Drugs for Rare Diseases on Their Way
Pharmaceutical and biotech companies are running patient tests of more than 5,400 potential new medicines, including many being tested for multiple conditions. Nearly 1,800 research projects are for rare diseases, and hundreds more are for disorders for which there’s been no new medicine in a decade or more….
[The companies are] trying to develop “specialty” or niche drugs meant for much smaller groups of patients but carrying six-figure price tags. And more companies are taking advantage of grants, tax credits and other incentives of the Orphan Drug Act passed in 1983. In the preceding decade, only 10 drugs for rare diseases had been approved, but more than 350 were approved from 1984 through 2011.
This is from USA Today. Study available here.
Exciting times.
“Nearly 1,800 research projects are for rare diseases, and hundreds more are for disorders for which there’s been no new medicine in a decade or more….”
Im sure this is encouraging news for people with very rare diseases. Im interested to watch the progress of these trials.
Call me a curmudgeon, but I’m skeptical. Orphan drug makers get a monopoly on the drugs they develop and can charge $100,000, $200,000 or even $300,000 per year for the therapy. It’s easy to feel good about providing drugs for exceedingly rare conditions. But it’s a problem all of us will ultimately pay for in the form of much higher premiums. It’s not that I don’t think rare diseases deserve research. But the current mechanisms allow the drug maker to extort just about any figure they can dream up and insurers are forced to pay it.
“In the preceding decade, only 10 drugs for rare diseases had been approved, but more than 350 were approved from 1984 through 2011.”
Wonder how much more this will trend will continue to grow…
Regardless of having research that focus on the niche market, there has to be some form of knowledge spillover from the research work itself. Added, the knowledge spillover may not be equal to the actual costs of conducting a research for the niche market.
I suspect that the expensive cystic fibrosis is on a par with a hospital stay that they inevitably have every year. So for an insurer it might actually be worth it. From another article, the drug is so effective it allows some to be taken off the transplant list and lead normal lives.
http://www.npr.org/blogs/health/2013/01/02/168353348/drug-fulfills-promise-of-research-into-cystic-fibrosis-gene