With the $1,000 genome fast approaching, the implications of Charley's post on "Knowing Which Patients Will Benefit First Hand" will become increasingly apparent.
In other words, drug and medical device companies are going to be slicing and dicing common and rare diseases into sub-populations with particular genetic variations that have a greater likelihood of benefiting from targeted therapies. As Charley points out, this is a win-win-win: for regulators, who can approve these drugs faster (and with less fear of being slammed by Congress later), for patients (who get more effective therapies), and companies (who can charge a premium price for even very small patient populations).
For instance, today Vertex Pharmaceuticals announed that it had submitted an application to the FDA for a new drug for cystic fibrosis, a devastating lung disease that kills people in the prime of their lives - their 30s and 40s - by basically drowning them from the inside out.
The drug, Kalydeco, targets the 4 percent of cystic fibrosis sufferers with a genetic mutation - that's 4 percent out of a total patient population of just 30,000 in the U.S. By the FDA's definition of an orphan disease (fewer than 200,000 patients in the U.S.) that's an orphan of an orphan.
Although the drug's benefits are modest (just a 10 percent improvement in lung function) it's still the first disease modifying agent available for CF. It's also expected to cost about $150,000 per year.
Here's a question for Charley (or anybody else). Is this model affordable in the long run? After all, increased efficacy is wonderful, and Kalydeco (assuming it pans out) sounds like a breakthrough innovation.
I think the model must be sustainable, but we're also going to have to revolutionize the process of drug development. It still took Vertex about 13 years to just get this application to the FDA. We've got to get much faster, cheaper, and more efficient in how we test and approve new medicines.
How do we get there? That's the big question.
I think what Paul describes here is certainly a trend, but it has its limitations.
If each drug takes $1 billion to reach the market and 10 million people use it over its patent protected lifetime, then each patient contributes, on average, $100 to the development of that drug. If we keep shrinking the denominator, then the economics become more difficult. Taken to the extreme of personalized medicine, with one specific drug for each person, we cannot expect that one person to cover the $1 billion development cost. Even if the development cost drops to $1 million per new drug, the economics don't work.
I think the development cost would need to drop to $10,000 per drug to be reasonable. To reach this price, we would need to exclude the FDA completely or allow the FDA to approve the process of drug development instead of each specific drug.
With this arrangement, the FDA would evaluate and approve the process of developing personalized medicines, but would then stand aside and let the drug companies deal directly with patients and physicians.
Just like drug companies which built their business models on blockbuster medicines, the FDA will need to rethink its approach in the era of micro-market and truly personalized drugs.