In the latest issue of Science, there’s an article about Trikafta (elexacaftor/ivacaftor/ tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation, which was approved by the FDA this past October.
In the U.S. alone, there are more than 30,000 people with cystic fibrosis, an inherited condition characterized by the buildup of thick, sticky mucus that results in progressive damage to the respiratory system and chronic digestive system problems, and that can have other complications as well. A generation ago, children with CF seldom lived to adulthood, and even today CF sufferers seldom live to age forty. Currently, there are more than 30,000 people in the U.S. with the disease.
In the interests of full disclosure, I have a personal interest in cystic fibrosis, because almost forty years ago one of my daughters was given a preliminary diagnosis of CF, which was indeed terrifying. Fortunately, additional tests determined that she didn’t have CF, but a combination of other factors mimicking it, which were treatable, and she recovered without lasting ill-effects. But that experience definitely made me aware of the devastating effects of cystic fibrosis.
So, the development of Trikafta struck me as a godsend for cystic fibrosis sufferers… until I saw the price tag, $311,000 a year [the “list” price], every year. That’s what it will cost to provide CF sufferers with the chance to live a close to normal and productive life.
Since Trikafta is designed to deal with the most common form of cystic fibrosis, it won’t work for all CF sufferers, but even if it only works for 60%, the annual cost to those people, or their insurors, would total nearly $6 billion.
Needless to say, some medical insurance companies have already turned down patients.
Somehow, I doubt that developing Trikafta cost anywhere near $6 billion, and it strikes me that, at least from everything I’ve seen published (and I hope I’m wrong), this is just another form of medical extortion.