Clinical trials are an important option in cancer care.

Cancer clinical trials are research studies that involve people who either have or are at risk of getting cancer. The goal of these studies is to find better ways to diagnose, treat, and prevent cancer so that people can live better and longer. The studies also give us more information about reducing cancer risk in healthy people.

Compared with the large number of children with cancer who take part in clinical trials, a very small number of adult patients do so—as few as three to five percent. So there is a large effort to help people understand the importance and benefits of clinical trials. Researchers are also reaching out to include more members of minority groups and the elderly in clinical trials.

Treatment guidelines are developed from the results of clinical trials. Doctors across the country and around the world follow these guidelines so they can deliver the best possible treatment to their patients. Today there are about 12 million cancer survivors in the United States, mainly because of the work done in clinical trials. Every cancer treatment used today is a result of clinical trials and the people who took part in them. That is why it is so important to continue this research.

There are many reasons why people take part in clinical trials, but most hope they will help themselves today and others in the future. These volunteers are doing something very important to improve cancer treatment.

How Do Clinical Trials Work?

A clinical trial often starts with a scientific idea based on results of laboratory research. Researchers who come up with these ideas may work in cancer centers, universities, community clinics, pharmaceutical company labs, or hospitals. Most funding for clinical trials comes from the government’s National Cancer Institute and from the pharmaceutical industry. Together, doctors and researchers design the studies and how they will work.

Clinical trials that test new drugs or other treatments are done in phases. Each phase has a different purpose and helps researchers answer different questions. If a new drug or treatment does not seem promising in the early phases, the research can be stopped. Most clinical trials are divided into one of three phases:

In phase I trials, researchers try to find the smallest dose of a new drug or drug combination that will be effective. Phase I trials usually include a small number of people (15 to 30). This type of study also looks at any side effects that might occur. These trials usually take place in research institutions, such as large teaching hospitals and cancer centers, where patients can be closely watched. In phase I trials, doctors may also study other types of treatment such as radiation.

In phase II trials, the new drug or treatment is given to a larger group of people (but generally less than 100) to learn more about its effectiveness and safety. Like phase I studies, phase II trials usually take place at research institutions. In some phase II studies, patients may be randomized. This means that patients in the trial are chosen by chance to get either the new treatment being studied or a standard treatment that is already being used. In order to avoid influencing the results, patients (and often their doctors) do not know which group they are in. Randomized clinical trials are considered the most reliable way of learning which treatments work best.

In phase III trials, the drug or treatment being studied is given to even larger groups of people (1,000 to 3,000). During this phase, researchers are able to:

  • Confirm how well the treatment works
  • Learn about side effects they might not have seen during earlier phases with smaller groups
  • Compare the new treatment with standard treatments
  • Collect information that will allow the new drug or treatment to be used safely

Patients in phase III trials are randomized, as described above. In cancer clinical trials, patients are generally not given a placebo (a look-alike pill or liquid that contains no active ingredient). A placebo is only used when there is no standard treatment against which a new treatment can be compared. If a clinical trial uses a placebo, people who are thinking about taking part in the trial are told in advance that they could get a placebo.

Phase III studies are often carried out in the community by local doctors in private practice, in community hospitals, or in special cancer centers. Because phase III clinical trials involve large numbers of people, doctors can better understand how a treatment will work in people with many different health factors.

In phase IV trials, researchers study drugs and other types of treatments after they have been approved by the U.S. Food and Drug Administration (FDA) and sold to the public. Phase IV trials are designed to learn more about the treatment’s risks and benefits and the best way to use it. These studies help doctors understand how safe and useful the treatment will be over the long term.

What Are the Benefits of Joining a Clinical Trial?

Being in a clinical trial lets you play an active role in your health care. Your own cancer doctor will still care for you, but in a clinical trial you also receive close observation by cancer experts. Because a clinical trial must follow a strict protocol, or plan, people in clinical trials are very closely observed. This is the only way researchers can be sure that the information they get from the study is accurate and complete.

