OBJECTIVE: This study quantified patient preferences for generalized myasthenia gravis (gMG) treatment features.
BACKGROUND: gMG is a chronic condition characterized by severe muscle weakness. Emerging gMG treatments have limited clinical differentiation but vary considerably in their administration.
DESIGN/METHODS: An online discrete-choice experiment survey was administered to adults living with gMG in the US, UK, and Germany. Respondents chose between experimentally-designed pairs of hypothetical treatments defined by the following 6 attributes: setting, mode, and frequency of administration, time until meaningful symptom improvement, and risk of mild-to-moderate and severe injection site reactions (ISRs). The pooled data were analyzed using a mixed logit model. Model estimates were used to (1) calculate the conditional relative attribute importance (CRAI) and (2) predict the probability respondents would select one hypothetical treatment profile over another using clinically relevant combinations of attributes.
RESULTS: The 200 respondents (US, n=150; UK, n=25; Germany, n=25) placed the most importance on changes in risk of ISR (from 40% to 0% [mild-to-moderate], CRAI=23.4%; from 10% to 0% [severe], CRAI=22.2%) and frequency of administration (from once daily to once every 8 weeks, CRAI=20.4%). While changes in setting and administration mode were the least important (CRAI = 10.3% and 10.0%, respectively), self-administration at home was preferred to administration by a doctor or nurse in a healthcare facility. Model estimates suggest respondents were more likely to choose a treatment self-administered at home once-a-day through a prefilled syringe with 2 weeks until onset of action (65.2%) over a treatment administered once every 8 weeks through a 1- to 2-hour IV at a medical facility with 4 weeks until onset of action (34.8%).
CONCLUSIONS: Respondents preferred gMG treatments with lower ISR risk, less frequent administration, and those self-administered at home. However, respondents were willing to trade off among treatment attributes, suggesting an important role for patient preferences in treatment selection.
