What is a clinical trial?
A clinical trial is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the safest and most effective way to find new methods of preventing, diagnosing, and treating breast cancer. Cancer clinical trials are divided into four main categories: prevention, screening, treatment, and quality of life.
Are clinical trials just for advanced cancer?
No, although this is a common misconception. Cancer clinical trials exist for all types and stages of cancer. In fact, as researchers learn more about the biology of the different types of breast cancers, there is increased interest in testing new therapies early in the course of the disease in order to see if they can be useful in preventing a recurrence. Cancer clinical trials also look at prevention, screening, and quality of life.
Do I need to live near a big hospital to take part in a clinical trial?
No. Many breast cancer clinical trials are conducted at local hospitals. Some also take place at local cancer clinics and doctor's offices.
Are there clinical trials for people who do not have breast cancer but who are at a higher risk of getting the disease?
Yes, there are. These are called cancer prevention trials. Prevention trials study ways to reduce the risk, or chance, of developing breast cancer. Most breast cancer prevention trials are conducted with healthy people who have not had cancer. Some trials are conducted with people who have had breast cancer and want to prevent the return of cancer or reduce the chance of developing a new type of cancer.
What is a clinical trial protocol?
Clinical trials are conducted according to a detailed plan, called a protocol. The protocol describes what types of patients may enter the study; how they will be assigned to which treatment group; the schedule of tests and procedures; the treatments and dosages that will be used; the length of study; and the outcomes that will be measured. Each person participating in the study must agree to the rules set out by the protocol.
What is the trial "phase?"
Cancer clinical trials are usually conducted in a series of steps, called "phases." Each phase is designed to answer a separate research question. Phase I trials evaluate safety. Phase II trials measure effectiveness. Phase III trials test the new drug against the best existing treatment (often referred to as the standard of care). Phase IV trials are conducted to further evaluate new uses or long-term effects of the treatment.
What does "standard therapy" or "standard of care" mean?
The terms "standard therapy" and "standard of care" are used to describe a treatment that breast cancer experts agree is appropriate, accepted, and widely used. Healthcare providers are obligated to provide patients with standard therapy. The terms "best practice" is also sometimes used.
What is an investigational treatment or procedure?
A treatment or procedure is considered to be investigational if it is being tested in people, but has not yet been approved for routine clinical care by the US Food and Drug Administration (FDA). To receive FDA approval, a series of clinical trials have to have shown that the treatment or procedure is safe and effective for the diagnosis, prevention, or treatment of a defined disease or condition. A treatment or procedure may be approved by the FDA for use in one disease or condition, but be considered investigational in other uses. An investigational treatment may also be referred to as experimental therapy.
What is a control or control group?
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given the standard of care. There is often a misconception that the control group is the group that gets a placebo. However, most cancer trials do not have a placebo group and a placebo is never used when a cancer patient would be put at risk by not having effective therapy for their cancer.
What is a randomized clinical trial?
A randomized clinical trial is a study in which the participants are assigned by chance to separate groups to compare different 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 (called the control group) or to an investigational treatment group or groups. These separate groups are referred to as the "arms" of the trial. In cancer clinical trials, the control group virtually always receives the current standard of care, which is often a treatment that would be a very reasonable choice for you to take if you were not participating in the clinical trial.
Why are patients randomized?
Randomization in a cancer clinical trial gives each patient an equal chance of being assigned to any of the groups. It is one method used to prevent bias in research.
What is a "blinded" trial?
In a blinded trial, participants are randomly assigned to receive the test product, or an existing, approved therapy, and they are not told which treatment they are receiving. In a "double-blinded" study, neither the doctor nor the patient knows who is getting the experimental treatment and who is getting the existing standard of care. It is another method used to prevent bias in research.
What is an "open" trial?
In open, or unblinded trials, both doctors and participants know what treatments are being given. This is the case for trials of surgical procedures or medical devices.
What is a "crossover" trial?
In a crossover trial, each participant gets both treatments being tested. Some participants are assigned at random to receive drug A for say, six weeks, and then, later, drug B, for six weeks. Other participants receive drug B, then, later, drug A.
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. Placebos are sometimes referred to as sugar pills.
Are placebos ever used in breast cancer treatment trials?
Placebos are never used when a cancer patient would be put at risk by not having effective therapy for their cancer. Patients who join cancer clinical trials that are testing a new cancer treatment are given either the standard of care (the best treatment available for their specific cancer), or receive a new treatment being investigated. Only in the rare instance that there is no treatment proven to be effective against a certain type of cancer would patients be randomized to a placebo.
