Trending News Today: Rare Disease Drug Manufacturer Pauses Launch Over Pricing Outrage

In light of the recent approval of deflazacort (Emflaza) for Duchenne muscular dystrophy, many patients, advocacy groups, and lawmakers have expressed concern over the price of the drug. While the generic version of the drug costs $1200 in other countries, Marathon Pharmaceuticals plans to charge patients $89,000 for the branded drug. Due to the outrage over the price, the manufacturer said that they plan to pause the launch of the drug, and plan to offer it through an expanded access program, according to Kaiser Health News.

Former FDA Commissioner Robert Califf, MD, recently spoke out about things to watch for in order to keep drugs safe under the new administration. He said that the 21^st Century Cures Act will likely streamline drug approvals in a positive way, according to Kaiser Health News. However, Dr Califf is concerned about maintaining safety standards for drugs that are explored through comprehensive clinical trials under the new administration.

David J Shulkin, MD, was unanimously confirmed to lead the Veterans Affairs (VA) Department, and is the only holdover from the Obama Administration, according to The Washington Post. Dr Shulkin’s confirmation also marks the first non-veteran chosen to lead the VA, which caused some outrage among veterans and advocacy groups, but did not disqualify him for the position. Dr Shulkin now must prove that he can transform the VA system to improve care for veterans.