Government Reform and Oversight Committee
February 4, 1998
Mr. Thomas Moore
Mr. Chairman, members of the committee.
The key issue today is an important one. Should consumers,
especially those with a serious or life threatening illness,
have the right to any drug or alternative medicine even though
it has not been proven safe and effective and approved by
the Food and Drug Administration?
Let me tell a story of what could happen if that were the
case. In this age of media hype, it is plausible that literally
millions of Americans could be persuaded to take a pill every
day that they hoped would prevent cancer-especially if it
included some natural ingredient or a vitamin.
Suppose that long after millions of people were popping this
cancer prevention pill, proper, expensive randomized clinical
trials were finally conducted to see if the hoped for benefits
in fact existed.
Now suppose that those clinical trials-the only real scientific
evidence we have whether drugs work or not-showed that these
anti-cancer pills either didn't work at all-or actually caused
lung cancer. Millions of Americans would be spending their
hard earned money on a remedy that at best was ineffective-and
at worst could give them cancer.
Am I telling you a fanciful, alarmist story? This is a true
story. It already happened-and the treatment involved was
beta-carotene supplement. Like so many new drug treatments,
it sounded promising but proved to be worthless or harmful
when tested.' 2
Humans have dreamed of powerful medicines since the dawn
of history. But for most of the last seven thousand years
consumers were mostly victims of hazardous, poisonous, or
merely unpleasant drugs. The era of modern beneficial drugs
began only a few decades ago when society began to insist
that drugs be tested for safety and efficacy in well controlled
clinical investigations. Real progress began only when we
used randomized clinical trials to separate beneficial drugs
from those that were worthless or harmful.
This morning you have heard some dramatic stories from individuals
who believe they were greatly helped-perhaps saved--by a treatment
that is not available in the United States. The question therefore
is should Americans have access to a medical treatment if
there are individuals who can personally testify that it is
valuable?
The most simple test case would be a remedy for obesity.
Here seems to be a treatment every consumer can judge. Either
you lose weight or you don't. Suppose for our test case that
the FDA had approved the drugs-so they had been subject to
at least modest levels of safety testing. Should not then
the consumer-and not government regulators or health authorities-be
the judge this treatment?
You all ought to know the answer to this question. This episode
also happened, and the result may turn out to be one of the
greatest drug disasters that our nation has experienced. Last
September the diet drugs Pondimin and Redux were hastily withdrawn
after the FDA received evidence that an astonishing 31 percent
of the people tested showed some evidence of damage to their
heart valves. 3 4 At the time, more than 5 million Americans
were taking these drugs.
Did the consumers notice? Could they judge for themselves?
They could not. Until it became very severe, the heart damage
had no symptoms. Did their doctors notice? They did not. Pondimin
was on the market for more than 20 years before two alert
medical workers in North Dakota noticed something suspicious.
What is the first lesson of the diet drug debacle?
Not only does it take systematic testing to discover whether
drugs work, it also takes systematic scientific study to discover
serious adverse effects that are potentially harming millions
of people. If we don't have the proper safety system in place,
people will be harmed for years or decades. In their potential
to harm millions of people there are few rivals for drug treatments-whether
they are mainstream prescription drugs or alternative remedies.
This is exactly why society has erected the safeguards now
being examined in this hearing.
Another aspect of the issue today is more difficult. Should
people with advanced cancer, or Parkinsons Disease or full-blown
AIDS have the right to any treatment they choose? Some of
these people might not live long enough for the kind of drug
testing I believe so important to protecting the public. Should
they not be entitled to take any risks they choose?
On the surface, the case for individual liberty seems compelling.
However, another example will illustrate the dark problems
underlying this seemingly straightforward idea.
Suppose you are dying of cancer, and I offer you this ghoulish
shell game. In one of my hands, I have hidden a treatment
that might save your life. In the other hand, is a quack medicine
that will make you so sick you can hardly get out of bed,
and will hasten your death. I can give you a free choice.
But which hand holds the lifesaving drug? The left hand? Or
the right hand?
