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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 supplements. Like so many new drug treatments, it sounded promising but proved to be worthless or harmful when tested [1,2].
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 of 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% 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 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% 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 . The use of so much experimental treatment may be one important reason we have had such disappointing results. Does the U.S. 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.