by US Army Africa
Philly Inquirer: Fresh take on the generic drug problem
[Via Knight Science Journalism Tracker]
It’s an old story: A generic drug looks promising, but because anybody can make it, nobody wants to.
Drug companies, unlike many other businesses, often refuse to sell a product unless they have monopoly control. I guess we should be grateful they still sell us aspirin.
As I say, it’s an old story. But Marie McCullough of the Philadelphia Inquirer has made this story fresh again with a nice piece on a generic multiple sclerosis drug that looks promising but is still years and millions of dollars away from approval.
She begins with that old standby, the anecdotal lede–but it’s a good one. It’s about a woman who has mostly avoided the worst symptoms of MS apparently because she was pregnant–six times. The lede, as an anecdotal lede should, directs us right to the heart of the story: A UCLA neurobiologist who studied the protective effect of pregnancy in MS has come up with a potentially useful drug that could possibly trigger that protection in women who aren’t pregnant.
McCullough gives us the good news: The biologist, Rhonda Voskuhl, has raised $5.6 million to study the drug, a generic called estriol. But in the very next instant, she cuts us off at the knees:
If this sounds like a poignant success story, it isn’t.
Ouch.
I like that line so much that I plan to steal it for one of my own stories at the first opportunity.
It turns out that the multimillion dollar study is not the one that could win FDA approval for this drug. It’s the second-last study. The last one could cost $20 million. And it’s years away. The results from the current trial won’t be available until 2013, and then Voskuhl will have to start raising the really big bucks. And she has already been working on this for a decade.
[More]
Development of a new drug costs a lot of money. This drug has not been proven to work, which is why it is undergoing clinical trials.
Phase I trials are to determine safety and dosage. Phase II are a small scale study to make sure the recommended dose is safe. It is really only Phase III, which are large scale studies with thousands of people, that are designed to determine efficacy. Most drugs fail at this point. This one might also. It might lessen the effects of MS but increase the incidence of all sorts of other problems. That is why large numbers of people need to be examined.
Few drugs are free of side effects. There could be major problems that only appear in 1% of the population taking the drug. The FDA really needs to know this in order for the advisory panels to make a good decision on the drug. To get good statistics at that level can require thousands of people followed over many years.
Phase III studies, because they can involved 1000s of patients with huge amounts of record-keeping, can be very expensive.
Someone has to pay the huge cost of the Phase III trials, which has usually been done by the drug companies. So, unless they can recoup the costs, something difficult with a generic, they will usually pass.
And the NIH has historically not been a big funder for clinical trials. It funds research, usually at less than $5 million a grant. It has only recently funded some development grants but it is just not in the position to fund large scale clinical trials at $20 million a pop or more.
So, we will have to find a better model for funding these trials if we want to get these sorts of drugs approved.
