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Introduction <p>Spinal muscular atrophy (SMA) is a rare autosomal recessive hereditary neuromuscular disease that leads to progressive muscle weakness and atrophy. Available treatments differ significantly in terms of effectiveness, administration, costs, and side-effect profiles. Currently, evidence regarding patient treatment preferences remains scarce, especially in developing countries. This study aimed to provide evidence on patient preferences toward SMA treatments.</p> Methods <p>We conducted a discrete choice experiment (DCE) among patients with SMA in China. Attributes were selected on the basis of a systematic literature review, in-depth patient and expert interviews, and validation through a focus group discussion. Five attributes were included: mode of administration, annual out-of-pocket costs, motor function, respiratory function, and incidence of side effects. A mixed logit model was applied for preference estimation. On the basis of the model results, we derived attribute relative importance (RI), conducted scenario analyses to predict the uptake of hypothetical treatment profiles, and performed willingness-to-pay (WTP) analyses to quantify the monetary value of attribute improvements.</p> Results <p>A total of 202 patients completed the questionnaire, with 190 included in the final analysis. Respondents had a mean age of 29.2 years, were predominantly diagnosed with type 3 SMA (67%), and mainly received parental care (70%). Annual out-of-pocket expenses (RI = 56%) emerged as the most influential attribute, followed by motor function improvements (RI = 20%), incidence of side effects (RI = 13%), respiratory function (RI = 8%), and mode of administration (RI = 3%). Patients expressed a substantial willingness to pay of renminbi (RMB) 32,217 annually for improvements in motor function. Scenario analyses indicated that 95% of patients would prefer a new treatment profile providing enhanced motor function, even at higher costs.</p> Conclusions <p>Adult patients with type 2 or type 3 SMA in China strongly prefer affordable treatments with oral administration, improved motor and respiratory functions, and minimal side effects. Understanding these preferences can guide clinical decision-making, supporting personalized treatment strategies that may ultimately enhance treatment adherence and clinical outcomes.</p>

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Treatment Preferences in Adult Patients with Type 2 and Type 3 Spinal Muscular Atrophy: Evidence from a Discrete Choice Experiment in China

  • 期刊论文

  • 发表日期：

    2026年5月4日

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  • Jiaqi Yuan,
  • Shunping Li,
  • Lei Dou,
  • Huanping Xing,
  • Liyong Lu,
  • Shan Jiang

摘要

Introduction

Spinal muscular atrophy (SMA) is a rare autosomal recessive hereditary neuromuscular disease that leads to progressive muscle weakness and atrophy. Available treatments differ significantly in terms of effectiveness, administration, costs, and side-effect profiles. Currently, evidence regarding patient treatment preferences remains scarce, especially in developing countries. This study aimed to provide evidence on patient preferences toward SMA treatments.

Methods

We conducted a discrete choice experiment (DCE) among patients with SMA in China. Attributes were selected on the basis of a systematic literature review, in-depth patient and expert interviews, and validation through a focus group discussion. Five attributes were included: mode of administration, annual out-of-pocket costs, motor function, respiratory function, and incidence of side effects. A mixed logit model was applied for preference estimation. On the basis of the model results, we derived attribute relative importance (RI), conducted scenario analyses to predict the uptake of hypothetical treatment profiles, and performed willingness-to-pay (WTP) analyses to quantify the monetary value of attribute improvements.

Results

A total of 202 patients completed the questionnaire, with 190 included in the final analysis. Respondents had a mean age of 29.2 years, were predominantly diagnosed with type 3 SMA (67%), and mainly received parental care (70%). Annual out-of-pocket expenses (RI = 56%) emerged as the most influential attribute, followed by motor function improvements (RI = 20%), incidence of side effects (RI = 13%), respiratory function (RI = 8%), and mode of administration (RI = 3%). Patients expressed a substantial willingness to pay of renminbi (RMB) 32,217 annually for improvements in motor function. Scenario analyses indicated that 95% of patients would prefer a new treatment profile providing enhanced motor function, even at higher costs.

Conclusions

Adult patients with type 2 or type 3 SMA in China strongly prefer affordable treatments with oral administration, improved motor and respiratory functions, and minimal side effects. Understanding these preferences can guide clinical decision-making, supporting personalized treatment strategies that may ultimately enhance treatment adherence and clinical outcomes.