Early in my scientific career I was in a meeting to decide what one of our department’s new drug discovery projects was going to be. A team made a presentation on a prospective target, which was scientifically solid but was shot down because the potential market was too small. Not enough people had the disease, so we’d never be able to recoup our costs of finding and developing a treatment. It was certainly an eye-opener to a young, naïve scientist.
Since then, my career has taken place in what you could call the Genome Age. And one of the great promises of the Genome Age has been more or less “When we know peoples’ DNA sequences, we’ll be able to tailor drugs to their specific needs”. These days, it’s known as “personalized medicine”.
As a patient, I always thought this sounded awesome, like something out of science fiction. But as a drugmaker (and perhaps one weaned on the bottom line calculations like those above), I thought this sounded like a terrible idea. I’d want to make a drug that worked on everyone, not just a small segment of the population.
An approval by the FDA this week highlights the tricky nature of making drugs for small diseases. The Agency approved Vertex’s drug Kalydeco, which treats a rare form of cystic fibrosis. About 1200 people in the US currently suffer from this version of the disease, which arises from a genetic mutation. The FDA approved the drug ahead of schedule and called it a “breakthrough” – language very very rarely uttered by The Man.
This is great news for this patient population. The drug has been shown to be remarkably effective at diminishing symptoms and limiting disease progression. Well, maybe it’s not great news. The yearly cost for a prescription of Kalydeco is slated to be $294,000.
Why so high? Well, we don’t have all the numbers, but a drug like Kalydeco probably took about $500,000,000 to discover, develop, and produce and the company needs to make that back and then some if they’d hope to see a reasonable profit (and this doesn’t begin to take into account the billions spent on R&D projects that never make it to the marketplace).
Because of the small patient population, Vertex claims that it will be able to assign a case-manager to each prescription to help the consumers navigate the financial aspects of taking the drug and in certain cases, it will pay up to $80,000 for patients who are commercially insured but facing high co-pays and/or co-insurance costs.
I’m going to watch this drug with a lot of interest, because I think it will be a bellwether of how personalized medicines might make it in market. Will people and (more importantly) insurance companies pay for it?
If not, what happens to the dream of personalized medicine and so-called “orphan diseases”? You can imagine a scenario where the government supports research for indications that might be “non-profitable”, but spending money on things that might or might not work and that will only benefit small portions of society seems to be out of fashion these days.
Perhaps I’m still naïve, but I’d like to think that somehow we – drug companies, researchers, government, insurers and society – will be able to figure out a way to make this work, because NOT finding a way to get cures to patients merely because enough people aren’t suffering seems so 20th century.