$2.1M Drug is Worth Every Penny

One time dose. Cures fatal genetic disease. Saves hundreds of thousands of dollars per year. Saves money for everyone.

30 babies each month can have a normal life.


This is remarkable, but I wonder if some families will simply be unable to afford it.

Expensive but apparently a net cost savings overall. I suspect that most gene therapies are going to be $$$ overall, considering the nature of the treatment.

Maybe larger cost reductions could be realized by genetic counseling.

Realize that by pricing the drug this way, Novartis can give the really small number of children with this disease free as a tax write-off. And if they can get some government funding to bring drugs of this type to a wide distribution of people for other disease they stand to make billions. This is a remarkable result. And will save the lives of 30 babies a month - PRICELESS. Time to pour billions more into gene therapy research because science works.

I am sure that no family is able to afford $2.1M pricetag. But I am also sure that all babies in the country have access to the drug. It is all about access and where to go, how to get it.

Every kid with this disease is on Medicaid or private insurance. Every kid is this diease will be covered, becuase this reduced costs for the insurance company.