The results of a new Cure SMA study showed that people with spinal muscular atrophy (SMA) and their caregivers are willing to tolerate a range of risks while seeking potential treatment benefits.
The advocacy association released its research report on March 6.
“In the 2022 Cure SMA Risk/Benefit Survey, we asked individuals with SMA and their caregivers how willing they were to live with certain possible treatment risks in exchange for certain potential treatment benefits,” the organization said. “We then compared the survey results to those from a similar 2017 survey.
“The survey results showed that the SMA community remains willing to tolerate a range of possible treatment risks in exchange for a variety of potential treatment benefits. These findings suggest that there are still important unmet SMA treatment needs.”
Three treatment options now available
The U.S. Food & Drug Administration (FDA) approved Spinraza (nusinersen) in December 2016 as the first drug to treat SMA, the leading genetic cause of death in infants. In 2019, the FDA approved Zolgensma, a genetic therapy to treat SMA. Evryski (risdiplam), an oral medication, was approved by the FDA in 2020.
Cure SMA partnered with Silicon Valley Research Group first in 2017 and again in 2022 to conduct surveys on the risks and benefits of SMA treatments.
“One of Cure SMA’s top priorities is to relay the SMA community’s treatment experiences and preferences to the FDA so the organization can make patient-centered decisions about new SMA drugs,” the most recent survey summary said. “In 2022, we carried out the Cure SMA Risk/Benefit Survey to learn about the SMA community’s current views on treatment risks and benefits. We compared these results with those from a similar survey we conducted in 2017 to determine if community perspectives have changed as more treatment options have become available.”
Cure SMA emailed survey invitations to SMA community members in the fall of 2022 to ask about how willing they were to tolerate 11 different treatment risks. In the following two months, the organization received 298 surveys completed by people living with SMA or caregivers for people with SMA. All participants lived in the United States and were at least 18 years old.
About 40% of the survey respondents were caregivers for infants and children with SMA. The rest of respondents were teens and adults.
One third (33%) of respondents had or were caring for someone who had SMA type 3; 39.4% had or were caring for someone with SMA type 2; 21.6% were caring for someone with SMA type 1. Type 4 SMA accounted for 0.7% of respondents, and 3.2% said they didn’t know the type of SMA involved.
Treatment risks most, least tolerated
Respondents were shown a list of 11 treatment risks and asked their willingness to tolerate the risk.
The three risks that respondents were “most willing to live with” were:
— Common side effects, such as nausea, vomiting, loss of appetite, headaches, back pain, fatigue, etc.
— Side effects of dizziness, which could increase fall risk.
— Possible need for general anesthesia to administer treatment.
— Possible need for invasive administration of treatment, such as infusions or injections.
The three risks respondents were least willing to tolerate were:
— Life-threatening allergic reactions.
— 1 in 1,000 risk of life threatening side effects to the heart, liver or kidneys that could result in organ failure.
— Worsening in quality of life, possibly due to drug’s side effects, worsening condition, etc.
The four remaining treatment risks were scored between “most willing” and “least willing” to tolerate:
— 1 in 100,000 risk of serious side effects to the heart, liver or kidneys that could affect normal organ function and require immediate medical attention.
— Increased risks of respiratory or other infections due to the medication.
— 1 in 100,000 risk of life-threatening side effects to the heart, liver or kidneys possibly resulting in organ failure.
— 1 in 1,000 risk of serious side effects to the heart, liver or kidneys that could affect normal organ function and require immediate medical attention.
Generally, Cure SMA said, the results of the 2022 survey were very similar to results from the 2017 survey, with a few exceptions, including less tolerance for invasive means of administering the treatments, such as via infusions or injections.
Cure SMA also said 2022 SMA type 1 respondents and caregivers “were much less likely to be willing to live with the possibility of ‘life-threatening allergic reactions’ for the potential treatment benefit of ‘improvement in the ability to communicate’” versus SMA type 1 respondents to the 2017 survey.
And 2022 SMA type 2 survey respondents and caregivers “were somewhat more likely to be willing to live with the possibility of ‘life-threatening allergic reactions’ for the potential treatment benefit of ‘lessening of symptoms’ severity (decrease in tremors, muscle weakness, etc.) than SMA type 2 survey respondents in 2017.”
“The 2022 Cure SMA Risk/Benefit Survey results show that people with SMA and their caregivers are still willing to live with many different treatment risks in exchange for a variety of possible treatment benefits,” the organization noted. “In addition, we found that the type of SMA an individual has may affect his/her/their risk tolerance. Together, these findings suggest that there are still unmet treatment needs in the SMA community, and that these needs may vary based on demographic factors like SMA type. Cure SMA will relay this important information to the FDA in hopes that it will support patient-centered decision making around SMA drug development and approval.”
Cure SMA’s annual conference takes place in Anaheim, California, June 26-29.
