preexisting treatment landscapes.
定量总结欧洲获批的复发缓解型多发性硬化症（RRMS）疾病修正治疗（DMT）选择中患者的偏好。为了识别和总结 RRMS DMT 的最重要特征，在德国、英国和荷兰进行了文献回顾、探索性医生访谈、患者焦点小组和验证性医生访谈。开展了一项离散选择实验（DCE），以衡量患者对最重要 DMT 特征的偏好。从英国和德国获得的 DCE 数据（分别为=799 和=363 名受访者）使用贝叶斯混合对数模型进行分析。使用另外 3 种分析方法总结了估计的个体水平患者偏好：使用个体水平估计值评估选择数据的质量，将可用 DMT 的个体偏好汇总为特定 DMT 的偏好份额，以及进行主成分分析以解释患者的选择过程。德国和英国 RRMS 患者的 DMT 使用情况有所不同，但总体患者偏好相似。在所有国家中，42%的患者更喜欢口服药物，38%的患者更喜欢输注药物，16%的患者更喜欢注射药物，4%的患者不使用 DMT。最常被选择的 DMT 是那他珠单抗（26%）和口服 DMT 克拉屈滨片（22%）。最不受欢迎的是米托蒽醌和β干扰素注射剂（1%-3%）。患者的偏好与患者的 MS 疾病持续时间和 DMT 经验密切相关，患者对 DMT 的偏好差异可以用 8 个主要成分概括，这些成分共同解释了患者对 DMT 偏好的 99%的变化。本研究总结了患者对纳入 DMT 的偏好，促进了在与 RRMS 患者相关的维度上进行共同决策，并在医学 DCE 文献中引入了理想的方法，可用于总结 DMT 引入对现有治疗方案的影响。