You may also receive access to new cancer treatments and techniques (such as a better way of delivering treatment) before they are widely available. Doctors perform clinical trials because they believe the drugs or techniques they are studying could be more effective or safer than the treatments they’re using now. But doctors also have to prove it scientifically. Only with this proof will the FDA approve the new treatments or techniques for other people with the same cancers.

As with all medical treatments, there may be risks associated with the treatments used in clinical trials. These risks include side effects and the possibility that the new treatment may not work well for you. Taking part in a clinical trial may also take up more of your time and attention than you expected. There may be trips to the hospital or clinic where the study is being done, hospital stays, or a complex schedule for taking medications. Before you agree to enter a study, your doctor or nurse will fully explain the possible risks and make sure you understand them, as well as the things that you are expected to do.

What Rights and Protections Do I Have in a Clinical Trial?

People who take part in clinical trials have both rights and protections to make sure their privacy and well-being are taken care of. One of the most important protections is called informed consent. This is a process to ensure that the participant understands all aspects of the study, including the risks and benefits. A written consent document is signed by a patient, stating that he or she has entered the trial of his or her own free will without being pressured. As a patient in a clinical trial, you must be able to follow study instructions, but you always have the right to leave a clinical trial at any time for any reason.

Besides informed consent, you will be asked to sign another form called a HIPAA Authorization. HIPAA stands for Health Insurance Portability and Accountability Act. This form allows doctors to use your health information as part of the report on the study, without using your name or other personal details.

Each research institution, hospital, or cancer center that conducts clinical trials has a committee called an Institutional Review Board (IRB). An IRB includes doctors, nurses, lawyers, and people like yourself. The IRB reviews each clinical trial to make sure it is safe and ethical, in order to protect the people who take part. They review the study on a regular basis to be sure it is being carried out properly.

If you have concerns about any part of being in a clinical trial, you may be able to speak with a patient representative at the institution where the trial is being conducted. The name and contact information for this person are usually included on the informed consent document. If a patient representative is not listed, ask if there is someone else you can speak with, such as a nurse or social worker, to answer any questions you might have.

Weighing the risks and benefits of a clinical trial is a very personal decision. There is no right or wrong answer. Only you can decide if a trial is right for you.

Frequently Asked Questions

Q. How do I find information about clinical trials?

A. Visit the website of the National Cancer Institute (NCI), www.cancer.gov, or call the NCI at 1-800-4-CANCER and speak with an Information Specialist about clinical trials. The National Cancer Institute recommends that you talk with your doctor about the right clinical trial for you.

You should know that all clinical trials have guidelines about who can take part. These guidelines are used to:

  • Identify the right patients for the trial.
  • Keep all participants safe.
  • Make sure that researchers will be able to answer the questions they plan to study.

Your doctor can advise you about whether you qualify for a study and help you contact the researchers involved.

Before you meet with your doctor for that discussion, do your homework by searching online or calling the toll-free numbers listed in the “Clinical Trial Resources” section on the right. Your doctor may not know about newer studies that are available for you. Some people with cancer have placed their names in databases for future clinical trials. If you let your doctor know that you would like to hear about new clinical trials, he or she can help you stay informed.

Q. I’m in a clinical trial now and have experienced side effects from the drug I’m taking. Do the doctors need to stick with the plan of the clinical trial?

A. All doctors in a clinical trial must follow protocols—that is, the plan for what treatment will be studied and how it will be given to patients. Safety is always the most important part of any study. That’s why the protocols include guidelines about how best to help patients with expected side effects. It is very important to let your health care team know about any side effects you are having. This is the only way they will be able to help you manage your symptoms so you can feel better. Knowing what side effects the drug causes is important for research purposes. You are helping yourself and the researchers by being as honest as possible.

Q. I live in a rural area. I can’t convince my doctor to sign me up for a clinical trial. What should I do?

A. Living in a rural area may make it more challenging to get the kind of care that you want. Often, there are not as many cancer specialists (oncologists) in rural areas as there are in a city or the suburbs. But there are other options you can explore. Perhaps there is another doctor in a nearby town where you could take part in a clinical trial.

