Just 12% of the 162,000 people estimated to be living with cystic fibrosis in nearly 100 countries are receiving a highly effective, but pricey treatment. And tens of thousands more are believed to be undiagnosed in dozens of other countries where the medicine is not available, according to a new study.
The findings suggest a sizable global disparity in treating cystic fibrosis, an inherited disorder that severely damages the lungs and limits life expectancy to 46 years in the U.S., according to the Cystic Fibrosis Foundation. The average life expectancy has climbed from 38 years a decade ago, due in part to the recent arrival of new medicines. But life expectancy can be half as much in low and middle-income countries, notably many in Africa.
The treatment, however, is available in 24 mostly wealthy countries, including the U.S., the U.K., France, and Canada. The medicine, a triple-combination therapy called Trikafta, is sold by Vertex Pharmaceuticals (VRTX) and is effective in treating roughly 90% of cystic fibrosis patients. The list price varies from country to country, but is nearly $327,000 per year in the U.S., before any discounts. Sales last year totaled $5.7 billion.
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