Adult patients with spinal muscular atrophy (SMA) type 2 and type 3 who cannot walk as well as their caregivers strongly prefer treatments that stabilize or improve motor and breathing function, according to a new study published in PharmacoEconomics.
They also prefer daily oral treatments vs other modes such as intrathecal injections and intravenous infusions. Finally, they prefer treatments that demonstrated effectiveness in all 3 types of SMA (type 1, 2, and 3) and want to avoid the risk of liver injury, fatigue, headache, and nausea.
These findings can help decision-makers identify the best treatment options for these patients in a more informed manner. Previous studies have explored the preferences of patients and caregivers for different SMA treatment options in the UK and US, but not in Europe.
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Researchers developed a discrete choice experiment survey to better understand the treatment preferences of patients and caregivers with SMA in the Netherlands, Belgium, Finland, Ireland, and Portugal. They then recruited participants using patient advocacy associations to complete the online survey.
A total of 65 participants completed the survey. The results showed that the participants were 4.8 times more likely to choose a treatment that stabilized motor function and 8.1 times more likely to choose 1 that improved motor function. Similarly, they were 4.3 times more likely to choose a treatment that stabilized breathing function and 5.8 times more likely to choose 1 that improved it.
The participants were also 1.6 times less likely to choose a treatment that had a risk of liver injury, fatigue, headache, and nausea compared to treatments that had a risk of diarrhea and rash, and 2.3 times less likely to choose treatments with demonstrated effectiveness in only SMA type 1 than those that demonstrated effectiveness in all 3 types of SMA.
Finally, participants were 1.8 times less likely to choose treatments administered intrathecally compared to those that were administered orally. “The present study findings demonstrate the importance of motor and breathing function to patients and caregivers of this SMA population,” the researchers concluded.
It also showed the importance they place on maintaining these functions and how they want to avoid intrathecal injections, treatments described as involving risk of liver injury in combination with fatigue, headache, and nausea, and treatments with demonstrated effectiveness in type 1 SMA only.
Lo SH, Lawrence C, Martí Y, Café A, Lloyd AY. Patient and caregiver treatment preferences in type 2 and non-ambulatory type 3 spinal muscular atrophy: a discrete choice experiment survey in five European countries. PharmacoEconomics. Published online December 13, 2021. doi:10.1007/s40273-021-01118-2