Food and Drug Law Institute
April 1, 2003

From Rep. Henry Waxman

Good morning. I'm pleased to be here. World events right now so overshadow other issues that it is tempting to focus on nothing else. But while terrorism and Saddam Hussein may pose serious threats to the health and safety of the American people, there are others that cannot be ignored. The epidemic of Severe Acute Respiratory Syndrome spreads with each passing day. And this year, preventable or treatable illness will needlessly kill hundreds of thousands of Americans.

In the coming months and years, the FDA will be front and center in dealing with the most pressing health challenges facing the United States. Today, I would like to make the case that success depends upon how well the agency performs its key role - supporting medical progress and public health through science-based regulation. I will argue that failure lurks where politics and ideology are allowed to skew the agency's mission.

In the field of military power, none of us would question that the Defense Department should select military equipment based on the best available science. There is no room for asking which missile manufacturer has the right religious views, or for even suggesting that troops in harm's way rely on equipment that has not been rigorously tested.

Yet, in the field of public health, and increasingly in the field of food and drug regulation, some are quite willing to look to turn away from science and towards politics, religion and ideology for answers. They are willing to expose the public to health-related products on the basis of preliminary data and unproven theories. Unfortunately, some of those people are decision-makers in this Administration.

Across cabinet-level Departments, expert appointments to scientific advisory boards and peer review panels are going to individuals with specific ideological viewpoints rather than scientific credentials. Scientific information that does not serve the Administration's political agenda has been suppressed or altered. Science-based regulation of significant health and environmental risks, from arsenic levels to industrial pollution, has been rolled back.

Within HHS, for example this Administration has altered CDC web sites on contraception and AIDS prevention, erasing key facts about condom use that could save lives. At the National Cancer Institute, officials changed a web page to read that abortion might lead to breast cancer, despite overwhelming evidence to the contrary. After Congressional pressure and a meeting of 100 experts, the NCI now acknowledges that experts have concluded there is no link between breast cancer and abortion, yet the misleading webpage remains up.

On lead poisoning, this Administration replaced a leading academic expert on a key advisory committee with a paid lead industry consultant who believes a lead level of 70 does not produce long-term effects on learning and behavior. This is a position that was universally rejected by the scientific community -- thirty years ago.

The FDA has been anything but immune from the Administration's willingness to sacrifice science for political ends.
 

As we approach the 100th anniversary of the agency, we must remember that the history of food and drug regulation has shown us over and over again that our nation's food and drug industries thrive only when there is a strong FDA protecting the public from unsafe and ineffective products. When consumers are exposed to scams, unjustified claims, or needlessly dangerous products, consumer confidence evaporates. Over the long term, patients and industry either suffer or advance together.

This idea is so obvious it is remarkable how often it appears to be ignored at today's FDA. I not going to sugar coat my message: Over the last two years, the FDA has embarked on a path that poses grave dangers not only to American consumers but ultimately to the companies that manufacture them and to scientific progress itself.

In December, this Administration appointed to the FDA advisory committee that oversees reproductive health drugs a physician whose qualifications for the position appear to have more to do with his opposition to abortion and his religious views than to his academic credentials. This is a doctor who apparently counseled women to read scripture as a treatment for PMS and who refused to prescribe contraceptives for unmarried women.
Some may think that one spot on one advisory committee won't make much of a difference. But I would argue that this appointment is a very ominous sign for an agency that is dedicated to science.

Once we accept the idea that ideological views can substitute for objective, scientific analysis, the review of health-related products has the potential to become a political process rather than a scientific one. I don't believe that any of us want that to happen.

It is a small step from appointing people based on ideology, to manipulating scientific information to fit an ideological preference. In January, an FDA press release claimed to describe the results of a scientific survey of physicians on direct-to-consumer advertising. To read the press release, one might have concluded that physicians thought DTC advertising was the greatest advance in patient care since padded examining tables.

In fact, it is quite clear that FDA significantly slanted the results of the survey in order to make a case for greater DTC advertising.

For example, the FDA Talk Paper states:

"According to the survey, one effect of DTC ads was to help educate patients about their health problems..."

In fact, the survey data showed that only ten percent of the physicians surveyed felt the patient was better educated or informed due to DTC advertising. By comparison, 75% of the physicians said the DTC advertisements caused patients to think a drug worked better than it did. And 60% of their patients understood only a little or had no understanding of possible risks and negative effects of an advertised drug.

Ignoring or minimizing these and other inconvenient facts revealed in the survey, the talk paper concluded,

"These new results confirm FDA's current understanding . . . Ads can and do help increase patient awareness about the availability of effective treatments for their health problems."

