Spark Therapeutics offers medicine for treatment of rare inherited form of blindness

Spark Therapeutics offers medicine for treatment of rare inherited form of blindness

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

Anticipating criticism, Spark promised to reimburse patients if the treatment proves not to be effective, and said it expects U.S. health authorities to authorize payment in stages.

Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases. It isn't hard to understand why these drugs are hitting the market with sky-high prices, but it is also undeniably problematic when a life-saving medication arrives with a price-tag that is out of reach to many.

"New technologies such as gene and cell therapies hold out the potential to transform medicine", FDA Commissioner Dr. Scott Gottlieb-said in a statement announcing the August approval of Kymriah, considered to be the first gene therapy to be approved for the United States market. Spark Therapies says $850,000 with a money-back guarantee for its Luxturna gene therapy.

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

These therapies are generally meant to be taken once, a fact which drug developers argue sets them apart from traditional drugs taken for months or years.

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

"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 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 gene therapy is widely expected to be the first in a wave of cutting-edge treatments that are targeted at fixing the causes of a wide range of genetic diseases.

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This restores the patient's ability to make the missing enzyme - dramatically improving their eyesight and restoring a large degree of independence, although not offering 20/20 vision.

The group said it would share the risk of the treatment failing by paying some health insurers a rebate linked to whether the treatment worked in the first 30 to 90 days, and also to whether it was still effective after 30 months.

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". "But ... what is a fair price that will maximize affordability and accessibility and provide a reasonable return for the drug?" The pharmaceutical company said that is now negotiating with other insurers for similar payment schemes linked to treatment outcomes.

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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