H.R. 971 / S. 1421: The OPEN ACT

Orphan Product Extensions Now ~ Accelerating Cures & Treatments

Bipartisan Legislation Supported by 166 Patient Organizations
including Genetic Alliance, Global Genes, National MPS Society, the National Organization for Rare Disorders (NORD), and the Pediatric Cancer Foundation

Issue:  Despite advances made by the Orphan Drug Act, 95 percent of the 7,000 rare diseases still have no FDA-approved treatment. Biopharmaceutical companies seldom consider repurposing already approved therapies to treat rare diseases because there is little incentive for them to do so.

Solution:  Modeled on the incentive programs in the Best Pharmaceuticals for Children Act (BPCA), the OPEN ACT establishes an exclusivity extension, which would provide an additional six months of market exclusivity for the drug being repurposed for rare disease treatment. The sponsor company must demonstrate that the repurposed therapy is designated to treat a rare disease and obtains an approved rare disease indication from the FDA on the drug label.   Repurposing drugs is faster, cheaper, and presents fewer risks than traditional drug development.

Outcomes:

  • Double the number of treatments for rare disease patients. Many of these drugs would be priced at major market drug prices, thus bringing down the average cost of rare disease drugs.
  • A surge in biotech investment, new jobs, and grants to research universities to conduct clinical trials.
  • Fewer rare disease patients using untested and potentially ineffective drugs off–label.
Legislative Status

The OPEN ACT passed the House of Representatives in July, 2015 as a part of the bipartisan-supported 21st Century Cures Act (H.R. 6).  The bill must now be passed in the Senate to make it to the President’s desk.


Take Action to ensure that the OPEN ACT becomes law.  For additional questions about the OPEN ACT, contact our Senior Director of Advocacy and Science Policy, Max Bronstein, at mbronstein@everylifefoundation.org.

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