Gene therapy treatment for hereditary eye disease will cost $850000


The FDA in December approved Luxturna, a new gene therapy that can treat the progressive blindness, and on Wednesday, its maker revealed the staggering cost: $425,000 per eye.

A landmark gene therapy from Spark Therapeutics to treat a rare, inherited form of blindness will cost $850,000.

An outcomes-based rebate arrangement with a long-term durability measure where Spark would share risk with certain health insurers by paying rebates if patient outcomes failed to meet a specified threshold, thereby linking payment to both 30-90 day efficacy and 30-month durability. The condition affects between 1,000 and 2,000 people in the United States.

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There are now about 1,000 cases of people who suffer from hereditary degeneration of the retina, and 10 to 20 new cases are expected to be added each year. There is often little explanation from the drug's manufacturer about why it would need to cost that much, save for them claiming that it's very expensive to develop and test the drugs.

Luxturna is the first true gene therapy approved by the FDA for an inherited genetic disease.

"The challenge is like going from being an apartment renter to a condo buyer and being shocked at [the] purchase price", he says.

Luxturna was just approved by the Food and Drug Administration last month after a dramatic hearing where teenagers spoke of seeing the stars in the night sky for the first time in years and at which eye doctors with no links to the company or the drug pleaded for its approval.

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Beyond these agreements, Spark has discussed with the Center for Medicare and Medicaid Services (CMS) a proposal allowing the company to offer payers the option to spread payments over multiple years in a payment-plan type model. It says it may give insurers a rebate if patients' vision gets worse within two and a half years, and it is working with Medicare and Medicaid so that the US government can pay in installments. The product works by delivering, via an eye injection, some 150 billion viral vector particles containing a correct copy of the RPE65 gene to retinal cells, restoring their ability to make the needed enzyme.

"The new payment models announced today are merely a way to disguise a price that is simply too high", said Patients For Affordable Drugs founder and president David Mitchell. However, some companies have begun to offer more detailed reasoning as the backlash against drug prices has grown more heated. We are waiting to see the price list of the next similar gene-targeting therapies.

To further allay concerns over the cost of the drug, Philadelphia-based Spark will use unconventional pricing models and schemes with insurers.

Express Scripts Chief Medical Officer Steve Miller, who is well known for his critiques of high drug prices, called Luxturna's $850,000 price tag "responsible".

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Marrazzo's not saying how much he's offering in rebates, but when I asked him why not a full refund for patients who don't respond adequately, he said that's not possible. "Our system can not handle unjustified prices like this, and the new payment models announced today are merely a way to disguise a price that is simply too high".