After a unanimous panel vote, will Spark’s novel gene therapy be a $1 million drug?

“A panel of independent FDA advisers is meeting today to consider the merits of Spark Therapeutics’ (ONCE) Luxturna, a one-time gene therapy meant to treat a rare, inherited form of childhood blindness.”

Major news for gene therapy but look at the price of this.

https://www.bloomberg.com/news/articles/2017-10-12/spark-s-gene-therapy-to-treat-blindness-gets-closer-to-approval

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Yeah, it doesn’t cost less to develop a drug/therapy for a few hundred or thousand people than it does to develop one for millions. But, probably the 1 million price is still less than the cost of lifelong disability.

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If I had to pay 1’000’000 USD to get cured of sz I’d find the money. It would take a year or two but I would find it.

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1mill is alot to ask of children… also how long would it take realistically take for most of the worlds population to have all at once 1 million dollars… if there was an affordable payment plan… how long would most of the worlds population be indebted… then take into consideration this is the price tag…1million usd. to stop a child from going blind… thats heartless… genetic modification may cause an unfixable rift between the uber wealthy and 90% of the population… this needs to go in the scifi book…sorry ramble…

I didn’t mean the child, or family, should pay, if it wasn’t possible. That’s why we need things like insurance and socialized medicine.

A premature birth or childhood cancer can cost a million, in the US many people do go bankrupt due to medical debt, but the care still is provided.

In other parts of the world, kids go blind for lack of a few dollars worth of vitamin A, so we have a long way to go. This isn’t a problem of vitamin companies not donating vitamins (they do) it’s more a problem of distribution.

What I meant, for the US, is that it will make sense for medicare to cover this service because it’s a hell of a lot cheaper than a blind person. Same for sz frankly. It is a lot cheaper to pay hundreds of thousands for a cure than millions for a lifetime of hospitalizations - but because there are millions of people with sz, not thousands, it would not cost as much as a therapy developed for a very small number of people.

At this early point in time, I think the main question was not cost, but simply “can it be done?”

It may also be that there waere people lobbying for this and those people had some deep pockets. I imagine The Foundation For The Blind and other older, larger charities with big financial donors had some influence.

Otherwise, why would they not put their efforts and money to develope a drug for a larger poplulation as a larger population with a illness would make them more $.

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Exactly. 15141312

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I believe they attempted (and succeeded, as it was approved) in this therapy suitable for a relatively small group of people because it’s a monogenic disease with a relatively simple target (as simple as gene therapy gets, anyway.)

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