Spark Therapeutics' gene therapy for a rare blindness gets $850K price tag

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

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

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.

Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases. Consternation over skyrocketing drug prices in the USA has led to intense scrutiny from patients, Congress, insurers and hospitals. However, "there are very few such patients, and the price of every treatment matters a great deal".

In August 2017 the FDA approved the first gene therapy for public use in the United States. Many investors expected Spark to charge $1 million or more for Luxturna, so the actual price will be considered a bargain by some.

Another company making pricey treatments has a plan to offer rebates if patients don't get better. The gene therapy - the first of its kind - was approved to treat a rare form of hereditary blindness caused by a mutation in the RPE65 gene, and only affects a few hundred people in the U.S. The hope is that such treatments could be cures, but at the moment, no one knows exactly how long the gene therapy's effects-which do not fully restore vision-will last.

Consternation over skyrocketing drug prices, especially in the USA, has led to intense scrutiny from patients, Congress, insurers and hospitals.

The first two novel gene therapies for cancer treatment passed through FDA approval earlier this year, first B-cell acute lymphoblastic leukemia (ALL) followed quickly by Yescarta for large B-cell lymphoma - a type of non-Hodgkin lymphoma. 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.

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. The treatment for one pair of eyes was expected by Wall Street to be around $1 million, Bloomberg Technology reported on Wednesday, with the final announced price coming in at $425,000 per eye - just short of the prediction.

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.

"Many were anticipating this was going to be over a million dollars because it's a small patient population", said Dr. Steve Miller, chief medical officer of Express Scripts, a company working with Spark Therapeutics on some of its services, according to Forbes. "But ... what is a fair price that will maximize affordability and accessibility and provide a reasonable return for the drug?"

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. "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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