Thanks to the hard work and commitment of advocates from the MDF community and many others, the 21st Century Cures Act has just been signed by U.S. President Barack Obama. This legislation is designed to enhance the drug review process and accelerate the approval of therapies to treat and cure rare diseases like myotonic dystrophy (DM). Thank you to all of you who who met with your members of Congress during our annual Hill Days, wrote your congressional leadership during our 500 Voices Campaign and helped raise the profile of the Act in Congress.

Bipartisan Effort

The 21st Century Cures Act was a bipartisan effort that originated in the U.S. House of Representatives under the leadership of Energy & Commerce Committee Chairman Rep. Fred Upton (R-MI) and ranking member Rep. Diana DeGette (D-CO). The legislation is intended to improve the Food and Drug Administration's (FDA) regulatory review process and provide an additional $8.75 billion over 5 years to the National Institutes of Health (NIH), to help put the NIH on a path to long-term growth and continued investment in medical research.

Funding and Regulatory Impact

Among the many stipulations of the Act, it:

• Authorizes the CDC to create the National Neurological Conditions Surveillance System to better track neurological diseases;
• Authorizes $500 million over 10 years to accelerate regulatory review through the initiatives included in Cures (below);
• Requires the FDA to issue data upon approval of a product stating how patient experience data informed the review process and decision;
• Requires issuance of additional guidance on how to collect and utilize patient experience data as a patient-focused drug development (PFDD) tool;
• Establishes a pathway for review and qualification of biomarkers/drug development tools;
• Allows applicants developing products for rare disease to include data from previously approved submissions involving the same or similar underlying technology to accelerate development and delivery of rare disease therapies; and
• Extends the Pediatric Rare Disease Priority Review Voucher to Sept. 30, 2020, and allows issuance of vouchers through 2022 if a designation for a candidate therapy was provided before Sept. 30, 2020.

Thank you!

Together we have made a difference! Thank you for all you do to drive Care and a Cure for DM!