Rep. Gus Bilirakis (R-FL) authored the OPEN Act after meeting a young woman who had been diagnosed with dysautonomia, which is a blanket term used by medical professionals for conditions marked by malfunctions in the autonomic nervous system.
The OPEN Act is included as part of the 21st Century Cares Act.
Although diagnosis of such conditions can take years, this particular young woman was diagnosed quickly. With a regimen including an orphan drug and experimental treatments, her condition and quality of life have been improving. Orphan drugs are medications used to treat a rare disease.
"My OPEN Act will help these orphan diseases that are not easily treated due to their rare status," Bilirakis said. "This bill will incentivize drug makers and innovators to 'repurpose' major market drugs for life-threatening rare diseases. This opens the door to the development of hundreds of safe, effective and affordable treatments for rare disease patients.”
