About CDER - Report

CDER Report to the Nation: 2000

Center for Drug Evaluation and Research
Food and Drug Administration
U.S. Department of Health and Human Services

CDER 2000 Report to the Nation

Improving Public Health Through Human Drugs

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Contents

Introduction

1999 Highlights

• Scientific Research

1 Drug Review

• New Drug Review
• New or Expanded Use Review
• Over-the-Counter Drug Review
• Generic Drug Review
• Manufacturing Supplement Review
• User Fee Review Performance

2 Drug Safety and Quality

• Drug Safety
• Drug Promotion Review
• Export Certificates
• Drug Recalls and Withdrawals
• Drug Product Quality

3 International Activities

4 Communications

• Where to Find More Information

Introduction

Who we are

The Center for Drug Evaluation and Research is America's consumer watchdog for medicine. We are part of one of the nation's oldest consumer protection agencies-the Food and Drug Administration. The FDA is an agency of the federal government's Department of Health and Human Services. We are the largest of FDA's five centers, with nearly 1,700 employees. Approximately half of us are physicians or other kinds of scientists. Many of us have experience and education in such fields as computer science, legal affairs and regulatory matters.

What we do

Our best-known job is to evaluate new drugs for safety and effectiveness before they can be sold. Our evaluation, called a review, makes sure that the drugs we approve meet our tough standards for safety, effectiveness and quality. We also make sure that you and your doctor will have the information you need to use medicines wisely. Once drugs are on the market, we monitor them for problems.

Reviewing drugs before marketing. A drug company seeking to sell a drug in the United States must first test it. We monitor clinical research to ensure that people who volunteer for studies are protected and that the quality and integrity of scientific data are maintained. The company then sends us the evidence from these tests to prove the drug is safe and effective for its intended use. We assemble a team of physicians, statisticians, chemists, pharmacologists and other scientists to review the company's data and proposed use for the drug. If the drug is effective and we are convinced its health benefits outweigh its risks, we approve it for sale. We don't actually test the drug when we review the company's data. By setting clear standards for the evidence we need to approve a drug, we help medical researchers bring new drugs to American consumers more rapidly. We also review drugs that you can buy over the counter without a prescription and generic versions of over-the-counter and prescription drugs.

Watching for drug problems. Once a drug is approved for sale in the United States, our consumer protection mission doesn't stop. We monitor the use of marketed drugs for unexpected health risks. If new, unanticipated risks are detected after approval, we take steps to inform the public and change how a drug is used or even remove a drug from the market. We also monitor manufacturing changes to make sure they won't adversely affect the safety or efficacy of the medicine. We evaluate reports about suspected problems from manufacturers, health care professionals and consumers. Sometimes, manufacturers run into production problems that might endanger the health of patients who depend on a drug. We try to make sure that an adequate supply of drugs is always available.

Monitoring drug information and advertising. Accurate and complete information is vital to the safe use of drugs. Drug companies have historically promoted their products directly to physicians. More and more frequently now, they are advertising directly to consumers. While the Federal Trade Commission regulates advertising of over-the-counter drugs, we oversee the advertising of prescription drugs. Advertisements for a drug must contain a truthful summary of information about its effectiveness, side effects and circumstances when its use should be avoided. We are monitoring the industry's voluntary program to provide consumers useful information about prescription drugs when they pick up their prescriptions. We are watching this program closely to see that it meets its goals for quantity and quality of information.

Protecting drug quality. In addition to setting standards for safety and effectiveness testing, we also set standards for drug quality and manufacturing processes. We work closely with manufacturers to see where streamlining can cut red tape without compromising drug quality. As the pharmaceutical industry has become increasingly global, we are involved in international negotiations with other nations to harmonize standards for drug quality and the data needed to approve a new drug. This harmonization will go a long way toward reducing the number of redundant tests manufacturers do and help ensure drug quality for consumers at home and abroad.

Conducting applied research. We conduct and collaborate on focused laboratory research and testing. Research maintains and strengthens the scientific base of our regulatory policy-making and decision-making. We focus on drug quality, safety and performance; improved technologies; new approaches to drug development and review; and regulatory standards and consistency.

Why we do it

Our present and future mission remains constant: to ensure that drug products available to the public are safe and effective. Our yardstick for success will always be protecting and promoting the health of Americans.

Getting consumer input. Protecting consumers means listening to them. We routinely consult the American public when making decisions about the drugs that they use. We hold public meetings about once a week to get expert, patient and consumer input into our decisions. We also announce many of our proposals in advance. This gives members of the public, academic experts, industry, trade associations, consumer groups and professional societies the opportunity to comment and make suggestions before we make a final decision. In addition, we take part in a series of FDA-sponsored public meetings with consumer and patient groups, professional societies and pharmaceutical trade associations. These stakeholder meetings help us obtain enhanced public input into our planning and priority-setting practices.

What is a drug?

We regulate drugs used to treat, prevent or diagnose illnesses. However, drugs include more than just medicines. For example, fluoride toothpaste, antiperspirants, dandruff shampoos and sunscreens are all considered "drugs." You can buy some drugs in a store without a prescription, while others require a doctor's prescription. Some are available in less-expensive generic versions.

Prescription drugs

Prescription medicines must be administered under a doctor's supervision or require a doctor's authorization for purchase. There are several reasons for requiring a medicine be sold by prescription:

• The disease or condition may be serious and require a doctor's management.
• The medicine itself may cause side effects that a doctor needs to monitor.
• The same symptoms may be caused by different diseases that only a doctor can diagnose.
• The different causes may require different medicines.
• Some medicines can be dangerous when used to treat the wrong disease.

