Based on the AveXis studies, the FDA approved Zolgensma Friday, making it only the second gene therapy ever approved for a genetic disorder. There are currently around 700 patients eligible for the treatment, according to Novartis, and roughly 30 babies are born each month with the disease.
The single biggest reason for the high cost of this drug is the fact that the number of doses the company will sell is very small Do the math. If it cost $1 billion to develop this drug and get it through the FDA approval process, then this cost has to be spread among the 700 existing patients and 30 new patients per month. If Novartis has 12 years of patent protection for this drug, then this $1 billion development cost has to carried by only about 5,000 patients. That's a $200,000 cost per patient just for this drug alone -- which means it doesn't include all of the other costs Novartis has to cover over those 12 years for the testing and research of drugs that are never approved by the FDA.
How much do you think an iPhone or a Toyota SUV or a box of Corn Flakes would cost if Apple, Toyota, and Kellogg's only sold 5,000 of them over 12 years?
The deeper question is, how much of a limited pool of taxpayer money and insurance premiums can go to support development of drugs that cost multi-billions to get to market and which help only 1/600 of one percent of Americans?