Clinical Trials
Clinical trials are research studies in which people agree to try new therapies (under careful supervision) in order to help doctors identify the best treatments with the fewest side effects. These studies help improve the overall standard of care.
Today, fewer than 5% of breast cancer patients receive treatment for their disease in a clinical trial. Why? One factor is that information about current trials and how to enroll in a trial are often not well understood. You can find information about cancer trials run by The National Cancer Institute (NCI) on the web or by calling the NCI Information Service at 1-800-4-CANCER (1-800-422-6237).
FAQs about Clinical Trials
Note: These questions are reprinted with permission from the Living Beyond Breast Cancer Newsletter.
Why aren't we moving faster in finding effective treatments for breast cancer?
One of the reasons is that advances in treatment depend on a combination of laboratory research and careful evaluation in patients with cancer. New drugs and procedures that look promising in test tubes, and even in mice, have to be evaluated in people. This process is known as a clinical trial. And with so few women with breast cancer participating in clinical trials — only 5% — it takes a long time to collect enough information to decide if a new treatment is better than the current standard treatment.
By comparison, over 90% of children with cancer are treated in a clinical trial setting. Cure rates for children in the early 1970's were one or two out of ten. Today, cure rates are seven out of ten. According to Dr. Anna Meadows, former director of the National Cancer Institute's Office of Cancer Survivorship, results of these clinical trials also revealed how to distinguish between patients who require more aggressive treatment and those who require less aggressive treatment, with fewer side effects.
Many of you have benefited from clinical trials that first compared radical mastectomy to modified radical mastectomy, and then compared modified radical mastectomy to lumpectomy and radiation, plus other studies that discovered Taxol/Taxotere and Herceptin. These advances were made because thousands of women participated in those trials over the years, giving many of you the benefit of these new treatment options.
You want me to be a guinea pig?
Not at all. Clinical trials provide the best treatment available. According to Dr. Larry Norton of Memorial Sloan-Kettering Cancer Center, people who participate in clinical trials do better than the those who don't.
In fact, participating in a clinical trial is one of the best ways to guarantee good care. Clinical trials are designed to compare a new treatment that has shown promise to the best available treatment. Under very strict scientific guidelines, a clinical trial treats large numbers of patients with either the standard therapy or the new therapy.
The idea of being the first to use a new drug or new treatment scares me.
A new drug or treatment approach undergoes very carefully controlled testing in several phases long before it would ever be offered to you. The large clinical trials in which most breast cancer patients can participate are called Phase III trials.
Before Phase III trials, the new treatment has already been tested successfully in many patients in Phase I and Phase II trials. Phase I trials establish how a drug should be administered, how often, and in what dosage. Phase II trials provide preliminary information on how well the drug works, and more information on its risks and benefits.
A new treatment is not even considered for a large-scale Phase III trial unless it looks like an improvement in treatment and seems to be as safe as the standard treatment. And most drugs take years of development and laboratory testing before the three phases of testing in people can begin.
Large clinical trials are done according to very strict guidelines, called "protocols". Protocols describe the goals of the study, who can be in the study, what drugs and tests participants will take, how many patients are to be enrolled, the information that will be collected, and the information people must be given when they are deciding to participate. Before the trial begins, the protocol is reviewed by groups of patient advocates, health care providers, scientists, and statisticians. These reviews are designed to ensure that the study makes sense, that patients' safety is safeguarded, and that upon completion of the study, the results will be valid.
First, patients are assigned at random to either the standard therapy group or to the new treatment group. This is called a randomized trial. If you are assigned to the standard group, you receive what experts view as the best treatment currently available. If you are assigned to the new treatment group, you receive a treatment that some experts think may have significant advantages over the standard.
There's got to be some risks involved in participating in a clinical trial.
Of course. There are well-known risks associated with many standard cancer therapies, which your doctor will explain to you before you embark on a particular course of treatment. And since there has been less experience with a new treatment, not all the risks can be anticipated ahead of time. But the people who design the clinical trial believe that the new treatment will be as good as or better than the current standard care, and that the potential benefits will outweigh the risks. By participating in a trial, you have an opportunity to be one of the first to benefit if the treatment is successful.
If there's something that might be better than the standard treatment, that's what I want.
That's a very common reaction. In fact, that's why thousands of breast cancer patients have undergone high dose chemotherapy (HDC) and bone marrow transplantation (BMT). It seems logical to think that if regular-dose chemo works, then maybe extra-strength chemo doses would work better. And many doctors offered it to their patients outside a clinical trial setting, also believing it was going to be more effective. Given how strongly people believed that HDC/BMT was better, it took a long time to enroll enough patients in the clinical trials that were done to evaluate its benefits, where half the patients received HDC/BMT and half received standard doses of chemotherapy. Four of the five studies reported no survival benefit to HDC/BMT compared to standard lower dose chemotherapy (American Society of Clinical Oncology, May 1999). While these results are somewhat disappointing, at least they provide real data that breast cancer patients can now use to make more informed and objective treatment decisions.
If I participate in a trial, will I know if I'm getting the new treatment or not?
That depends on the study. In some trials, it will be obvious which treatment you're getting. For others, neither you nor your medical team will know. This approach is called a double-blind study, and is used in an effort to eliminate any bias that you or your doctor might have about the effectiveness of one treatment or the other. Bias in this context means any preconceived ideas about a treatment that could affect how you take the medicine, which in turn could influence the study results. The medicine that you receive is labeled with a code that is held in confidence by a small group of people not involved in your treatment. The code is revealed at the end of the study when the results are evaluated. Whenever possible, clinical trials are both randomized and double-blind.
When might I get just a "sugar pill" instead of real medicine?
