Expensive gene therapy can cure the rarest blindness

Drugmakers see a pricing blueprint in an $850,000 gene therapy

Spark Therapeutics has devised a set of payment and access programs to make Luxturna more accessible while removing additional costs and risks that may face insurers and hospitals, CNBC News said.

It's the first true gene therapy approved in the US for an inherited genetic disease - vision loss caused by mutations in a gene called RPE65. The Post says it went with the lower figure in part because insurers indicated $500,000 per eye would shrink the pool of those who qualified, and the company is discussing rebate and payment plan options.

The US Foundation Fighting Blindness estimates there are around 18 therapies in development for other inherited forms of blindness. But gene therapies are given only once, with potentially permanent effects.

Luxturna, as approved by the FDA, specifically targets a retinal dystrophy that is named biallelic RPE65.

Drug prices are not regulated in the USA, as they are in many other countries, so drugmakers can price their goods like any other manufacturer.

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.

But even compared to other one-time gene therapies Luxturna is still an outlier.

Given Luxturna's federal approval and strong study results, experts say US insurers will likely cover the drug.

The manufacturer said that already had entered into an agreement in principle with New England insurance company Harvard Pilgrim Health Care, according to which Spark will partially recover the money if the patient does not respond to the specified tests that will take place 90 days after the treatment and again 30 months later. 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 USA who suffer from this kind of inherited retinal disease caused by a mutant gene.

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. "The mutation affects both eyes, usually at the same pace, so most patients would need treatment for both eyes, a Spark Therapeutics spokeswoman said", she writes.

The company has opted for a slightly lower price of $425,000 per eye and has announced three programmes making the one shot per eye treatment more manageable for United States payers. That idea would apply to government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly.

The company also plans to arrange for a new type of contracting model with payers to help ease the potential burden of upfront costs on providers.

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

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".

Spark's Luxturna, for instance, involves inserting a functioning copy of a missing gene directly into a patient's eye, where it encourages the body to produce a protein essential for sight. While it's less than the $1 million price tag that analysts had expected, it's still among the most expensive genetic therapies in the world.

To be sure, the list price of a drug is not necessarily what patients actually pay.

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

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