One of Cure SMA’s top priorities is to relay the SMA community’s treatment experiences and preferences to the United States Food and Drug Administration (FDA). This helps the FDA make patient-centered decisions about new SMA drugs.
This is a critical time for patient input on SMA treatment, as a variety of new “add-on” therapies and variations on approved disease-modifying treatments approach the later stages of clinical trials.
In our new report to the FDA, “SMA community risk tolerance: Comparison of 2022 and 2017 SMA Risk/Benefit Survey Data,” we communicated important insights about how the risk tolerance of the SMA community has evolved following the increased availability of disease-modifying therapies.
In the 2022 Cure SMA Risk/Benefit Survey, we asked people with SMA and their caregivers how willing they were to live with certain possible treatment risks in exchange for certain potential treatment benefits. We then compared the survey results to those from a similar 2017 survey, which was conducted shortly after the approval of the first disease-modifying treatment for SMA.
The comparison revealed that the SMA community is still willing to live with a range of possible treatment risks in exchange for a variety of potential treatment benefits. These findings support what we have already learned from the SMA community---that there are still important unmet treatment needs for people of all ages who are living with SMA.
The findings in our new report will support the FDA in making decisions about new treatments that reflect the SMA community’s current needs.
To read the complete report to the FDA, visit this link.
To read a summary of the survey findings, visit this link.
This survey and report were funded by the Cure SMA Industry Collaboration. The Cure SMA Industry Collaboration (SMA-IC) was established in 2016 to leverage the experience, expertise, and resources of pharmaceutical and biotechnology companies, as well as other nonprofit organizations involved in the development of spinal muscular atrophy (SMA) therapeutics to more effectively address a range of scientific, clinical, and regulatory challenges. Current members include Cure SMA, Biogen, Novartis, Scholar Rock, Genentech/Roche Pharmaceuticals, NMD Pharma, and SMA Europe. Funding for this research was provided by the 2022 SMA-IC; members included Cure SMA, Biogen, Genentech/Roche, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, and SMA Europe.