Most states have at least one comprehensive cancer center where clinical trials are underway. (See the list of resources on the right for information on finding a center near you.) A comprehensive cancer center is a large hospital that specializes in caring for people with all types of cancer, conducting clinical trials, and teaching the public about cancer. The National Cancer Institute selects these centers; there are 66 centers in 33 states. These centers may be far from your home, but sometimes the sponsor of the trial or organizations such as CancerCare may be able to help you with travel expenses.

Q. I’m 72 years old. Can I still take part in clinical trials?

A. We used to think that because of their age, older adults would not be able to tolerate strong treatments. We now know that a person’s overall health, rather than his or her age, is more important when it comes to clinical trials. Sometimes a clinical trial might be studying only patients in a certain age range. But in general, age alone should not prevent you from entering a trial. Researchers are encouraging older adults to take part in clinical trials so that seniors can receive the treatment benefits. Doctors also want to be sure that the medicines are, in fact, safe and effective for older people with cancer. Clinical trials are the best way to find out.

Q. I’ve been told I can’t be in some clinical trials because I have high blood pressure. Can trials exclude people with certain medical conditions?

A. Yes, sometimes patients with certain conditions are excluded. The safety of people taking part in clinical trials comes first. If the researchers believe that high blood pressure or some other medical condition puts a person at risk, they will not enroll him or her. However, you should talk to your doctor about your particular case. Each trial is different, and sometimes these decisions are made on a case-by-case basis. So you still might qualify for some clinical trials.

Q. I’m in treatment and doing well. Should I enter a clinical trial?

A. When treatment is going well, a person with cancer may still wonder if he or she should join a clinical trial. Talk with your doctor about whether the treatment you would receive in a clinical trial may improve upon the treatment you’re already getting. Some trials study new treatments that may prevent the return of cancer after your initial treatment is complete. Your doctor may suggest you take part in one of these clinical trials in the future.

CancerCare Can Help

When you are being treated for cancer and are thinking about entering a clinical trial, you may have many concerns about the process. It’s perfectly normal to feel confused or nervous about clinical trials. But the more you learn about what’s involved and what to expect, the better you’ll feel about your decision. Help is available to you as you consider your options. Your health care team, family members, and friends will likely be your main source of support. CancerCare is also here to help. We offer the following free resources:

Counseling Often, when people are thinking about enrolling in a clinical trial, they need someone to talk with who will help them sort through the emotional and practical concerns that come up. Oncology social workers provide emotional support, help you find ways to stick with treatment and cope with side effects, and guide you to resources. CancerCare offers free counseling from professional oncology social workers on staff.

Support groups Talking with other people who have taken part in clinical trials can help reduce the feeling that you are going through it alone. These groups provide reassurance, suggestions, insight—a safe and supportive place where people can share similar concerns. At CancerCare, people with cancer and their families take part in support groups in person, online, or on the telephone.

Connect Education Workshops In these workshops, you can hear about the latest treatments and clinical trials directly from leading medical experts in one-hour presentations. You can listen live by telephone or you can download podcasts of past workshops from our website.

Publications Free booklets and fact sheets from CancerCare provide up-to-date, easy-to-read information about the latest on clinical trials, treatments, managing side effects, and coping with cancer.

Financial help For those who qualify, CancerCare can provide financial assistance to help with some costs that might arise during a clinical trial, such as transportation and child care. Social workers and case managers are knowledgeable about financial issues and will work closely with you to get you the help you need.

Referrals to resources CancerCare can help you learn about other organizations in your community and nationwide that can assist you in finding a clinical trial that is right for you.

To learn more about how we help, call 1-800-813-HOPE (4673) or visit www.cancercare.org.

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This e-booklet was made possible by an educational grant from GlaxoSmithKline Oncology.

Last updated January 23, 2012

The information presented in this publication is provided for your general information only. It is not intended as medical advice and should not be relied upon as a substitute for consultations with qualified health professionals who are aware of your specific situation. We encourage you to take information and questions back to your individual health care provider as a way of creating a dialogue and partnership about your cancer and your treatment.