Clinical research trials are carefully-controlled research studies that test how well new medical approaches work in people. They are designed to answer scientific questions about vaccines, new therapies or new ways of using known treatments and are used to determine whether those new drugs or treatments are both safe and effective.
Clinical trials are sponsored or funded by a variety of organizations such as pharmaceutical companies, medical institutions, and foundations, as well as federal agencies such as the Department of Defense, the National Cancer Institute, or the National Institutes of Health.
Clinical research trials give scientists and doctors vital information about the safety and effectiveness of new treatments and procedures. Although much can be learned from laboratory studies and scientific research, only clinical trials can show whether new treatments actually benefit people. Information gained in these trials allows researchers and doctors to develop new and improved ways to prevent, diagnose and treat diseases.
Clinical trials have sets of very well-established rules (called protocols) which define who can participate. These protocols spell out the exact state of a participant’s health and may specify other conditions, such as age and gender. Rules to qualify to participate in a clinical trial are very strict, for several reasons. To ensure safety, researchers must gather very specific information about people with a certain kind of disorder and medical history. They also want to recruit people who are likely to be helped by the new treatment and who have the lowest risk of possible side effects.Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB). The Board, which includes doctors, researchers, community leaders, and other members of the community, reviews the protocol to make sure the study is conducted fairly, ethically, and participants are not likely to be harmed. The IRB also decides how often to review the trial once it has begun. Based on this information, the IRB decides whether the clinical trial should continue as initially planned and, if not, what changes should be made. An IRB can stop a clinical trial if the researcher is not following the protocol or if the trial appears to be causing unexpected harm to the participants. An IRB can also stop a clinical trial if there is clear evidence that the new intervention is effective, in order to make it widely available.
In addition to having access to promising new medical approaches that are not often available outside of a clinical trial, the new medical therapies being studied may also be more effective than the standard approach and participants receive regular and careful medical attention from a research team of doctors and other healthcare professionals. However, because the treatments are considered experimental, they may not be effective or may cause unpleasant or possibly dangerous side effects. Participating in a clinical trial may also be time consuming because the study may require more visits to the doctor.Ultimately, your doctor is the best person to determine whether you should participate in a clinical research trial, so you should consult with him or her before deciding to enter a study. If you decide to participate, it is critical that you follow the doctor’s instructions very carefully.
People who participate in clinical trials do so for many different reasons. They may want to play a more active role in their own health care or gain access to new medical treatments that aren’t currently available to them. Some people participate because they feel good knowing that the information obtained from their participation in a study will ultimately benefit others with the same condition. In addition, participants can receive medical evaluations, study-related lab tests and study medications at no cost and are usually compensated for their time and travel.
Clinical trials usually fall into four categories. Each phase has a different goal and is designed to answer a different question:
Phase 1: Phase 1 clinical trials are designed to test the safety of a new drug or medical procedure. They are usually quite small and may only involve a few dozen people.
Phase 2: Phase 2 clinical trials are designed to further test the safety of the new treatment and learn if it is effective. They usually involve several hundred people.
Phase 3: Phase 3 clinical trials are intended to confirm whether a new treatment is effective as well as gather information about its side effects. They usually involve 1,000 people or more.
Phase 4: Phase 4 clinical trials are usually undertaken after a new drug has been approved for use by the FDA. In these trials, additional information is gathered about the drug’s long-term benefits and risks, how the drug compares to other drugs already available on the market, or the best ways to use the new drug.
Just as participation in a clinical trial is voluntary, withdrawal is also voluntary. A participant can leave a clinical trial at any time, but should always inform the research team and let them know the reasons why, as that information may be important to the trial results.
After a Phase 1 or Phase 2 clinical trial is over, the researchers carefully examine the data collected and decide whether to move on to the next phase or discontinue the study because the therapy was not safe or effective. When a Phase 3 trial has been completed, researchers will look at the data and decide if the results have medical importance. Results of Phase 1 through Phase 3 trials are usually published in peer-reviewed scientific or medical journals where experts can review the reports to make sure the conclusions are sound. Once a new therapy is proven safe and effective, it may become the new standard of practice.