Spark sets precedent with outcomes models, $850K gene therapy price tag

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Spark Therapeutics Inc., the Philadelphia-based maker of voretigene neparvovec, announced Wednesday in a statement that it reached an agreement in principle with Harvard Pilgrim and affiliates of-Express Scripts to make the one-time treatment available to patients with a rare genetic defect that often leads to blindness.

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

Spark said it will offer rebates to insurers if the treatment doesn't work.

The therapy treats the genetic variation which affects some of thousand people in the US.

Gene therapies are an undeniably exciting new frontier for medicine.

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.

In August 2017 the FDA approved the first gene therapy for public use in the United States. The manufacturers say the price is a bargain. Hemophilia treatments for example can now cost up to $300,000 per year, for the lifespan of a patient, so a single dose of a cure priced at $1 million dollars doesn't seem entirely unreasonable. The product, approved in December by the Food and Drug Administration, is the first US -approved gene therapy for an inherited disease.

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That makes the treatment, called Luxturna and made by Spark Therapeutics, the most expensive medicine sold in the US, ranked by sticker price.

"Spark CEO Jeffrey Marrazzo's comments, implying that $850,000 represents something other than greed, are absurd and obscene", Maybarduk said. The one-time injection will cost patients $425,000 per eye and reverses the disease called retinal dystrophy. The company is also offering new ways for patients and insurers to pay for the drug. As a result of current government drug price reporting rules, the company can not offer an installment plan without being forced to sell the drug to Medicaid at an unsustainably low price.

"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.

Hardly anyone pays the full price of drugs out of pocket, however.

Luxturna's price is less than what many analysts and outside investors expected, and commercially insured patients likely won't have to pay for the treatment.

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". "Gene therapy is also being used to correct deficiencies in the production of dopamine, such as in Parkinson's disease, correct some immune system problems, and restore components needed for normal blood cell function in those with certain blood diseases, such hemophilia and beta-Thalassemia".

Spark's Luxturna, for instance, involves inserting a functioning copy of a missing gene directly into a patient's eye, where it encourages the body to produce a protein essential for sight. "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".

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