It won’t be just Mr. Barons case, should he develop PML, that would bring down this drug.

This is a cancer drug. My understanding is that as long as the facts and risks are known and documented, then this drug could come to market with a BBW.

You cannot look at this case as some other class of drug. It is not a birth control drug for example. Developing PML as a side effect for a birth control drug would be unacceptable. There are other cancer drugs that cause CHF and a whole host of other life threatening problems.

I do think the data will be reviewed. But here is the catch, why are we willing to say that if a few people develop PML and the drug doesn’t come to market that we have done a great dis service to the countless thousand others who must or at least, should access this drug. Why can we think that the majority of these patients will not develop PML? Therefore, if that is the case, why bring this to the market for them at all?

In other words, denying him access on the basis that he MIGHT develop PML and that would be a disaster for all the other patients is not a strong argument.

I don’t assume that, but I do assume that if Mr. Baron gets PML, this drug will be even more tainted with PML than it is now. And hundreds of Mr. Baron’s law partners will sue the heck out of biogen everytime someone develops PML, assuming this drug even makes it to market for MME. In case you haven’t noticed, the FDA is very focused on safety these days …

Tysabri seems to be wonder drug for MS patients and I am hoping that it will become a wonder drug for Multiple Myloma patients as well.

Gooe luck and God bless,

George

I said the government is the best, not the health system being the best.

In fact, I stated,”underfunded health funds”.

By the way, my surmising has just become fact, Mr. Baron will get his drug therapy.

You are ASSUMING that if the patient gets the drug and develops PML, that it will prevent patients getting the drug within the confines of the clinical trial from further treatments.

That is the wrong argument. The FDA has given permission for this to go ahead and have obviously looked at the risks involved. The patient knows the risks, it does not jeopardize the clinical trial. It provides more data, but the experience of one patient will not stop the trial, especially when so many patients may, MAY, benefit from it.

As for the idea that patients in the MM arm can be randomized to not receive this drug, that is the way it goes. However, HOWEVER, in most cases if the drug is showing clinical effect in the arm for the patients getting the drug, with this type of critical disease, patients are allowed to cross over.

The article further states that at this time, there is no data on the use of this drug in Human trials. So, here is a chance to capture some firsthand data.

You can make a good point about this being an “elitist” issue, in my view, that is the only leg you have to stand on.

Yet, I know countless patients who are ordinary citizens and have jumped the cue. What can you say about those people?

So, we all partake in denying Mr. Baron access to this drug, based on baseless arguments, when really it seems to be more because he is a “somebody” and some of us just can’t handle that?

As for the update that Ed has provided, I rest my case, that was exactly what I said it was. The results of Mr. Baron’s experience cannot be lumped in with the rigorous clinical trial, but it does provide more data.

Nathan, your remark about FDA approval becoming meaningless, cracks me up. Are you sure you really meant that? Compassionate use as this is has been going on for a long time, and must continue. For the sake of the patients these companies try to make us believe they provide drugs for and try to help them prolong their lives.

Funny how Biogen figures they can pass up any opportunity to get more data…

The people at Biogen do not have the necessary skills to negotiate this issue, so they are blowing it off. Political posturing on Biogen’s part - they get no sympathy from me.

I have been involved with the biotech industry for many, many years and our democracy is based on a capitalistic structure.

I admit it may be unfair to those who can’t get any drug they want for nothing or because it’s in a trial, and they maybe do not have the close contacts to gain access to the therapies but, that is the unfortunate and only way our country works but for a few government underfunded health funds.

I would like to see everyone treated equally but that isn’t reality. We have created the American Way and so far, it’s a lot better than any other government on this planet.

It is also right to point out that while there are often instances where terminally ill patients receive a drug through compassionate use, it is rarely a drug in phase 1 development.

That makes this elitist by nature, right? Only those rich enough to afford it can have access to the experimental drugs. This completely flies in the face of FPME’s point above when he says “This isn’t about access for the elite either.”

Now to Steve’s non-response. You really didn’t answer any of my question. You can say I’m being cold-hearted. I say I’m being practical. I don’t see any easy answers to the questions I posed.
1) If dying patients could get easy access to experimental treatments, then why should they enroll in the clinical trial? It doesn’t make any sense. I would shake the very foundations of cancer clinical trials.
2) What happens to the data (good OR bad) that comes from these patients that are using the unapproved drugs? Can/should the FDA take that data into consideration?
3) If the company charges huge prices for the drug, then they conceivably could make enough money simply through “compassionate use” and never actually COMPLETE a real, controlled clinical trial to prove efficacy. (this is especially true of small companies, which is where many of these experimental drugs originate)
4) Is the company COMPLELLED to supply the drugs to the dying patients, or is it up to their discretion? Remember that companies typically manufacture only enough drug to complete the clinical trial. Why should they spend millions of $$$ to make more GMP-quality compound? These drugs are cheap to make on a ton-scale for millions of people — but incredibly expensive to make small quantities of.

You can call me cold-hearted if you like — but these issues are very real. I’d love to hear some detailed responses to each of the points I raise. I don’t see any easy way around any of them.