This conclusion was an ideological one, not a scientific one.
A fair reading of the survey results is that physicians had both positive and negative views about DTC ads, in about equal measure. But FDA did not provide a fair reading of the survey. Instead, it published something very close to a puff piece for DTC advertising.

Again, you may question whether distorting the results of a survey on DTC advertising really matters. It does. It shows a disdain for scientific truth that can infect far more important decisions.

Public health agencies like the FDA run tremendous risks when they play fast and loose with scientific data. They run those risks with their own credibility, with the credibility of the products they regulate, and ultimately with the lives of the American people.

Yes, there are lives very much at risk when ideology trumps science at FDA. I encourage everyone here to read Philip Hilts book on the history of the FDA entitled Protecting America's Health. This book tells the story of a steady stream of tragedies that shaped the agency and the people who have run it.

Today, I am very concerned that an agency that does not understand this history will be condemned to repeat it. I am referring to this Administration's approach to regulating advertising claims, arguably the core function of FDA for the past century.

The Federal Food, Drug, and Cosmetic Act doesn't permit companies to advertise unapproved health claims for foods or drugs. The Act presumes that promoting unapproved health claims is inherently misleading. Because preliminary evidence is often misleading. And because neither patients nor physicians are in a position to evaluate the quality of the data supporting unapproved claims.

This Administration has signaled an interest in loosening the rules on promotion of foods and drugs, particularly on advertising of unapproved uses of approved drugs. In fact it has already substantially lowered the level of evidence needed to make a health claim about a food.

The ideological principle behind lifting the restrictions on advertising of foods and drugs is that commercial speech should not be restricted any more than political speech.
Underlying that principle is the view that more information is better than less, and that even if the information is based on preliminary data or unproven theories, no one is harmed as long as the information is not false or misleading. In fact, according to the FDA, waiting for proof of safety and effectiveness could itself be harmful by depriving people of useful information that could enhance their health.

(I will let someone else comment on the irony that this is the same Administration that is energetically suppressing scientific evidence on the effectiveness of condoms.)

According to the FDA, lifting advertising restrictions would be a step forward. But what the FDA is really contemplating is turning back the clock several decades. And history has already fully documented the harm to American lives and health when you permit companies to advertise health claims without demonstrating their effectiveness.

Before 1962, when drug companies were first required to demonstrate the effectiveness of their products before approval, they were subject to a regime much like that apparently favored by the Administration. Industry could make unapproved effectiveness claims about their products, as long as the claims were truthful and not misleading.

Congress held several years of hearings on the drug industry in the late 1950s and early 1960s. The hearings detailed a marketplace rife with drugs that were promoted on the strength of preliminary evidence or nice theories, but that were ultimately shown to be ineffective and sometimes dangerous for their claimed uses. The hearings revealed that:

" Drugs were being promoted for indications far beyond any responsible evidence of their effectiveness and even for indications for which they were known to be ineffective. This was borne out by a review of marketed drugs in 1962, which revealed that one-third of all drugs on the market were ineffective for any use, and that a much higher percentage of secondary indications lacked evidence of effectiveness;

The hearings also found that:

" In the absence of an effectiveness requirement, manufacturers rarely carried out adequate effectiveness tests;

And that:
" Truthful information was impossible to separate from misleading information because promotional material distorted the available evidence in ways that made harried physicians believe they had adequate information to make prescribing decisions; and

The record also showed that:

" The use of ineffective drugs subjected patients to severe harm. Patients suffered serious side effects without any corresponding benefit. And the use of ineffective drugs delayed or prevented them from getting effective treatment for serious and life-threatening conditions.

Let me give you an example of the harm caused by the marketing of a drug based on an unproven theory rather than on dispositive scientific studies. The drug DES, a synthetic estrogen, was promoted to millions of healthy women for preventing miscarriage in the 1950s and 60s, on the strength of the theory that miscarriage was caused by inadequate estrogen production.

Twenty years later, it was learned that DES was responsible for thousands of cases of unusual cancers and serious reproductive abnormalities in the children of women given DES. Perhaps the greatest tragedy of DES is that none of the harm to those young people was necessary: scientific studies ultimately disproved the theory on which DES was marketed and showed that DES was ineffective for preventing miscarriages.

But, you say, the FDA could stop claims that were false or misleading. In reality, the hearings showed that enforcement actions against misleading claims were virtually futile because the FDA couldn't begin to police them all. When the FDA or FTC did bring an action, it took months and more often years, while the products continued to be promoted and to cause harm.

