It's ridiculously expensive because it's a rare disease.
The total investment to bring this drug to market is in the range of hundreds of millions of dollars. Recouping that expense when there are only hundreds of patients in the US or thousands across the global requires a price that is far higher than normal medications.
Then layer on top the 1 in 20 chance that a drug actually makes it to market after that investment and the math starts to make sense.
> The total investment to bring this drug to market is in the range of hundreds of millions of dollars. Recouping that expense when there are only hundreds of patients in the US or thousands across the global requires a price that is far higher than normal medications.
So lets assume the drug cost 180 million to develop. There are currently 30,000 people with CF in united states. At the current price point they would recoup that cost within 2 months just from usa sales. This is a drug cf suffers would have to take for the rest of their life.
Its reasonable its more expensive than you average drug, but the $310,000 per year for these pills still seems excessive. Especially when its basically a death sentence without them so their customers are really held over a barrel.
"So lets assume the drug cost 180 million to develop."
The trouble is, you not only have to cover expenses for development of this, very successful drug, but also for 50 others that you never heard of, because they were duds, but the necessary trials still cost money.
Pharma is a numbers game. Most attempts fail, and you need to cover the cost of all the failures, otherwise you can't try out anything new.
It is possible that AI will help here, modelling drugs in virtual environments and recommending the most hopeful molecules. If it can be done, the ratio of failures to successes could improve.
There are currently 30,000 people with CF in united states.
This drug can only be used by CF patients with a specific mutation, not the entire population.
And I wouldn't assume $180M since this treatment is a combination of 3 different drugs all with their own development pathway and safety testing.
Then you need to look at the actual cost of manufacturing the drug, packaging it, delivering it to patients and all the salaries of the people it takes to do that. Sales of $180M is not profit, it's revenue.
Don't get me wrong, they are making a profit, but pharma company profit margins are usually in the 20-30% range and that's for the successful ones, not including all the companies that spend $100M and fail with nothing to show for it.
Then add on top the remaining patent life which is usually less than 10 years, after which the cost of the drug will plummet and the originator company will stop making money.
Tens of thousands of patients in USA, and given the massive longevity improvements, that count will go up. And add to that a likely increase in childbirths by CF patients.
People can save a bundle of money if they take advantage of the fact that it's a CYP-3A4 substrate, but I'm not going there and don't officially recommend this. But if you're lost in the woods and there's some grapefruits around....
The total investment to bring this drug to market is in the range of hundreds of millions of dollars. Recouping that expense when there are only hundreds of patients in the US or thousands across the global requires a price that is far higher than normal medications.
Then layer on top the 1 in 20 chance that a drug actually makes it to market after that investment and the math starts to make sense.