On the recordFebruary 16, 2017
Mr. President, I recently read a story in the Wall Street Journal that I thought was so alarming it demanded action. Here is the headline: ``Marathon Pharmaceuticals to Charge $89,000 for Muscular Dystrophy Drug After 70-Fold Increase.'' Yes, that is $89,000 a year, and, yes, that is a 70-fold increase-- 70-fold, as in 7,000 percent. For those of you who have not read the article, here is the story. There is a rare disease called Duchenne muscular dystrophy. It affects about 12,000 young men in the United States. Most of them, unfortunately, end up dying in their twenties and thirties because of it. We don't have a cure yet for Duchenne. Until recently, there was not even a treatment with FDA approval. So, for many years, patients and parents have been importing a drug called deflazacort, a steroid, from other countries. Even though it is not a cure, it at least helps treat symptoms and has been a welcome relief to many families. Well, technically it is illegal to import a drug that doesn't have FDA approval. But there is a catch. The FDA does not quite enforce the ban against all unapproved drugs. In fact, it has issued regulatory guidance saying that you can get an exemption and buy an unapproved drug from overseas if you meet five conditions. First, you have to have a serious illness for which there is no other treatment available. Second, you can't sell the drug. Third, you can't pose an unreasonable risk to your health. Fourth, it has to be for you and you alone.…





