When you have an incurable disease, as my wife does, you are
forced to become informed about your options. And sometimes what
your doctor would like to prescribe is simply not available here in
the United States, although it may be in wide use around the
globe.
That was the case with my wife when the drug she was taking to
halt the progress of multiple sclerosis ceased to be effective. Her
doctor wanted her to take the new drug, Rebif, that was in wide use
in Europe and Canada, but was then unavailable here in the U.S.
Because time was critical in stopping the advance of MS before
irreversible damage was done, we took action. After consulting with
Kathi’s doctor and our lawyer, we imported the drug from Canada
until it was eventually approved in March of 2003. We both believe
the progress of the disease would have been drastic had she not had
access to Rebif during this time.
The problem my wife and others had in gaining access to Rebif
highlights the problem faced today by sufferers of many incurable
diseases. Fortunately, drug companies have poured hundreds of
millions of dollars into research to find new drugs which promise
to halt or delay the progress of diseases such as MS, AIDS,
arthritis, Alzheimer’s, and the like. But unfortunately, the U.S.
lags behind the rest of the modern world in making many of these
drugs available to those suffering from these diseases.
Not only did my wife face this problem with the treatment of her
disease, but more recently, my mother-in-law faced similar
difficulties when the drug she was taking for arthritis was pulled
from the market.
The fact is that those who suffer from incurable diseases should
have the right to decide on the basis of their own individual
situation if the risk of taking a new drug (that has successfully
completed clinical trials and is in wide use elsewhere) should be
taken. Someone who has MS or any other incurable disease must have
the right to conduct their own risk analysis because it is his life
that is on the line. While politicians squabble and the media fret,
time is running out for those who have MS, AIDS, Alzheimer’s and
similar diseases.
Unfortunately, a patient at the mercy of a disease often suffers
additionally from decisions made at the Food and Drug
Administration (FDA).
Falling victim to bad press after the removal of COX-2
inhibitors from the market when serious side effects were reported,
the FDA has grown a thin skin with regard to any press reports of
complications in the use of newer drugs. What the FDA seems to
ignore in its response to these public reports is the due
diligence, awareness of risk and subsequent informed choices made
by patients with no other hope of recovery.
Who ends up safe, and who ends up sorry?
The FDA often seems more concerned with weathering political
pressure and scrutiny from a headline-obsessed media than
practicing kind and prudent regulation. While the agency must
remove products proved unsafe in wide use, the real tragedy is what
happens when FDA reacts too quickly, and beneficial treatments end
up being removed from the market without solid cause.
Such was the case with Lotronex, a treatment for Irritable Bowel
Syndrome, and may also be the case with Tysabri, the only new MS
treatment available in the last eight years. Manufacturers of both
voluntarily pulled their product from the market in response to FDA
concerns. Lotronex ultimately was allowed back on the market,
albeit with additional restrictions. Tysabri currently is
undergoing a safety review, and recently published data that
confirm that the drug works hopefully will help FDA put it back on
the market too.
MS sufferers, including my wife, have fallen victim to the
culture of “analysis paralysis.” Federal regulators, fearing legal
action and bad press, have increasingly taken action absent
sufficient cause, which limits patient access to useful and needed
drugs. It forgets that this is a patient population among the most
informed in the world, and in the most desperate need of new
treatment options. The FDA should not confuse activity with
accomplishment. Analysis paralysis has had a profound negative
impact on the entire American health care system.
Although my wife was not taking Tysabri, her current medication
may well become less effective over time. I believe her limited
options should remain as wide as possible.
Prior to yanking widely effective drugs such as Lotronex and
Tysabri off the market, the FDA must consider all factors that lead
to complications, such as mixing medications or dosage deviations
often made by individuals desperate for relief. Genetics also play
a role in individual complications; some people can’t even tolerate
aspirin. Most important, consideration must be given as to whether
removing a drug from the market is worse for the larger cohort of
patients than its actual risk.
Patient access and informed consent are cornerstones in the
treatment of serious and debilitating conditions. When the FDA
removes a drug based on a minimal number of unfortunate but
reasonably anticipated adverse reactions, they remove what may be
the most important choice of a lifetime away from those who truly
should be making it.
Nothing in life is certain — except the results of untreated
disease.
