Clinical Trials

To believe in something not yet proved and to underwrite it with our lives; it is the only way we can leave the future open.

— Lillian Smith

For many people, clinical trials are unknown, mysterious and perhaps even somewhat intimidating. This may be because information about people who participate in clinical trials is kept strictly private. Furthermore, it often takes many years and many participants before the results of any clinical trial are presented. The most important reason many patients do not know about clinical trials may be that some doctors do not routinely tell their patients about clinical trials or do not suggest ones that may be appropriate for them.

Clinical trials are the best way to find new ways to detect, treat and hopefully cure breast cancer.

The standard ways to treat breast cancer patients were developed mainly from information gained in clinical trials. When you receive treatment for breast cancer, you are benefiting from the willingness of thousands of women to volunteer for clinical trials or other high-quality research studies. Currently, only about 3 percent of adult cancer patients participate in clinical trials. If more people took part in clinical trials, breakthroughs in breast cancer prevention, diagnosis, treatment and quality of life would happen faster.

Studies show that breast cancer patients who take part in clinical trials tend to do better than those who do not. This may be because clinical trial patients are given excellent care and follow-up, no matter what treatment they get. Also, hospitals that do clinical trials have access to state-of-the-art equipment and follow precise guidelines when giving treatment.

If you have had one or more kinds of treatment, you may still be eligible for certain clinical trials. Some clinical trials test new follow-up treatments that may help prevent your cancer from returning. Others test “second-line” or “third-line” treatments if your first treatment did not work.

In the following section we attempt to take some of the mystery out of clinical trials, explain who can participate, and how to find a clinical trial.

What are clinical trials?
Clinical trials are research studies that are carefully regulated and controlled. Clinical trials are used to find out if new treatments work better, the same, or worse than the usual treatment for the same disease. Sometimes they also look at prevention, screening or quality of life issues. Carefully run clinical trials are the fastest and safest way to find treatments that work. Many drugs which have been tested in clinical trials are now in common use, such as tamoxifen for breast cancer.

Before using any new treatment with patients, scientists perform studies on laboratory animals to find new drugs that are most likely to work well and be safe. If these studies, called “pre-clinical testing” are promising, then the drug can be tested in people in a clinical trial.

How are clinical trials designed?
Clinical trials of new treatments happen in three steps, called “phases.”

Phase I trials are the first studies of treatment in humans. Researchers test the treatment on a small group of people, sometimes just a dozen. The trial looks at how a new drug should be given, how often, and what dose is safe.

Phase II trials build on the knowledge gained from a Phase I trial. The new treatment is given to a larger group of people. This trial continues to test the safety of the drug, and to find out if the new treatment works against a specific cancer.

Phase III trials build on Phase I and II trials. The new treatment is given to an even larger group of people. These trials test the new drug or procedure in comparison to the current standard. This is usually done by assigning patients to the new group or to the standard group at random (“randomization”). Patients cannot choose which group they will be in.

What do clinical trials mean for breast cancer patients and survivors?
For breast cancer patients, participating in a clinical trial could mean:

• studying new drugs (chemotherapy, hormonal therapy, biologic therapies or some complementary or alternative therapies);
• studying current drugs (testing aspects such as dosage amount or frequency to increase effectiveness or decrease side effects);
• studying new surgical methods (i.e., sentinel node biopsy);
• comparing surgical methods (i.e., lumpectomy vs. mastectomy);
• using new equipment or devices (i.e., digital mammography vs. traditional mammography);
• supportive care studies (better ways to manage side effects and relieve pain, help patients cope emotionally, or improve quality of life and well-being in other ways); or
• prevention and early detection studies.

I’m thinking about clinical trials. What are my first steps?
Talk with your doctor. Your primary care doctor, oncologist (cancer doctor), or surgeon, or other healthcare provider may know about a clinical trial for you to consider. They can help you decide whether a clinical trial may be a good option for you.

You do not always need a referral from your doctor to join a clinical trial. If you are eligible to join a trial, the final decision is up to you. However, you should consider the professional opinion of your doctor. He or she may have specific reasons why a particular trial would not be helpful to you.

