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About Clinical Trials

What is a clinical trial?

A clinical trial is a research study that tries to find better ways to prevent, screen for, diagnose, or treat a disease.  Clinical trials are an essential part of cancer research.  They may provide possible treatment alternatives to patients who have not had success with standard and approved therapies.  People who take part in cancer clinical trials have an opportunity to contribute to the knowledge of, and progress against, cancer.

What is an investigational drug?

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.  It 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.

What are the phases of clinical trials?

There are 3 types of clinical trials that are most often conducted when studying investigational drugs:

  • First step in testing an investigational drug in people
  • Often the first time a drug is given to humans
  • 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
  • Performed in a small group of patients (less than 40)
  • Test how well a new therapy works in a particular group of patients.
  • Performed in larger groups (40 to 100 patients)
  • The largest (more than 100 patients) and often include at least 2 randomized groups:
    • One group of patients receives the standard of care, which means the best bladder cancer treatment available today
    • The other group receives a new therapy that has shown promise in earlier-phase studies

What is a randomized clinical trial?

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; neither the researchers nor the participants choose which group each participant is entered into. 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 3 trials.

What is a “placebo?” If I enter a cancer clinical trial, is there a chance I will receive a “placebo” instead of a therapy that could treat my cancer?

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.

What is the difference between “standard therapy” and “experimental therapy” in a clinical trial?

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.

What is informed consent?

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. This form is not a contract and signing it 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.

Do insurance companies, Medicare and Medicaid pay for clinical trial participation?

Health insurance and managed care providers may or may not cover the treatment costs associated with a clinical trial. This is why it is important to check with your insurance company before enrolling in a clinical trial to see if they will cover the costs of participating in a specific clinical trial. 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).

Where can I go for more information about clinical trials?

Additional information about clinical trials can be found at:

National Cancer Institute (www.cancer.gov)
American Cancer Society (www.cancer.org)

How do I know if I am eligible to participate in a clinical trial?

Clinical trials are typically designed to test investigational drugs for specific conditions, for example 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 by using BCAN/Emerging Med’s cancer clinical trial matching and referral service.