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need this kind of testing done? I am wondering, do you set up the
types of regulations that actually inhibit research?
Dr. CROUT. That is a very difficult question because there clearly
is a balance between wanting to get a lot of information about a
drug on the one hand and on the other seeing costs escalate.
What you are describing is not really a matter of regulation.
That is a matter of negotiation of an individual protocol with the
firm.
We do have a high degree of flexibility in determining what
particular toxicological tests need to be done, how long the trial
runs, how it is designed, how many patients, and that sort of thing.
Those are the things that we will try and negotiate with the firm
in the most efficient and least costly way possible for any new
trials that need to be done on DMSO.
MS. OAKAR. But you have a lot of flexibility in determining that.
Dr. CROUT. Yes.
Ms. OAKAR. Well, I think if you adhere to what the chairman is
saying-and that is, if you take a certain degree of initiative in
assisting to get the issues settled once and for all, then you would
not put uncompromising types of regulations in the way of those
who are doing the research, would you?
Dr. CROUT. No, ma'am.
MS. OAKAR. OK. Thank you, Mr. Chairman.
The CHAIRMAN. Well, Dr. Crout, Dr. Gyarfas, and Dr. Finkel, we
thank you all very much for coming here today. We are going to
watch this matter with the keenest interest and hope that the
public interest will be served in what shall be done.
We will call Dr. Scherbel back for a moment.
Dr. SCHERBEL. I appreciate this. I am leaving in a few minutes. I
would like to make one or two comments to what Dr. Crout has
said.
He indeed has a very difficult position, and for him to make
some of these decisions, I just don't envy him one bit. I am sure he
is doing the very best he can. This is not criticism toward him.
The study on interstitial cystitis, that was carried out and ap-
proved, was done on a very simple protocol, with historical con-
trols. In other words, the patient was ill, the patient didn't improve
spontaneously, treatment other than DMSO was ineffective. The
patients then received DMSO and they improved. That was the
study. Historical controls in a very simple fashion.
Investigators studying the effect of DMSO in patients with inter-
stitial cystitis had an entirely different advisory committee than
we had. Yet, it was the same drug. But the criteria, the restrictive
elements that entered our study, did not occur in that study.
We had a group of FDA monitors throughout our study until the
time came to present our data. Then we had a second group of
FDA officials who were highly critical of the study design as well
as our conclusions regarding treatment.
The last point I would like to make is that the FDA consulting
committee had only one expert on scleroderma. He had never used
this drug.
The study and treatment of connective tissue disease is a subspe-
cialty of medicine. When I talked about sclerodermatous ulcers,
there is not a chapter in a textbook on sclerodermatous ulcers.