If your doctor does not discuss clinical trials with you, you can find one by calling the National Cancer Institute (NCI) or looking on the Internet (See the Resources at the end of this chapter). Before you begin looking for a trial, you will need to know the details about your diagnosis (size and type of tumor, location, stage, etc.) to figure out if you qualify for a clinical trial. Your doctor, nurse or social worker can help you with this.

Why should I join a clinical trial?
Clinical trials offer breast cancer patients and survivors a number of reasons to consider joining:

• It may be the best way to be sure you get good treatment. Studies have shown that just being in a clinical trial (no matter what treatment group) increased patients’ chances of surviving.
• You may get a promising, new treatment earlier. Or, you may have a chance of treating your breast cancer if standard treatments have not worked.
• Clinical trials are the best way to find new treatments for breast cancer and, hopefully, one day find a cure for the disease. The more patients who join clinical trials, the more quickly research moves forward and helps other women who develop breast cancer in the future.
• You can play an active role in your own health care by providing input to physicians about side effects and quality of care.

Are there reasons NOT to join clinical trials?
When you take part in a clinical trial, you are followed carefully before, during and after the study. This usually involves regular tests and careful monitoring of how you are feeling. You may need to go to the hospital or research center more often. You should ask how many extra visits may be needed, and weigh how convenient it will be for you.

If you are in a Phase I trial, not much is known about how the drug or therapy will behave in humans. Although you will be watched carefully, there is a chance the treatment may turn out to be harmful, unsafe or have serious side effects.

In Phase II trials, more is known about the treatment, but there is no guarantee that it will work. In Phase III trials, there is some evidence that the therapy may work. However, there is no guarantee that you will be picked to receive the new treatment versus the standard treatment, since patients are “randomized” (assigned by chance) to each group. Also, you may not know which treatment group you are in.

Who can participate?
In order to participate in a clinical trial, a person must meet certain eligibility requirements. Clinical trials have different participation guidelines. These requirements often include age, gender, previous treatment history, nature of the tumor, location of the tumor, other medical conditions, and other factors. If you think clinical trials may be an option for you, find out about each trial’s participation guidelines ahead of time. Some clinical trials may not take patients who have already had a certain drug or treatment.

Participation in clinical trials is entirely voluntary and confidential. You can choose whether or not to participate, and you may withdraw from a study at any time. You should not allow anyone to pressure you into making a decision without time to think about it.

If you decide to enter a clinical trial, you will be asked to read and sign an Informed Consent form. It explains what you will be doing and what can happen to you in the trial. Make sure you read through the form carefully before you sign. Ask questions if you do not understand any part of it. Even if you sign the consent, you can still withdraw from the trial at any time.

How can I find a clinical trial?
There are three main ways to find clinical trials. However, no one method will give you all the information you need about every trial. Use all the tools you can.

• Ask your doctor or healthcare provider. If the doctor doesn’t know of any trials available for you, he or she will at least know that you are interested in finding one and can be on the lookout for new information.
• The National Cancer Institute (NCI) maintains a current list of cancer clinical trials. You can call them or look up trials on the web site (See Resources).
• The Internet is a good source for information on many clinical trials. No single web site will list them all, so you may want to look at more than one. See the Resources at the end of this chapter for web sites to find clinical trials. Be careful to check the sponsors of the trial and verify the credentials.

Before participating in any clinical trials, ask your doctor for his or her opinion. When talking to your doctor, it is helpful to have a “protocol summary” of the trial, which explains what will be done in the trial, how and why, and the locations of the trial.

Will it cost anything to be part of a clinical trial?
Some insurance plans don’t cover these costs once you join a trial. Consult your health plan, if you have one, or see “States that Require Health Plans to Cover Patient Care Costs in Clinical Trials” in the Resources to see if your plan must provide such coverage.

The trial sponsor (which could be the National Cancer Institute, a drug company, or some other organization) usually pays the cost of the drug or treatment being studied. However, you or your insurance plan would be responsible for paying “routine patient care costs.” These are the costs of medical care (e.g., doctor visits, hospital stays, X-rays and other tests) that you would receive whether or not you were in a clinical trial.

