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Welcome to the Senior Health Section of RetirementCommunity.com. This easy-to-use website features health and wellness information for older adults from the National Institutes of Health.

 

Taking Medicines

Testing Medicines

Testing potential new drugs in people is done through a process known as clinical trials. A clinical trial is a research study with people to find out if a new drug or treatment is both safe and effective. This type of medical research has led to treatments for many diseases, such as Hodgkin's lymphoma, arthritis, heart disease, and childhood leukemia.

Testing Medicines - Developing Medicines

The Food and Drug Administration, or FDA, is the federal agency responsible for making sure that foods and cosmetics are safe, and that drugs and medical devices are safe and effective. Clinical trials are a key part of drug discovery and development, and the FDA requires that new drugs be tested in clinical trials before they are put on the market.

Clinical trials are sponsored or funded by pharmaceutical companies, federal agencies like the National Institutes of Health, foundations, individuals, and voluntary groups. Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.

Along with seeking new therapies, researchers sometimes perform clinical trials to compare existing drugs. This way, they can see which medication or combination of medications is the safest or most effective for treating a particular ailment.

Both prescription drugs and over-the-counter drugs are tested in clinical trials before they become available to the public. However, according to a 1994 law, ingredients used in dietary supplements no longer have to be tested like drugs before they go on the market, so they are not tested in clinical trials. Also, there are no regulations that govern the way these substances are manufactured.

How Medicines are DevelopedScientists usually do years of experiments in the laboratory and in animals before they can even consider testing an experimental medication in people. Most of this early research occurs at universities and medical centers across the country. The National Institutes of Health funds much of this basic research.

According to the Pharmaceutical Research and Manufacturers of America, most of the medicines tested in scientists' laboratories fail in laboratory or animal studies. On average, only 5 out of every 5,000 potential medicines actually make it to clinical trials. And only one of these five is eventually approved for use in patients.

In 2003, the Tufts University Center for the Study of Drug Development found that a pharmaceutical company typically spends $802 million over 10 to 15 years to bring a new medicine from the laboratory to your local pharmacy.

It is true that developing drugs is a time-consuming and expensive process. Yet, clinical trials are the only way doctors can know for sure whether medicines are both safe and effective in people.

Enrolling in a clinical trial offers benefits and risks. If you choose to participate in a clinical trial, you may get therapies not yet available to most patients. Also, patients in clinical trials are watched very closely, and they typically benefit from a high standard of care.

Importantly, scientists who wish to test drugs in people must follow strict rules that are designed to protect those who volunteer to participate in clinical trials. Special groups called Institutional Review Boards, or IRBs, evaluate all proposed research with humans to determine the potential risks and expected benefits.

The goal of an IRB is to make sure that the risks to humans are minimized and that they are reasonable compared to the knowledge researchers expect to gain by doing the study. Clinical studies cannot go forward without IRB approval.

Quiz

1. Clinical trials help scientists find new treatments for a variety of health problems.

TRUE is the correct answer. After testing potential drugs in the laboratory and in animals, researchers have to see how drugs work inside the human body. Researchers sometimes perform clinical trials to compare existing drugs, so they can see which medication or combination of medications is the best possible treatment for a particular ailment. Treatments for many diseases are found through this important type of research.

2. Most drugs tested in clinical trials eventually become available to the public.

FALSE is the correct answer. Most of the medicines tested in scientists' laboratories fail in laboratory or animal studies. On average, only 5 out of every 5,000 potential medicines actually make it to clinical trials. And only one of these five is eventually approved for use in patients.

3. The Food and Drug Administration monitors the safety and effectiveness of foods, drugs, and dietary supplements.

FALSE is the correct answer. The Food and Drug Administration, or FDA, makes sure that foods and cosmetics are safe, and that drugs and medical devices are safe and effective. However, according to a 1994 law, ingredients used in dietary supplements no longer have to be tested like drugs before they go on the market, so they are not tested in clinical trials. Also, there are no regulations that govern the way these substances are manufactured.

4. Strict rules protect people who participate in clinical trials.

TRUE is the correct answer. Special groups called Institutional Review Boards, or IRBs, evaluate all proposed research with humans to determine the potential risks and expected benefits. The goal of an IRB is to make sure that the risks to humans are minimized and that they are reasonable compared to the knowledge researchers expect to gain by doing the study.

Testing Medicines - Clinical Trials

People in clinical studies must agree to the terms of a trial by participating in a process called informed consent and signing a form, required by law, that says they understand the risks and benefits involved in the study.

Throughout a study, patients are kept informed about anything that may affect their willingness to continue. Clinical trial patients may withdraw from a study at any time, even after signing the consent form.

Scientists conduct clinical trials in three steps, called phases I, II, and III. Each phase provides the answer to a different question about a potential new drug: Is it safe? Does it work? Is it better than the standard treatment?

Phase I studies test a drug's safety in a few dozen to a hundred people and are designed to figure out what happens to a drug in the body -- how it is absorbed, metabolized, and excreted. Phase I studies usually take several months.

Phase II trials test whether or not a drug produces a desired effect. These studies take longer -- from several months to a few years -- and can involve up to several hundred patients.

A phase III study further tests how well a drug works, as well as whether it is better than current treatments. Phase III studies involve hundreds to thousands of patients, and these advanced trials usually last several years.

Many phase II and phase III studies are randomized, meaning that one group of patients gets the experimental drug being tested while a second, control group gets either a standard treatment or a placebo. A placebo often comes in the form of a "dummy" pill or injection. The use of placebos helps researchers compare the effects of a given treatment against no treatment.

Usually, phase II and phase III studies are "double-blinded" -- neither the patients nor the researchers know who is getting the experimental drug. This helps to prevent bias on the part of the researcher or the patient.

Finally, once a new drug has completed phase III testing, a pharmaceutical company can request approval from the Food and Drug Administration to market the drug. Once a drug goes on the market, it undergoes a period called phase IV, or "post-market surveillance."

During this time just after a new drug becomes available, uncommon and unexpected problems with a new medicine may occasionally occur. This is because rare problems, which range from mild to severe side effects, can only surface when hundreds of thousands to millions of people begin taking the medication.

To find out more about participating in a clinical study, talk to your doctor or search on-line for the clinical trials that are available. For more information visit the federal Web site: ClinicalTrials.gov.

Quiz

1. Testing drugs in people usually takes a few months.

FALSE is the correct answer. Scientists conduct clinical trials in three steps, called phases I, II, and III. The entire process usually takes several years.

2. The first step in the clinical trial process determines whether a drug is safe for use in humans.

TRUE is the correct answer. Early clinical trials, known as phase I studies, test a drug's safety in a few dozen to a hundred people and are designed to figure out what happens to a drug in the body -- how it is absorbed, metabolized, and excreted.

3. Your doctor can enroll you in a clinical trial without your knowing about it.

FALSE is the correct answer. Participating in a clinical trial is completely voluntary. People in clinical studies must go through a process called informed consent and sign a form, required by law, that says they understand the risks and benefits involved in the study. Patients can withdraw from a trial at any time, even after they have signed the consent form.

4. In randomized clinical trials, study patients receive any of several different types of medicines at random.

FALSE is the correct answer. In randomized clinical trials, one group of patients gets the experimental drug being tested while a second, control group gets either a standard treatment or a placebo. The use of placebos helps researchers compare the effects of a given treatment against no treatment.

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