We all know it: rare diseases, especially progressive pediatric disorders like Duchenne, are different than common diseases. The FDA knows it too, but they don’t have a plan for how to treat them differently. They are working on different ways to weigh the benefits and risks of treatments for rare disease versus treatments for treat common diseases. We are aggressively engaged to keep the community voice in that process.
PPMD, together with our partners in Washington, FaegreBD, have laid out the differences between benefit/risk assessment in common and rare diseases in a new white paper: Benefit-Risk Assessments in Rare Disorders: The Case for Therapeutic Development in Duchenne Muscular Dystrophy as the Prototype for New Approaches. It suggests ways the FDA might make decisions differently for rare diseases, and especially for Duchenne.
What is the ultimate goal with an effort like this?
We want to encourage the FDA to apply appropriate flexibility in the review process. This includes when applying the use of expedited pathways like accelerated approval. This kind of data helps to further our case for speeding the development of potential therapies.
Yes, a “white paper” may seem like a passive action... just words. However, these words lay an important groundwork that we can use to push hard for change in the FDA. Together with other families and organizations, we are making a case for Duchenne as a model for altering the benefit/risk equation. Through our benefit/risk survey, we provided the FDA with the sort of data they can’t ignore about your treatment preferences. In the white paper, we propose a new path. We will aggressively partner with the FDA for changes that allow the approval process to move faster, with more and earlier access to treatments.
We continue to hold meetings with FDA on this and all matters regarding accelerating treatments for Duchenne. More interactions are planned for the fall that you will be hearing about in the near future. Stay tuned, stay engaged.
Tell your story to the FDA
We encourage families to use the PPMD “share your story” site. Please tell us what you’d like the FDA to know about your family and your needs. We will continue to collect these stories, compile them, and share them with the FDA and others who benefit from understanding your experiences. So far, the stories seem to be having an impact, the FDA has made that clear and we’d like to have more to share.
We need to continue to give the FDA information that will make a difference in the benefits/risk equation and make the case for flexibility when they review potential therapies for Duchenne.