North Carolina passed a bill in 2002 that requires all health insurance plans in North Carolina to cover the routine medical costs of a clinical trial. This law covers people diagnosed with a life-threatening condition who choose to enroll in a Phase II or III clinical trial. Not all clinical trials may be covered. Check beforehand with your insurance company to make sure the clinical trials sponsor is on its approved list. The approved list includes:

• National Institutes of Health (NIH)
• NIH cooperative group or center
• U.S. Food and Drug Administration (FDA)
• Centers for Disease Control and Prevention (CDC)
• Agency for Healthcare Research and Quality (AHRQ)
• U.S. Department of Defense (DOD)
• U.S. Department of Veterans Affairs (VA)

Make sure you carefully read and understand your health insurance coverage before you begin a clinical trial. Ask questions of your health plan provider if you are unsure about what is covered. Also, work closely with your doctor to complete the paperwork he or she submits to your health plan. If there are any problems with coverage, the doctor may need to send supporting materials about the new treatment’s or procedure’s safety, benefits and medical appropriateness.

Material for this section was adapted from the National Library of Medicine’s Clinicaltrials.gov, the National Breast Cancer Coalition Fund’s Guide to Quality Breast Cancer Care (www.stopbreastcancer.org/nbccf), and National Cancer Institute publications (www.cancer.gov).

RESOURCES

Organizations

American Cancer Society (ACS)
800.ACS.2345 or 866.228.4327 (TTY)
www.cancer.org
Provides information and services for all forms of cancer, diagnosis, treatment and many other topics. Educational materials available on a wide variety of topics relating to cancer, including clinical trials.

National Breast Cancer Coalition Fund
202.296.7477
www.stopbreastcancer.org
NBCCF publishes the excellent Guide to Quality Breast Cancer Care, available free by calling 866.624.5307 or on www.stopbreastcancer.org/nbccf. The Guide includes helpful information on clinical trials.

National Cancer Institute’s Cancer Information Service
800.4.CANCER (800.422.6237)
www.cancer.gov
One of the best resources available for cancer patients, this government organization provides the toll-free hotline above in English and Spanish for questions about any type of cancer. The web site has an excellent clinical trials section, including how to search for a clinical trial. To access “States That Require Health Plans to Cover Patient Care Costs,” see www.cancer.gov/clinicaltrials/learning/laws-about-clinical-trial-costs.

Books and More

Cancer Clinical Trials: Experimental Treatments and How They Can Help You (Patient-Centered Guides), by Robert K. Finn (1999). Guides cancer patients through the clinical trials process, from understanding clinical trials to financial and other issues.

The Activist Cancer Patient, by Beverly Zakarian (1996). The author, a cancer survivor, describes how to be an advocate for one’s cancer care. Clinical trials are covered, explaining how trials work, describing the risks involved with unproven drugs, and giving tips.

Web Sites

Cancer Trials Help, from the Coalition of National Cancer Cooperative Groups, Inc.
www.cancertrialshelp.org
Has many resources for patients, including a clinical trials finder called “TrialCheck”, information about clinical trials, a patient toolkit, questions to ask and a glossary.

Cancer411.org
http://cancer411.org
This non-profit organization’s web site includes a clinical trials finder.

Centerwatch: Clinical Trials Listing Service
www.centerwatch.com
Comprehensive resource on clinical trials. Lists available clinical trials and has an e-mail notification service that can inform you of new clinical trials for your condition.

Clinical Trials Discussion List
www.acor.org
This public online support group provides information and resources to its members.

ClinicalTrials.gov, from the National Library of Medicine
http://clinicaltrials.gov
This site, a service of the National Institutes of Health, includes a clinical trials finder and resources to learn about clinical trials.

EmergingMed
www.emergingmed.com
Provides a free, confidential, clinical trials matching and referral service for cancer patients.

People Living with Cancer, from the American Society of Clinical Oncology (ASCO)
www.plwc.org
Site offers oncologist-approved information on many types of cancer and treatment, side effects, coping, clinical trials and more.