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Gene Therapy Optimism Combines With Fear of Cost

Treatments Aimed at Rare Diseases Are a Double-edged Sword

More and more miracle drugs are coming to the market, giving a niche group of patients hope where once there had been none. But with this hope comes a caveat: the prohibitive cost. How can the health system bear such a price tag?

For instance, the Wall Street Journal (WSJ) reports on a new gene-therapy treatment, Luxturna, which helps those born with the eye disorder Leber congenital amaurosis. The condition degrades the retina, and infants born with the condition will lose all sight by the time they are teenagers.

But not if they are treated with Luxturna, manufactured by Spark Therapeutics near Philadelphia. It is the first (but will certainly not be the last) gene-therapy treatment targeting a single gene to enter the U.S. market. According to the WSJ, “The therapy entails inserting a functional gene through harmless, virus-like particles into the retinal cells to compensate for the faulty gene causing his disease.”=

Four-year-old Caspian Soto, who walks with a cane and has “never seen the stars,” is one of the first patients in the country to receive the treatment. His doctors believe it will prevent further deterioration of his retina. But the miracle treatment is not cheap: Luxturna costs about $850,000 to treat both eyes. Eventually, the family’s insurance company (not named in the article) approved it, and Spark Therapeutics, the manufacturer, offered to pay the $4,000 out-of-pocket costs.

According to the WSJ, the Institute for Clinical and Economic Review, which conducts cost-effectiveness analyses on new therapies, concluded that Luxturna’s price exceeds standard thresholds for cost-effectiveness. This is partly due to its long-term benefits remaining unknown.

For Caspian, it’s a huge gamble. He received the treatment over the last couple of weeks and his family will have to wait for at least a month to find out if it has worked.

Source: The Wall Street Journal, September 24, 2018

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