What you're referring to is called a "placebo." If the goal of a clinical trial is to find out if doing something is better than doing nothing, then some individuals would be given a placebo. An example of this is the recently completed breast cancer prevention trial, in which some women took tamoxifen and others received a placebo. The women enrolled in this study were not breast cancer patients, but rather women who were at higher than average risk for developing breast cancer. The question being asked was, "can a woman's risk of developing breast cancer be lowered by taking tamoxifen?" The alternative to taking tamoxifen in this instance was to take nothing, so half the women in this study received a placebo. A placebo would not be appropriate for a Phase III cancer treatment trial. These trials are designed to answer the question: "Is the new treatment better than the best standard treatment that is currently available?" So some of the patients would receive the new treatment, and others would receive the standard treatment.
There's a lot to remember. Will any of the details be written down, so I can refer to them at home?
It does sound pretty complicated, and yet you should make every effort to understand it. When you review with your doctor the pros and cons of treatment, consider taking a close family member or friend along for support and for help in asking questions, expressing concerns, and recording answers. Some people even take a tape recorder along so they can listen to the conversation again later.
Your doctor or nurse will give you a written consent form that describes the key facts you need to know about the study: its goals, its potential risks and benefits, the medicines and tests you will be given, and alternative methods of treatment. You should take this form home with you, read it carefully, and discuss it with your family, friends, and anyone else you choose, before you make your decision. Feel free to call the doctor or nurse from home if you think of more questions, or if there is something you don't understand. Keep asking questions until you feel you have enough information to make a sound decision.
If you decide to participate in the trial, you will be asked to sign the consent form, indicating you understand what's written there and agree to participate. Be sure to keep a copy of the form so that you can refer back to it at any time.
Once the trial starts, can I change my mind?
Yes, you can withdraw your consent at any time, but it's best to take the time you need before you start treatment to make the best decision possible. Questions may come up during the study that you didn't think about before you agreed to participate. Don't hesitate to ask these questions of any member of the research team to get answers and resolve concerns.
Can I be treated at my doctor's office or local hospital, or do I have to go to a big medical center?
That depends on the study. Most studies are available at community hospitals as well as at the central university hospitals. Exceptions arise if a very scarce piece of equipment is required by the study, or if the trial involves a new specialized technique familiar only to a few doctors.
How do I find out about clinical trials?
- Ask your doctor.
- Call 1-800-4CANCER to reach the Cancer Information Service, a program of the National Cancer Institute. They can give you clinical trial information over the phone, or send it by mail.
- Call a cancer center in your area.
- The National Cancer Institute has a site specifically devoted to clinical trials. One of the choices on that site allows you to search the Physicians Data Query (PDQ) database for trials by cancer type and by geographical area.
- All of the major cancer centers also have web sites. Most of these provide information on clinical trials being carried out at that center. One of the best all-around cancer web sites is the University of Pennsylvania's OncoLink. Its Clinical Trial page has information on current trials. Other cancer centers can be found through the NCI site.
I found out about a clinical trial, but my doctor tells me I'm not eligible. I feel rejected. Why was I excluded?
Clinical trials are planned very carefully to answer certain questions. Part of the process involves enrolling patients who are alike in certain ways. For example, a trial might be designed to answer questions about treating patients who have a particular stage of breast cancer, or who have already received a certain type of chemotherapy. In order for the results to make sense at the end of the study, only those patients who meet these criteria will be enrolled.
Another consideration is patient safety. A new drug may be safe only in people with normal kidney or liver function, for example. So people with poorly functioning kidneys or livers would not be accepted into the study. Also, people who are at a high risk for a drug's side effects are ineligible: e.g., women with a history of serious blood clots should generally avoid taking tamoxifen. People who have had another kind of cancer in the past are usually not accepted because this history can complicate the study's results.
Why hasn't my doctor told me about participating in a clinical trial?
There could be several reasons. Your doctor may be aware of the clinical trials that are enrolling patients, and knows that the trials don't apply to your situation, or knows you are not eligible to participate for other reasons. Or your doctor may not have the necessary staff to do all the extra record-keeping required for participation in a clinical trial.
Don't assume anything. If you are interested in available studies, ask your doctor.
How much will it cost me to participate in a clinical trial? Who pays for the drugs and for all those tests?
Some studies have a sponsor, such as a pharmaceutical company or a biotechnology company, that will pay for the new drug being tested. But rarely do they cover the costs of hospital and doctor visits, x-rays, blood tests, and other procedures. You or your insurance company will most likely be responsible for these. The doctor or nurse who talks to you about the trial should give you that information. If it's not clear to you, be sure to ask. And when thinking about the expense, be sure to factor in any travel costs if you have to go to a center far away from home.
Will my insurance cover the costs?
Whether any of these costs will be covered by your health insurance plan depends on the company that provides your insurance, the particular plan you have, and the study you hope to participate in. If the drugs, procedures, tests, and doctor's visits associated with the clinical trial are about the same as what you would receive if you were not participating in a trial, your insurance company may pay. A recent study indicates that in general there is no difference in the cost of patient care between clinical trials and standard care. You'll need to discuss this with your insurance company BEFORE getting involved in a trial.
So if participating in a clinical trial costs me money, has possible unknown risks, if no one knows if the new treatment is any better than the current standard treatment, and if I won't know which treatment I'm getting until the study is over, then why should I consider a clinical trial?
Participating in a clinical trial can offer you many advantages. There is a chance that the new treatment will shrink or eliminate your tumor, and that it will reduce your symptoms, that it will enhance and extend your life. Be assured that you will be monitored very closely, and that you will receive the best of care during the trial. And there's the gratification of helping others: your participation will advance the treatment possibilities for breast cancer patients who follow you.
Whatever you decide about participating in a clinical trial, make sure your decision is based on as much information as you can gather and that it is the decision that gives you high quality care along with the most peace of mind.