Too Ornery to Die
Until 2012, if you had a rare genetic disorder, there didn’t seem to be much hope for a cure. The science just wasn’t there, and creating drugs for small populations made little financial sense for big pharma.
The story of one cystic fibrosis drug is proof: not only is treating the root cause of a rare genetic disorder possible; it can be profitable.
But the way this new drug was made is causing a stir among some of the very scientists and doctors who helped to create it.
This week: what happens when a charity dips its toe into the risky world of venture capitalism to speed the search for a cure — and the result is a drug with a list price of about $300,000 a year?
Scientist Paul Quinton, 72, who discovered the root problem in cystic fibrosis patients, called the price tag “unconscionable.” He is one of 28 doctors and scientists who wrote a letter to the pharmaceutical company pushing back on the price tag. He said he’s in a difficult position.
“I’ve had friends tell me that they would shake hands with the devil if it meant that we would get a cure for this disease,” Quinton said.
But in the case of this particular drug, everybody wants to know: who gets to decide how much it costs to save a life?
Next month, we’re doing a show about not feeling at home in your own skin. And we want to hear your stories: when did your body betray you and how did you learn to live with it? Call and leave a message at 803-820-WNYC or leave a comment below.