by: Ryan Fischer, Chief Advocacy Officer
When I think about Gene Therapy, I think about the possibilities. I think about those who fight every day so that Gene Therapy may become a safe reality for many. I think about a future where people with Duchenne live stronger, longer lives than those that came before them. I also think about countless families who paved the way, but for whom the therapeutic developments we see today were too far in the future to be reached.
Drug development involves much more than only funding research, we need the approval of proven therapeutics in order to move beyond clinical trials and increase opportunities for the entire Duchenne community. A big part of this process is PPMD’s work with the FDA and the incorporation of patient and caregiver views and perspectives into benefit-risk analysis. While progress has been made over the past 10 years, the FDA is not currently required to explain how they incorporate patient and caregiver perspectives into their final decisions on approved therapies.
Including your voice makes these trial outcome measures about so much more than a percentage point on a graph. It’s about your abilities, your independence, and your quality of life. Make your gift to help PPMD turn obstacles into possibilities. Thanks to the Small Heroes Foundation, every gift will be matched dollar-for-dollar up to $50,000 through April 30.
To remedy this issue, our Senate champions have introduced the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, which will require the FDA to include this valuable patient-centric information.
We also need to ensure therapies are developed efficiently and led by the community voice. In 2014, we came together as a community to ensure that our perspectives were top of mind by submitting the first-ever patient advocacy-initiated Draft Guidance to the FDA, to help accelerate drug development for Duchenne. In 2018, the FDA finalized their own Duchenne Guidance, and the drug development pipeline subsequently grew. Since then, we’ve made more progress and now need to revisit the document to ensure it reflects that progress, including advancement in Gene Therapy. This will help drive even more innovation in Duchenne, and carve a path toward our ultimate goal of accessible therapies for all patients.
PPMD has, and will always, continue to take every action necessary to ensure that we support the entire Duchenne community. It is not enough to have therapies for some when there is the potential to have therapies for all.
Thank you for your support, your contributions, and most importantly lending your voice to patient-focused drug development.
Ryan Fischer
Chief Advocacy Officer
