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The last 15 years or so have seen a dramatic increase in the cost of drugs for rare diseases. The reason these medications, in particular, are so expensive is that so few patients use it.
For example, about 30 babies are born every month in this country with spinal muscular atrophy. At the same time, Novartis paid $ 8.7 billion to acquire the biotech company behind the development of Zolgensma. For the value proposition to work, companies like Novartis need to recover their costs in chunks.
Without attractive prices for pharmaceutical companies, potential markets are so small that drug manufacturers will simply ignore rare diseases. Instead, they will spend time and money targeting conditions that affect more people.
The emergence of gene therapies such as Zolgensma has complicated the problem. Since doctors only administer these treatments once, companies have only one mouthful of apples to recover their development costs.
This, of course, differs from the model we are used to, where patients receive regular doses of ongoing treatment – and they (and their insurers) continue to pay for this treatment.
In the future, we can expect to see more single-dose gene therapies like Zolgensma. Already, there is Luxturna, a gene therapy that treats a rare form of blindness and costs $ 850,000. Cell therapy therapy for leukemia, also by Novartis, costs just under $ 500,000. By the year 2024, more than 60 gene therapies are expected to be commercialized.
Where all this leaves doctors, patients, insurers and the health system remains to be seen. But it should be noted that the proposals the Congress is considering to control drug prices would not affect Zolgensma or other gene therapies.
Two million dollars for a drug may seem like madness – until you think that we, as a society, are willing to pay to give a child sentenced to death a full and complete life. disease.
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