Novartis believes its new gene therapy is worth more than $4 million for a one-time dose, and the company has some data to back that up. But, with a global spotlight on the escalating cost of medicine, is it politically viable to set a new record for the world’s most expensive drug?

The treatment, called AVXS-101, has demonstrated dramatic effects in spinal muscular atrophy, a rare genetic disorder whose most severe form is fatal for almost all patients before age 2. In a 15-patient trial, infants with SMA who got AVXS-101 had a 100 percent survival rate after 24 months, data that convinced Novartis to pay $8.7 billion for the gene therapy’s inventor.

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  • Something fundamentally wrong with the argument that high price for a gene therapy is justified because it is a “one-time treatment”. If that argument would have been applied 50 years ago when antibiotics were emerging, that also were “one-time treatment” for the patient with an (usually lethal) infection, we would not have antibiotics today. Most probably within few years from now most non communicable diseases will have a “one-time” gene based therapy, therefore, the pricing/marketing strategy should not be based on the “one-time” dose or regimen but rather on the size (number) of the patients that would benefit from the gene based technology, that probably would be similar independently of the disease.

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