Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA

By Cure SMA | Published On May 14, 2019

Your voice has been heard! 

Cure SMA has published a paper, titled, Evaluating Benefit-Risk Decision-Making in Spinal Muscular Atrophy: A First-Ever Study to Assess Risk Tolerance in the SMA Patient Community in the Journal of Clinical Therapeutics.

Our community’s perceptions of benefit-risk are essential to the evaluation of potential therapies for SMA. To bring this critical information to regulators, Cure SMA launched a first-ever Benefit-Risk Survey for spinal muscular atrophy (SMA). The goal of the survey was to better understand risk-benefit tradeoffs in SMA, associated with the benefits of a potential treatment.

The survey was conducted as a follow-up to an externally-led Patient Focused Drug Development (PFDD) meeting that was held with the FDA and the SMA Community. Key findings from this survey were included, and submitted to FDA, as an addendum to the Spinal Muscular Atrophy Voice of the Patient (VoP) Report to bring your voices, experiences, challenges, and hopes to regulatory authorities making key decisions on future therapies for SMA.

Key Learnings

Overall results and key learnings from this study reveal the following:

  • A total of 298 responses were evaluated (28% affected adults and 72% caregivers, mostly parents). Most respondents were diagnosed more than five years ago (67.3%), with 22.1% SMA type I, 45.6% SMA type II, and 27.9% SMA type III.
  • No diffrerences in risk tolerance were found based on SMA type, stage of disease progression, respondent type, gender, quality of life assessment, or rated levels of independence.
  • Risks rated as “most tolerable” included common side effects, such as dizziness, and risks associated with the administration of a drug, such as the need for anesthesia.
  • Risks rated as “least tolerable” included side effects that would worsen a patient’s condition or quality of life.

With the recent approval of the first SMA treatment and several more on the horizon, these findings should inform future SMA drug development and approvals. Results from this survey will inform payers and FDA about the risks our community may be willing to accept in exchange for specific treatment benefits, and how those risks are weighed.

What Led Up to the Benefit-Risk Survey

Cure SMA Launches Benefit-Risk Survey to Address Important Issues in Treatment Access and Future Drug Approvals in SMA

SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report

SMA Community's Voice Heard “Loud and Clear” at Last Week's Patient Focused Drug Development Meeting with the FDA

Topics: Support & Care, Research, Front Page News

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