Enthusiastic Parents Daunted by $740,000 Epilepsy Drug Price Tag

Parents of children with a rare type of epilepsy were hopeful when they heard about Ravicti, an FDA-approved drug that could help. However, the drug’s $740,000 yearly price tag could mean some parents may not be able to afford the drug. Some parents are enrolling their children in clinical trials to obtain treatment for epilepsy and other rare diseases, highlighting a serious national problem as parents find themselves unable to afford healthcare treatment. Rare disease drugs are particularly costly.

“A growing number of other parents are raising money for clinical trials — as much as $4 million to $7 million for research on cures, such as gene therapies. They’re also funding drug repurposing studies, which aim to find existing drugs to stabilize their children during the long hunt for cures. These trials are usually much cheaper and faster than developing new drugs. Parent foundations are paying $320,000 for the Ravicti trial, which has been in the making for three years. In contrast, developing a new drug can cost billions of dollars over a decade or more. Freed calls the trial — for which the drug’s maker is providing Ravicti for free — a “steal of a deal.”’ Read more here.

(Source: Erika Check Hayden, STAT, 6/3/21)