Gene Therapy Cure For Blindness Gets $850K Price Tag

A research scientist works in Spark Therapeutics’ laboratory.				Spark Therapeutics Inc

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

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 USA market. The rare condition only affects two to three people per 100,000. Luxturna will be distributed through just six to eight treatment centers in the USA initially and is expected to be available later in the first quarter. It isn't hard to understand why these drugs are hitting the market with sky-high prices, but it is also undeniably problematic when a life-saving medication arrives with a price-tag that is out of reach to many.

In an article, Bloomberg's Michelle Cortez noted that the schemes are very different from the approach offered by pharma companies, and could set a precedent for how expensive gene therapies could get to market in the future.

Critics, however, say Luxturna's price is still too high, adding that people without insurance and those who have high deductibles will face the brunt of the cost.

Like most prescription medicines in the US, most of the immediate costs of Luxturna will be borne by insurers - not patients - including private plans and government programs.

We're just going to have to wait and see what impacts the price of Luxturna has on the drug manufacturer, as well as on health insurers.

Spark Therapeutics CEO Jeff Marrazzo co-authored a recent blog post in the journal Health Affairs that said "the current payment system - accustomed to ongoing treatment of chronic diseases - creates significant challenges" for gene therapies and other treatments.

It will retail for $425,000 (Rs 2,69,89,625) per eye, which is less than the $1 million price tag that had been expected before the company received FDA approval in mid-December. 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. And the company is also working on covering patients' out of pocket costs as part of the overall price. But the Spark pricing team has worked it out so that under one model Spark can offer payers unspecified rebates at 30 days and 30 months - which is about the average amount of time a patient stays in a commercial plan - if Luxturna falls short of established efficacy goals on vision. 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.

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. "We're at the infancy of what I think could be a lot of innovations in how we pay for these really expensive drugs", Express Scripts' Chief Medical Officer Steve Miller told CNBC. "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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