American consumers benefit from having access to the safest and most advanced pharmaceutical system in the world.
The main consumer watchdog in this system is the U.S. Food and Drug Administration's Center for Drug Evaluation and Research (CDER). The center's best-known job is to evaluate new drugs before they can be sold. The center's evaluation not only prevents quackery, but also provides doctors and patients the information they need to use medicines wisely. CDER ensures that drugs, both brand-name and generic, work correctly and that their health benefits outweigh their known risks.
What Is a Drug?
Consumers usually think of drugs as the medicines they take to treat illnesses, but most Americans use CDER-regulated drug products every day. Drugs include more than just medicines. For example, fluoride toothpastes, antiperspirants, dandruff shampoos, and sunscreens are all considered drugs. Some medicines can be purchased over the counter (OTC) without a prescription, while others require a doctor's prescription.
Vitamins and dietary supplements can be sold without prior approval from the FDA and are regulated by the FDA's Center for Food Safety and Applied Nutrition.
Prescription medicines must be administered under a doctor's supervision or require a doctor's authorization for purchase. There are several reasons that medicines are required to be sold by prescription. The disease or condition may be serious and require a doctor's management. The same symptoms can 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 diseases, and some drugs are abused.
As with prescription drugs, CDER closely regulates OTC drugs to ensure that they are safe, effective, and properly labeled. The FDA has been evaluating the ingredients and labeling of OTC products that have been marketed for many years. In some cases, familiar brands or products have been withdrawn from the market because they were ineffective or unsafe.
A generic drug is a chemical clone of a drug sold under a brand name. There are generic versions of both prescription and OTC medicines. For example, ibuprofen is the generic name of the anti-inflammatory drug sold under the brand names Motrin or Advil. The biggest difference between a generic drug and a brand-name drug is usually price. A generic drug often costs about 30 percent less than the brand-name drug. Widespread use of generics helps control medical costs and insurance premiums.
Drug Development and Review
Drug companies seeking to sell a drug in the United States must first test it. The company then sends CDER the evidence from these tests to prove the drug is safe and effective for its intended use. A team of CDER physicians, statisticians, chemists, pharmacologists, and other scientists reviews the company's data and proposed labeling. If this independent and unbiased review establishes that a drug's health benefits outweigh its known risks, the drug is approved for sale. The center doesn't actually test drugs itself, although it does conduct limited research in the areas of drug quality, safety, and effectiveness standards.
Before a drug can be tested in people, the drug company or sponsor performs laboratory and animal tests to discover how the drug works and whether it's likely to be safe and work well in humans. Next, a series of tests in people is begun to determine whether the drug is safe when used to treat a disease and whether it provides a real health benefit.
Reforming the U.S. Drug Review Process
Until this century, prescribing and taking drugs was a risky business for doctor and patient alike. Little was known about drugs, no scientific standards existed, and sometimes medicines caused illnesses rather than curing or preventing them. The U.S. drug review process assures that drugs are safe and effective. It has been lauded for years for the scientific and manufacturing quality it ensures in our drugs.
Getting beneficial drugs on the market quickly is just as much a part of CDER's public health mandate as keeping unproven and dangerous drugs off. Early in the 1990s, CDER started reforming the drug review process to speed the delivery of new drugs to consumers while preserving high standards of quality and safety.
To obtain added resources for reform, the FDA, Congress, and the pharmaceutical industry negotiated the Prescription Drug User Fee Act of 1992. These added resources come from the drug companies and are called user fees. As a result, the center has been able to hire more scientists to review marketing applications for drugs. As part of the agreement, CDER set ambitious performance goals regarding drug reviews, standardized policies, improved communications, and streamlined many burdensome rules and regulations. CDER has created a review process that not only honors sound scientific principles, but sound management principles as well.
Helping Everyone Benefit From Drugs
Congress passed the Orphan Drug Act of 1983 to provide incentives to companies to research and develop medicines for people who have disorders that affect fewer than 200,000 Americans (orphan diseases). The most powerful incentive in the law is marketing exclusivity. Once the FDA approves a company's product for a designated orphan disease, competitors are legally blocked from introducing an identical competing product for seven years. Other provisions provide grants, help from the FDA in designing research protocols that will meet regulatory requirements, and tax credits.
