An $850000 Price Tag on Gene Therapy Shouldn't Freak You Out-Yet

Spark Therapeutics' Luxturna will cost $850,000 the company announced Wednesday

Maybe if Spark focused its attention on developing a gene-therapy treatment for a disease less rare than the blindness Luxturna is supposed to treat, the company wouldn't have to charge such an exuberant price.

Luxturna is still more expensive than almost every other drug on the global market, including two other gene therapies approved earlier a year ago.

Spark already has a program in place with Boston insurer Harvard Pilgrim Health Care, by which Spark will get a full payment immediately after treatment and offer a rebate if patients do not see an immediate benefit to their eyesight, measured at 30 days or 30 months. The rare condition only affects two to three people per 100,000. Misa was 4-yearsold when he received his gene therapy treatment.

Many experts predicted Luxturna (voretigene neparvovec-rzyl) would ring in at about $1 million because it's designed for a rare genetic disease and a small patient population. "To be very frank, they've hit on a responsible price".

"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".

A proposal to CMS under which payments for Luxturna would be made over time, which is not now available "due to current government price reporting requirements", according to Spark.

Like most prescription medicines in the USA, most of the immediate costs of Luxturna will be borne by insurers - not patients - including private plans and government programs. The product, approved in December by the Food and Drug Administration, is the first US -approved gene therapy for an inherited disease.

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Mark Trusheim, who directs MIT's New Drug Development Paradigms program, says gene therapies are moving medicine from a model of "renting" treatments to one of "buying" long-term health improvements.

"Spark CEO Jeffrey Marrazzo's comments, implying that $850,000 represents something other than greed, are absurd and obscene", Maybarduk said. It will cost $425,000 per eye, and while $850,000 is steep, it's lower than the $1 million at which many expected the treatment to be priced.

And it's in negotiations with the Centers for Medicare and Medicaid Services, the federal government health insurance agency, to come up with ways to let insurers pay by installments.

And on the other side of the Atlantic the Association of the British Pharmaceutical Industry (ABPI) has often suggested that expensive treatments could be funded by instalments, or vary price per patient according to effectiveness.

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. "We're at the infancy of what I think could be a lot of innovations in how we pay for these really expensive drugs", Express Scripts' Chief Medical Officer Steve Miller told CNBC.

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. The therapy is also created to only be given once.

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