Blindness Gene Therapy Becomes Most Expensive Medicine In US

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Philadelphia-based Spark Therapeutics said yesterday that it planned to charge $425,000 per eye for Luxturna, the first gene therapy for an inherited disease to reach the US market. Between 1,000 and 2,000 people in the United States are estimated to have the disease.

Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases. Luxturna will be distributed through just six to eight treatment centers in the US initially and is expected to be available later in the first quarter.

But others said that the company had behaved in a responsible way by keeping the price tag below the million-dollar price mooted when it was approved, and offering ways to make the cost more tolerable through the rebate and instalment payment proposals. 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".

Given Luxturna's federal approval and strong study results, experts say USA insurers will likely cover the drug.

One more gene therapy squeaked in before year's end, not for cancer treatment, but an inherited form of vision loss that can lead to blindness called biallelic RPE65 mutation-associated retinal dystrophy.

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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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. It will cost $425,000 per eye, and while $850,000 is steep, it's lower than the $1 million at which many expected the treatment to be priced.

The question for Orkin, who has written extensively about how society should pay for gene therapy, will be just how Spark determines whether an individual patient treated with Luxturna has "failed" and thus merits a rebate.

As well as outcome-based pricing models, Spark is working with the US Centers for Medicare and Medicaid Services (CMS) to establish multi-year payment plans to help make the cost more accessible to private-paying patients. Ultimately, if you price it at a point that is too high, and you don't have don't have patients who get therapy and get access to this one time treatment.

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

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. The gene therapy is widely expected to be the first in a wave of cutting-edge treatments that are targeted at fixing the causes of a wide range of genetic diseases. "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".