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An essential part of cancer research, a clinical trial is a study conducted with cancer patients, usually to evaluate a new treatment. Clinical trials may provide possible treatment alternatives to patients who have not had success with standard and approved therapies. You may want to discuss clinical trial options with your physician prior to beginning therapy.
A clinical trial is a research study that tests how well a new medical approach works in people. Each study answers scientific questions and tries to find better ways to prevent, screen for, diagnose, or treat a disease. Most cancer clinical trials study how well different drugs work in people. People who take part in cancer clinical trials have an opportunity to contribute to knowledge of, and progress against, cancer.
An investigational drug is a drug that has not been approved for marketing by the U.S. Food and Drug Administration (FDA) and has not yet been proven to be both safe and effective for the diagnosis, prevention or treatment of a defined disease or condition. An investigational drug can also be a drug that has been approved by the FDA and available to the public, but is being studied for a different disease or condition. Most clinical trials involve an investigational drug in order to study the drug’s safety and effectiveness.
There are four types of clinical trials that are most often conducted when studying investigational drugs:
Phase I trials are the first step in testing an investigational drug in people and are often the first time a drug is given to humans. In these studies, researchers evaluate side effects of the drug, what dose is safe, how the drug should be given (by mouth, injected into a vein, or injected into the muscle), and how often the drug should be administered/taken. Phase I trials usually involve a small number of participants.
Phase II trials study the safety and effectiveness of an investigational drug and evaluate how it affects the human body. Phase II studies usually focus on a particular type of cancer.
Phase III trials continue to study the safety and effectiveness of an investigational drug. These studies often compare an investigational drug with the current standard therapy. In most cases, studies move into Phase III testing only after they have shown promise in Phases I and II. Phase III trials often include large numbers of participants across the country.
Phase IV trials are conducted to further evaluate the long-term safety and effectiveness of a drug. They usually take place after the drug has been approved for standard use. Several hundred to several thousand people may take part in a Phase IV study.
A randomized clinical trial is a study in which the participants are assigned by chance (like picking a number from a hat) to different groups in order to compare different treatments. In cancer clinical trials, participants are often randomized to either a standard treatment group (control group) or the investigational drug group. Randomization mostly occurs in Phase III trials.
A placebo is an inactive substance (often called a “sugar pill”) that looks the same as, and is given the same way as, an active or investigational drug being tested. The effects of the active/investigational drug are compared to the effects of the placebo.
The use of a placebo in cancer clinical trials is very uncommon. Generally, in cancer clinical trials “standard therapy” (best treatment available for a specific cancer) is given in place of a placebo.
In medicine, standard therapy (also called “standard of care” or “best practice”) is the treatment that experts agree is appropriate, accepted, and widely used. Healthcare providers are obligated to provide patients with standard therapy.
In clinical trials, experimental therapy (also called “investigational therapy”) is most often an investigational drug (which could be a new drug, new dose, combination with other drugs, or route of administration) that has undergone basic laboratory testing and needs to be tested in humans to see if it is safe and effective. Experimental therapy has not been approved for marketing by the U.S. Food and Drug Administration (FDA) and is usually not available to the public outside of a clinical trial.
Informed consent is a process by which people learn the important facts about a clinical trial to help them decide whether to participate. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. The research team also provides a written informed consent form that includes details about the study, such as its purpose, duration, required procedures, risks, potential benefits and key contacts. Potential participants can then read over the information, discuss it with family members, and ask the research team questions in order to help them decide whether to participate or not.
People who agree to take part in the clinical trial are asked to sign the informed consent form. The informed consent form is not a contract and signing the informed consent form does not mean participants must stay in the trial. Participants can leave the trial at any time—either before the trial starts or at any time during the trial.
The informed consent process also continues throughout the study. If new benefits, risks, or side effects are discovered during the study, the researchers must inform participants. Participants may be asked to sign additional informed consent forms that contain new information if they want to stay in the study.
Health insurance and managed care providers may or may not cover the treatment costs associated with a clinical trial. It’s important to check with your insurance company before enrolling in a clinical trial to see if they will cover the costs of participation. In many cases, the research team can help you contact a representative of your health plan and find out about coverage. Medicare reimburses patient care costs for its beneficiaries who participate in clinical trials designed to diagnose or treat cancer. Information about Medicare coverage of clinical trials is available at www.medicare.gov or by calling Medicare’s toll-free number for beneficiaries at 1–800–633–4227 (1–800–MEDICARE).
Additional information about clinical trials can be found at:
Clinical trials are typically designed to test investigational drugs for specific conditions, such as a certain type or stage of cancer. Before you may participate in a clinical trial, the research team will ask you many questions to determine your eligibility. If you are interested in participating in a clinical trial, you can also find ongoing cancer trials that may be suitable for you here: Clinical Trials