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.
