The purpose of a clinical trial is to find a better way to prevent, diagnose, or treat cancer. A clinical trial is designed to test if a particular anti-cancer therapy (for example, a new chemotherapy drug) will be better than the treatments currently available.  Clinical trials identify promising cancer treatments and cures, design cancer prevention techniques and new screening methods to help detect cancer. Through cancer clinical trials,  physicians are continuously testing new treatments, new drug combinations, and new uses for existing treatments so that the best care possible can be provided to all cancer patients.

Find out MORE about clinical trials at Cancer.Net.

First and foremost, clinical trials lead to the discovery of new cancer treatments and cures. They are a key step within a comprehensive research process and, typically, they build upon the results of previous trials. Patients can benefit from clinical trials through improved health and quality of life. As physicians gain more and more knowledge through clinical trials,  patient care is revolutionized. Specifically, research trials are helping to improve cancer treatment and prevention options, not only for patients currently seeking treatment, but for generations to come.

Before a new drug can be tested through a clinical trial, a comprehensive protocol, or research plan, is written. Depending on who is doing the research it may be written by a pharmaceutical company, government agency, physician, or research group. The protocol includes details about how the treatment will be administered, how patient safety will be maintained, the type of patients who would be eligible for the treatment, baseline requirements for participants, the purpose of the trial, and the exact procedures that will take place during the trial. The protocol must go through a strict regulatory approval process. Once a protocol is approved, it can begin the clinical trial process.

Clinical trials go through a series of research phases. The initial phase, also called pre-clinical research, tests new therapies in a laboratory. This stage of research is designed to obtain preliminary information regarding how long a drug may stay in a patient’s system, the effectiveness of the drug, and any potential side effects. From there, promising therapies will enter the clinical research phase. Clinical research is broken down into four phases:

• Phase I – This is the first phase in which new treatments are tested in humans. Typically, this type of trial will involve 20-100 healthy volunteers. The volunteers are usually confined to a research center for a period of time so that they can be closely observed. During this phase, the treatments are being tested for many of the same things that were tested during the pre-clinical phase. Initially, however, the dosage levels start much lower than what would be used in laboratory studies. Additional preliminary information is obtained regarding the safety of the drug, how the body metabolizes the drug, which dosage method is best (i.e. by mouth, injection, or IV drip), and the effect, if any, of eating food with the drug.
   
• Phase II – Once the initial safety of the drug is determined in Phase I, the trial moves into Phase II. In this phase, the drug is tested on a larger group of volunteers (20-300). Researchers assess how effective the drug is and at what dosage levels. If the development of a new drug fails, this is usually the phase in which failure occurs.
   
• Phase III – During this phase, the experimental drug is compared with the standard treatment currently on the market. This phase of the trial usually involves multiple research sites and 300-3,000 participants. Although Phase I and II trials typically involve healthy volunteers, Phase III trials allow patients to test out new treatments that may be beneficial to them. Typically, a drug must pass at least two Phase III trials in order to be approved to go on the market.
   
• Phase IV – After a drug/treatment is available on the market, the research does not stop. Phase IV trials are also referred to as “Post Marketing Surveillance Trials.” During this phase, the newly-approved treatments are studied for how they interact with other drugs, how different populations (i.e. pregnant women, children, cardiac patients) respond to the drug, and are continued to be monitored for safety. If a drug fails safety requirements at this point, it can be pulled off the market.

Knowing which phase the research trial is in will help you understand the goals of that trial. In most cases, the results of these clinical trials are published in scientific or medical journals. If you are interested in seeing the results of previous testing for the trial in question, you can ask the doctor, nurse, or research coordinator in charge of your treatment for that information. Keep in mind, however, that it is sometimes many months or even years until the trial results are published.

There are clinical trials for most if not all types of cancer. This site lists trials available to patients in Nebraska;  there is additional clinical trials taking place all throughout the United States and around the world.  If you are not finding a trial on this website to help, you can visit the National Cancer Institute website for additional help.

Before a clinical trial can be offered to a patient as a treatment option, the trial must be approved by the National Cancer Institute (NCI) and the National Institute of Health (NIH). These organizations review a detailed clinical trial protocol that discusses the required tests, treatments, and patient follow-up. They also review what the expected results are and what previous research can do to help predict the outcome of this trial.

Only after this rigorous review and approval by NCI and NIH will cancer clinical trials be opened up to patients. An approval of a trial means it is believed that the treatment is at least as effective, if not better, than the treatments currently in use.

