A: Clinical trials, also called research studies, look at whether a new medicine, treatment or approach is safe and effective in people. They compare the effectiveness of a standard treatment or approach to a new treatment or approach to see which works better or causes fewer side effects.
As a recently diagnosed woman, your doctor may talk with you about treatment clinical trials. Treatment trials are available for all types of breast cancer and may study chemotherapy treatment, hormonal therapy, targeted therapy, vaccine therapy, surgeries, radiation treatments or other therapies.
New treatments are tested in clinical trials only after several years of research in the laboratory and with animals. Those data are reviewed and the federal Food and Drug Administration (FDA) must agree that the treatment is safe before human testing can begin.
A: Clinical trials are conducted in four phases, or steps—each is a separate trial. Data from each phase must be approved by the FDA before the next phase can begin.
- Phase I: First human test step. Measures safety; best dosage and delivery method (by mouth, injected); side effects. Enrolls small number of participants.
- Phase II: Determines effectiveness; watches for side effects. Has 300 or fewer participants.
- Phase III: Compares new treatment to current standard; measures side effects. When phase III is completed, new treatment may receive FDA approval. More than 1,000 participants needed.
- Phase IV: After FDA approval. Monitors effects of long-term use and compares new medicines to others. Many times, there is no phase IV trial.
A: When you take part in a breast cancer clinical trial, your safety is vitally important. The researchers are ethically bound to protect you. What’s more, federal laws specify that the study be closely monitored and that data on side effects and outcomes be reported to safety regulators, government agencies and the sponsor. Those safeguards mean that independent groups are watching and evaluating what is going on.
Today, every clinical trial is regulated by the FDA and an Institutional Review Board, a diverse group of people that reviews and approves trials with your safety in mind. You will also take part in an approval process called informed consent, in which your doctor or nurse will explain the trial to you in detail and give you a paper document with details about the treatment, tests, side effects, possible risks, benefits and costs.
During the trial, a Data Safety and Monitoring Board, an independent group of experts not involved with the trial, reviews the information produced so far on the treatment, side effects and structure of the trial. Should they find anything wrong with the trial, they will stop it. You may also stop participating in the trial at any time.
Taking part in a clinical trial is a serious decision, but doing so may help other women in the long run. All the progress we have made in cancer research comes from people like you who tried the latest, most novel treatments, tests or procedures with the hope of improving outcomes for themselves and generations to follow.
A: No— clinical trials are not just for people who are gravely ill. People with early-stage breast cancer can participate; even people who have never had breast cancer can take part in specially designed clinical trials. Clinical trials simply offer you the option of a treatment that may work as well as the standard treatment but cause fewer side effects, or perhaps be more effective and safe.
When clinical trials compare breast cancer treatments, they compare the standard treatment against the new treatment. Placebos (inactive substances) are rarely used alone in clinical trials for breast cancer treatment unless no other effective treatment exists, so you can feel confident that you will get treatment that meets the current standard of care if there is one available. You will always be told if the study uses a placebo instead of or in addition to active treatment.
A: Some clinical trials are randomized, meaning you and other trial participants will be assigned to one of the specified treatments by random computer selection. Many trials also are double-blinded, which means neither you nor your doctor know which treatment you are receiving.
Randomized, double-blinded trials ensure the results of the study are accurate and have not been influenced by beliefs you or your doctors may have about the outcome of the treatment.
Sometimes trials are unblinded, which means you and your doctor know which treatment you are receiving. If your trial involves medicine, this might also be called an open-label trial.
A: You can learn more about clinical trials from your doctor and nurses, from nonprofit organizations like Living Beyond Breast Cancer and from the Internet. Here are some resources to learn more about clinical trials:
- Visit the National Cancer Institute’s section on finding clinical trials. You can search by state and type of trial.
- Go to LBBC’s Clinical Trials Resource Center, presented in partnership with CenterWatch.com.
- Visit the Coalition of Cancer Cooperative Group’s website cancertrialshelp.org.
- Go to clinicaltrials.gov from the National Institutes of Health for information on understanding clinical trials and finding studies in your community.
- Emerging Med: (877) 601-8601, emergingmed.com
All FAQs reviewed by by Rebecca S.Trupp, RN, OCN, CBPN-IC