Rare Disease Report

The Most Transformational Biomedical Legislation in the Past 40 Years

DECEMBER 09, 2016
RDR Staff
Congressman Fred Upton, who led to charge to get 21st Century Cures Act passed, delivered this weekly address about the bill that recently passed the House and Senate.

As Upton notes, “This is this the most transformational biomedical legislation in the past 40 years.”

Some of the highlights of the bill that are of interest to the rare disease community include:
  • $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)
  • 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
  • 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
One item that was dropped from the act was The Orphan Products Extensions Now, Accelerating Cures and Treatments (OPEN ACT) which would have provided an incentive for companies to repurpose existing drugs for rare disease indications.
It is expected to be signed into law next week by President Obama.

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