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Our staff and advisory committee felt, to the contrary, that
improvement of the untreated hand raised the strong possibility
that the general improvement trend in the whole trial was attrib-
utable to a nonspecific effect of DMSO. Everyone agreed that the
trial showed that DMSO may be effective, but few felt that the
trial proved the point.
Because the statutory standard for approval of a drug is substan-
tial evidence of effectiveness as shown by well-controlled trials, not
simply the possibility of effectiveness, we are unable to approve
DMSO for this indication at this time.
I think this illustrates a fundamental confrontation, if you will,
between the quality of the scientific evidence available and the
statutory standard for approval. Again, from our perspective, the
only thing holding up an approval of DMSO for any of its indica-
tions is the availability of well-controlled trials that meet statutory
standards.
It is the science that is deficient as far as DMSO is concerned.
Because of the possibility that DMSO may be useful in treating
the hands of patients with scleroderma, we believe a properly
controlled study should be conducted to demonstrate whether the
drug is effective or not.
We have, therefore, contacted representatives of the Cooperative
Systemic Studies for Rheumatic Diseases Group, which is support-
ed by a contract from the National Institutes of Health. This is an
academic group of rheumatologists, supported under a contract
from NIH.
It is our understanding that a new trial of DMSO for sclero-
derma is being organized by the group and will be conducted in the
future. This trial will be controlled and blinded in such a way as to
obviate the problems of past trials.
We look forward to receiving the results of this new trial, but I
must acknowledge in all honesty that a definitive scientific answer
is probably at least 2 years away.
Let me turn now to the IND's; that is, the investigational appli-
cations that are on file with us. These are the applications for new
research.
At present the FDA has on file 16 active investigational new
drug applications for DMSO. Conditions under study include sclero-
derma, joint injuries and spinal cord injuries. There are no active
IND's at the present time for the study of DMSO in rheumatoid
arthritis or osteoarthritis.
There is no regulatory barrier to the evaluation of DMSO for
these or any other indication. Our sole interest is in seeing that
research on DMSO is well controlled scientifically so that meaning-
ful data result and that studies are conducted safely under the
usual ethical and scientific standards that apply to all human
research.
In summary, DMSO is a solvent that crosses body membranes
with ease and appears to have analgesic effects when applied local-
ly. There is much testimonial evidence to suggest that DMSO re-
lieves pain after local application to injured or inflamed tissues.
This is an effect similar to that we usually associate with lini-
ments. Properly controlled studies to prove this point are not avail-
able but are technically possible to perform.
