A Word About Clinical Trials

When you or a loved one is diagnosed with cancer, it's important to know all of the treatment options available to make the best decision about your cancer therapy. Your options may include clinical trials.

Clinical trials are research studies designed to evaluate the safety and effectiveness of new drugs the U.S. Food and Drug Administration (FDA) hasn't yet approved or test new uses for already approved drugs. Cancer clinical trials also test unique approaches to surgery and radiation therapy, and new combinations of existing treatments.

Such trials offer you the opportunity to receive promising new cancer therapies not otherwise available. These new therapies could be more effective, or have fewer side effects, or be more convenient than the current standard treatment. Clinical trials also offer another option if your cancer has become resistant to treatment or has reoccurred. You may want to participate in a trial because you can help further medical research not only for yourself but for other cancer patients as well.

While there are potential benefits to participating in a clinical trial, it is important to realize clinical trials are not designed specifically to treat or cure patients. They are experiments, first and foremost, and there can be risks involved for participants in clinical trials as well.

At any time, thousands of clinical trials are under way looking at the safety and effectiveness of new cancer drugs, treatments, or combinations of existing treatments. Many trials may be within your geographic area.

In the United States, the U.S. Food and Drug Administration (FDA) oversees the conduct of clinical trials. This government agency makes certain that federal regulations governing research are being followed. It also reviews research outcomes and determines whether the new drug will be approved and made available to patients in the United States. Until a drug receives FDA approval, it cannot be sold and the drug company may only provide it to patients through clinical trials. 

How do clinical trials progress?

Before clinical trials with humans can begin, a new medication must undergo extensive testing in the laboratory and with animals. The FDA must also give its permission.

Human clinical trials occur in a long process that has four phases designed to determine specific information, such as risks, safety, and effectiveness compared with a standard drug or therapy. The hope is that the new therapy will be an improvement over the previous standard therapy. Each trial phase is strictly regulated and evaluated, and the safety of the participants is always the top priority. If there are any safety concerns, officials will halt the trial.

Phase I Trials: This phase is probably the most important step in the development of a new drug or therapy. Phase I therapy may produce anticancer effects and a small number of patients may benefit; however, the primary goals of this phase are to determine safety issues, which include:

• The maximum tolerated dose of the treatment.
• The manner in which the drug works in the body.
• The toxic side effects related to different doses.
• Whether toxic side effects are reversible.

Phase I trials usually involve a small number of patients for whom other standard therapies have failed or no known alternative therapy is available. Upon completion of phase I trials, the information that has been gathered is used to begin phase II trials.

Phase II Trials: Phase II trials are designed to determine the effectiveness of the treatment in a specific patient population at the dose and schedules determined in phase I. These trials usually require a slightly higher number of patients than phase I trials. In general, all of the patients participating in a phase II trial will receive the treatment that is being investigated. Drugs or therapies that are shown to be active in phase II trials may become standard treatment or be further evaluated for effectiveness in phase III trials.

Phase III Trials: Phase III trials compare a new drug or therapy with a standard treatment to determine proof of effectiveness. Patients are assigned at random to the new drug or therapy or the best standard treatment. Phase III trials require a large number of patients to measure the statistical validity of the results because patient age, sex, race, and other unknown factors could affect the results. To obtain an adequate number of patients, several physicians (investigators) from different institutions typically participate in phase III clinical trials.

Phase IV Trials: Once the drug or treatment is approved by the FDA and becomes part of standard therapy, the manufacturer of the drug may elect to initiate phase IV trials. This phase includes continued evaluation of the treatment effectiveness and monitoring of side effects as well as implementing studies to evaluate usefulness in different types of cancers.

What type of care do I get in a clinical trial?

When you volunteer for a cancer clinical trial, you are guaranteed to receive either the treatment under investigation or the best available standard treatment for your cancer. Unlike clinical studies in other therapeutic areas, cancer clinical trials do not give a "sugar pill" placebo to anyone.

Patients in cancer clinical trials also receive increased monitoring and attention from nurses and doctors.

How do I qualify for a clinical trial?

Each clinical trial has specific criteria that patients must meet to be included. The study might be looking at only a certain cancer type and stage. Enrollment might also be dependent upon patient age, treatments already received, or present health condition. If you're interested, talk with your physician. Cancer doctors are usually aware of ongoing studies. Very often, they are helping to conduct a trial by having some of their patients participate. Clinical trials are conducted in hospitals, universities, cancer centers, clinics, and doctors' offices.

Are clinical trials safe?

All medical treatments carry risks, even those that have been used by thousands of patients. Not all treatments in clinical trials prove to be better than the standard of care and may produce additional side effects. Yet all clinical trials must meet rigorous guidelines designed to protect you.

You will be monitored very closely during the trial for any potential side effects or problems. Also, the health care professionals involved in the study will explain any known or anticipated risks to you before you commit to the trial. This is part of the informed consent process.   

What is informed consent?

Informed consent is your right as a trial participant. A document will outline the purpose of the study, the exact treatments you will receive, all possible side effects, and your right to withdraw at any point. Discuss the informed consent with your doctor and those who are close to you. Ask the study professionals any questions you may have (see list below). Make sure you understand what costs-including treatment, medical care, travel, and others-are covered by the trial. Check what your insurance covers for trial  expenses that may be your responsibility.

Signing the informed consent form acknowledges that the trial was explained to you and you understand it. However, you can withdraw from a clinical trial at any time, even after signing the form.

Questions to Ask Before Participating a Clinical Trial

Choosing to be in a clinical trial is an important personal decision. Discuss the following questions with your physician or the health care professionals involved in the study to make an informed choice:

• What is the purpose of this trial?
• How will I benefit?
• What are the potential risks?
• What is required of me and my family?
• What kinds of tests and procedures are involved?
• Do I have to change doctors to be in the trial, or can I stay with my own doctor?
• How will I learn the final result of the study?
• Is there any cost to participating in the study or are all costs covered?
• Is there any payment to patients?
• What follow-up is involved once my treatment ends?
• What treatment will I receive if I do not participate in this clinical trial?

Finding a Clinical Trial

The resources listed below offer descriptions of many different trials. They also provide contact information that you can use to enroll in one of these trials.

National Cancer Institute (http://www.cancer.gov/clinicaltrials)—NationalCancer Institute (NCI) is part of the National Institutes of Health. NCI is an agency of the federal government and it provides funding for most cancer clinical trials. The site lists open and closed clinical trials that are funded by NCI, as well as other government agencies, international organizations, and pharmaceutical companies.

TrialCheck (http://www.trialcheck.org/cancertrialshelp/cancertrialshelp.aspx)—TrialCheck is a search tool offered by the Cancer Cooperative Groups, a network of cancer clinical trials specialists. TrialCheck asks you several questions and then finds trials that might be appropriate for you.

CenterWatch Clinical Trials Listing Service  (http://www.centerwatch.com)—This site was developed by a publishing and information services company. It lists more than 41,000 clinical trials sponsored by the federal government and private companies.

Can I get an unapproved drug if I don't qualify for a clinical trial?

Occasionally, a cancer patient who is not eligible for a clinical trial may receive a promising unapproved drug if the patient's doctor, the drug company, and FDA each agree. Talk with your doctor about this.