The OPEN ACT passed the House in July of 2015, but was not signed into law. The bill was reintroduced in the House on February 27th, 2017. The bill will be reintroduced in the Senate in early 2017. Click here to download an informational OPEN ACT one-sheet.
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.
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.