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Clinical Trials Q & A

Harold Burstein, M.D., Ph.D.Harold Burstein, M.D., Ph.D. Medical Oncologist, Dana-Farber/Brigham and Women’s Cancer Center; Associate Professor of Medicine, Harvard Medical School.

What are clinical trials?

Clinical trials are treatments or other aspects of medical care that are different from accepted “standard” therapies. Clinical trials for those with advanced breast cancer often center on studying new drug treatments. They might also study new doses or schedules of drugs, or new ways of measuring treatment response, or new types of supportive care. A clinical trial could also be a survey of patient attitudes toward treatments, or of patients’ relationships with their families.

The principal distinguishing feature of a clinical trial is that data are collected in a specific, organized way to allow the researcher both to learn from the patient’s treatment and to share the information with others. As a result of clinical trials, doctors understand better how to take care of cancer patients — which treatments work, which do not, and what the side effects of therapy will be.

Who sponsors clinical trials?

The effort to collect and analyze data and deliver new treatments is expensive. Clinical trials, therefore, are usually paid for (sponsored by) large organizations including drug companies, the Federal government through the National Cancer Institute, major cancer centers, or cooperative groups such as the CALGB* or NSABP**.

*CALGB: Cancer and Leukemia Group B, a national clinical research group sponsored by the National Cancer Institute.
** NSABP: National Surgical Adjuvant Breast and Bowel Project, a clinical trials cooperative supported by the National Cancer Institute.

When is the best time for me to try a trial?

There are clinical trials for all stages and treatment moments in breast cancer management. It is possible that any time could be the appropriate time for you to participate in a study; it is also possible that there would never be a right moment for you. Clinical trials are usually written with “eligibility requirements” that specify exactly what kind of patients (tumor stage, prior therapy, general health) the study will focus upon. The trial is written to exclude patients who aren’t at that specific moment in their treatment.

How is a trial different from my ordinary care?

As noted, the goal of the clinical trial is for the investigators to learn something new in such a way that the knowledge can be analyzed and shared. This means that you may be required to undergo extra tests or scans and that your treatment must be delivered exactly as specified in the protocol (the document that provides the details for the study). Thus, clinical trials may be less convenient than ordinary care.

Because a clinical trial differs from ordinary care, you must provide your consent to be treated on the study. You will be asked to sign a written document indicating that you understand why the study is being done, what the possible risks are, and how the data will be analyzed.

On a clinical trial, you may get extra monitoring for safety and toxicity of the drugs. You may meet new members of the health care team, such as research nurses or clinical research assistants who are specially trained to help in the care of patients on study.

What are the benefits & risks of participating in clinical trials?

Participating in clinical trials offers you access to the latest in innovative care. As the slogan goes: you are getting tomorrow’s treatments today. In addition, you may find it gratifying to share in the research process. Not all new treatments are better, however, and some are worse. You are not guaranteed that the trial drug is more effective or safer than other options. Fortunately, seasoned breast cancer experts write most trials, so the trials are intended to align with your clinical needs. You rarely “burn bridges” by participating in trials, even if the new treatments don’t prove helpful for you.

How is my oncologist involved while I’m in a trial?

Usually, the oncologist remains very involved in administering the treatment. If you are on a study at a facility other than your primary cancer center, the new doctors should send regular updates to your primary oncologist.

Why go on a trial and take a risk when there are known treatments that have worked for many with my diagnosis?

Patients with advanced breast cancer face a paradox: there are many known treatments, but we certainly could do better. The search for better treatments motivates doctors and patients to seek out newer options. You should go into a trial only if that trial’s treatment makes sense for you. Known, effective treatments may be a better choice for you than the new therapy offered in a given trial. A well-developed clinical study should be something that complements, not detracts from, your care.

What is the possibility of being in a trial yet getting the actual treatments at my own cancer center?

If the study is not open at your cancer center, then it is very difficult to arrange for study treatment at that site. To start treating patients on a study, a cancer center must go through an elaborate review and approval process to make sure that the center has the correct resources to care for patients on the clinical trial. You can ask your doctor to open more trials or to arrange for a specific study to be opened at your center, but that is a time-and-effort-consuming process.

Can I change my mind and drop out after starting a trial?

You can change your mind at any time.

What do I need to know to make a decision?

First, you should ask your oncologist if the trial is a good idea for you given the details of your situation and the treatments discussed in the study. Then you have to determine whether the logistics of treatment on that study are realistic for you. Most studies are written to be cost neutral; that is, it shouldn’t cost you more to participate in the study than it would to receive standard care. Because trials collect data in a detailed, organized way, you may be asked to take tests or trips to the clinic that would not be needed if your care were not part of the study.

What are the pros and cons of traveling to another site?

Traveling to another site for participation in a trial may enable you to tap into innovative, state-of-the-art treatment not available at your own cancer center. On the other hand, you have to be realistic about the time, effort, and costs for travel and lodging.

What is informed consent?

Informed consent is the requirement that anyone who participates in a clinical trial understand the experimental nature of his or her therapy, the rationale for the study, and the possible risks to the patient. A study participant must sign an informed consent form, usually a 10-to-20-page document that reviews these matters. It is an ethical requirement that anyone who participates in a study understand the nature of the trial and how it differs from ordinary care.

What is compassionate care and use?

Compassionate care is the administration of a new drug (or device) to a patient in desperate need, done outside of a clinical trial but before the drug is commercially available. This sometimes happens when new drugs have generated exciting new data, but the FDA has not yet approved the drug for sale. Compassionate access used to be more common than it is right now. For a variety of reasons, there is little compassionate use of drugs before commercial availability. This is often a frustration to patients.

What do I do if my doctor never mentions trials?

When patients are deciding how best to proceed, it is very helpful for the medical team to outline what the standard choices are, and what, if any, trials are appropriate. If you are never hearing about trials, you might ask your doctor why. Your doctor may not have any trials to offer, or may not think you are right for any at this time. It never hurts to ask.