Have you read about the furor over Fred Baron’s quest to get Tysabri to attempt to treat his myeloma?
According to his son’s blog, they believe it could help prolong his life. Andrew Baron says that Biogen, the maker of Tysabri (a drug used to treat MS), will not allow him to have the drug. It is, however, being tested on MM patients in a phase I trial.
I still have to do some reading on this drug. The side effects are potentially serious, but, when you have only weeks left to live, I don’t suppose that matters.
Andrew’s blog: http://dembot.com/post/54498664/open-letter-to-james-c-mullen-ceo-of-biogen
My understanding was that whatever happened with the drug that it would count against the drug company in it efforts to get the drug approved for this and other diseases. The FDA would not indemnify the drug company.
In January 2008 grandpa started feeling a bit weary and sick. 3 months later he weighted about 120 lbs.
– Culprit? MM
He’s rocked it though and pullin’ through quite well. Needs to gain a bit more weight though.
I think the trouble with the approval process is that there can’t really be a “one size fits all” process. In the MM world, we saw Velcade get fast tracked, and that was a good thing.
For patients who literally have only days or weeks to live, I think THEY should get to make the choice. For someone like me, at this stage of disease, caution makes perfect sense.
Unfortunately, there are people who would seek to benefit from an unfavorable outcome. That company has to protect itself from litigation.
I have a love/hate relationship with the FDA. :) We can’t just allow manufacturers to make claims about drugs that aren’t proven, and we can’t just let loose a flood of bad drugs onto the market. It’s a terrible condundrum. How much is too much and how much is not enough regulation in these kinds of cases.
In the case of Tsyabri, it was the manufacturer, Biogen, who would not allow the prescription of the drug initially. From what I’ve read, Mr. Baron now has it.
I was in a phase I trial once and was also in an EAP study so I could get a drug before it was approved.
“I still have to do some reading on this drug. The side effects are potentially serious, but, when you have only weeks left to live, I don’t suppose that matters.”
Isn’t this the same for everyone who enters the company’s Phase I trial? Why should anyone have to go into a drug trial and risk not getting the drug? Why should we even have the FDA approve drugs?
Oh! Yes, MM is an orphan disease.
Word has it that he did get the drug. We hope to hear good news.
This is such a frustrating story. One can understand the legalese at the pharms end, but the need at the patient’s end ought to outweigh legalisms. Could he not sign some sort of waiver releaving the company of responsibility?
Does MM count as one of the group of illnesses that have orphan drugs?