Published: Fri, January 12, 2018
Finance | By Loren Pratt

Gene therapy for rare retinal disorder to cost $425000 per eye

Gene therapy for rare retinal disorder to cost $425000 per eye

A first-of-its kind genetic treatment for blindness will cost $850,000, less than the $1 million price tag that had been expected, but it's still among the most expensive genetic therapies in the world.

A single treatment of Luxturna nearly immediately reversed vision loss in most of the 21 volunteers who have received the drug.

"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.

"I feel like we made the right middle ground decision", Marrazzo said in an interview, balancing the company's desire to capture the economic value of Luxturna while ensuring patients will have access to the therapy.

Drug companies usually make money by selling drugs over and over again to the same patients and to new patients.

Luxturna is one of an emerging breed of gene therapies that differ from more established medicines administered over a period of time.

Spark said that government regulations meant it could not offer to sell the drug under instalment plans that would allow insurers and patients to bear the cost over time.

Steve Miller, chief medical officer at Express Scripts, said: "For one-time therapies like Luxturna, we believe non-traditional payment and distribution models are needed".

Express Scripts has a deal with Spark to distribute Luxturna.

Spark Therapeutics, perhaps aware of the problematic PR that could come its way after announcing the almost $1 million dollar price tag, also delivered a press release outlining a series of new payment models that could establish a novel precedent for how we deal with the financial reality behind these breakthrough, one-off gene therapy treatments. First, like Novartis Pharmaceuticals' one-time treatment Kymriah, for some, Luxturna's cost will be rebated if the sight of those receiving the treatment doesn't improve sufficiently. Speaking to CNBC Miller openly says, "We've got to figure this out, because - let's be frank - there are going to be more of these drugs coming to the marketplace for even bigger populations".

Some patient groups are already pushing back.

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.

"Luxturna offers hope to hundreds of patients".

After the FDA approval, speculation turned to its price. "The answer to that is certainly not the $850,000 price tag announced today", he added.

If insurance companies pay for patients to be treated, they'll pass along the costs to other patients in the form of higher premiums, Public Citizen's Peter Maybarduk said. The company's CEO, however, has said the effects of treatment could be "life-long".

"Our health care system is cracking under the strain". Developers in Europe found this out the hard way when they priced gene therapy treatments at upwards of $1 million, and were unable to market the drugs.

Like this: