Gene Therapy Sets $850000 Price With Programs To Make It 'Accessible'

Dr. Albert Maguire right checks the eyes of Misa Kaabali 8 at the Children's Hospital of Philadelphia. Misa was 4-years-old when he received his gene therapy treatment

Cost of treating rare genetic blindness: $425000 per eye

Spark Therapeutics, the company producing Luxturna, has just revealed the new therapy will cost $850,000.

Reactions to the announcement yesterday range from the headline "U.S. drug firm says it can cure blindness - but it's going to cost an arm and a leg" in the South China Morning Post to Bloomberg "Gadfly" columnist Max Nisen pointing out that the price tag is less than had been expected and that a performance-based discount program for a drug therapy is "a risky move".

The pharmaceutical company Spark Therapeutics asks 850,000 Dollars to cure a rare inherited form of blindness, which is one of the top in ever-increasing medicine prices in the United States. Misa was 4-yearsold when he received his gene therapy treatment.

"Our work is not done, but we believe that the offerings we are announcing today will help ensure that eligible USA patients have the coverage and financial support they need to gain access to both Luxturna and the specialized medical care required to deliver the product at treatment centers".

"To be very frank, they've hit a responsible price".

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

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

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

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The drug is intended for patients with retinal dystrophy due to a mutation of the RPE65 gene.

To soften the pressure on the health system, and potentially set a precedent for other companies developing one-time therapies, Spark also set up several different payment plans. The company hopes its $425,000-per-eye treatment may eventually lead to new ways to pay for extremely expensive new therapies. It says it may give insurers a rebate if patients' vision gets worse within two and a half years, and it is working with Medicare and Medicaid so that the USA government can pay in installments.

"For a one-time therapy, like Luxturna, a non-traditional payment and distribution model is necessary to ensure needs of all parties - patients, payers and providers - are addressed", said Jennifer Luddy, a spokeswoman for Express Scripts.

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.

Alongside the pricing announcement for Luxturna, Spark also unveiled several different pricing programs created to help mitigate the burden on patients and payers. Ultimately, if you price it at a point that is too high, and you don't have access...you don't have patients who get therapy and get access to this one time treatment.

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

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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