V.

Medical Research

Research is one of the most important fields of medicine. It provides health care professionals with new knowledge and technology for better diagnosis, treatment, and prevention of disease.

Medical research often combines medicine with related fields of biology, and is called biomedical research. Research can be basic or applied. Basic, or fundamental, research has no immediate practical application. Basic cancer research, for instance, may try to identify gene mutations that turn a healthy cell malignant. While this information does not have immediate clinical value, it generates knowledge that often leads to better care for patients. Applied research has a specific practical goal, such as development of a better drug for breast cancer. The early stages of biomedical research usually occur in a laboratory. As scientists gain more knowledge in a particular area, they begin studies on humans. These studies often take place in hospitals or clinics and are called clinical research.

Clinical research usually is performed by multidisciplinary teams, rather than by individual scientists working alone. These groups of men and women have knowledge and skills in different areas, or disciplines, of science. A multidisciplinary biomedical research team may include biochemists, geneticists, physiologists, and physicians. Each team member approaches the problem from a different side and shares knowledge with the group. This multidisciplinary approach increases the chances of solving a problem or developing a new treatment.

A.

Clinical Trials

One of the greatest advances in medicine was the introduction of a new research technique in the mid-1950s called the controlled clinical trial, which is used to determine if new drugs and other treatments are safe and effective. In the controlled clinical trial, one group of patients, the treatment group, receives the new drug or new treatment. Another group, the control group, is given an inactive pill (a placebo) or the best standard treatment. Researchers then compare the two groups over a period of time. The data collected is put through rigorous statistical techniques to determine whether the new treatment is safer and more effective than standard therapy or no treatment.

Most clinical trials are conducted on a blind or double-blind basis. In a blind trial, patients do not know whether they receive the new drug or a placebo. In a double-blind trial, neither patients nor physicians know who is receiving the new treatment. This secrecy is important because patients who know they are taking a powerful new drug may expect to feel better and report improvement to doctors. Researchers who know that a patient is receiving the test treatment may also see improvements that really do not exist.

Clinical trials usually are randomized. Researchers put patients into the treatment group or control group at random. This helps to assure that neither group contains an excess of patients with severe disease. A drug may appear more effective if the treatment group were packed with patients who had only mild symptoms.

The results of clinical trials are subjected to peer review. Researchers publish their results in scientific journals or present them to an audience of other scientists, who are their peers. This gives scientists not involved in the research a chance to spot potential errors.

B.

Research Funding

Until World War II (1939-1945), most money for medical research in the United States was donated by wealthy individuals, industry, and universities. Scientists resisted government funding because they feared losing the intellectual freedom to study as they chose. Since the 1940s, however, the Federal Government has taken a major role in funding biomedical research.

The National Institutes of Health (NIH) in Bethesda, Maryland, is the biggest government source of research funds. NIH is an agency within the U. S. Department of Health and Human Services. In 2001, the NIH planned to spend about $20.3 billion on biomedical research, distributed to scientists in colleges and universities to conduct specific research projects.

The pharmaceutical industry spent about $26 billion on research in 2000. The next largest source of funds is the Howard Hughes Medical Institute (HHMI), which spends about $554 million annually. Other major funding sources are private foundations and voluntary health organizations. Private foundations are organizations established by wealthy individuals. Among those active in biomedical research are the Charles A. Dana Foundation, the Lucille P. Markey Foundation, and the Whittaker Foundation. Voluntary health organizations are charities supported by contributions from members and the public. Major voluntary health organizations include the American Cancer Society, the American Heart Association, and the American Diabetes Association.

C.

Research Costs

Research is expensive. During the late 1990s the NIH often spent more than $130,000 per year to fund an average research project. Drug manufacturers estimate that they spend an average of $359 million to develop one new drug.

The availability of funding often determines what medical research is conducted. Voluntary health organizations and other groups act as advocates in urging or lobbying the government to spend more on their own particular disease. Governments in developed countries usually spend most heavily on diseases that affect their own citizens, and these diseases are typically different than those commonly found in developing countries. Pharmaceutical companies also emphasize development of the most profitable new drugs, usually for diseases that occur in developed countries.

As a result, little research is done on diseases that kill millions of people in developing nations. In 1998, for instance, the NIH planned to spend only $116 million on malaria and other tropical diseases. While rare in industrialized nations with developed health care programs, malaria kills 1.5 million to 2.7 million people in developing countries each year.