What is it like to receive treatment in a
study?
When you participate in a clinical trial, you receive treatment in a hospital, clinic, and/or doctor's office. Doctors, nurses, and other health professionals may be part of your treatment team. These people follow your progress closely and in addition, a data safety monitoring board (DSMB) reviews the results of the drug to ensure it is safe and effective. During a clinical trial, you may have more tests and doctor visits than you would if you were not taking part in a study. You will follow a treatment plan your doctor prescribes, and you may also have other responsibilities such as keeping a log or filling out forms about your health. Some studies continue to check on patients even after their treatment is over.

How is the research carried out and how are patients protected?
In clinical trials, patient well-being is very important. To help protect patients and produce sound results, research with people is carried out according to strict scientific and ethical guidelines. These guidelines include:

1. Each clinical trial has an action plan (protocol) that explains how it will work.
   

   The study protocol explains to the doctor what will be done in the study and why. It outlines how many people will take part in the study, what medical tests they will receive and how often. The same protocol is used by each doctor that participates in the clinical study by enrolling patients.

   For patient safety, each protocol must be approved by the organization that sponsors the study and an Institutional Review Board (IRB). Members on the IRB include consumers, clergy, and health professionals. The IRB reviews the protocol to ensure the research will not expose patients to extreme or unethical risks.

2. Each study enrolls people who are alike in key ways.

   Each study's protocol describes the characteristics that all patients in the study must have. Called eligibility criteria, these guidelines differ from study to study, depending on the research purpose. They may include age, gender, the type and stage of cancer, and whether cancer patients who have had prior cancer treatment or who have other health problems can take part.

   Using eligibility criteria is an important principle of medical research that helps produce reliable results. During a study, they help protect patient safety, so that people who are likely to be harmed by study drugs or other treatments are not exposed to the risk. After results are in, they also help doctors know which patient groups will benefit if the new treatment being studied is proven to work. For instance, a new treatment may work for one type of cancer but not for another, or it may be more effective for men than women.

3. Cancer clinical trials include research at three different phases.

   Each phase answers different questions about the new treatment.

   • Phase I trials are the first step in testing a new treatment in humans. In these studies, researchers look for the best way to give a new treatment (e.g., by mouth, IV drip, or injection? how many times a day?). They also try to find out if and how the treatment can be given safely (e.g., best dose?); and they watch for any harmful side effects. Because less is known about the possible risks and benefits in Phase I, these studies usually include only a limited number of patients who would not be helped by other known treatments.
   • Phase II trials focus on learning whether the new treatment has an anticancer effect (e.g., Does it shrink a tumor? improve blood test results?). As in Phase I, only a small number of people take part because of the risks and unknowns involved.
   • Phase III trials compare the results of people taking the new treatment with results of people taking standard treatment (e.g., Which group has better survival rates? fewer side effects?). In most cases, studies move into Phase III testing only after a treatment shows promise in Phases I and II. Phase III trials may include hundreds of people around the country.

    

4. In Phase III trials, people are assigned at random to receive either the new treatment or standard treatment.

   Researchers assign patients by chance either to a group taking the new treatment (called the intervention group) or to a group taking standard treatment (called the control group). This method, called randomization, helps avoid bias: having the study's results affected by human choices or other factors not related to the treatments being tested.