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Motor function and the stabilization or improvement of breathing – as well as oral administration – are the factors that adult patients and caregivers of children with spinal muscular atrophy (SMA) type 2 or 3 who cannot not walking appreciate the most when deciding what treatments are available, according to a British study. .
Avoiding long-lasting side effects was also important for caregivers, but less so in adult patients.
These findings highlight the tradeoffs 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.
Taken together, the data underscore that treatment choices shouldn’t be guided by efficacy alone, the researchers noted.
The study, “Preferences and utilities for treatment attributes in spinal muscular atrophy type 2 and non-ambulatory type 3 in UK, ”Was published in the Pharmacoeconomics.
Currently, three disease-modifying therapies are available for AMS: Spinraza from Biogen (nusinersen), gene therapy from Novartis Zolgensma (onasemnogene abeparvovec-xioi) and Evrysdi from Roche (risdiplam).
While they all work to restore levels of SMN – the missing protein in patients with ADS – they do so through different mechanisms, are delivered by distinct routes and regimens, and have different safety profiles. different.
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 providers, must make treatment choices. These decisions, when not constrained by eligibility criteria, are likely based on preferences for specific treatment attributes and tradeoffs between them.
A previous US study showed that caregivers of pediatric patients and patients with all types and levels of SMA motor function “highly value treatments that improve motor and respiratory function, an indication at all ages, oral infusion or single and minimal risk. profiles, ”the researchers wrote.
Now, researchers from Roche and Acaster Lloyd Consulting have assessed treatment preferences for AMS in the UK in a smaller group of patients – those with type 2 or 3 AMS who are not ambulatory. – and 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 any attributes associated with Zolgensma, which is available for a minority of this group of patients.
The researchers also estimated the health utilities associated with AMS, which are cardinal values of the health conditions that patients may experience.
Public health services can be used to estimate the health-related quality of life of patients and therefore the impact of treatment, which helps guide funding decisions in countries with publicly funded health systems , like the UK.
The researchers conducted two experimental surveys of discrete choices: one to assess treatment preferences and the other to analyze health services. This type of survey is increasingly used in health care to elicit respondents’ preferences, including treatment characteristics, without asking them directly to indicate their preferred options.
Survey participants were presented with a series of scenarios and asked to choose between sets of two hypothetical drugs with varying characteristics, forcing them to distinguish their preferences for the characteristics given 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 pain; monitoring, contraception (female patients only) and overall survival (general public only).
The first survey was conducted among 84 adult patients (mean age 33.9) and 83 caregivers of pediatric patients (mean age 8.4 years), recruited through TreatSMA and SMA UK.
Most of the patients (73% to 92%) in both groups had type 2 disease. Most of the children could sit independently and about half needed breathing assistance, while the adults did. most often with support and relied less on respiratory support (27%).
Almost two-thirds (65%) of pediatric patients and one adult patient had been treated with Spinraza. Most (69%) of adult patients were not receiving treatment for their ADS.
Adult patients were twice as likely to favor treatment that improved (as opposed to stabilizing) their motor or respiratory function, while caregivers were three to nine times more likely to promote improved motor function, according to the results. .
Treatment linked to worsening (versus stabilization) of motor or respiratory functions was four to five times less preferred by adult patients, but only four times less by caregivers.
These results highlight that caregivers “were more willing to make compromises to improve motor function and – to a lesser extent – respiratory function, while adult patients were more focused on preventing deterioration of respiratory function and improving respiratory function. motor function, ”the researchers wrote.
Adult patients “may place a relatively higher value on stabilization because it can allow them to maintain 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 therapy over spinal or intrathecal injections three times a year.
While reactions 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.
The treatment choices of patients and caregivers were not determined by the avoidance of monitoring or contraception.
The second survey, designed to estimate health services related to ADS, was carried out by 506 adults representative of the UK general public.
The results showed that the health utility values significantly decreased with worsening of motor and pulmonary function and, to a lesser extent, with intrathecal therapy and adverse events. This underscored the importance of maintaining an ability to sit and breathe without mechanical ventilation and to avoid spinal injections and reactions to treatment.
These findings highlight “the value caregivers and adult patients place on motor function, respiratory function, and oral administration in the context of AMS treatments,” the researchers wrote, adding that Health disutilities (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 efficacy alone, but be guided by many factors”, as patients and caregivers “valued avoiding intrathecal injection and caregivers preferred that their children avoid longer lasting treatment reactions, ”the team added.
“Data on the preferences of caregivers, patients and the general public can also be used to inform decision-makers at the national level concerned with understanding the value of improvements in treatment,” the researchers concluded.
Three of the six authors of this study are employees of Roche and one is a former employee; the company sponsored this work.