Today, President Obama signed into law the 21st Century Cures Act, a game-changing bill for medical innovation. The bill includes many provisions that will improve the discovery, development, and delivery of orphan therapies for rare disease patients, including:
- An extension of the Rare Pediatric Disease Priority Review Voucher Program, which incentivizes the development of new therapies to help the more than 15 million children with rare diseases;
- Streamlining of U.S. Food and Drug Administration (FDA) review of genetically targeted and protein variant therapies for rare diseases;
- Creation of funds in the amount of $4.8 billion over 10 years for the National Institutes of Health (NIH) to fund the Precision Medicine Initiative, BRAIN Initiative, and the Cancer Moonshot;
- Further expansion of the Patient-Focused Drug Development Initiative and requirements for the FDA to report on how patient experience data was used in regulatory review.
“Passage of the 21st Century Cures Act is the culmination of several years of hard work and advocacy by many rare disease patient advocates and patient advocacy organizations,” said Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD). "We echo the President and Vice President's sentiments today that this bill offers new hope to millions of Americans. We thank President Obama, Vice President Biden, and members of the House and Senate for their commitment to working together on this legislation that has the potential to help nearly every American family, including the 1 in 10 Americans with rare diseases who desperately need treatments and cures.”
Read more about 21st Century Cures.