This is not a meaningful choice. Without extensive drug testing
we just can't tell which hand holds a dangerous poison, and
which conceals the life saving drug. Without proper testing
even a potentially life saving treatment may be harmful if
given in the wrong dose, or to the wrong patients. I want
people to have choices too. But they should be real choices,
involving scientific data about how much harm and good various
treatment alternatives can be expected to achieve.
We have a only one proven solution. We need public policies
to promote more drug testing, not still more new loopholes
that could endanger the heath and safety of millions of people.
Some may ask, "But aren't people going denied a life-saving
treatment for the several years it takes for human testing
and drug evaluation?" My answer is that we don't know that
it is a life-saving drug until it is tested. Even if proven
life-saving, we can't truly hope to save lives until we have
done enough testing to know how to use it properly. The history
of modern drug treatment includes many cases of valuable drugs
that proved ineffective or harmful because they were used
in the wrong patients, or at the wrong time in the progression
of a disease. 5 6 Until it is tested, and we know how to use
it, a drug cannot properly considered a life-saving treatment.
Alternative medicines pose special problems that deserve
the attention of this committee. They are falling between
the cracks of the system we have devised to search for new
medicines. Large drug companies are expected to invest millions
of dollars in the elaborate drug testing we wisely require.
In return they are granted patents that are so lucrative that
a single blockbuster drug can sustain an entire multinational
pharmaceutical giant. This system has provided many beneficial
medicines, but at a price. Only large firms can afford the
extensive testing required by law. Large organizations tend
to follow conventional thinking; daring innovators often work
alone or in small firms. It is certainly possible there are
neglected therapies that involve common molecules or natural
ingredients that cannot be readily patented. Also there may
be promising scientific avenues of advance that were ignored
or abandoned by mainstream medical research and its partners
in the pharmaceutical industry. The tiny office in the National
Institutes of Health devoted to alternative therapies doesn't
have even a fraction of the resources needed to investigate
the most promising leads.
What is needed is money and a structure to target research
and assign priorities. The funds could be come from general
tax receipts-as do the funds for the National Institutes of
Health. Or the research could be financed by a small tax paid
by industry. I believe that consumers would be willing to
pay an extra amount to insure they got a product that might
benefit their health rather than harming it. The policy problem
is to figure out how to get the necessary scientific testing
done. The solution is not to expose more Americans to untested
and possibly ineffective or harmful compounds.
Finally, I would like to address the issue of the FDA and
experimental cancer treatments. My main concern is that there
is already too much experimental treatment of cancer patients-rather
than not enough.
A survey by the General Accounting Office showed that 23
percent of all cancer patients receive an experimental treatment;
another GAO study estimated that about 56 percent of cancer
patients receive a drug for off-label use-which can be considered
quasi-experimental use of an approved drug.7 8 Despite the
billions we spend on research and treatment, the mortality
rate from cancer is higher today than it was in 1970, despite
dramatic declines in most other major causes of death. 9 The
use of so much experimental treatment may be one important
reason we have had such disappointing results. Does the US
Congress want to expose more patients to experimental cancer
agents without the safeguards required for formal National
Cancer Institute protocols or human drug testing studies under
FDA supervision?
Finally, some people seem to believe that heartless FDA bureaucrats
are somehow keeping valuable drugs away from people in life
or death situations. I have published articles and books filled
with criticism of the FDA, detailing many failings and numerous
ways it could do a better job. But I also am here to testify
that after 20 years in Washington I have not found a group
of more capable public servants more sincerely dedicated to
protecting the American public. By the large, they work at
a thankless task under very difficult circumstances, and I
for one, have great respect for their efforts.
In conclusion, I believe the central issue before the committee
today is not access to treatment, but assuring that the proper
and necessary drug testing is conducted to insure that both
mainstream medical therapies and alternative medicines help
rather than harm people. That is easier said than done. But
with sound public policies, we can move towards this goal.
However, if Congress abandons the essential safeguards of
drug testing, there is no limit to the harm that may occur.
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