What are clinical trials?

Clinical trials are research studies in which people help doctors find ways to improve health and cancer care. Each study tries to answer scientific questions and to find better ways to prevent, diagnose, or treat cancer.

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Why are there clinical trials?

A clinical trial is one of the final stages of a long and careful cancer research process. Studies are done with cancer patients to find out whether promising approaches to cancer prevention, diagnosis, and treatment are safe and effective

• Treatment trials test new treatments (like a new cancer drug, new approaches to surgery or radiation therapy, new combinations of treatments, or new methods such as gene therapy). See Taking Part in Clinical Trials: What Cancer Patients Need to Know.

•    Prevention trials test new approaches, such as medicines, vitamins, minerals, or other supplements that doctors believe may lower the risk of a certain type of cancer. These trials look for the best way to prevent cancer in people who have never had cancer or to prevent cancer from coming back or a new cancer occurring in people who have already had cancer.

• Screening trials test the best way to find cancer, especially in its early stages. See Understanding Screening Trials for additional information.

• Quality of Life trials (also called Supportive Care trials) explore ways to improve comfort and quality of life for cancer patients. See Understanding Supportive Care Trials for additional information.

• Genetic Studies are sometimes part of another clinical trial.  The genetics component of the trial may focus on how genetic makeup can affect detection, diagnosis, or response to cancer treatment.
  

• Population- and family-based genetic research studies differ from traditional cancer clinical trials. In these studies, researchers look at tissue or blood samples, generally from families or large groups of people, to find genetic changes that are associated with cancer. People who participate in genetics studies may or may not have cancer, depending on the study. The goal of these studies is to help understand the role of genes in the development of cancer.
  

• The National Cancer Institute (NCI) sponsors many clinical trials through several programs, including the following.

• The Cancer Centers Program provides support for research-oriented institutions, including those that have been designated as NCI Comprehensive or Clinical Cancer Centers for their scientific excellence. More information is available in the NCI fact sheet The National Cancer Institute Cancer Centers Program, which is available at http://www.cancer.gov/cancertopics/factsheet/NCI/cancer-centers on the Internet, or from the NCI’s Cancer Information Service (CIS) at 1–800–4–CANCER (1–800–422–6237).

• The Clinical Trials Cooperative Group Program brings researchers, cancer centers, and doctors together into cooperative groups. These groups work with the NCI to identify important questions in cancer research, and design and conduct clinical trials to answer these questions. Cooperative groups are located throughout the United States and in Canada and Europe. For more information, refer to the fact sheet NCI’s Clinical Trials Cooperative Group Program. This fact sheet is available at http://www.cancer.gov/cancertopics/factsheet/NCI/clinical-trials-cooperative-groupon the Internet, or from the CIS.  
  

• The Cancer Trials Support Unit (CTSU) makes NCI-sponsored phase III treatment trials available to doctors and patients in the United States and Canada. Doctors who are not affiliated with an NCI-sponsored Clinical Trials Cooperative Group (see above) must complete an application and credentialing process to become members of the CTSU’s National Network of Investigators. CTSU members can enroll patients in clinical trials through the program’s Web site, which is located at http://www.ctsu.org on the Internet. General information about the CTSU is also available on the program’s Web site, or by calling 1–888–823–5923.     
  

• The Community Clinical Oncology Program (CCOP) makes clinical trials available in a large number of communities across the United States. Local hospitals throughout the country affiliate with a cancer center or a cooperative group. This affiliation allows doctors to offer people participation in clinical trials more easily, so they do not have to travel long distances or leave their usual caregivers. The Minority-Based Community Clinical Oncology Program is a CCOP that focuses on encouraging minority populations to participate in clinical trials. More information about the CCOP can be found in the NCI fact sheet Community Clinical Oncology Program, which is available at http://www.cancer.gov/cancertopics/factsheet/NCI/CCOP on the Internet. The fact sheet can also be obtained from the CIS.

• The National Institutes of Health (NIH) Clinical Center, a research hospital located in Bethesda, Maryland, is part of the NIH. Trials at the Clinical Center are conducted by the components of the NIH, including the NCI. The NCI fact sheet Cancer Studies at the National Institutes of Health Clinical Center: Questions and Answers has more information about the Clinical Center. This fact sheet is available at http://www.cancer.gov/cancertopics/factsheet/NCI/clinical-center on the Internet, or from the CIS.

• Drug and biotechnology companies sponsor trials of their products. They may conduct these trials in collaboration with universities, the NCI, and/or doctors in private practice.

Most clinical research that involves the testing of a new drug progresses in an orderly series of steps, called phases. This allows researchers to ask and answer questions in a way that results in reliable information about the drug and protects the patients. Clinical trials are usually classified into one of three phases:

• Phase I trials: These first studies in people evaluate how a new drug should be given (by mouth, injected into the blood, or injected into the muscle), how often, and what dose is safe. A phase I trial usually enrolls only a small number of patients, sometimes as few as a dozen.

• Phase II trials: A phase II trial continues to test the safety of the drug, and begins to evaluate how well the new drug works. Phase II studies usually focus on a particular type of cancer.

