Washington, D.C., December 7, 2016 — Today, the United States Senate overwhelmingly approved the 21st Century Cures Act, a game-changer for medical innovation, by a bipartisan vote of 94-5. The President is expected to sign the bill into law later this week.

“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), which has supported the bill. “The 21st Century Cures Act is a landmark medical innovation package 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.”

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.

NORD has participated in the 21st Century Cures Initiative since its inception in the winter of 2014 by submitting policy proposals for the initiative and testifying in hearings before the House Energy and Commerce Committee to provide the rare disease patient perspective. The organization has also published multiple position statements, led and joined coalition letters, and organized advocacy alerts to rally patients and advocates.

Added Saltonstall, “We were proud to help lead the charge to reauthorize the Rare Pediatric Disease Priority Review Voucher program, help author the provision to streamline review of targeted therapies for rare diseases, bring attention to the importance of NIH funding, and advocate for greater transparency of expanded access policies.

“After several years, we are excited to celebrate the Congressional enactment of the 21st Century Cures Act. Once again we wish to convey our deep gratitude. This bill would have never passed without the hard work of patient advocates over the past several years. There is still work to be done, but this is a major victory. Progress has been made, and with the ongoing effort of patient advocates working together for a better future, progress will continue,” Saltonstall concluded.

There are an estimated 7,000 rare diseases, which are defined as a disease affecting 200,000 or fewer Americans. Of the nearly 1 in 10 people with a rare disease, more than half are children. Nearly 95 percent of rare diseases have no treatment and the majority of these diseases are life-altering or life-limiting.

The National Organization for Rare Disorders (NORD) is a 501(c)(3) independent charity and the leading independent advocacy organization representing all patients and families affected by rare diseases.

Read the statement at


The MSUD Family Support Group is currently funding several research projects and we are proactively looking for researchers interested in developing new treatments or finding a cure for MSUD. Significant funding is necessary if we are to accomplish this goal.
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