“There are millions of Americans who are still holding out that chance and still want that opportunity to change the lives of their loved ones, to change the lives of themselves.” ![]() “Rare diseases really aren’t that rare,” said Heidi Ross, vice president of policy at the National Association of Rare Disorders. Now there are over a thousand, but an estimated 90 percent of rare diseases still don’t have a treatment, advocates say. Before the ODA, there were less than 40 treatments for rare diseases. The 1983 Orphan Drug Act provided a framework for developing treatments for these rare diseases through financial incentives for drug manufacturers and expedited approvals. There are estimated to be about 10,000 rare diseases impacting 30 million Americans, half of whom are children, according to the National Institute of Health (NIH). Rare diseases are defined as those with fewer than 200,000 patients. ![]() “There’s a need to modify and move the Orphan Drug Act into the next generation.” “Quality and access are the two largest challenges for the next few years,” Thomas Crawford, a pediatric neurologist at Johns Hopkins Children’s Center, said. As the Orphan Drug Act turns 40, advocates want Congress and the Food and Drug Administration (FDA) to be more flexible when supporting the development of treatments and cures for rare diseases. ![]() Strict regulations on drug safety and testing make it difficult to develop new treatments for rare, obscure and deadly diseases, according to experts.
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