• Phase III trials: These studies test a new drug, a new combination of drugs, or a new surgical procedure in comparison to the current standard. A participant will usually be assigned to the standard group or the new group at random (called randomization). Phase III trials often enroll large numbers of people and may be conducted at many doctors' offices, clinics, and cancer centers nationwide.

• After a clinical trial is completed, the researchers look carefully at the data collected during the trial before making decisions about the meaning of the findings and further testing. After a phase I or II trial, the researchers decide whether to move on to the next phase, or stop testing the agent or intervention because it was not safe or effective. When a phase III trial is completed, the researchers look at the data and decide whether the results have medical importance.

• The results of clinical trials are often published in peer-reviewed, scientific journals. Peer review is a process by which experts review the report before it is published to make sure the analysis and conclusions are sound. If the results are particularly important, they may be featured by the media and discussed at scientific meetings and by patient advocacy groups before they are published. Once a new approach has been proven safe and effective in a clinical trial, it may become standard practice. (Standard practice is a currently accepted and widely used approach.)

• People can locate the published results of a study by searching for the study’s official name or Protocol ID number in the National Library of Medicine’s PubMed® database. PubMed is an easy-to-use search tool for finding journal articles in the health and medical sciences. PubMed is available at http://www.ncbi.nlm.nih.gov/PubMed on the Internet.
  

Should I take place in the clinical trial?

Only you can make the decision about whether or not to participate in a clinical trial. Before you make your decision, you should:

•  learn as much as possible about your disease and the trials that are available to you. See the Cancer Information section of this Web site for general cancer information. Read the User's Guide for PDQ Clinical Trials Search for guidance on how to find current clinical trials that might be appropriate for you; the PDQ database includes the National Cancer Institute's list of ongoing cancer clinical trials.

• Then, talk about this information and how you feel about it with your doctor and/or nurse, family members and friends to help you determine what is right for you.

What are the potential risks and benefits of clinical trials?

Potential benefits include:

·       Health care provided by leading physicians in the field of cancer research.

·       Access to new drugs and interventions before they are widely available.

·       Close monitoring of your health care and any side effects.

·       A more active role in your own health care.

·       If the approach being studied is found to be helpful, you may be among the first to benefit.

·       An opportunity to make a valuable contribution to cancer research.

The potential risks include:

·       New drugs and procedures may have side effects or risks unknown to the doctors.

·       New drugs and procedures may be ineffective, or less effective, than current approaches.

·       Even if a new approach has benefits, it may not work for you.

How are participants protected?

The government has a system designed to protect human research subjects. Before a government-funded clinical trial can begin, the trial plan (also called a protocol) must be approved. During the trial, review committees make sure that the plan is being followed and participants are being protected.

• See the Protecting Participants in Clinical Trials Digest Page for more information.

•   There is also a related Web-based CME/CEU course called Human Participant Protections: Education for the Research Team.

Regulations require the researchers performing studies to thoroughly inform patients about a study's treatments and tests and their possible benefits and risks before a patient decides whether or not to participate in any study. This process is called informed consent.

What is informed consent?

Informed consent is a process in which you learn the key facts about a clinical trial before you decide whether or not to participate. In addition to talking about these facts with the research doctor or nurse, they will be included in a written consent form that you can take home to read and discuss. The consent form will include details about:

• the study approach

• the intervention given in the trial

•  the possible risks and benefits

• the tests you may have

Don't hesitate to ask questions until you have all the information you need (see Participating in a Trial: Questions to Ask Your Doctor). While informed consent begins before you agree to participate in a trial, you should feel free to ask the health-care team any questions you have at any point. Informed consent continues as long as you are in the study. You can change your mind and leave the study whenever you want -- before the study starts or at any time during the study or follow-up period.

• See A Guide to Understanding Informed Consent for additional information about this important process.

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Could I receive a placebo?

In treatment trials involving people who have cancer, placebos ("dummy" pills that contain no active ingredient) are very rarely used. Many treatment trials are designed to compare a new treatment with a standard treatment, which is the best treatment currently known for a cancer based on results of past research. In these studies, patients are randomly assigned to one group or another. When no standard treatment exists for a cancer, a study may compare a new treatment with a placebo. However, you will be told about this possibility during informed consent, before you decide whether or not to take part in the study.

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What are eligibility criteria and why are they important?

• Each study’s protocol has guidelines for who can or cannot participate in the study. These guidelines, called eligibility criteria, describe characteristics that must be shared by all participants. The criteria differ from study to study. They may include age, gender, medical history, and current health status. Eligibility criteria for treatment studies often require that patients have a particular type and stage of cancer.
  

• Enrolling participants with similar characteristics ensures that the results will be due to what is under study and not other factors. In this way, eligibility criteria help researchers achieve accurate and meaningful results. These criteria also make certain that people who could be made worse by participating in the study are not exposed to the risk. 

If you decide to participate in a clinical trial, you will work with a research team. Team members may include doctors, nurses, social workers, dieticians, and other health care professionals. They will provide your care, monitor your health carefully, and give you specific instructions about the study.

