Research Clinical Trials

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.