Let me quote then Secretary of Health, Education, and Welfare, Abraham Ribicoff, on the adequacy of the regulatory regime he oversaw, where the burden was on the FDA to prove that promotional claims were false or misleading:

"If claims for effectiveness are made which the FDA believes are groundless, a proceeding must then be brought to take the drug off the market as a misbranded product. . . . And throughout the period of time it takes for the FDA to prepare its case and secure relief in the courts, the manufacturer will have foisted his product upon an unsuspecting public.

" . . . . where public health is involved it is intolerable to permit the marketing of worthless products under the rules of a cat-and-mouse game where a manufacturer can fool the public until the Food and Drug Administration finally catches up with him."

Forty years after DES, why would we possibly want to return to this cat and mouse game? Two recent cases illustrate the folly of even considering this idea.
Hormone replacement therapy, or HRT, was approved for treatment of the symptoms of menopause and prevention of osteoporosis. But for years HRT was widely believed, on the basis of observational studies, to protect women against heart disease. The first prospective, well-controlled trial, however, was stopped when it revealed that HRT not only failed to protect women against heart disease, it increased their risk of heart attack and stroke. Most of the medical community was stunned.

In January, a large safety study of the asthma drug Serevent was halted when it was discovered that use of the drug was associated with an increased risk of life-threatening asthma episodes and deaths, especially in African-Americans. Serevent is approved for use only when taken with other asthma medications. The increased risk of death was particularly evident in patients taking the drug by itself, an unapproved use.

What makes these cases particularly striking is that the manufacturers of both HRT and Serevent had previously been stopped by FDA from promoting their products for the unapproved uses in question.

Do I think that either of these companies was deliberately promoting its product for a dangerous use? No. In all likelihood, the companies were suffering from the same problem that faces anyone trying to understand the uses of a drug based on preliminary evidence or appealing theories: they were misled because they did not have access to adequate scientific data.

Let's ask what would have happened in a legal system where the burden was on FDA to prove that claims for these unapproved uses of HRT and Serverent were false or misleading. Before the definitive studies were done, could FDA have proven that the companies' claims were false or misleading? No again.

In other words, until adequate scientific data were available, the company couldn't know whether its product worked as advertised, and FDA couldn't have proved that it didn't. So under a system where FDA must prove a claim to be false or misleading, these drugs could have been widely promoted for their unapproved uses. We can count ourselves lucky that we don't live under such a system.

Permitting companies to promote unapproved uses on the strength of preliminary evidence or unproven theories may satisfy an ideological preference for unrestricted commercial speech. It does not make good public health sense. And it is not consistent with the role Congress has assigned FDA. HRT, Serverent, and DES all show that we can't know whether it is wise to broadly promote a product until we have adequate scientific data to judge its safety and its effectiveness.

At the beginning of my comments, I drew an analogy between our military and the public health.

I think we can all agree that it would be terrible if we selected the technologies and weapons relied upon by our troops based on preliminary data suggesting the weapons might work, or worse, on the ideological leanings of their manufacturers. Why do we insist that military technology be proven to work? Because the lives of American soldiers depend upon their equipment working, and the lives of the American people depend upon our military effectively protecting them.

In the field of public health, however, the Administration has begun to take a less scientific approach. I believe that this approach is entirely unacceptable, for the same reason that it would be unacceptable in the military. Because human lives depend upon our public health system as well.

I don't intend to stand by idly and let this happen, and neither should you. I'm sure that as Americans who care about their country, all of you want our health policy to be as effective as possible.

I will be critical of FDA's actions where they appear to undermine science and the public health, as I have been today. But I also will stand up for products manufactured by the industries represented here today in the face of unfair criticism. For example, I have consistently fought to make sure discussion of the safety of vaccines is informed by science, not speculation about, for example, their possible relationship to autism. I am committed to preventing these vital weapons against disease from being undermined by unsubstantiated and unscientific allegations.

We must all continue to insist that vaccines and other products be judged on scientific evidence, not on unproven theories.

I know that those in the food and drug industry who are here today share this interest in avoiding unscientific attacks. And so I challenge you, just as you ask the public not to judge your products on poor science, ask the FDA to rigorously apply science to the manufacture and marketing of your products.

Do not be seduced by the promise of a bigger market in the short term at the cost of shaking the foundation of consumer confidence in food and drugs. It will be a bad day for the food and drug industry if the public comes to see our health policy as based on political interests rather than science. Because on that day, the public will come to distrust government decisions approving products for sale.

In this crucial arena, the interests of industry and the public overlap. And I hope that you will join me in working for both of them and fighting this trend towards the politicization of health science.