On the recordMarch 7, 2017
as cochairs of the Rare Disease Caucus, Senator Klobuchar and I have worked hard to bring more hope to patients and their families who are coping with rare diseases on a daily basis. Today 1 in 20 individuals worldwide is living with one or more of the more than 7,000 rare diseases, 95 percent of which do not have an effective treatment. While the incentives provided by the Orphan Drug Act, first championed by me in 1983, has led to the approval of nearly 600 orphan drugs, much more needs to be done. Many patients living with rare diseases rely on the FDA to evaluate and approve treatment options for their conditions. That is why it is so important for the FDA to use its authority to accelerate the evaluation and approval of drugs for treating rare diseases and for Congress to ensure that proper incentives exist for research to discover and make affordable treatments and cures available for this community. To address this issue, Congress passed the FDA Safety and Innovation Act of 2012, which refined and strengthened the tools available to FDA to accelerate the evaluation and approval of new drugs targeting unmet medical needs for rare conditions. I have been paying close attention to how this new authority translates into advances for patients suffering from conditions such as Duchenne muscular dystrophy, atypical hemolytic uremic syndrome, Bertrand-N-glycanase deficiency, and other rare diseases.…
