Remarks by:
Jane E. Henney, M.D.>
Commissioner of Food and Drugs
U.S. Food and Drug Administration
for
University of Kansas Medical Center
March 27, 2000
Good morning. I am delighted to be back for a visit to this institution where I spent many memorable and enjoyable years, and to see many of my old friends and colleagues again. For those of you who are wondering about the title of my talk...allow me to explain. Looking back, I have observed that being on the faculty at the University of Kansas Medical Center was a little like being in the proverbial frying pan. Little did I know that I would eventually land in the fire, as a regulator at the Food and Drug Administration. But that's just what happened, and I must say that leading this organization of individuals who are so committed to preserving and protecting the health of the American people has been a challenge well worth accepting.
I would like to focus this morning on the importance of having a strong scientific capacity at the FDA in order to handle each and every issue we face. I'll be covering just a few of those this morning.
As you well know, cutting edge science and technology are providing us with a never-ending supply of new challenges and new opportunities. Over the past few decades, and increasingly in just the last few years, we have seen major investments by both the public and private sectors in this country in biomedical research. These investments are paying off in an abundance of new products that must be evaluated by FDA before they are introduced into the marketplace.
Assessing the safety and efficacy of these increasingly complex products requires a talented and energetic scientific staff--and the necessary resources--committed to meeting these and other scientific challenges of today. Our decisions must be firmly grounded in science, and our actions should continue to meet our very high standards, be timely and predictable, and open and transparent to the public. It is important for scientists, health professionals, patients and industry to understand our decision-making processes and have the opportunity to provide comment and feedback.
The benefits of a strong scientific regulatory agency are many. Most important are the benefits to patients and consumers who need safe and effective drugs, biologics, and medical devices, and who depend on a safe and wholesome food supply. There are benefits from an economic standpoint as well. A credible, high-performing regulatory agency stimulates innovation, enhances U.S. competitiveness in global markets, provides a predictable and level playing field for industry, and strengthens the domestic economy as a whole by inviting increased foreign investment in this country.
Up until the mid-1990's, the agency had been criticized for delaying the availability of medical therapies to patients. This is clearly no longer the case. With additional funding provided through the Prescription Drug User Fee program FDA has proven that it can meet extremely demanding performance goals. We are now reviewing drugs in the U.S. as fast or faster than anywhere else in the world, without compromising the very stringent standards that Americans and the rest of the world have come to expect. The average review time for drugs is less than 12 months and for drugs for serious and life-threatening conditions less than 6 months. This is occurring in spite of the increasing number and complexity of products reviewed today.
Cancer patients may stand to benefit the most from FDA's 1999 product approvals. Eight drugs were approved for the treatment or diagnosis of cancer, including early stage breast cancer, relapsed anaplastic astrocytoma, and non-small cell lung cancer that is refractory to cisplatin-based chemotherapy. Also noteworthy was the approval of a new biologic treatment for patients with advanced or recurrent cutaneous t-cell lymphoma, and a drug-device combination for the treatment of precancerous skin lesions.
We also approved a number of non-cancer-related medications including a new protease inhibitor for the treatment of HIV infection in both children and adults; a new epilepsy drug that helps control partial onset seizures in adults; a plasma derived biologic to treat severe cases of von Willebrand's disease; the first antibacterial drug for infections associated with vancomycin-resistant Enterococcus aecium bacteremia; a new immunosuppressant drug to prevent acute organ rejection in patients receiving kidney transplants; a new non-sterioidal antiinflammatory drug for treatment of osteoporosis, menstrual pain and other acute pain in adults; and a new lipase inhibitor to treat obesity.
Patients with hepatitis, pneumonia and Lyme disease can be treated sooner and with greater assurance thanks to four new tests approved in 1999. They include the first home test for hepatitis C virus and an improved supplemental test to confirm screening test results for antibodies to the hepatitis C virus, which is the nation's most common blood-borne infection and a leading cause of liver damage. Also available now are a urine test for detecting Streptococcus pneumoniae, one of the leading causes of pneumonia, and a simple blood test for Lyme disease that can be used in a doctor's office.
Additional products are now available to help people with diabetes better manage their disease and improve their quality of life, including a new glucose monitoring system--the first device that provides physicians with continuous measures of tissue glucose levels in adults.
Flu sufferers can get relief from two new drugs approved last year: Oseltamivir phosphate (Tamiflu), an oral anti-viral drug for adults whose flu symptoms have not lasted more than two days; an inhaled anti-viral drug for adults and adolescents aged 12 or above with uncomplicated flu.
From the Center for Devices and Radiological Health, we have seen the new T-Scan, an imaging device that will help radiologists determine whether a woman should be evaluated further when the results of her mammograms are ambiguous. In addition, cardiac patients can benefit from three new devices approved by FDA last year. Two of them, Ancure Tube and Bifurcated Endovascular Grafting Systems, are designed to treat aortic aneurysms in the abdomen without major abdominal surgery; the third one, AngioJet, has been approved for removing blood clots from blocked cardiac arteries or bypass grafts prior to angioplasty.
