We are committed to working with key stakeholders, including the US Food and Drug Administration (FDA), to help accelerate the development of life-changing therapies. Below are our priorities to accelerate therapeutic development.

MDA's Accelerating Therapy Development Priorities

Advocate for innovative ways to accelerate therapeutic development and FDA regulatory review so patients can gain access to life-changing treatments sooner.

• Work with Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher, which incentivizes companies to invest in therapies for rare diseases. 
• Ensure the successful implementation of the many FDA and clinical trial reforms MDA advocates helped pass in 2022.
• Ensure the continued success of the Orphan Drug Act, which has led to many vital therapies for rare diseases.
• Ensure the FDA hears from the neuromuscular community's perspective to gain an appreciation for the factors that are taken into account by patients when a treatment is chosen.
• Host and support Patient-focused Drug Development (PFDD) meetings, including our July 2020 meeting on Pompe disease, as well as Patient Listening sessions.
• Ensure the federal government continues to invest in medical research, particularly through a well-funded National Institute for Neurological Disease and Stroke.
• Break down barriers to successful neuromuscular disease basic and translational research

Ways to Take Action

• Action Alert: Urge Congress to Support NIH & FDA Funding

Key Resources

• Webinar: Threats to Research Development