Clinical Trials 101
Posted 8/26/2012
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Remember this: Every single thing that is known about effective treatment for breast cancer (or any other cancer) is because of clinical trials. This also means that we all should be forever grateful to the women who participated in trials through the years. To me, this also means that, when it is appropriate, we should consider enrolling in trials ourselves.
People sometimes think that clinical trials are reserved for otherwise hopeless situations. In fact, although surely some trials are designed to test new drugs in the care of advanced disease, many other trials look at some aspect of drugs or care in early stage cancers. For example, I participated in 1993 in a trial that examined timing of radiation and adjuvant chemotherapy. Specifically, I was in the arm of the trial where the first cycle of chemo was given, then, on day 14, six weeks of radiation commenced. During and concurrent to the radiation, chemotherapy continued on the usual schedule. Interestingly enough, I have never learned the outcome of this trials (and that is a whole other topic: should participants be later advised of the results? I think "yes"), but this schedule clearly is not the standard of care, so I am guessing it did not turn out to improve survival and surely was at the cost of greater side effects.
Every week, I am in a number of discussions with women who are considering enrolling in a trial. The range is huge: a trial involving monthly injections to shut down ovaries so a young woman can be given an AI instead of tamoxifen, a trial to test a new drug rather than use a standard chemotherapy for someone with newly diagnosed metastatic disease, etc. Important to know: cancer clinical trials do not have a placebo arm, so a participant never gets "nothing." The question is always whether New Medication X will turn out to be better than Known Medication Y.
This is a good summary from CureToday about clinical trials. Here is the beginning and then a link:
Before Treatment: Understanding Clinical Trials
What you need to know to decide if a clinical trial is right for you
Clinical trials provide data that may prove a new, experimental treatment is better than the standard therapy. They offer a source of hope if you have few options or if you're seeking treatments with the potential to be more beneficial than the standard treatment, one with fewer toxicities or one that is more convenient, such as oral medication or shorter treatment times. You may also be motivated to join a clinical trial to further cancer research and help future cancer patients.
Although an increasing number of investigational cancer drugs are being approved by the Food and Drug Administration each year, the process of drug testing and approval is still lengthy and complex. Most clinical research of a new drug progresses in an orderly series of steps, called phases.
Phase I trials enroll a small number of patients and evaluate how a new treatment should be given (for example, if a new drug is best taken orally or injected into the bloodstream or muscle), how often it should be administered, and the most effective dose with the fewest and least severe side effects. Most patients who enter phase I trials have limited therapeutic options or do not improve with standard therapies. The primary goals of phase I trials are studying side effects and establishing a safe dosage.
Phase II trials continue to test the safety of a treatment, and also begin to evaluate how well it works. These trials are usually limited to a specific cancer that showed benefit with the treatment in earlier trials.
Phase III studies either test an experimental drug, combination of drugs, regimen of radiation therapy, or surgical procedure in comparison with the current standard. Enrollment is often in the thousands across multiple locations, and the treatment is more likely to be effective. Typically, a participant is randomly assigned to the standard treatment or the new treatment (called randomization). Those patients who are not randomized to the experimental treatment will receive the best standard treatment available.
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