Fact #2: This medicine will not save his life
He has a very serious disease and is in late stages. This medicine is approved for other diseases and there is no data to tell him or his doctors that this is going to save his life. IN FACT the company has a clinical trial going on testing the drug for this very disease in patients just like Mr. Baron.
Of course, any terminally ill patient and their family will wan to to try anything. But false hopes and passing blame to a “greedy company” are not going to save his life. In, fact see #3 below for how the company is doing the right thing here already.

Fact #3: Why the company has an ethical issue on their hands
Everyone loves to jump on fraud, corruption, etc. Believe it or not, many of the people employed by Biogen and FDA are actually MDs and PhDs that want to help people live longer and better.
The problem here is that Biogen has been doing the “right thing” and has a Phase I/II trial underway with Tysabri in Myeloma patients (http://www.clin...loma&rank=1). The implication that his doctor ran down to his lab and “discovered” that Tysabri could work here is not really true — this has been being studied for this disease for a few months.
How do we test a medicine and see if it is going to work — in particular if randomizing some patients to receive standard/existing therapies — if the rich and well-connected and do an end-run and just get the experimental medicines they want?

Fact #4: What this will mean to you
You and I (you know, Joe the Plumbers of the world) need Biogen to conduct and finish their trial showing if the drug actually works for Myeloma. We need them to then bring the data to FDA if it works, and get the label expanded to cover the disease. Rest assured, Biogen is very motivated for this to happen as this is how they MAKE THEIR MONEY.
You and I WILL NOT get access to Tysabri if you or a family member develop Myeloma because we are not friends with Kennedy/Clinton/Armstrong/etc. We NEED the research to get done and the approval to happen.

I know I am an ass

PML strips the myelin off your nerve cells, basically removing all their insulation so they can no longer transmit signal. This leads to disorientation, weakness, inability to speak, loss of vision, confusion — basically like Alzheimer’s, only it happens over months instead of years.

Personally, I want to be able to recognize the people I love when I’m on my deathbed.

But on the flip side, there are rules about drug injections for a reason. Things have to be tested in a lab, tested on rats, tested in limited trials. Results have to be gathered, and success measured. Being rich and powerful does not mean you get to have your name at the top of the list to get this drug, when hundreds or thousands of other patients who are not rich are told “no, it’s not approved for general use yet”.

Either you say yes to everyone, and terminally ill patients have the right to whatever treatment they wish no matter if it’s approved or not… or you do things by the rules. And it goes without saying that the drug company, doctors, and hospital should not in any way be liable if this guy dies 20 minutes after taking this drug. You can’t have it both ways.

You do realize that despite the unsubstantiated claims that the FDA has given its approval, allowing Mr Well Connected to receive this medication could jeopardize its availability or the label for the group of patients the drug has been approved (MS and Crohns) and even its potential as a future treatment for multiple myeloma patients don’t you? Any associated AEs that are reported could have a very real impact on this drug in the marketplace or in clinical trials, like the one that is underway in MM.

You should be taking your torches and pitch forks and heading to Gaithersburg, not Cambridge. Given the current regulatory climate, the quickness with which pharm/biotech is painted in broad strokes as pure evil, the tort environment and other factors… there are a number of reasons why you all should not be jumping to conclusions.

There are some things very wrong with drug development and drug regulation in this country and its not all EVIL Pharma. Having lost my father to an aggressive form of brain cancer with limited treatment options I feel the pain of this family but this man, Mr Baron aka Well Connected actually has had a hand in shaping the environment in which a company like Biogen has nothing to gain and everything to lose by providing him their medication. From the media to lawyers to drug company CEO and the FDA, there are many to blame for us being where we are right now.

If you are all outraged by this situation join up with Frank Burroughs and the Abigail Alliance and work towards policy change that may bring about a change in the dynamics of situations like this. Until then you sound like uniformed sheep being lead by a letter that leaves out much in the way of context.

I am friends with at least a half dozen MS patients, one of whom has 3 young children and a rapidly progressing form of the disease. If Tysrabi is withdrawn from the market, some of these people are going to die. (The first withdrawl was almost fatal for my friend.) The company owes it to these people to protect the availability of the drug for them. The good of the many…

To the FDA: I think it’s awful that you’d let the political influence contramand your insistence on TOUCH. You ensured TOUCH would be air tight. You insisted. Why should this guy get in?

And in conclusion: Ain’t Karma a B!tch?

Like most government agencies, the primary objective of the FDA over time has become the protect their jobs and cover their *ss.