Speeches & Testimony

2005 Wiley Lecture
Delivered by Abbey Meyers on April 6, 2005, at the annual meeting of the FDA Alumni Association in Washington, DC

I am truly honored to be here today, not only because of the Wiley Award, but because it is so unusual for a professional society to honor a consumer. For me it is an honor simply to be in the same room with you who have dedicated so many years of your professional lives to public service.

I understand that next year marks the 100th anniversary of the Pure Food and Drugs Act of 1906. Subsequently, the FDA became the premier consumer protection agency for the entire world. Recently, however, FDA has found itself in the midst of great controversy and turmoil. Dedicated public servants at the agency are under fire, and morale is wilting under unremitting attacks by investigative reporters and congressmen, who were the very ones responsible for starving the agency of adequate resources to do its job. Yet the FDA, and not Congress, finds itself in a major credibility crisis.

Let me tell you about my first contact with the FDA. In the late 1970s, I was a simple housewife from Connecticut with children who have a rare genetic disorder. My oldest son was severely impacted, and we tried many medications to no avail. Finally he was put on an investigational drug, and it worked. But a few months later the manufacturer decided to stop development of the compound. I did not know at the time that the decision was based solely on economic - not medical - reasoning. The drug was being developed for a prevalent disease and it was not effective for that condition. The manufacturer didn't care that it worked for my son's disease because the market was too small to be sufficiently profitable. In other words, it was an "orphan drug."

Since I had no answers as to why we couldn't get the drug, I phoned the FDA. Eventually I spoke to a woman in the neuropharmacology division and asked why development of the drug was being stopped. In particular, I wanted to know if my son was in danger; for example, did FDA find out that it caused a serious side effect such as cancer and therefore ordered the sponsor to discontinue the clinical trials?

The woman on the phone said, "I can't talk to you until I speak to a Freedom of Information Officer," and she promised to call me back. A few hours later she did call me back. She said, "I spoke to the Freedom of Information Officer, and he said I cannot talk to you."

Needless to say, I hung up the phone in disbelief. That phone conversation, however, was the very beginning of a battle that culminated in passage of the Orphan Drug Act of 1983. I spoke with numerous rare disease support groups who felt the orphan drug dilemma needed to be solved, and that coalition evolved into the National Organization for Rare Disorders (NORD) dedicated to the identification, treatment, and cure of rare diseases through programs of education, advocacy, research, and services forpatients and families.

Ultimately the American orphan drug program became the model for an international effort to alleviate rare diseases. The U.S. program was initiated because of the research, documentation, and concepts of Dr. Marion Finkel, an FDA alumnus, and its unbelievable success can be credited almost exclusively to Dr. Marlene Haffner, a very dedicated and committed public servant.

When morale at the agency flags, any FDA staff person can point to the FDA's orphan drug program with great pride. The Office for Orphan Products Development can share its accomplishments with the rest of the agency, as it continues to mentor regulatory agencies in the rest of the world. This is one area of FDA that has no me-too drugs, no life-style drugs, and no inconsequential drugs. You can all be very proud of the millions of lives that have been saved and improved by orphan drugs and humanitarian medical devices.

Let me tell you, from a consumer's perspective, the major changes at the agency in the past 25 years that I believe have led up to the current controversies.

The first FDA commissioner I dealt with was Donald Kennedy, who is currently the Editor-In-Chief of Science. He was a young energetic man who was committed to monumental changes at the agency. He didn't get most of what he wanted, and I clearly remember that he wanted something we still desperately need today: mandatory understandable patient information leaflets with all prescription drugs. Needless to say, if patients could read the warnings, side effects and contraindications every time they received a prescription, a lot of adverse events could be avoided, and many lives would be saved.

When Dr. Wiley launched the agency in 1906, through the FD&C Act in 1938, to the 1960s when Dr. Francis Kelsey prevented Thalidomide from being marketed in the United States, medicine was a paternalistic endeavor. Patients never questioned what doctors told them. Today, however, we are an Internet society and patients often question their diagnosis and treatments. They go home and do their own research; they get second and third opinions; and they demand access to investigational treatments when nothing else works for a serious or life-threatening disease.

Today, FDA must serve two very distinct constituencies. One is the general public, which is largely healthy and requires medicines for temporary and benign conditions such as the common cold or headaches. These people do not want to be exposed to risks, and they often expect the FDA to ensure that drugs are absolutely safe and reasonably effective.

The other segment of FDA's constituency is people with serious or chronic diseases who want new treatments as quickly as possible. These people are often willing to bear substantial risks in exchange for possible efficacy - for example, cancer patients who are willing to take toxic chemotherapy drugs with serious side effects in exchange for hope of recovery.

These two groups bring tremendous political pressure on the agency. FDA is pressured to approve new drugs quickly for very sick people, but it is also compelled to review drugs with more careful deliberation to avoid serious risks for healthy people.

This conflict emerged with the AIDS epidemic in the 1980s, and Commissioner Frank Young was faced with these disparate points of view. He came up with a concept that was applauded by the patient community: Drugs for serious and life-threatening diseases could be fast-tracked so they could be reviewed in six months. Other drugs would be given a standard review of one year.

Then in the 1990s came the Prescription Drug User Fee Act (PDUFA) and its amendments, which changed everything. The pharmaceutical industry pressed for changes that would speed approvals for all new drugs. Suddenly the FDA's performance was being measured on speed, not on scientific excellence. Congress, which never in my experience had adequately financed the FDA, found a good way to avoid its fiscal responsibility on a permanent basis. The pharmaceutical industry, which lobbied hard for PDUFA, was not careful about what it wished for.

