Myotonic has been working with partners in other rare disease organizations, Congress, and biotech/pharma in order to improve the regulatory review and drug approval processes in the US. Below, in their own words, our partners explain the need to come up with new approaches informing the FDA about research and our respective communities.

"Patient advocates have worked tirelessly with Congress, over time through legislation, to increase the engagement of the patient voice within the regulatory review process for therapeutics...PPMD is now engaging Congress on the 21st Century Cures Initiative and proposing the development of a patient engagement assessment tool in order to quantify how FDA reviewers use - or do not use - available patient-focused drug development tools and data. The patient community needs transparency about whether increased engagement is yielding an impact on outcomes. Otherwise, we are wasting time and resources that could be directed elsewhere in drug development."

- Ryan Fischer, Senior Vice President of Community Engagement, Parent Project Muscular Dystrophy

"For me, the question has been how to accelerate the approval process so patients can have better and faster access to the drugs that they so desperately need. 21st Century Cures and other legislation are really about applying the best science and safety that we know about to expedite treatments for our community."

- Woodie Kessel, MD, MPH, Former Assistant US Surgeon General and Myotonic Board Member

For more information on 21st Century Cures, please see the accordion above.