Family, healthcare professional, and societal preferences for the treatment of infantile spinal muscular atrophy: A discrete choice experiment

Aim To understand the factors that most influence decision-making in the treatment of infantile spinal muscular atrophy (SMA). Method 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. Results 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. Interpretation 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.

Automated summary

This study aimed to understand the factors that influence decision-making in the treatment of infantile spinal muscular atrophy (SMA). The results showed that treatments with a higher chance of improving functioning and mobility were preferred, while treatments with higher costs, invasive delivery, and risk of adverse events were less preferred. The study also revealed that cost influenced treatment choices for healthcare professionals and the general population, while change in mobility and mode of administration were most influential for parents.