Company Says Its Treatment for Blindness Will Cost $850K

Dr. Albert Maguire right checks the eyes of Misa Kaabali 8 at the Children’s Hospital of Philadelphia. Misa was 4-yearsold when he received his gene therapy treatment. AP FILE

Company Says Its Treatment for Blindness Will Cost $850K

Spark Therapeutics, its maker, said that Luxturna is priced fairly, noting it is a one-time treatment and that treatments for rare diseases are usually expensive because of small patient populations.

Pharmaceuticals firm Spark Therapeutics has said it will charge a hefty $850,000, for its new drug to treat a rare form of blindness, the highest list price of any drug.

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 affects between 1,000 and 2,000 people in the United States.

Today, finally, is the beginning of another important chapter in the gene therapy story.

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.

They include a treatment for haemophilia and another for so-called "bubble baby" syndrome, where sufferers have to live in a sterile environment, which is to be offered on the NHS despite a £500,000 price tag.

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The company has grown sales at a 6.80% annual rate over the past five years, putting it in the medium growth category. And then on the other side of the spectrum one analyst entrenched in the bullish camp has a target as high as $82.

CEO Jeffrey Marrazzo stated that we wanted to balance the value and the affordability concerns with a subject amount that would ensure access to patients, in an interview with The Associated Press.

Luxturna is just the first of many one-time gene therapy treatments likely to hit in the market over the coming years, and therefore will necessitate changes in how payment and reimbursement are conducted. Spark Therapies says $850,000 with a money-back guarantee for its Luxturna gene therapy. For instance, Spark wants to offer discounts based on whether or not the drug initially works and remains effective; there are roughly 1,000 to 2,000 patients in the US who suffer from this kind of inherited retinal disease caused by a mutant gene. Last year, a European-approved gene therapy called Glybera flopped because it treated a very rare disease and cost $1 million. It costs $425,000 per injection. The company is also offering new ways for patients and insurers to pay for the drug.

Most prescription drugs in the U.S. are paid for by insurers, including private plans and government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly. 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.

"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. Steve Miller, Express' chief medical officer, said that "Many people were anticipating this [retinal disease treatment] would be more than a million dollars..."

After the FDA approval, speculation turned to its price.

However, even though the cost is lower than the $1 million price tag that analysts were expecting, there remain concerns that the drug is overpriced.

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