Spark sets off gene therapy debate with $850K sticker on Luxturna

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

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

Spark Therapeutics, the company behind the treatment, had previously claimed the treatment was worth US$1million, citing the cost of a lifetime of blindness in lost earnings and wages for carers.

Spark Therapeutics' new gene therapy, called Luxturna, was reportedly lowered in price from $1 million after health insurers voiced concerns about their ability to cover the expensive treatment.

Spark announced it will ease the cost burden of Luxturna (vertigene neparvovec-rzyl) with an outcomes-based rebate scheme with a long-term durability measure, a contracting model that aims to reduce risk, and allowing taxpayer-funded payment schemes to make payments over time. The rare condition only affects two to three people per 100,000.

Gene Therapy: The Future Of Medicine?

Dr Peter Bach, director of a policy centre at Memorial Sloan Kettering Cancer Center in NY quoted regarding the issue, "The company very cleverly convinced everyone that they were going to charge a million dollars, so now they are being credited for being reasonable".

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.

"The product is just phenomenally innovative, and we've been talking about gene therapy for over 20 years; we're now at the threshold of having gene therapy reaching patients".

How much of the $850,000 cost of Luxturna would be returned to insurers if patients don't benefit from treatment was not disclosed.

Unlike traditional drugs, which tend to be taken for months or years at a time, gene therapies are meant to be one-off treatments that tackle a disease at its source, repairing faulty DNA so the body can fix itself.

As questions swirl around the cost and effectiveness of gene therapy, it could not be doubted that Luxturna is a breakthrough in the development of this kind of medicine.

The company said it had agreed bespoke deals with U.S. insurers, which cover the cost of most USA prescriptions, and they will get a refund if the drug doesn't work as expected.

It's clear that Spark is working hard to get the treatment to market - but the headline price is still likely to cause controversy in a country where the patient pays some, and sometimes all of the costs of drugs from their own pocket. "The therapy was controversially priced at $475,000 per treatment", Rich Haridy reminds us on New Atlas.

True to his meticulous nature, Marrazzo wants to carefully explain the multi-tier payment model Spark's team has been crafting and the objective behind it all: Not just steering the first gene therapy to an approval, but making sure that payers will cover it so that patients will be able use it to save their vision.

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

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.

Gene therapy involves delivering a healthy copy of a gene to replace a deficient one, in this case the RPE65 gene. "The answer to that is certainly not the $850,000 price tag announced today", he added.

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