Last month, the FDA approved a gene therapy called Luxturna.  Luxturna can treat a rare eye disease that causes blindness.  But it’s kind of pricey.  It will cost $425,000 per eye.  Yes, that’s super expensive, but it was originally estimated that it was going to cost $1 million for the treatment.  In light of the price, Spark Therapeutics (the company that developed Luxturna) is working on some unconventional payment structures.  To start, Luxturna’s cost will be rebated back to some individuals if their vision doesn’t improve after receiving the treatment.  I should hope so!  I realize that this is a new drug, but at $850,000 for a treatment, this should literally cure all forms of blindness.  But this rebate is only for “some”individuals. This is the same kind of offering that you would see from Novartis Pharmaceuticals for their treatment Kymriah.

In addition, they have partnered with Express Scripts so that insurers can buy Luxturna directly. This is instead of treatment centers having to purchase it first.  That way, treatment centers don’t have to worry about buying an expensive product and waiting for reimbursement and insurers can avoid any price markups set by the treatment centers.  They are also looking at spreading out the costs over time.  Which would make this price tag a little bit better.  Spark CEO, Jeff Marrazzo said that they are discussing Luxturna coverage with a number of insurers including the US Centers for Medicare and Medicaid Services.


What’s interesting about this treatment (as well as many others) is that the price tag and how it’s paid for are tied very closely to their success.  This is also a huge part of their availability.  Which means, boatloads of money can be put into researching and developing these much-needed treatments and drugs, but their availability is tied to price.  Feel free to call me too liberal for the rant I’m about to go on.  But think about this concept for a moment, will you?  So, let’s say there is a “cure” discovered for breast cancer.  And I’m not talking chemotherapy and a whole battery of drugs and treatments.  I’m talking one pill that is taken, one time, and boom, your cancer is gone.  If that pill is going to cost too much money, it doesn’t matter because it will never see the light of day.

Do you see where I’m going with this?  Pharmaceutical companies will spend a lot of money to research and develop the drug, but if they can’t get any money for it, what happens?  Well, it sounds like it just sits on the shelf, so to speak.  This kind of sounds like they don’t want to actually help people.  I’m not saying they should give the drugs away for free, or anything like that, but there has to be a better system, doesn’t there?  Maybe I’m painting big pharma in a bad light, but damn, this sounds really harsh.  Especially when you think about what this particular drug can do.  Essentially it replaces a mutated gene found in people with retinal dystrophy with a normal copy of the gene.  The non-mutated gene can then produce a necessary protein that allows the retina to convert light into electrical signals.

The bottom line is this treatment would give some people the ability to see again.  Is it worth the heavy price tag?  I can’t answer that as I don’t live with this mutated gene.  Should insurance companies cover the treatment?  Of course, they should, but will they is the big question that we have to ask.  I’m shocked to learn how they treat pharmaceuticals in terms of their availability.  I didn’t realize that’s what stopped certain drugs or treatments from making it to market. I would like to see more of these kinds of treatments become available so people have options, and don’t have to take a mortgage out to get the treatment.