Since PDUFA, and particularly the 1997 amendments, FDA has been fast-tracking most New Molecular Entities (NMEs). When Vioxx, Celebrex, and even Viagra were reviewed in six months, the writing was on the wall. Each catastrophic pharmaceutical event has eroded the public's trust in the FDA: The Cox-2 inhibitors, antidepressants for children, Estrogen, Fen-Phen, Baycol, Rezulin, etc. The news has gone from bad to worse.

Shockingly, at a public meeting when I heard an FDA official refer to the pharmaceutical industry as "our customers," it seemed obvious that disaster was waiting in the wings.

The absence of a permanent FDA Commissioner during the past four and one-half years, for all but 18 months, is only one problem plaguing the agency. The main problem in my opinion is that the agency is not funded by a congressional health committee, but rather an agriculture committee. This is because 100 years ago the main health problems were caused by adulterated foods. So today FDA's appropriations compete against diseases of peach trees, catfish farms, and the tobacco subsidy, to name a few.

Many consumers feel the growing reliance on user fees makes a mockery of FDA's mission of protecting and improving the public's health. The agency can only use user fee revenues for new drug reviews. Because Congress has abdicated its responsibility to adequately fund the agency, FDA has very scant resources for post-marketing surveillance, or even to monitor pharmaceutical advertising. If companies blatantly violate FDA's policies, the agency has inadequate enforcement authority. This is why so few Phase IV studies are done, and why the agency "negotiated" for more than one year to change the labeling of Vioxx. How many people needlessly died during the "negotiations"?

Direct-to-consumer advertising is another ridiculous change that I personally find very hard to tolerate. The pharmaceutical industry says that the ads are "educational." I have to agree. When my 10-year old granddaughter asked me what a four-hour erection is, it was very educational.

Companies that develop direct-to-consumer ads can broadcast them before FDA reviews them. If the agency eventually finds that the ads are inaccurate or misleading, FDA can order withdrawal of the ads. But by that time millions of Americans have seen the ads and been influenced by them before they are pulled off the air.

Congressional hearings have shown that the agency's post-marketing surveillance system is seriously flawed. Again, this is an example of an unfunded mandate. Congress expects FDA to monitor adverse events to detect patterns of side effects, but Congress has not appropriated sufficient funding for the agency to do so, and has prohibited use of user fees for this purpose.

Thinking back to my first conversation with an FDA employee, it reinforces my conviction that the agency's lack of transparency is still one of its leading problems. I believe the FDA is here for us - the consumers. We cannot be expected to write Freedom of Information letters every time we ask a question that may affect a family member. FDA's secrecy may be appropriate for Homeland Security or the CIA, but not for an agency that regulates goods that amount to 25 cents out of every dollar the American consumer spends each year. Because of the agency's insistence that even benign information is a "trade secret," consumers have become increasingly suspect that important facts that could affect their health are being purposely hidden. Paranoia will continue to grow until this policy is changed.

Additionally, because of this secrecy policy, FDA has never been able to defend itself appropriately when it comes under fire. The public's perception that the agency is beholden to the industries it regulates continues to grow. The agency's decision makers should be reminded that they are not running the Defense Department with national security concerns. FDA must be free to answer consumers' questions readily and factually.

I continue to believe that public health catastrophes would be less likely to occur if the agency were substantially strengthened and had the full support of key congressional committees. The FDA needs greater enforcement capabilities, and a substantial increase in funding to allow it to respond to public health controversies. Its performance should be measured not on speed, but scientific evidence and excellence. Fast-track reviews should be used only for treatments addressing serious and life-threatening diseases. Since no one ever died from osteoarthritis or erectile dysfunction, there has never been a medical reason to speed reviews of other types of drugs.

If Congress gives the FDA the tools, the agency will secure the public's trust. For all the talk about less government and smaller government, the FDA is one area of government that the public wants more of, not less. People want assurances that the food on their table will not make them sick. They want to be certain that medical devices will not break when they are used. They want to be confident that the medicines they take will enhance, not destroy, their health.

It is up to Congress to reinforce America's trust in the FDA, so it can guard our nation from health catastrophes that are much more of an immediate real threat than bioweapons. Even more importantly, Congress has to learn what every housewife knows, "You get what you pay for." Eventually Congress will recognize that taxpayers are willing to foot the bill for a strengthened FDA simply because we as a society cannot afford not to.

In this sense, I have given you a snapshot of the past 25 years the way I see it. The pendulum has swung from a restrictive unbending agency consumed with absolute safety to the AIDS epidemic when sensible changes were made for the sake of desperately ill people with serious and life-threatening diseases, to now when tens of thousands of deaths are being blamed on arthritis pain medicines because the pendulum has swung too far in the other direction. As a consumer advocate, I want the pendulum to swing back to the middle. I want the curtains of secrecy to open wide so that FDA can answer questions truthfully, and defend itself when attacked by irate public relations firms who tell half truths in their effort to protect their clients.

To us, the Orphan Drug Program works so well only because we recognized that we cannot manufacture orphan drugs ourselves. We need the industry. There are many wonderful and dedicated people in the industry who want to do the right thing. They should not suffer because of the outlaws who abuse the system.

This is why we need a strengthened FDA as the public's policeman for drugs, biologics, medical devices, veterinary medicines, foods and cosmetics. If Congress does nothing, medicines may soon be sold like nutritional supplements, making any claim for any health condition, with tablets that don't even contain the labeled ingredients. The fact is, the agency will not be able to return to its former glory - the point at which the pendulum hung dead center - unless Congress gives it the resources and authority it needs to do its job. In this sense, I believe all of the catastrophic health events of the past few years have finally made the public and its elected representatives realize how important the FDA's mission really is.

Thank you.