Trending News Today: Legislators Target Abuse of Orphan Drug Act

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Sen Chuck Grassley (R-Iowa) has launched an inquiry into potential abuses of the Orphan Drug Act that may contribute to high drug costs, according to Kaiser Health News. In a statement by Grassley­­—–chairman of the Senate Judiciary Committee­­––he said the inquiry is based on a 6-month investigation by KHN that discovered the program was being manipulated by drugmakers, in order to secure lucrative incentives and gain monopoly control of rare disease markets. According to KHN, many of the drugs that have gained orphan status are not completely new, with more than 70 drugs first approved by the FDA for mass market use.

A steroid treatment that has long been used outside of the United States was approved by the FDA last week for the treatment of Duchenne muscular dystrophy (DMD). However, patients with DMD were able access to deflazacort (Emflaza) for years by importing it from abroad for an average of approximately $1200 per year, according to The Washington Post. Babar Ghias, chief financial officer of Marathon Pharmaceuticals, told the Post that the new list price for the drug will cost $89,000 per year, which after rebates and discounts, will drop to $54,000 per year. However, with insurance and financial assistance offered by Marathon, patients will have either no or low out-of-pocket expenses. Aaron Kesselheim, an associate professor of medicine at Harvard Medical School and a critic of the system, told the Post that it appears to be yet another example of “gaming the system.” Kesselheim said in the report, “Instead of making the price at a level that is reasonable for patients, they make it a very high price and offer this pathway that patients may not qualify for, they may not know about, there may be limitations on it. So it’s a marketing move and not really a public health solution.” Ghias countered, saying that Marathon carefully came up with a price that was far lower than other rare disease drugs, according to the Post.

On Monday, the House Oversight Committee will vote on whether to send a resolution to the House floor that would block the “Death with Dignity” law in the Washington, DC. According to The Washington Post, oversight chairman Jason Chaffetz (R-Utah) has vowed to stop the law, saying it’s both immoral and misguided. The Death with Dignity law allows terminally ill patients to end their lives with a physician’s help. The law was signed last year by Democratic Mayor Muriel Bowser, and so far, 5 states have similar laws, according to the Post.