Learn More About Clinical Trials

What are clinical trials?

Before a new drug or new treatment is approved by the U.S. Food and Drug Administration (FDA) it must be shown to be both safe and effective. Typically, this is accomplished via clinical trials-carefully controlled studies designed to test and evaluate new drugs and new treatment plans. These trials are carried out to learn more about the new treatment and to find better therapies.

Why do people participate in clinical trials?

People may be interested in clinical trials for a variety of reasons. Some people who are very sick or are not responding to standard treatments join clinical trials in order to receive new, investigational treatment. They are hoping that this treatment-possibly a new drug or a new combination of drugs-will work better for them than standard therapy. This is not always the case as new treatments strategies may not work or may have side effects. For this reason, all people in clinical trials are carefully monitored. Frequent medical exams and tests are performed to assure their safety.

Other people participate in clinical trials to contribute to medical science and to help doctors and researchers find better ways to help others.

Are there risks in clinical trials?

The process of evaluating new treatments can involve some risk. All drugs used in clinical trials have been extensively tested in laboratory experiments. However, some side effects do not become apparent until the treatments are given to humans. Side effects can vary from patient to patient. It is important to remember that clinical trials can carry unknown dangers as well as possible benefits.

The progress of patients in clinical trials is constantly monitored. Periodic reviews of test results and other statistics are carried out while the trial is underway. If, at any time, a new treatment is found to be harmful to the study participants, the trial will be terminated.

What is informed consent?

Before enrolling in a clinical trial, the doctors and nurses will discuss with all potential participants the possible risks and benefits involved. These issues are summarized in a lengthy form that explains the trial procedures and the possible side effects. This is called an informed consent form and all clinical trial participants are asked to read, understand, and sign it before beginning treatment. It is important to ask the doctor or nurse to explain any part of the form that is unclear.

Can clinical trial participants leave a trial?

Yes. Even though someone has agreed to participate in a trial by signing the informed consent form, they are free to leave the trial at any time.

How are clinical trials conducted?

The doctors and researchers running a clinical trial develop a written plan detailing exactly how the trial will be conducted. This plan, also called a protocol, explains how the trial will be run, what information will be gathered, and what new things the researchers hope to learn.

Many clinical trials compare a new treatment to a standard treatment. In these trials, participants are divided into two groups. One group receives the new treatment and one group receives the standard treatment (the “control” group). Various statistics are then gathered using blood, x-ray, and other tests to assess how well each group responds to the two kinds of treatment.

In order to prevent biased results or interpretation, many trials assign participants by chance to one of these two groups. This process, known as randomization, is only used when it is not known which treatment option will work better.

Can clinical trial participants still see their regular doctor?

Yes. Clinical trials do not replace normal health care. In fact, the regular doctors of trial participants should be updated regularly by the trial coordinators.

What are the different phases of clinical trials?

Clinical trials are traditionally divided into different phases. Each phase is designed to gather specific information about the drug or treatment being studied.

Phase I trials

These trials are the first human tests of new drugs or therapies. They typically involve small numbers of volunteers and are designed to determine the best dose of the drug and to check for any side effects. Because Phase I trials use treatments that have never been tested in humans, they may involve significant risks.

Phase II trials

If a treatment is shown to be safe and well tolerated, it moves on to Phase II trials. These trials involve many more volunteers and are designed to see how well the treatment works, usually in a small group of patients.

Phase III trials

If the treatment is effective in Phase II trials, it moves on to Phase III trials. Hundreds or even many thousands of patients may participate in these studies. They are aimed at further testing how well the treatment works. Often, Phase III trials compare the new treatment to an already existing, standard treatment, in a randomized fashion.

Phase IV trials

These trials are conducted after a drug has been approved by the FDA and is on the market. Phase IV trials typically involve a very large number of participants and are designed to evaluate new uses of existing therapies or to detect side effects that were not apparent during Phase III studies.

Who pays for the treatment in a clinical trial?

For the most part, the drugs used in clinical trials are given free of charge to the study participants. However, there may be other costs associated with the trial, such as blood work, x-ray tests, and travel expenses. Health insurance may cover some of these items and some may be offered free of charge. Medicare now covers all routine patient care costs associated with clinical trials. The financial details should be discussed prior to enrolling in a trial.

What questions should be asked about a clinical trial?

The following questions might be helpful in discussing a clinical trial with a healthcare provider (Adapted from the NIH booklet What Are Clinical Trials All About?).

  • What is the purpose of the trial? What kind of tests and treatments are involved? What is likely to happen in my case with, or without, this new research treatment? What are alternative treatment options and their advantages and disadvantages? How might this trial affect my daily life? What side effects might I expect from the study? How long will the trial last? Will hospitalization be required? Who will pay for the treatment? If I were harmed as a result of the trial, what treatment would I be entitled to? What type of long-term follow-up care is part of this study?