Earlier this week, PPMD hosted a webinar to discuss our FDA “aggressive collaboration” approach and the results from our recent benefit/risk survey. We conducted a rigorous survey of more than 100 parents whose children have Duchenne, gathering invaluable perspectives as to treatment priorities and risk threshold based on realistic but hypothetical treatments – information that PPMD is encouraging regulators to use to accelerate review time and, ultimately, to speed access to effective therapies.
To help keep the survey realistic, the treatments described in the experiment did not include a cure—the best outcomes that participants saw were stopping the progression of weakness, and adding 5 years to the child’s lifespan. In the experiment, we found that the parents were not willing to accept unlimited risk, but the presence of side effects and even a serious risk could be compensated for by a treatment that stops or slows progression of muscle weakness. If you missed this week’s webinar, watch the video below to learn more about the results and how you can still share your story with the FDA.