On Nov. 20, 2014, Rep. Gus Bilirakis (R-FL) introduced the “Orphan Product Extensions Now Accelerating Cures and Treatments Act of 2014” (H.R. 5750), also called the OPEN Act. This legislation would provide an additional six months of exclusivity for approved drugs that are subsequently approved for a rare disease indication. The legislation was co-sponsored by Rep. G.K. Butterfield (D-NC), Rep. Michael McCaul (R-TX), and Rep. Alcee Hastings (D-FL) and referred to the House Committee on Energy and Commerce.
Extended exclusivity was also part of the Best Pharmaceuticals for Children Act for conducting pediatric clinical trials for drugs that may be prescribed to children. This has helped to increase research and development on the safest, effective doses for children. If legislative history repeats, rare disease patients will have more treatments available as pharmaceutical companies test and find approved products that can be used to treat some of the more than 7,000 rare diseases.
- OPEN Act Introduced to Repurpose Drugs for Rare Diseases: https://www.orphan-drugs.org/
2014/11/25/open-act- introduced-repurpose-drugs- rare-diseases/
- H.R.5750 – Orphan Product Extensions Now Accelerating Cures and Treatments Act of 2014: https://www.congress.gov/bill/
- Developing Safe and Effective Medicines for Children: https://www.nlm.nih.gov/
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