Clinical Trials

A cancer clinical trial is a medical research study in which people participate as volunteers to test new methods of prevention, screening, diagnosis, or treatment of a disease. A cancer clinical trial can also be referred to as a cancer clinical study. Some cancer patients may fear that enrolling in a cancer trial means they chance receiving no cancer treatment at all, but the reality is that patients in clinical trials receive either the best cancer treatment currently known for their cancer, or a new, and possibly more effective, therapy.

Where do cancer clinical trials take place?

Cancer clinical trials are conducted in doctors’ offices, cancer centers, other medical centers, community hospitals and clinics, and veterans’ and military hospitals in cities and towns across the United States and around the world. Cancer clinical trials may include participants at one or two highly specialized centers or they may involve hundreds of locations at the same time.

What are the phases of a cancer clinical trial?

Cancer clinical trials are usually conducted in a series of steps, called “phases.” Each clinical trial phase is designed to answer a separate research question. Phase I trials evaluate safety, Phase II trials measure effectiveness, and Phase III trials test against best existing cancer treatment. Phase IV trials are conducted to further evaluate new uses or long-term effects of the treatment.

What is a randomized cancer clinical trial?

A randomized cancer clinical trial is a study in which the participants are assigned by chance to separate groups to compare different cancer treatments; neither the researchers nor the participants choose which group the patient is entered into. People are assigned by chance to either a standard treatment group (control group) or investigational treatment arm. In cancer clinical trials the control group invariably receives the standard treatment currently available.

Why are “randomized trials” important in developing new treatments for cancer?

Randomization in a cancer clinical trial gives each cancer patient an equal chance of being assigned to any of the groups. It is one method used to prevent bias in cancer research.

What is standard therapy?

In medicine, standard therapy is a cancer treatment that experts agree is appropriate, accepted, and widely used. Healthcare providers are obligated to provide patients with standard therapy. The terms standard of care or best practice are also used. 

What is an investigational drug?

A drug is considered to be investigational if it is being tested in people, but has not yet been approved for marketing by the US Food and Drug Administration (FDA) by proving to be both safe and effective for the diagnosis, prevention, or treatment of a defined disease or condition. A drug may be approved by the FDA for use in one disease or condition, but be considered investigational in other uses.

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

In medicine, standard therapy is the treatment that experts agree is appropriate, accepted, and widely used. Healthcare providers are obligated to provide patients with standard therapy. Standard therapy is also called standard of care or best practice. In cancer clinical trials, experimental therapy refers to a drug (including a new drug, new dose, combination with other drugs, or route of administration) or procedure that has undergone basic laboratory testing and received approval from the FDA to be tested in human subjects. A drug or procedure may be approved by the FDA for use in one disease or condition, but may be considered experimental in other diseases or conditions. Experimental therapy is also called investigational therapy.

What is a “placebo?”

A placebo is an inactive substance or treatment that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo. The use of placebos in cancer treatment studies is very uncommon and only with patient consent. Generally, in cancer trials “standard therapy” is given in place of a placebo.

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

This misunderstanding is why some cancer patients are reluctant to enter cancer clinical trials. Placebos (also called sugar pills) are rarely used in cancer clinical trials and are never used in place of treatment. Patients who join cancer clinical trials are either given the standard of care, (best treatment available for their specific cancer) or receive a new treatment being investigated. Remember that although you are the only one who can decide whether to take part in a cancer clinical trial, your doctor and the rest of the healthcare team are important and valuable sources of balanced information about the risks and benefits of participation.

How would my cancer clinical trial “experience” compare with my “standard of care” treatment?

An important benefit to participating in a cancer clinical trial is the high level of patient care, as patients in cancer clinical trials are followed more closely by their doctors and nurses. This monitoring is an integral part of the cancer clinical trial process, and undoubtedly provides cancer patients with a high quality experience, whether they are in the treatment or control group.

Are cancer survivors satisfied with their cancer clinical trial experience?

Yes, very. Survivors who participated in a study conducted by the Coalition reported they were very satisfied with the cancer clinical trial experience, with 91% indicating they would recommend participating in a trial to a friend. Additional study results showed:

• 97% felt they were fully informed on risks and benefits
• 96% felt they were treated with dignity and respect
• 92% had a positive experience
• The majority of cancer patients (more than 90%) rejected the idea that participants felt like a guinea pig or that they were subjected to more tests and procedures than were necessary.

Are cancer clinical trials just for advanced cancer?

No, although this is a common misconception. Cancer clinical trials exist for all types and stages of cancer, as well as for cancer prevention.

Are there cancer clinical trials for people who do not have cancer, but who are at a higher risk of the disease due to their family history?

Yes, there are cancer clinical trials for those at higher risk of the disease due to their family history. These are called cancer prevention trials. Prevention trials study ways to reduce the risk, or chance, of developing cancer. Most prevention trials are conducted with healthy people who have not had cancer. Some trials are conducted with people who have had cancer and want to prevent the return of cancer or reduce the chance of developing a new type of cancer.