How much is life or $ 2.1 million per injection: a wonderful gene therapy

On a Sunday evening by chance, although not, probably after all because I sometimes look at the news releases of Artemy Lebedev, Youtube algorithms recommended me another Artem Lebedev Design Lynch. In the beginning, Artemy, as always, announced that half of the funds raised, and collected in 3.5 hours a little less than 300 thousand rubles, will go to charity. And this charity will help Adrian:

Today we collect for the injection the most expensive medicine in the world for Andrian with a diagnosis of spinal muscular atrophy –

It is simply amazing that such amounts have to be allocated for treatment, and that several hundred thousand euros in emergency reserve is already “not money”, and does not guarantee access to a particular procedure. What is the reason for such a cost of one dose and why are such prices generally possible in the modern world? When, in order to treat ONE patient, it is necessary to spend sufficient funds to save the lives of more than one hundred or even thousands of other patients with less expensive diagnoses. And who, alas, are also sometimes forced to raise funds or die because of the inability to provide treatment. How can a dose of a drug be comparable with the cost of a Tier IV data center?

The fact is that the development of “breakthrough” drugs is a very expensive procedure and can take decades, while only 14% of drugs according to statistics receive final approval, and not in all countries. So the price here is not only the thirst for pharmacists to earn, but also a need that allows you to recoup costs and follow the interests of investors.

Spinal muscular atrophy is a rare genetic disease that affects every 8,000 newborns according to statistics. But it’s not so rare, if you imagine that 125,000 cases happen per billion newborns. People with the most severe form of SMA have difficulty eating, drinking (difficulty swallowing), raising their heads, or breathing. They suffer from frequent respiratory infections and usually die before the age of two. Those with a “moderate” SMA can survive to adolescence or early adulthood, but never gain the ability to stand.

Until recently, the disease was not treatable, since 2016 Spinraza received approval, which was used to stop the disease and, of course, access to such treatment was and is limited, since its cost ranged from 625,000 to 750,000 US dollars in the first year and then $ 375,000 annually for life. Everything changed when Novartis released Zolgensma. Now, in the case of type 1 SMA, only one injection is required before the age of 2 years, which in almost all cases results in complete recovery. The only negative is the price of $ 2,125,000, which makes the drug virtually inaccessible to many needy patients and challenges medical support programs in many countries. However, in recalculation, treatment with a new drug is cheaper than others that need to be taken for life annually.

There are many scandalous discussions on the topic of cost on the English-language Internet. Someone argues that the cost of the new drug is greatly overstated and it may well be lower and ranges from 300 to 900 thousand US dollars. Others say that billions of dollars were invested in the development of the drug, namely 2.6 billion and over 10 years in time. It is also worth noting that Novartis did not develop this medicine, but acquired AveXis for $ 8.2 billion, which is why a small number of patients do not allow to offer treatment at a lower cost.

In turn, Novartis, to make treatment more accessible, organized a lottery for patients from countries where the drug is still not approved and promised to distribute up to 100 doses for free annually. Nonetheless, patient advocates are concerned that the lottery is not aimed at those who need treatment in the first place. Although, apparently, they simply forgot that the birth rate around the world is over 130 million annually, which means that according to statistics, about 16,250 patients should be formed per year who have only 2 years to receive the necessary treatment and fully full life.

At the same time, critics predicted low returns, since treatment is catastrophically expensive. Nevertheless, Novartis exceeded expectations by 60% and earned over 160 million US dollars in just a quarter. Nevertheless, it is expected that the next drug for the treatment of SMA will become cheaper, drugs can not always be expensive, since there is competition. At the same time, CEO Novartis proposed introducing a payment model in which patients and insurance companies pay the full price only if the drug works. A 2016 study by MIT and the Dana-Farber Cancer Institute suggested financing expensive drugs on a mortgage basis, which would allow patients to pay for essential types of treatment for long periods of time and only if they work.

The high prices of such drugs ensure that medical researchers have the capital they need to fund their research, which creates such wonderful drugs as Zolgensma. Limitations of these prices by regulators would actually stop medical innovation – and prevent the development of future Zolgensma.

Unfortunately, any progress has side effects, and alas, many patients around the world do not receive the treatment they need. Having studied the problem, I saw that there are dozens of people in the “fundraising” funds in the Russian Federation alone, and it’s very sad to see when deadline is 2 months, and out of the necessary 160 million rubles for treatment, only 6 or 60 are collected at best. I hope that the development of IT infrastructure will be able to reduce the cost of research and make treatment more affordable in countries where state support is not particularly hoped for. In the meantime, we, the hosting provider, taking this opportunity, want to once again draw the attention of the IT community to this problem and wish everyone health and lack of such problems.

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