Ok everyone needs to understand the reason why it “costs” $2.1 million. AveXis, the company that makes the therapy and a subsidiary of Novartis, is doing groundbreaking work. This is gene therapy, not your basic drug manufacturing. It’s going to cost this much.
Imagine, they have to build facilities all over the US. Fully staffed with specialists trained to culture the viral vector, maintain it in cell lines, periodically maintain its integrity, make sure its not contaminated. It’s a thousand times more labor intensive than manufacturing a chemical drug (which you can simply store in a warehouse when you’ve done it). All of this work, for a condition which only affects 500 children per year. There is no other decent treatment, children born with SMA rarely live past 3, and can’t even sit up straight without assistance. This extremely sophisticated therapy works miraculously well. Many of these kids (with only a one time injection) are even walking and running as if nothing was wrong.
Eventually, as more gene therapy treatments for more conditions are available, the costs will drop. You can have 5 fully staffed facilities working on treatments meant for millions of patients rather than 500 per year. So $2.1 million treatment today may become a few hundred thousand in a decade.
At the end of the day, our issues with cost mainly come down to insurance companies, not these new gene therapy companies. No patient should ever have to pay for this. Society as a whole should split the costs, which is basically what insurance plans are. Don’t look at this and think “big Pharma” that’s a totally misrepresentation of the situation. Their work is extraordinary.
Edit: some have said “recouping the investment” is the reason for the price. For drug manufacturers that’s true, but for gene therapy treatments it’s more about “actually paying for the thousands of specialists that are needed to manufacture it.” The research was done by public universities, that’s not the issue. Gene therapy is a revolution in medicine, and it’s expensive as hell, but with health insurance reform in the US, we could split the cost as a society.
Edit Edit: Before more people start reddit-fighting each other, let me point out that healthcare for many life threatening conditions is crazy expensive. Take cancer. My dad overcame kidney cancer when I was in middle school. He had to be monitored for months on end, by dozens of nurses, doctors, specialists etc. It took advanced drugs, expensive medical technology, and two surgeries to cure him. We had insurance, which covered the more-than $15 million it took in labor, equipment, treatment, etc.
**This is how insurance works. This is why we have it.** We know that some diseases are ridiculously difficult to cure. That in order to save a patient, it would cost several millions of dollars. But we also know that they are rare. This allows us to become part of an insurance group. Where many individuals pay into a pool, so that when one gets sick, and requires several millions of dollars, they can take from that pool of money. Now, of course there are scandals and flat out evil behavior at drug companies from time to time. We can all work to prevent that by requiring transparency, or reevaluating patent laws. That’s not the case here. This treatment is expensive, because it is so labor intensive to make.
In the US, we have a pretty horribly inefficient health insurance system. It’s a collection of thousands of administrative workers, hundreds of public and private companies, that form a patchwork system of employer mandated insurance, which isn’t universal since its often unaffordable unless your employer is paying for it. We can reform this system in many ways, perhaps by simplifying the administration by creating a single national insurance program that could more easily cover costs like this $2.1 million cure. But it’s well worth it to save a life, and because it’s so rare, we can use an insurance system to cover this so that no parent needs to fear having a baby with SMA would destroy them financially. Nobody needs to face that if we pool the costs like we do with cancer. This is a miraculous achievement, since before this drug, SMA type 1 was a heartbreaking death sentence which kills within the first few years.
And to some of our European friends making that “only in America” comment, it costs the same for you too. You just are never burdened by noticing the costs, since most of your countries have you on a national insurance program which pools money and handles it for you. But this treatment is about to come to market in the EU, and your country’s insurance system will be paying $2.1 million for the treatment to save that child, and you’ll never see a price tag.
>Before the drug won Food and Drug Administration approval on May 24, AveXis had been providing it free to some patients under its compassionate use protocol. But the program was limited to children with Type 1 SMA, the worst form of the disease. Based on Natalia’s medical records, the Novartis subsidiary concluded that she had Type 2, a milder form, and denied her application.
>But after receiving more documentation from Natalia’s neurologist at Massachusetts General Hospital after the Globe story was published, AveXis concluded the toddler did indeed have the worst form and was entitled to receive Zolgensma as an experimental drug.
>The FDA approved Zolgensma for all forms of the disease, but only for children up to the age of 2. The compassionate use program made the drug available free to Type 1 patients older than 2 if they weighed less than 29 pounds and met other criteria. Natalia weighs 26 pounds.
As for the price tag, the medicine is for a rare disease, and it’s a one-dose shot, leading to the obscene price tag. To explain simply – for the drug company to recoup the investment, they need to charge that much since it’s not in demand and there’s only a need to administer once.