•  Health insurance and managed care providers often do not cover the patient care costs associated with a clinical trial. What they cover varies by health plan and by study. Some health plans do not cover clinical trials if they consider the approach being studied “experimental” or “investigational.” However, if enough data show that the approach is safe and effective, a health plan may consider the approach “established” and cover some or all of the costs. Participants may have difficulty obtaining coverage for costs associated with prevention and screening clinical trials; health plans are currently less likely to have review processes in place for these studies. It may, therefore, be more difficult to get coverage for the costs associated with them. In many cases, it helps to have someone from the research team talk about coverage with representatives of the health plan.

• Health plans may specify other criteria a trial must meet to be covered. The trial might have to be sponsored by a specified organization, be judged “medically necessary” by the health plan, not be significantly more expensive than treatments the health plan considers standard, or focus on types of cancer for which no standard treatments are available. In addition, the facility and medical staff might have to meet the plan’s qualifications for conducting certain procedures, such as bone marrow transplants.

• Many states have passed legislation or developed policies requiring health plans to cover the costs of certain clinical trials. For more information, visit the NCI’s http://www.cancer.gov Web site at http://www.cancer.gov/clinicaltrials/developments/laws-about-clinical-trial-costs on the Internet.

• Federal programs that help pay the costs of care in a clinical trial include those listed below:

• Medicare reimburses patient care costs for its beneficiaries who participate in clinical trials designed to diagnose or treat cancer. Information about Medicare coverage of clinical trials is available at http://medicare.gov on the Internet, or by calling Medicare’s toll-free number for beneficiaries at 1–800–633–4227 (1–800–MEDICARE). The toll-free number for the deaf or hard of hearing is 1–877–486–2048. Also, the NCI fact sheet More Choices in Cancer Care: Information for Beneficiaries on Medicare Coverage of Cancer Clinical Trials is available at http://www.cancer.gov/cancertopics/factsheet/support/medicare on the Internet, or by calling the CIS (see below).

• Beneficiaries of TRICARE, the Department of Defense’s health program, can be reimbursed for the medical costs of participation in NCI-sponsored phase II and phase III cancer prevention (including screening and early detection) and treatment trials. Additional information is available in the NCI fact sheet TRICARE Beneficiaries Can Enter Clinical Trials for Cancer Prevention and Treatment Through a Department of Defense and National Cancer Institute Agreement. This fact sheet can be found at http://www.cancer.gov/cancertopics/factsheet/NCI/TRICARE on the Internet. It is also available from the CIS.

•     The Department of Veterans Affairs (VA) allows eligible veterans to participate in NCI-sponsored prevention, diagnosis, and treatment studies nationwide. All phases and types of NCI-sponsored trials are included. The NCI fact sheet NCI and VA Make It Easier for Veterans To Enter Studies, Get Advanced Care for Cancer has more information. It is available at http://www.cancer.gov/cancertopics/factsheet/NCI/VA-clinical-trialson the Internet, or from the CIS. 

The Study

1. What is the purpose of the study?
2. Why do the researchers think the approach being tested may be effective? Has it been tested before?
3. Who is sponsoring the study?
4. Who has reviewed and approved the study?
5. What are the medical credentials and experience of the researchers and other study personnel?
6.   How are the study results and safety of participants being monitored?
7.   How long will the study last?
8. How will the results be shared?

Possible Risks and Benefits

1. What are the possible short-term benefits?
2. What are the possible long-term benefits?
3. What are the short-term risks, such as side effects?
4. What are the possible long-term risks?
5. What other treatment options are available?
6. How do the possible risks and benefits of the trial compare with those of other options?

Participation and Care

1. What kinds of treatment, medical tests, or procedures will the participants have during the study? How often will they receive the treatments, tests, or procedures?
2. Will treatments, tests, or procedures be painful? If so, how can the pain be controlled?
3. How do the tests in the study compare with what people might receive outside the study?
4. Will participants be able to take their regular medications while in the clinical trial?
5. Where will the participants receive their medical care? Will they be in a hospital? If so, for how long?
6. Who will be in charge of the participants’ care? Will they be able to see their own doctors?
7. How long will participants need to stay in the study? Will there be followup visits after the study?

Personal Issues

1. How could being in the study affect the participants’ daily lives?
2. What support is available for participants and their families?
3. Can potential participants talk with people already enrolled in the study?

  Cost Issues

1. Will participants have to pay for any treatment, tests, or other charges? If so, what will the approximate charges be?
2. What is health insurance likely to cover?
3. Who can help answer questions from the insurance company or health plan?
   

• People interested in taking part in a clinical trial should talk with their health care provider. Information about clinical trials is also available from the CIS (see below). Information specialists at the CIS use PDQ, the NCI’s cancer information database, to identify and provide detailed information about specific ongoing clinical trials. PDQ includes all NCI-funded clinical trials and some studies conducted by independent investigators at hospitals and medical centers in the United States and Europe.

• People also have the option of searching for clinical trials on their own. The clinical trials page of the NCI’s Web site, located at http://www.cancer.gov/clinicaltrials/ on the Internet, provides information about clinical trials and links to PDQ.

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