A series of bioethical reforms in the 1960s resulted in federal government rules that protect people who volunteer to take part in medical tests. These reforms sharply curtail the unnecessary risks faced by volunteers and prevent the exploitation of vulnerable groups, such as charity patients, prisoners, or people in the military. An unintended side effect of these reforms, however, was to stunt the development of scientific knowledge about how drugs worked in children, minorities, women, and older people. The center has begun implementing a series of reforms to make sure that these groups are included in clinical trials and that knowledge about the effects of existing drugs in these groups is collected and developed.
Along those lines, the Best Pharmaceuticals for Children Act of 2002 reauthorized pediatric exclusivity. This economic incentive provides extended protection from market competition for pharmaceutical companies that conduct pediatric studies requested by the FDA. In addition, the act requires the National Institutes of Health to develop and prioritize a list of drugs that need to be tested for use in children. The list is developed by the National Institute of Child Health and Human Development, along with the FDA and experts in pediatric research. It identifies gaps in drug use information for which studies would be needed for specific ages of children and specific diseases. The list is updated each year.
On the Watch for Drug Problems
Once a drug is approved for sale in the United States, CDER's consumer protection mission doesn't stop. The center monitors the use of marketed drugs for unexpected health risks. If new, unanticipated risks are detected after approval, CDER takes action to inform the public, change a drug's label, or even remove a product from the market. In addition to evaluating regular reports from manufacturers, the FDA's MedWatch program enables health care professionals and consumers to report suspected problems with their drugs.
Protecting Drug Quality
The center also promotes public health by regulating the manufacture of drugs and by setting standards for drug quality. CDER works closely with FDA field inspectors to make sure that manufacturers comply with current good manufacturing practices. Before a drug is approved, investigators determine whether the manufacturing data in the application are accurate. Once a drug is approved, another inspection is required to show the firm can consistently make a drug in large quantities. Periodic inspections check a firm's overall operation.
As the pharmaceutical industry has become increasingly global, the center is involved in international negotiations with Japan and the European Union to harmonize standards for drug quality and data needed to approve a new drug. This standardization will go a long way toward reducing the number of redundant tests and will help to ensure drug quality for consumers at home and abroad.
In 2002, the FDA began a significant initiative to enhance the regulation of pharmaceutical manufacturing and product quality and to bring a 21st century focus to this FDA responsibility.
The initiative focuses on the FDA's current good manufacturing practice (cGMP) program and will cover veterinary and human drugs, including human biological drug products such as vaccines. This initiative is designed to improve public health promotion and protection by focusing on three major goals that will augment the FDA's pharmaceutical product quality assurance programs across the board.
The first goal will be to focus the agency's cGMP requirements more squarely on potential risks to public health by providing additional attention and resources on those aspects of manufacturing that pose the greatest potential risk.
The second goal is to help ensure that the FDA's establishment and enforcement of pharmaceutical product quality standards do not impede innovation and the introduction of new manufacturing technologies in the pharmaceutical industry.
The third goal is to enhance the consistency and predictability of the FDA's approach to assuring production quality and safety.
Drug Information and Advertising
Accurate and complete information is vital to the safe use of drugs. While drug companies have traditionally promoted their products directly to physicians, more and more they are advertising directly to consumers. Advertising of OTC drugs is regulated by the Federal Trade Commission, but CDER oversees 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.
In addition to its efforts to improve the information that accompanies OTC drugs, CDER monitors a voluntary program that seeks to provide consumer information for prescription drugs in the pharmacy. The center watches this program closely to ensure that it meets its goals for quantity and quality of information.
Getting Consumer Input
CDER routinely consults with the American people in making its decisions about the drugs that they use. It holds public meetings to incorporate expert and consumer input into its decisions. The center also announces many of its decisions in advance so that members of the public, academia, industry, trade associations, consumer groups, and professional societies can comment and make suggestions before decisions become final. In addition, CDER holds annual public meetings with consumer and patient groups, professional societies, and pharmaceutical trade associations to obtain enhanced public input into its planning and priority-setting practices.
Over the years, policies have changed and laws have gotten stronger, but the center's present and future mission remains constant: to ensure that the benefits of drug products made available to the public outweigh all known risks.