On December 1, 2016 the House of Representatives passed the 21st Century Cures Act 392 – 26) and on December 7 this legislation passed through the Senate (94 – 5). With an overwhelming majority in favor in both houses of Congress the bill is now headed to President Obama who has pledged to sign it into law as soon as it reaches his desk.  This updated version of 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

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.

Senator Lamar Alexander, R – Tenn., who leads the committee involved in writing the bill, called the measure on Monday a “Christmas miracle” that would “help us take advantage of the breathtaking advances in biomedical research and bring those innovations to doctors’ offices and patients’ medicine cabinets around the country.”[1]

Unfortunately, the updated legislation left out one of the key bipartisan 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 and was included in the House-passed version of the 21st Century Cures Act, but was dropped from the new bill.  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 critical to accelerating 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.[2]

[1]Andrew Siddons, CQ Roll Call, December 7, 2016

[2] Faster Track Newsletter, December 2016, EveryLife Foundation for Rare Diseases, info@EveryLifeFoundation.org