NEVER doubt that your voice as a rare disease advocate matters! President Obama just signed the 21st Century Cures Act into law, after it passed both the House and Senate with broad bipartisan support. This would not have been possible without advocates from EveryLife Foundation for Rare Diseases, Global Genes, National Organization for Rare Disorders, Inc. (NORD) and many disease-specific organizations who called, emailed and met with Members of Congress in the past year and a half.
The 21st Century Cures Act 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
These provisions and additional funding would boost our nation’s research capacity and help modernize the drug review and approval process at the FDA.