Yesterday the House of Representatives passed the draft of the 21st Century Cures Act. It is slated for a vote in the Senate early next week.




The new draft includes:

  • $4.8 billion in new funding for the National Institutes of Health (NIH)
  • $500 million in new funding for the Food and Drug Administration (FDA)
  • Formally establishes the Precision Medicine and Cancer Moonshot initiatives
  • Reauthorization of the Rare Pediatric Disease Priority Review Voucher program through 2020
  • Funding for the establishment of a national neurological disease surveillance system coordinated by the Centers for Disease Control and Prevention (CDC)
  • Improved biomarker qualification
  • Allowances for the FDA to recruit and retain additional specialized employees
  • Strengthened patient engagement at the FDA through the Patient Focused Impact Assessment Act
  • A regenerative medicine designation to allow such products to qualify for priority review and accelerated approval
  • Provisions to foster programs to improve mental health and deter substance abuse

Funding for the NIH and FDA will be appropriated at the discretion of Congress via an “innovation fund” mechanism.  These provisions and additional funding would boost our nation’s research capacity and help modernize the drug review and approval process at the FDA.

This is good news for Duchenne muscular dystrophy and other rare diseases.

Unfortunately, Congress left out one of the key bi-partisan provisions that would have brought needed treatments for the 30 million Americans with a rare disease.  The Orphan Products Extensions Now, Accelerating Cures and Treatments (OPEN ACT; HR 971/S 1421) has been endorsed by 173 national patient organizations, including CureDuchenne, and was included in the House-passed version of the 21st Century Cures Act, but was dropped from the new bill.

The OPEN ACT is life-saving legislation that could double the number of therapies available to rare disease patients.  As a rare disease community we are disappointed that the OPEN ACT was not included.  We need as much flexibility to get drugs approved in order to help find a cure for all those with Duchenne.  The first FDA approved drug for Duchenne only helps 13% of Duchenne patients. That means 87% of those with Duchenne have no treatment. We need to continue to accelerate treatments and ultimately find a cure for all those with Duchenne.

The OPEN ACT would provide an incentive for companies to repurpose existing drugs for rare disease indications, which is substantially faster and more cost-efficient than traditional drug development. This legislation is key to supercharging the drug development process and helping ensure the translation of new research into life-saving treatments for patients. The updated draft of 21st Century Cures is a missed opportunity to save and substantially improve the lives of patients with rare diseases.

Thank you to EveryLife Foundation, Global Genes, NORD and other rare disease advocates who helped advance 21st Century Cures.

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