Gene Therapy Sets $850000 Price With Programs To Make It 'Accessible'

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The treatment called Luxturna is manufactured by Spark Therapeutics and helps treat Leber congenital aumaurosis, an inherited condition that leads to blindness.

A first-of-its kind genetic treatment for blindness will cost $850,000, less than the $1 million price tag that had been expected, but it's still among the most expensive genetic therapies in the world.

Harvard Pilgrim, a non-profit healthcare group, which sells health plans in New England, has signed up to the scheme, and Spark said it was in "active discussions with other commercial insurers". This particular genetic blindness only affects a few thousand people in the USA, making the market for it rather niche.

Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases.

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. "To be very frank, they've hit on a responsible price". Following the announcement, FDA commissioner Scott Gottlieb, MD, said he believed gene therapies like Luxturna will soon be mainstays "in treating, and maybe curing, many of our most devastating and intractable illnesses". Many investors expected Spark to charge $1 million or more for Luxturna, so the actual price will be considered a bargain by some. Under the agreement, payers would expedite benefits processing and cap patient out-of-pocket costs at in-network limits.

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The company estimated that between 1,000 and 2,000 people in the USA have vision loss due to the mutation the drug is meant to treat.

The drug will be available in the United States in early spring, with an estimated 1,000 to 2,000 people in the USA standing to benefit from this treatment, according to Spark. The pharmaceutical company said that is now negotiating with other insurers for similar payment schemes linked to treatment outcomes. The therapy is also created to only be given once. He cited CAR-T therapies for cancer, which cost hundreds of thousands of dollars, and newfangled immuno-oncology treatments with similar price tags. The company also stated that its proposal to the Centers for Medicare and Medicaid Services, the government program that covers about 100 million Americans, would allow installment payments for the drug. Ultimately, if you price it at a point that is too high, and you don't have access...you don't have patients who get therapy and get access to this one time treatment.

"We are also eager to work with CMS to enable more meaningful rebates as part of the pay-for-performance model", Marrazzo said.

Steve Miller, CEO of Express Scripts, is partnering with Spark Therapeutics to help make Luxturna more accessible to insurers and individuals.

This restores the patient's ability to make the missing enzyme - dramatically improving their eyesight and restoring a large degree of independence, although not offering 20/20 vision. "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?"

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