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.

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 chose, 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.