Gene therapy for rare form of blindness: $850K

Spark Therapeutics

Gene therapy for rare form of blindness: $850K

Luxturna, a new gene therapy drug used to treat blindness, carries a price tag of $850,000.

For some time Marrazzo has resisted the idea of offering rebates for patients who fail to benefit following treatment.

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

"But it's the right move", he continues, "and highlights a recent trend toward (relative) pricing moderation with big implications for the drug industry".

Just like two other gene therapies which got approved a year ago in the in the US, Luxturna is more expensive than nearly every medicine across the global market. Additionally, in partnership with Express Scripts, Spark has arranged it so that insurers can buy Luxturna directly instead of treatment centers having to purchase it first. "A green light from the EMA is a prerequisite for approval by the National Institute for Health and Care Excellence, the U.K.'s healthcare costs watchdog", Rob Davies reports in The Guardian.

"We wanted to balance the value and affordability concerns with a responsible price that would ensure access to patients", CEO Jeffrey Marrazzo told AP. Misa was 4-yearsold when he received his gene therapy treatment. With the burgeoning field of gene therapy finally taking hold of medicine, this is something that certainly needs to be managed.

Spark will also offer patient assistance programs with the aim that all eligible patients have access to Luxturna. On January 3, Luxturna's maker Spark Therapeutics gave the medical community its answer: $850,000 for a complete, one-time operation, or $425,000 per eye. And knowing full well just how much today's closely-watched marketing plan will be reviewed by the healthcare system, Spark's pricing team has come up with a mix of rebates and proposed staggered payment models created to ease past barbed payer barriers that can cripple any drug launch.

The sky-high price of some drugs - and so-called price-gouging by drug firms - became an issue in the USA presidential election after Martin Shkreli, a United States hedge fund entrepreneur, bought the drug Daraprim, used in the treatment of Aids and cancer, and hiked its price from US$13.50 to US$750.

The FDA then approved Luxturna a couple of weeks ago. The disorder usually makes the individuals blind in the adulthood. The firm said it has entered into an agreement with Massachusetts-based insurer Harvard Pilgrim to offer a partial refund if the drug doesn't work in the first three months, and is in discussions with other companies as well.

"Although much of this may still sound like the realm of mad scientists tinkering with the human body, gene therapy is an accepted experimental technique that is now being used to help patients with certain types of cancer to "target" specific antibodies that can be used to fight the disease", according to a blog post by Science Care, which runs a whole body donation program.

The advent of single-time gene therapies - and of new cancer cell therapies from Novartis and Gilead that are also administered once - has prompted a debate over how much drugmakers should charge for scientific breakthroughs and whether society can afford them. "Gene therapy holds promise for treating a wide range of diseases, including cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS".

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