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Ideal SMA therapy helps motor skills and breathing, is oral: UK survey

Motor function and stabilization or improvement of breathing – along with oral administration – are the factors that adult patients and caregivers of children with spinal muscular atrophy (SMA) type 2 or 3 who cannot walking valued the most when deciding on available treatments, according to a UK study. .

Avoiding longer-lasting side effects was also important for caregivers, but less so for adult patients.

These findings shed light on the trade-offs patients and caregivers are willing to make in terms of treatment attributes, and add to similar findings from a previous US study involving a larger patient population.

Together, the data underscore that treatment choices should not be guided by efficacy alone, the researchers noted.

The study, “Preferences and utilities for treatment attributes in type 2 and non-ambulatory type 3 spinal muscular atrophy in the UKwas published in the PharmacoEconomics.

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Currently, three disease-modifying treatments are available for SMA: Spinraza from Biogen (nusinersen), Zolgensma gene therapy from Novartis (onasemnogene abeparvovec-xioi) and Evrysdi from Roche (risdiplam).

Although they all work to restore levels of SMN – the missing protein in patients with SMA – they do so through different mechanisms, are delivered by different routes and regimens, and have different safety profiles.

Spinraza is injected directly into the spinal canal three times a year, Zolgensma is given as a single intravenous infusion, and Evrysdi is taken daily as an oral solution.

As such, patients, as well as their caregivers and healthcare providers, must make treatment choices. These decisions, when not constrained by eligibility criteria, are likely based on preferences for specific treatment attributes and trade-offs between them.

A previous US study showed that caregivers of pediatric patients and patients with any type and level of motor function “place a high value on treatments that improve motor and respiratory function, an indication at all ages, a oral or single infusion and minimal risk”. profiles,” the researchers wrote.

Now researchers from Roche and Acaster Lloyd Consulting have assessed SMA treatment preferences in the UK in a more limited group of patients – those with type 2 or 3 SMA who are not ambulatory – and in taking into account the specific security issues associated with Spinraza and Evrysdi. Members of the general public were also recruited to participate.

The study did not include attributes associated with Zolgensma, which is available to a minority of this patient group.

The researchers also estimated SMA-related health utilities, which are cardinal values ​​of health states that patients may experience.

Public health services can be used to estimate patients’ health-related quality of life and, therefore, the impact of treatment, which helps guide funding decisions in countries with publicly funded health systems. public funds, like the UK.

The researchers conducted two experimental discrete-choice surveys: one to assess treatment preferences and the other to analyze health services. This type of survey is increasingly being used in the healthcare field to learn about responders’ preferences, including treatment characteristics, without directly asking them to indicate their preferred options.

Survey participants were presented with a series of scenarios and were asked to choose between sets of two hypothetical drugs with varying characteristics, forcing them to distinguish their preferences for the given characteristics in each choice scenario.

Variable characteristics included treatment outcomes such as motor function and respiration; Delivery method; adverse events or side effects such as fever, headache, vomiting and body aches; surveillance, contraception (patients only) and overall survival (general public only).

The first survey was completed by 84 adult patients (mean age 33.9 years) and 83 caregivers of pediatric patients (mean age 8.4 years), recruited through TreatSMA and SMA UK.

Most patients (73% to 92%) in both groups had type 2 disease. Most children could sit independently and about half required respiratory support, while adults were most often seated with support and relied less often on respiratory support (27%).

Almost two-thirds (65%) of pediatric patients and one adult patient had been treated with Spinraza. Most (69%) adult patients were not on any treatment for their SMA.

Adult patients were twice as likely to favor treatment that improved (versus stabilized) their motor or respiratory function, while caregivers were three to nine times more likely to favor improved motor function, according to the results.

Treatment related to worsening (versus stabilization) of motor or respiratory function was four to five times less preferred by adult patients, but only four times less preferred by caregivers.

These results highlight that caregivers “were more willing to compromise to improve motor function and – to a lesser extent – ​​respiratory function, while adult patients were more focused on preventing deterioration of respiratory function and motor function,” the researchers wrote.

Adult patients “may place a relatively higher value on stabilization, as it may allow them to retain their independence and therefore could be seen as a marker of treatment success,” they added.

Patients and caregivers were two to three times more likely to prefer daily oral treatment over spinal canal or intrathecal injections three times a year.

While responses to treatment were not important for adult patients, caregivers preferred to avoid adverse events that lasted three to four days, while being less concerned about those that lasted one to two days.

Patients’ and caregivers’ treatment choices were not driven by avoidance of monitoring or contraception.

The second survey, intended to estimate SMA-related health services, was completed by 506 adults representative of the general British public.

Results showed that health utility values ​​decreased significantly with deterioration of motor and lung function and, to a lesser extent, with intrathecal treatment and adverse events. This underscored the value of maintaining an ability to sit and breathe without mechanical ventilation, and avoiding spinal canal injections and treatment reactions.

These findings highlight “the value caregivers and adult patients place on motor function, respiratory function, and oral administration in the context of SMA treatments,” the researchers wrote, adding that the disutilities for health (utility reductions) generated by the general public” were substantial for SMA disease outcomes and aspects of care.

The data also suggests that treatment choices “should not be guided by clinical effectiveness alone, but be guided by many factors”, as patients and caregivers “valued the avoidance of intrathecal injection and caregivers preferred that their children avoid longer-lasting treatment reactions”. added the team.

“Data on the preferences of caregivers, patients and the general public can also be used to inform policy makers at the national level concerned with understanding the value of treatment improvements,” the researchers concluded.

Three of the six authors of this study are Roche employees and one is a former employee; the society sponsored this work.

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