Nearsighted people can be helped with two new devices approved by FDA last year: KeraVision Intacs-tiny acrylic crescents that can be implanted into the eye to correct mild nearsightedness; and an eye laser for use in correcting myopia with or without astigmatism in adults.
The common denominator in all these product review decisions is the importance of assuring safety and demonstrating efficacy. Efficacy is the most obvious. It is the safety part of the equation where the agency spends a great deal of time weighing the benefits and risks of medical products.
During the course of a medical product safety review, it is simply not feasible for the agency to identify every potential risk associated with the use, and abuse, of the product. A key issue is how extensively a product should be studied to uncover adverse reactions before approval. Balancing whether knowing everything is worth keeping patients who need therapies absolutely risk-free. Obviously there is less certainty with a study of 500 people than one with 5,000 or 50,000. Still there could be risks that we won't discover during clinical trials because some adverse events are very rare or may only be associated with a subpopulation that is unusually sensitive compared to everyone else.
Determining the risk in a population does not necessarily predict the risk for the next individual. And from a practical standpoint, having to include 150,000 human subjects in a study could be a major obstacle to getting drugs on the market in a timely fashion, especially for rare diseases.
A less obvious reason that risks unseen in clinical trials appear when the drugs are used in the real world is that in clinical trials, the drugs are only studied for the condition for which the drug was developed, and with all other factors carefully controlled. Once the drug is on the market, it may be prescribed for a condition other than that for which it was approved, given using a different dosing regimen, or it may be used in conjunction with a variety of other drugs, foods, or dietary supplements that could interact with the drug. And the product will be used in numbers of people far greater than were used in the clinical trials, potentially revealing side effects not previously seen.
These factors must all be taken into account when we work with sponsors of new products to make sure they are safe and effective, and available to patients in a timely manner...and we must be alert to new findings after a product is on the market.
The FDA monitors the adverse reactions of drugs for both predictable and unexpected side effects. This requires effort on the part of scientists and medical officers in FDA; physicians and other healthcare professionals in the community; drug manufacturers and the patients themselves, to report any side effects, particularly previously unknown side effects associated with the use of a product.
In May of 1999, FDA issued a report entitled, "Managing the Risks from Medical Product Use: Creating a Risk Management Framework," which was developed by the FDA Task Force on Risk Management. The Task Force assessed risk management practices within the overall health care delivery system, focusing on the roles and responsibilities of each participant.
The Task Force concluded that FDA and the many other participants in health care delivery act to maximize the benefits and minimize the risks associated with using medical products, but often the actions of the participants are insufficiently integrated. Our common goal would be greatly advanced if the participants in the system worked together to gain an understanding of these activities within a systems framework. To do so we need a better understanding of the risks involved and their sources, and we need to clarify our individual roles and ensure that our roles are well integrated.
In examining FDA's role, the Task Force concluded that the agency's pre- and postmarketing risk assessment systems are performing well. However, they did conclude that additional emphasis should be placed on the quality assurance of our premarketing programs, and we have done so. In addition, the task force recommended that the FDA's postmarketing programs should be expanded to ensure that we are able to meet the challenges of the current regulatory and health care environment.
The management of medical risk received renewed attention with the recent Institute of Medicine report, "To Err is Human." This report indicates that medical mistakes are responsible for the deaths of up to 98,000 people every year.
It is important to point out that the majority of injuries and deaths in the U.S. that are attributable to medical products are from known side effects, and not from unexpected ones. The findings of this report confirmed the findings of our earlier report.
This is somewhat reassuring but I think we must conclude that more can and should be done by those of us in the health care community and by patients themselves, to prevent medical errors. There is room for improvement.
Most medical products have inherent risks associated with their use, some greater than others. As these products enter the market place it remains the FDA's responsibility to communicate the known risks to physicians, nurses, pharmacists, patients, consumers, and anyone else who may be affected by those risks or their management. And we must all be alert to previously unknown side effects or interactions with other drugs, foods, or dietary supplements, and prepared to communicate this new information to those involved with administration or use of the product.
The entire health care delivery system must have adequate safeguards in place. This begins with the development, manufacture, and approval of a product, and continues with physician prescribing practices, pharmacy dispensing, and proper administration of the product to patients in a hospital setting, or at home by the patients themselves.
FDA is responsible for the approval decision--determining that a medical product's benefits outweigh its risks. Before marketing, FDA also strives to minimize certain risks by assessing potential name confusions or interactions. We evaluate the adequacy of labels and package inserts for conveying information to the consumer. But, once a product is approved, management of the risks associated with the product in use is primarily in the hands of health care professionals and the patient.
