New gene therapy to treat blindness has a price tag: $850K

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Spark Therapeutics, the company behind the treatment, had previously claimed the treatment was worth US$1million, citing the cost of a lifetime of blindness in lost earnings and wages for carers.

The first commercial gene therapy available in the US will cost $850,000, Endpoints News reports.

Spark already has a program in place with Boston insurer Harvard Pilgrim Health Care, by which Spark will get a full payment immediately after treatment and offer a rebate if patients do not see an immediate benefit to their eyesight, measured at 30 days or 30 months.

The condition is rare, affecting between 1,000 and 2,000 people in the U.S. About 10-20 babies are born each year with RPE65-mutated retinal disease, Spark estimates. Developers in Europe found this out the hard way when they priced gene therapy treatments at upwards of $1 million, and were unable to market the drugs. Consternation over skyrocketing drug prices in the US has led to intense scrutiny from patients, Congress, insurers and hospitals.

"To be very frank, they've hit a responsible price".

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

A proposal to CMS under which payments for Luxturna would be made over time, which is not now available "due to current government price reporting requirements", according to Spark. The Philadelphia-based company said it would also pay transport costs not covered by insurance to help patients get access to treatment centres.

Clinical trial evidence suggested that the novel gene therapy holds that potential for patients with biallelic RPE65 mutation-associated retinal dystrophy, which often progresses to blindness.

Spark, in fact, says there will be three distinct "payer programs" to ensure that US patients who need treatment can get it: "an outcomes-based rebate arrangement with a long-term durability measure, an innovative contracting model and a proposal to [Centers for Medicare and Medicaid Services] under which payments for Luxturna would be made over time".

To soften the pressure on the health system, and potentially set a precedent for other companies developing one-time therapies, Spark also set up several different payment plans. He cited CAR-T therapies for cancer, which cost hundreds of thousands of dollars, and newfangled immuno-oncology treatments with similar price tags.

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To be sure, the list price of a drug is not necessarily what patients actually pay.

"As far as the price, and the structures to pay the price, I think it's all pretty much in line with what we're seeing in other innovative therapies", said Dr. Stuart Orkin, a pediatric oncologist at the Dana-Farber Cancer Institute and Boston Children's Hospital.

Luxturna is delivered to patients just once.

Nevertheless, the drug has received approval and is touted as the nation's first gene therapy for an inherited disease. "But the question that must be asked is this: What is a fair price that will maximize affordability and accessibility and provide a reasonable return for the drug? The answer to that is certainly not the $850,000 price tag announced today".

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