Sept. 21, 2009 | Modern medical treatments, whether they involve new drugs, devices or procedures, undergo extensive testing, including a process called a clinical trial. Clinical trials are research studies that test how well new medical approaches work in people. Each study answers scientific questions and tries to find better ways to prevent, screen for, diagnose or treat a disease. Clinical trials may also compare a new treatment to a treatment already available. Every clinical trial has a protocol, or action plan, for conducting the trial. The plan describes what will be done in the study, how it will be conducted, and why each part of the study is necessary. Each study has its own rules about who can participate. Some studies need volunteers with a certain disease while others need healthy people. In the U.S., a group of physicians, statisticians and members of the community must approve and monitor the protocol to make sure the risks are small and worth the potential benefits.

Sept. 21, 2009 | All clinical trials have guidelines about who can participate. Using inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. The factors that allow someone to participate in a trial are called “inclusion criteria” and those that disallow someone from participating are called “exclusion criteria.” These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied, while others need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people but rather are used to identify appropriate participants and to insure their safety during the clinical trial. The criteria help ensure that researchers will be able to answer the questions they plan to study.

Sept. 21, 2009 | Clinical trials are conducted in phases. Each phase has a different purpose and help scientists answer different questions. In Phase 1 trials, researchers test an experimental drug or treatment in a small group, somewhere between 20 and 80 people, to evaluate its safety, determine a safe dosage range, and identify side effects. In Phase 2 trials, the experimental study drug or treatment is given to a larger group of people, usually between 100 and 300, to see if it is effective and to further evaluate its safety. In Phase 3 trials, the experimental study drug or treatment is given to large groups of people, no more than 3,000, to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely. In Phase 4 trials, also known as post marketing studies, will provide additional information, including the drug’s risks, benefits, and optimal use.

Sept. 21, 2009 | Like many forms of medical treatment, there are certain benefits as well as risks associated with clinical trials, even those that are well designed and well executed. Such trials can play an active role in the health care of the participants as well as providing them with access to new research treatments before they are widely available. Clinical trials allow the participants to obtain expert medical care at leading health care facilities during the trial, not to mention the fact that they help others by contributing to medical research. Unfortunately, there may be unpleasant, serious or even life-threatening side effects to experimental treatments. The experimental treatment may not be effective for the participant. Those taking part in a clinical trial may find that the protocol requires more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.

Sept. 21, 2009 | Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board, or IRB, to make sure the risks are as low as possible and are worth any potential benefits. An IRB is a committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical, participants’ safety is insured and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research. Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the Department of Veterans Affairs. And clinical trials can take place in locations like hospitals, universities, doctors’ offices or clinics.