Do any of the trials listed on BCT use placebos?
None of the trials listed on BCT that are for patients who are in need of immediate treatment for their breast cancer use a placebo. You might, however, see a study comparing whether drug A, the current standard of care, works better when combined with drug B that has one group of patients being given drug A and drug B, and the other group being given drug A and a placebo. Note, though, that even though this trial is using a placebo, everyone is still getting the standard of care.
BCT also includes trials that address treatment-related side effects. For example, we might have information about a trial that is studying an investigational drug researchers hope will reduce hot flashes in women taking tamoxifen by randomizing women to either the investigational drug or to a placebo. Researchers are permitted to use a placebo in this type of trial because not getting the treatment does not put the patient at risk, as the trial is looking at an effective treatment for hot flashes, not cancer. In every trial listed on BCT, potential participants are always told before they agree to enter a trial whether placebos are going to be used in the study.
Shouldn't my doctor decide whether I enter a clinical trial?
Your doctor and the rest of your care team are important and can be valuable sources of information about the risks and benefits of taking part in a clinical trial. However, doctors aren't always aware of all the trials that are currently taking place in your region or in other areas of the country. Also some physicians may, for some reason, not support a certain research approach. Ultimately, the only person who can decide if a trial is right for you, is you.
Have cancer patients had good experiences in clinical trials?
Yes, very. Survivors who participated in a study conducted by the Coalition of Cancer Cooperative Groups
reported they were very satisfied with the cancer clinical trial experience. In fact, 92 percent said they had a positive experience, and 91 percent said they would recommend participating in a trial to a friend. In addition, 97 percent of the study participants said they were fully informed on risks and benefits and 96 percent felt they were treated with dignity and respect. Importantly, more than 90 percent of the participants rejected the idea that participants felt like a guinea pig or that they were subjected to more tests and procedures than were necessary.
What is informed consent?
Before you enroll in a trial, you will receive information about the trial from the research staff. This will allow you to learn the key facts about the trial, including any and all risks and benefits, before deciding whether you want to participate. The term "informed consent" is used to describe the process in which you learn this information, ask questions, and sign a written agreement that explains you have been told the purpose of the trial, what treatments are being used, the type of information that will be collected, and how that information will be used. The purpose of informed consent is to protect participants by giving them information that can help them make informed choices about whether to take part in research. It also makes you aware of your rights as a participant.
Can I ask about "results to date?"
Results to date is a term that is often used to describe early results or preliminary data analyses of clinical trial findings. If you are asking about "results to date" from earlier trials, you can frequently find out from the research team what has been learned about the treatment's effectiveness in Phase I or Phase II trials. However, when Phase I or Phase II trials are being conducted at multiple trial sites, the research team may not be able to provide "results to date" as they may not have information about how well the treatment is working for patients at other enrollment sites. In large Phase III trials, interim analyses are often performed and then announced in press releases or at conferences as "results to date." These results may provide some insight into a new treatment's effectiveness. But because they are based on incomplete data, there is no guarantee that the "results to date" will be confirmed in the final data analyses.
Can I ask about side effects the research staff has observed?
The research team should be able to provide you with information about side effects and safety issues that have come up during the trial so far.
What happens if I decide I don't want to stay in the trial?
You are free to change your mind and not participate in the trial at any time for any reason.
What happens when the trial is over?
If the results of a Phase I trial appear promising, the treatment will move into Phase II trials. If those look promising, then it's on to Phase III. If the Phase III trials show that the new treatment is safe and effective, its manufacturer can apply for FDA approval. To receive this approval, the drug manufacturer submits all the data it has collected from Phase I, II, and III trials to the FDA. The FDA reviews the data to assess the treatment's safety and effectiveness. It can choose to reject the manufacturer's application, request more data, or approve the drug. If the drug is approved, the manufacturer can begin marketing it and doctors can start prescribing it.
What happens if I was doing well on the drug? Can I keep using it while the manufacturer seeks FDA approval?
There are some instances in which the FDA allows pharmaceutical companies to distribute drugs prior to approval. This is called compassionate use. To find out if there is a compassionate use program for a drug you are interested in, you will need to contact the company that manufacturers that drug. In some cases, you can get this information from the clinical trial staff. Another good source is CancerActionNow.org
Some of the information on this page was used or adapted with permission from "Learn About Cancer Clinical Trials"
by the Coalition of Cancer Cooperative Groups.