Orphan Product Extensions Now ~ Accelerating Cures & Treatments (OPEN ACT)
The OPEN ACT passed the House in July of 2015, but was not signed into law. The bill was reintroduced in the House as H.R. 1223 on February 27th, 2017. The bill has been reintroduced in the Senate as S. 1509.
Problem: Despite advances made by the Orphan Drug Act, 95 percent of the 7,000 rare diseases (including childhood cancer) 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.
The OPEN ACT is bipartisan legislation and supported by 177 Patient Organizations
including Genetic Alliance, Global Genes, National MPS Society, the National Organization for Rare Disorders (NORD), and the Pediatric Cancer Foundation
Use this link to contact your Representative. Take action to support the OPEN ACT – Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 1223). The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases and pediatric cancers.
For additional questions about the OPEN ACT, contact our EveryLife Foundation, Senior Director of Advocacy and Science Policy, Max Bronstein, at email@example.com.