A survey on preferences and utilities for treatment attributes in spinal muscular atrophy (SMA) type 2 and nonambulatory type 3 revealed that both patients and caregivers value motor function, breathing function, and oral administration in the context of treatments, according to a study published in PharmacoEconomics.

“Patients with Type 2 SMA are unable to stand or walk without support, whereas patients with Type 3 SMA can stand and walk, although these abilities are often lost as the disease progresses,” the research team explained.

The researchers designed a survey for participants recruited from TreatSMA and SMA UK, 2 SMA organizations based in the UK. The survey quantified patient and caregiver preferences for different characteristics of SMA treatments.

Continue Reading

Read more about SMA etiology

“The online survey first provided descriptions of each treatment attribute, followed by 16 choice questions, each asking participants to choose between two treatments (‘A’ or ‘B’),” the researchers wrote. Caregivers were also asked EQ-5D-5L questions.

In addition, the researchers developed a discrete choice experiment survey which was adapted for general public participants. It described attributes such as motor function, breathing function, treatment administration, treatment reactions, eyesight monitoring, contraception (patient only), and overall survival (general public only). 

The results demonstrated that participants viewed motor function, breathing function, and oral administration as important in the context of SMA treatments. Moreover, caregivers demonstrated that they were more willing to make trade-offs to improve motor function and breathing function. The survey demonstrated that adult patients were more focused on breathing function while avoiding deterioration and motor function.

Eyesight monitoring requirements and the need for contraception did not significantly impact the treatment choices of caregivers and patients. Lastly, the researchers discovered that disutilities (decrements to utility) generated from the UK population were significant for SMA care aspects and disease outcomes.

The approved treatments for SMA are nusinersen, a disease-modifying therapy given via intrathecal injection, risdiplam, which is given orally, and onasemnogene abeparvovec, which is administered intravenously. 


Lo SH, Gorni K, Sutherland CS, et al. Preferences and utilities for treatment attributes in Type 2 and non-ambulatory Type 3 spinal muscular atrophy in the United KingdomPharmacoeconomics. Published online October 18, 2021. doi:10.1007/s40273-021-01092-9