For the past several years, we have been working to bring your voices, experiences, challenges and hopes to inform regulatory authorities as key decisions are made on future therapies for SMA. Most recently, on April 18, 2017, the SMA community gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA to do just that. On this day, over 400 individuals — representing patients and caregivers of all types, ages and stages of SMA, industry and regulators came together to speak of the profound impact of SMA on every aspect of their lives and their expectations and hopes for current and future SMA treatments.

The PFDD Meeting was an important landmark for our community. For a whole day, leaders and regulators at FDA had their eyes and ears on you! And they got to witness firsthand the bravery, resilience, optimism and power of our community.

The PFDD meeting also helped us to identify areas where further information and research is needed. That is why we’re launching this Benefit-Risk survey! The purpose of this survey is to determine how individuals and families with SMA make risk assessments when presented with specific benefits of a potential treatment. Results from this survey will inform payers and FDA about the risks you may be willing to ‘take on’ in exchange for specific treatment benefits.

Learnings from this survey will provide critical information that we will share with insurance companies and Medicaid to maximize insurance coverage for all.

We are encouraging every family in our community to complete this survey. An analysis of this survey will be submitted along with a summary of the PFDD meeting (called a “Voice of the Patient Report”) to the FDA. Help us to create a compelling case for the assessment and ultimate approval of new therapeutics in SMA.

Who Can Participate in the Survey

  • Individuals with SMA who have reached the age of majority. (The age of majority is 19 in Alabama and Nebraska, 21 in Puerto Rico, and 18 in all other states.)
  • Caregivers of individuals with SMA (all ages and types), including those who cared for an individual who has since passed away. Please note that caregivers must also have reached the age of majority in order to participate.

How to Participate

Please use the link below and you will be directed to the survey. In this survey, you will be asked to rate the value of many treatment benefits and risks against each other. Given the diversity of presentation in SMA, the list of benefits and risks is a bit long as we want to make sure all SMA types and stages are represented in the findings. We appreciate your understanding when answering it, as this the required format for collecting benefit-risk information. The survey should take approximately 15 minutes to complete.

We want to hear from you!

Take the survey


The survey will close on Friday, October 27th.

Take the survey

For Assistance with Survey

Silicon Valley Research Group administering our survey. If you have any questions or problems accessing the survey or need assistance filling it out, please contact Mike Wasnock, Senior Research Analyst at [email protected].