Over-the-counter drugs

You can buy OTC drugs without a doctor's prescription. You can successfully diagnose many common aliments and treat them yourself with readily available OTC products. These range from acne products to cold medications. As with prescription drugs, we closely regulate OTC drugs to ensure that they are safe, effective and properly labeled.

Generic drugs

A generic drug is a chemical copy of a brand-name drug. There are generic versions of both prescription and over-the-counter drugs. Generic drugs approved by the FDA have the same therapeutic effects as their brand-name counterparts. The biggest difference between a generic drug and its brand name counterpart is usually price. A generic drug may be priced anywhere between 20 percent and 75 percent of the cost of the brand-name version.

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We are pleased to present our fifth performance report. Our work last year offered many Americans new or improved choices for protecting and maintaining their health or new ways to use existing products more safely.

Drug review

Children have especially benefited from our approvals in 2000. Also, people with HIV, AIDS, cancer, heart disease and other serious conditions all benefited. We met our obligations to Congress for prompt and thorough review of drug applications supported by user fees. Our reviews of generic drugs have been prompt and predictable . We approved 98 new drugs, including 27 new molecular entities. New molecular entities contain an active substance never before approved for marketing in any form in the United Sates. We also approved 134 new or expanded uses of already approved drugs, 11 new or expanded uses for over-the-counter drugs and 244 generic drugs.

Drug safety and quality

All medicines have risks. With modern, state-of-the-art tools and techniques, we are able to detect rare and unexpected risks more rapidly and take corrective action more quickly. Last year, we processed and evaluated nearly a quarter million reports of adverse drug events. We issued more than 1,000 letters to help ensure that the promotion of drug products presents a fair balance of risks and benefits and isn't false or misleading. We mandated that five drug products be dispensed with specific consumer information that will help ensure the products are used safely and effectively. We issued a proposed rule to revise prescription drug labeling to improve its accessibility and enhance safe and effective prescribing and use. Our reviews of the safety profile of three approved drug products resulted in their voluntary withdrawal. We issued a public health alert about a once widely used decongestant that may cause strokes. The alert resulted in its withdrawal from the market by manufacturers.

International activities

We worked closely with our colleagues in Japan and the European Union on finding ways to make the drug development process more efficient and uniform. We agreed to a final version of the Common Technical Document that can be used for seeking approval to market new drugs in the United States, the European Union and Japan. We began harmonization efforts among the countries of North and South America. We led the U.S. consultations with the European Union to allow for reciprocal reliance on manufacturing plant inspections.

Communications

We continued our efforts to include greater input into our planning and decision making from consumers, patients, health-care professionals, academia and industry. We met almost weekly with outside experts on difficult scientific and public health issues. We responded to more than 58,000 individual requests for information.

Our Internet information site averaged more than 406,000 visitors and about 6.7 million hits per month. We developed public education campaigns in areas such as risk management and buying prescription drugs over the Internet.

Drug withdrawals influence benefit-risk assessment

The recent market withdrawals of some drugs resulted, in part, from the U.S. health care system's inability to manage known and preventable risks. These experiences have catalyzed an evolution in our thinking on risk management and the evaluation of new drugs for approval. Our risk assessment must evaluate both a drug's intrinsic safety profile as well as the ability of the health care system to adequately manage known toxicities.

Unless effective risk management strategies and methods are brought to bear, additional effective drugs are likely to be withdrawn, and some drugs may never become available in the first place.

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Scientific Research

We focus on advancing the scientific basis of regulatory policy by developing new scientific methods and regulatory testing paradigms and by providing scientific support for the development of regulatory policy. We have focused specifically on creating tighter linkage between nonclinical and clinical studies, enhancing the methodology for assuring product quality, building databases for improved drug development and review and providing regulatory support through laboratory testing.

Linking nonclinical and clinical studies

Key achievements in this area include:

• Establishing that biological markers for heart damage, such as troponin, are useful both experimentally and clinically to predict the incidence and extent of heart damage from exposure to certain drugs.
• Identifying unique biomarker proteins found in blood that cause heart damage and that are generated after treatment with certain drugs.
• Elucidating the molecular mechanisms operating in alternative transgenic mice that are used to test the cancer-causing potential of drugs. This understanding will make these models more rapid and reliable assessment tools.
• Developing an experimental model for detecting reactive metabolic products of drugs in cultured liver cells. This will facilitate mechanistic analysis of certain types of liver damage.

Database building

Our work included:

• Developing and implementing an Internet-enabled pilot of FDA's toxicology knowledge base. This database is linked to an expanded chemical substance dictionary that permits searches using unique chemical structures as well as structural similarities.
• Developing and distributing new computational software that predicts birth defects from drugs in rodent laboratory models. This was done under a commercial research and development agreement.
• Developing computational toxicology software to predict the human no-adverse-effect level for a substance based on its chemical structure and extrapolation of information about the maximum therapeutic dose.
• Developing computational models that use the pharmaceutical properties and molecular structure of drugs to predict their bioavailability in humans when administered orally.

Leveraging scientific resources

The Product Quality Research Institute is a unique and innovative collaboration among our scientists and those from academia and industry. PQRI conducts research to establish better testing methods, standards and controls for assessing product quality and manufacturing and management processes.

The institute's research will help us develop consistent and reasonable requirements for product quality information in regulatory filings. Leveraging scientific expertise in this way contributes to streamlining the drug development and approval processes for industry and ourselves while ensuring the highest level of product quality for American consumers.

Deming Award recognizes our scientific training

Our program of scientific training based on core competencies received the prestigious W. Edwards Deming Outstanding Training Award for 2000 presented by the Graduate School of the Department of Agriculture.

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