Communication, feedback and correction--throughout the entire system--about the benefits and risks of a drug or other product for a particular use, is critical to the health care system's effort to reduce the incidence of medical errors. This two-way communication must take place between drug companies and physicians, between physicians and patients, between pharmacists and patients, on the product label and package insert and in any advertisements for a particular product.
The agency is now committing considerably more resources to post-market surveillance of FDA-regulated products than it ever has in the past, and we have recently requested from Congress additional resources so that we can be more effective. Doctors, pharmacists, and patients can report serious adverse events to the FDA through our Medwatch system, or they can report side effects to the manufacturer who, in turn, must report the incidents to the FDA. In addition, the agency frequently sends "Dear Health Professional" letters to inform doctors, physicians, and others of new safety issues. In some cases, new information warrants a change in the product's labeling, or less frequently, when all of these corrective safeguards have proved insufficient, the product's withdrawal from the market.
Managing risks also extends to venues that never existed before. We now have a tool with enormous possibilities, but also substantial concern--the Internet. The sale of consumer products--"E-commerce"--over the Internet is growing exponentially. In the health area alone, there are a seemingly limitless number of medical products--both legal and illegal--available to anyone with an Internet service provider and a credit card.
There are clearly many benefits to consumers of legitimate prescription drug sales on the Internet. Greater availability of drugs for people in rural areas or others who find it difficult to get to a drugstore; the convenience of being able to order prescription drugs online, 24 hours a day, and privacy where purchasing can be done from your own home and not in line at the corner drug store.
Many reputable online pharmacies allow patients to consult with a pharmacist and physician from the privacy of their homes. Many online pharmacies are also able to provide customers with written product information and references to other sources of information much more easily than in a typical drug store. It is conceivable that, as the use of this technology to transmit prescriptions from doctors to pharmacies expands, it may even help reduce the incidence of medical errors.
Yet, the Internet has its dark side too. Not all Internet purveyors of medical products and advice are as scrupulous as the reputable pharmacist in your corner drug store. Traditionally there have been high standard safeguards to protect consumers against unsafe drugs--including the requirement that drugs be dispensed only for valid prescriptions from a health provider, and in most states new prescriptions issued only after a physical examination. Unfortunately, the Internet transaction can bypass all of the public health safeguards. We are aware of pharmacy web sites that offer drug prescriptions on the basis of a questionnaire alone. In this scenario, lost are the doctor-patient interaction, a thorough history of the patient, the physical exam, an accurate diagnosis, and the joint decision-making to determine the best treatment for the patient. Inherent in this is the risk that a person will buy a product that is inappropriate for the condition, or is actually contraindicated for that person because of a misdiagnosis or an underlying condition. Hence, the risk increases for serious side effects, drug interactions, and treatment failures.
Other risks include the possible ill effects of impure or unknown ingredients found in drugs manufactured under substandard conditions.
Fraudulent medical claims abound on the Internet--this is not news to any of you--and we are making special efforts to warn consumers about the particularly risky ones that we know of. FDA is warning consumers not to purchase certain unapproved products that pose significant, possibly life-threatening, health risks. To give you an example...one product that is offered for sale on the Internet is a female self-sterilization kit. Clearly, there is a market of unsuspecting women for this type of product.
The self-sterilization kit claims to use a method similar to inserting an IUD and to have a much lower risk than that associated with surgical sterilization. The kit uses pellets of quinacrine hydrochloride, an unapproved drug, which can cause ectopic pregnancy, abnormal pregnancies, and permanent damage to a woman's reproductive organs.
FDA is approaching the problem of illegal online prescription drug sales from two angles. The first is through education. We have expanded our public education efforts to warn consumers about dangerous practices involving Internet sales of drugs that are not FDA-approved, or sites that offer prescription drugs with little or no involvement or interaction with a health care professional. We want consumers to be "internet-savvy" and less likely to be taken in by erroneous health information--better able to evaluate the credibility of the source, and to spot illegal or unethical drug sales practices.
Our other approach is to expand enforcement activities against online sales of drugs through collaboration with interested parties. We have developed partnerships with State Boards of Pharmacy and Boards of Healing Arts. We are also working with State Attorneys General in order to take swift action against the illegal sales of prescription drugs in the U.S. The Attorneys General in both Kansas and Missouri have been particularly active in this area.
Most of these matters have traditionally rested with the states, with minimal federal presence, but state jurisdictions end at the state line. A consumer may be in one state, using an Internet site emanating from a computer in a second state, to order a drug actually dispensed in a third state, under a prescription from a doctor in a fourth state. Over the Internet sellers can easily change the location and appearance of their Internet sites. Clearly, it is important to have a federal presence, whose jurisdiction can span state boundaries, to stop these illegal activities.
We are optimistic that our dual approach of education and enforcement will protect consumers from some of the dangers associated with online prescription drug sales. I encourage your participation as well, in educating your patients and the health community at large, about the benefits--and the risks--of buying their prescription drugs online.
Thank you for the opportunity to be here today.
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