School of Women's and Children's Health, UNSW Medicine, UNSW Sydney, Sydney, NSW, Australia.
Department of Neurology, Sydney Children's Hospital, Randwick, Sydney, NSW, Australia.
Dev Med Child Neurol. 2022 Jun;64(6):753-761. doi: 10.1111/dmcn.15135. Epub 2021 Dec 28.
To understand the factors that most influence decision-making in the treatment of infantile spinal muscular atrophy (SMA).
A discrete choice experiment was conducted among parents of people with SMA (parents), healthcare professionals (HCPs), and members of the Australian general population (GenPop). Respondents were asked to accept/reject treatment for an infant newly diagnosed with SMA in eight hypothetical scenarios, characterized by different combinations of the attributes of the treatment offered. The results were analyzed using probability analysis.
Completed responses were provided from 1113 individuals (1024 GenPop, 21 parents, 68 HCPs). Respondents were more likely to accept treatments that improved functioning and mobility. Treatments with higher costs, invasive delivery, and risks of adverse events were accepted less often. Cost most affected treatment choices by HCPs and GenPop, while change in mobility and mode of administration were most influential for parents.
These results highlight the importance of understanding value for money and clinical impact in affecting treatment choice, which are crucial for effective planning of healthcare and the successful implementation of treatment programmes for SMA. What this paper adds Spinal muscular atrophy (SMA) treatments with a higher chance of improving functioning and mobility are preferred by the general population, parents, and healthcare professionals. Treatments with higher costs, invasive delivery, and risk of adverse events are less preferred. Willingness to pay for SMA treatments increases with impact on functioning.
了解影响婴儿脊髓性肌萎缩症（SMA）治疗决策的主要因素。
在 SMA 患者的父母（父母）、医疗保健专业人员（HCP）和澳大利亚普通人群（GenPop）成员中进行了离散选择实验。要求受访者在 8 种假设情景中接受/拒绝为新诊断患有 SMA 的婴儿提供的治疗，这些情景的特征是所提供治疗的属性具有不同的组合。使用概率分析对结果进行了分析。
1113 名个体（1024 名 GenPop、21 名父母、68 名 HCP）提供了完整的回复。受访者更有可能接受改善功能和活动能力的治疗方法。成本更高、侵入性给药和不良事件风险的治疗方法接受程度较低。成本对 HCP 和 GenPop 的治疗选择影响最大，而移动性和给药方式的变化对父母的影响最大。
这些结果强调了理解物有所值和临床影响对影响治疗选择的重要性，这对于有效规划医疗保健和成功实施 SMA 治疗计划至关重要。本文的新增内容：具有更高改善功能和活动能力机会的 SMA 治疗方法受到普通人群、父母和医疗保健专业人员的青睐。成本更高、侵入性给药和不良事件风险的治疗方法不太受欢迎。对 SMA 治疗的支付意愿随对功能的影响而增加。
