Rare Disease Report

An Update on the 21st Century Cures Act

AUGUST 25, 2015
Guest post by NORD
In a Congress that is sharply divided on many fronts, the 21st Century Cures Act received notably bipartisan approval in July with a 344-77 vote in the U.S. House of Representatives. The bill, which includes broad-ranging measures to advance innovation in the discovery, development and distribution of medical therapies, now has gone to the Senate for discussion and review.

Over the months that the bill was debated in the House, NORD representatives provided both oral and written testimony on several occasions. At the time of the House approval, NORD President and CEO Peter L. Saltonstall issued a statement praising the Act’s mandatory funding for NIH and provisions to streamline and incentivize development and review of orphan therapies.

House Energy and Commerce Committee Chairman Fred Upton of Michigan originally introduced the bill, in collaboration with Representative Diana DeGette of Colorado. Specific measures within the legislation include:
  • annual increases in the NIH budget along with establishment of an NIH Innovation Fund to counteract the effects of sequestration and budget cuts that have reduced the purchasing power of NIH in recent years
  • collection of patient experience data to inform risk-benefit determinations as part of the FDA Patient-Focused Drug Development initiative
  • reauthorization of the priority review voucher program for rare pediatric diseases
  • codification of the priority review program for breakthrough medical devices and the lifting of the Humanitarian Use Devices cap from 4,000 uses to 8,000 uses
  • exemption of prescription drug user fees, an important source of funding for FDA, from any future government sequestration
  • incentives for companies to repurpose existing treatments for rare disease indications
  • de-identified data from NIH clinical trials would be more available to researchers
  • FDA would be required to create a more structured process for the review of biomarkers and other surrogate measures in assessing efficacy of drugs and medical devices

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