The practical cost will be negligible if you have appropriate health insurance, and Marathon contends that this is a low price for a rare disease drug. FDA approval should also increase access to a drug that may be too expensive to buy outright for some, even in its generic form.

However, the price hike underscores a problem with American pharmaceutical companies exploiting the orphan drug label to reap massive profits from medicine. The category was originally meant to encourage companies to develop treatments for rare conditions, but it's hard to make that case for Emflaza when far less expensive alternatives have been available for years. Moreover, Marathon could sell the review voucher for hundreds of millions of dollars. As helpful as the approval is, it suggests that reform is needed to strike a balance between rewarding scientific research and keeping prices down to Earth.