Center for Drug Evaluation and Research

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The issues in distribution, therefore, may or may not be comparable because in the case we are discussing with multiple sclerosis and spinal cord injury, clearly, we are talking about, as I said, 10,000 or on that order of magnitude and, again, from our perspective, we certainly are not equipped as a company nor is Elan and nor would I say are many, even major pharmaceutical companies equipped on their own to run these kinds of studies. These are special circumstances and even in the cases of some of the major pharmaceutical companies who ran studies for HIV drugs, expanded access studies for HIV drugs and so on, they worked with some of the contract research organizations that we are also talking with now.

So, from our perspective, this is very much manageable, using an expert group that is outfitted to do this and has experience in doing it and really our obligation in that case is to work closely with them to exchange the appropriate information, to supply the drug in the appropriate form, which we can do with Elan as our partner and then to collect the data from them and assimilate it and pass it on to the agency under our IND.

MR. CATIZONE: I would speak in favor of not listing this product on the list of substances approved based solely on the safety data presented by the FDA prior to this meeting. I am uncomfortable accepting the data that was presented at this meeting regarding the compounded products by pharmacists because I feel that that data may be biased and unfair.

I am also disturbed by entities, which hold patients hostage and this committee hostage in situations where patients can be at risk and would commit that NADP would urge its members and work with its members to take whatever legal actions it could to ensure that patients' medications and therapies would not be interrupted in situations like this.

DR. GILMAN: I think there is a big contrast between 3,4-DAP and 4-AP, with respect to both efficacy and safety. The studies with respect to efficacy are highly questionable with respect to how good this medication really is, how much function do patients, most patients, really get. They may show some improvement on a scale. That doesn't mean that any but the unusual patient is really that much better with the medication.

And we are hearing about very variable levels of blood levels and dosage levels that provoke seizures. Here, I think, we really need systematic, carefully collected data with good reporting with respect to adverse events, as well as efficacy.

So, this one is much more clearcut from my perspective than DAP. I think this certainly ought not to be on the list.

DR. JUHL: Other comments?

Are we ready for the question?

DR. COHEN: Could I make one more comment in response to something that was said?

You know, the term "holding patients hostage" was used earlier and I certainly don't want to get into an escalation of that particular debate. I do want on the record to say that at the end of the day, we at Acorda and Elan are interested in the welfare of our patients. We have very strong relationships with the community patient groups, who work in this area. You have heard from some of them today. There are others out there. We try to be as responsible and good citizens in this regard as we possibly can in terms of supplying them with information about what we are doing and doing the right thing.

I just wanted to illustrate for the committee or for the panel one illustration of why this is for us not so clearcut in terms of how and when, under what circumstances one supplies patients with an experimental compound. I will tell you and I will go out somewhat on a limb because we have not yet submitted this to the FDA, but I do want to try and share this with the panel, that in our studies, even in chronic spinal cord injury, we were very surprised to find -- these were placebo controlled, double blind study -- we were very surprised to find the extent to which patients apparently experienced remarkable benefits under placebo, never having had the drug at all.

I am talking about people who were injured for five years, who suddenly one week opened their hand for the first time and grabbed a glass of water. I am sure that all of you have the same response to this that we did, which is as soon as we saw it before we broke the blind, we said my goodness, that is fabulous. We have just a remarkable drug effect here.

There was no question that it was placebo. There was no mistake. The blood levels, the plasma levels showed it. It was the same sequence. So, I think when we are talking about how we address our patient population here and taking into account their welfare, I just want to put a plea out that we all remember that these things in clinical medicine are hardly ever cut and dried and one sees remarkable things that one would never imagine you would see under other than the influence of the drug itself, but you do see it.

So, we want to be as careful as possible. Frankly, the idea of investing tens of millions of dollars to try and get a drug approved that we may or may not be able to get approved is not -- it is not the most appealing occupation in the world, but we are doing it because we do believe in the drug. We believe we will be able to show these things, but we also believe we need time. We need to do the right studies. We need to cooperate with the Agency and we need to do it in the right way and that putting this drug out ad lib for anybody in an uncontrolled fashion is not going to be doing anyone any favors and least of all our patients.

So, I just wanted to put that on the record. Thanks.

DR. ALLEN: 4-AP is similar to the 3,4-DAP. It is not the anybody is putting something out there. It has been out there for years, in fact, 10 or 11,000 patients are on it right now. Again, what this does is it alters their method of obtaining the drug, whether or not they can obtain it. That would be one of the things, you know, that I would be concerned about.

DR. JUHL: Seeing no more questions, let's call the question with the same two options. Option No. 1, recommendation to list the drug on the bulk compounding list. Option No. 2, recommend the drug not be listed and ask that the FDA would pursue an expanded access program for the drug that would not inconvenience patients who are already receiving it.

MR. TRISSEL: Dr. Juhl, just one point for clarification.

If we vote for the latter option and the program does not materialize, what recourse do we have at that point because now we voted to put it on this list and the program that we were banking on didn't happen. Is there an alternative to that?

DR. JUHL: I believe that the FDA would pursue something and not just let the patients all go wanting.

DR. BEHRMAN: Again, as I tried to say before, we believe there are two questions here. One, is it appropriate for the list and that question has to be answered. Then if the answer is "no," you have our assurance that we will make every effort that patients who need access -- that doesn't necessarily mean everyone who wants it, but depending on the appropriateness, the safety of the drug, et cetera, we will make every effort to make sure that such a program is in place. Again, the Agency cannot require programs.

I can think of very few companies that have initiated large programs this early in development, but it has been done and has been done successfully.

DR. JUHL: Ready? Okay. Those who favor Option No. 1, please raise your hands. We see two votes. Those favoring Option No. 2, raise your hands. Nine favoring Option No. 2. Okay.

We are six minutes over budget. Again, for those members of the staff who weren't here earlier, I want to thank you for the efforts that you put in preparing us for today's meeting and certainly my thanks to the committee for bearing with us, as well as those guests who made presentations to us.

The committee will adjourn and meet again tomorrow morning at 8:30.

[Whereupon at 5:06 p.m., the meeting was recessed, to reconvene at 8:30 a.m., the following morning, Friday, May 7, 1999.]
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