77
There are very few articles written about sclerodermatous ulcers.
The FDA officials and members of the consulting committee are
not authorities on treatment of sclerodermatous ulcers.
Now, out of all these patients-and I was able to get these
patients from a seven- or eight-State area-we had just a few who
would fit this very unique criteria of having persistent symmetrical
ulcers on both hands. This is very rare.
For those of you who don't understand scleroderma, patients
might have one ulcer on one finger, one on another which may last
3 months, while another lasts 2 weeks.
When a study group starts putting patients like this together,
they better have 200 patients, and not 19.
The result will be DMSO has been a therapeutic failure, it does
nothing for these ulcers, and therefore we cannot approve it.
Thank you.
The CHAIRMAN. Doctor, do you make the point that on this panel
that makes the original recommendation there are not enough
people who know something about arthritis and those kinds of
diseases?
Dr. SCHERBEL. No; I didn't mean to state that. That is not what I
meant. I was trying to say this is a very difficult problem because
the drug is unique. There is no drug that works like this.
When we submitted our material, this was again unique because
nobody has talked about a group of sclerodermous patients with
bad ulcers.
Now, in the group I was referring to, when I submitted my
report, I presented patients with ulcers that were persistently pres-
ent. Ulcers were not appearing and disappearing. I presented pa-
tients with ulcers that were constantly present for 1 year or longer.
An investigator does not need a large number of patients to
determine effectiveness if these ulcers begin to heal. At the end of
the study these patients were not having a serious problem with
ulcers.
In my mind treatment with DMSO was very effective. It was
very surprising to me when we went to FDA and were told DMSO
effectiveness was questionable. I have talked to very competent
clinical pharmacologists in this country who tell me that there are
many ways of determining if a drug is effective. One does not have
to have the type of study that FDA insists upon we have with this
drug to prove efficacy.
There are many ways of providing an effectiveness with a drug.
But if one insists on a double blind study this drug will never
become available. The investigator knows who is getting the drug,
and who is not.
The dose of the drug varies so greatly. It is difficult to determine
beforehand who is going to tolerate 30 percent concentration, there
are many of my patients I have increased from 30 percent to 50
percent to 70 percent rapidly, and others very slowly.
You have to individualize these patients. So, if one says, well, the
drug doesn't work because 30 percent didn't work, I take great
issue with this. The authorites who are evaluating DMSO are not
the investigators who have used this drug for 10 or 15 years to
learn these little idiosyncracies.
63-078 0 80-6