Sites offering clinical trials to patients must work with an Institutional Review Board (IRB).  This board is made up of medical and non-medical individuals who review the clinical trial one last time to ensure minimal harm to patients who agree to participate.  

Plaebo may be used if the drug being studied has yet to prove benefit. Patients are never given less than the standard treatment – the best accepted treatment for the kind of cancer they have – during the trial process. A lot of clinical trials are designed to compare new treatments with standard treatments. Often, patients are randomly assigned to one of the two treatment groups.

Before you decide to participate in a clinical trial, you should speak with a doctor, nurse, or other member of the trial’s research team to see if the trial is right for you. Each trial has specific guidelines on which types of patients could benefit from that particular trial. The research team will help you determine if you are eligible for a trial. This process usually involves reviewing your medical history, recent lab work, and past surgeries or biopsies to see if you are eligible. Sometimes the eligibility process will involve running new tests that are required prior to participation.

When deciding to participate in a clinical trial, consider these possible benefits and risks:

Risks

• New cancer treatments may have risks or side effects unknown to the doctors.
• The experimental treatment may not be effective for you.
• The trial may require more of your time than an off-trial course of treatment. It may require frequent trips to the trial site, additional treatments, hospital stays, or complex dosage requirements.

Benefits

• You can play an active role in your own health care.
• You can receive healthcare from leading physicians in the field of cancer treatment.
• You can gain access to new research treatments before they are widely available.
• You can obtain expert medical care at leading health care facilities during the trial.
• You can help others by contributing to medical research.

If you decide to take part in a trial for which you are eligible, you will be asked to sign a consent form in order to proceed. A member of the research team will review the form with you. The consent form includes detailed information on everything that will be required of you for the trial. It explains the purpose of the trial, the treatment you will be receiving, any procedures that will be conducted, how often you will need to see your doctor, the duration of the trial, and any potential side effects. This process should help you make a final decision on whether to participate in the trial. When reviewing the consent form with a research team member, you should ask any questions that you might have regarding the trial. 

If you decide to participate in the trial, you will either receive the best accepted treatment for the kind of cancer you have (we refer to this as “standard treatment”), or you will receive a treatment that doctors hope will be better than the standard treatment. Patients are randomly assigned to a treatment group. You will not be able to choose the group to which you are assigned. On some studies, you will know right away which treatment you are getting, and on other studies, the exact treatment you are getting will not be revealed to you until after you have completed the trial. As each person responds differently to treatment, it is not known for sure if the new treatment will help you more than the standard treatment.

In general, the clinical trial’s sponsor will pay for anything that is not considered part of “routine patient care.” Any experimental treatment you are taking should be provided by the sponsor. Also, if the trial requires any special testing or extra doctor’s visits, those will also be covered by the sponsor. Procedures that are considered “routine patient care” (i.e. doctor’s visits, x-rays, laboratory tests, non-experimental medication) will be billed to you/your insurance company.

Usually your healthcare plan or Medicare will cover the cost of routine patient care while you are on a trial, but some plans will not. It is important that you talk with your healthcare plan provider to find out if it will cover these costs. Work closely with your doctor and research team to find out what your insurance will cover.

Here are some tips while working with your insurance company (Provided by the National Cancer Institute, www.cancer.gov):

• Find out if there is a social worker or financial counselor associated with the trial who can help you contact your insurance company.
• Keep your own copy of any materials sent to your insurance company.
• Ask your insurance company if they cover the cost of “routine patient care” if you are on a clinical trial.
• If they do cover it, ask if they need to authorize the clinical trial in advance.
• If they need to authorize the trial, be sure to ask them exactly what you need to send to them. You should also send them information from your doctor about why this trial is necessary for you. This may include medical journals that show how patients benefit from this trial, a letter of medical necessity from your doctor, or support letters from patient advocacy groups.
• If an authorization is not required, ask your insurance provider to send you a letter confirming this.
• Every time you speak with your insurance company, make a note of who you are speaking with, the date, and the time.
• You can also try working with your employer’s benefits manager. He or she may be able to help you work with your insurance company.

If you experience an illness or injury while on the trial, any necessary services will be provided to you at the usual charge. There are not funds set aside to compensate you in the event of an injury.

Anyone considering participation in a clinical trial should take some time talk to the research team about your concerns. Sometimes it is helpful to bring a family member or friend with you to be another set of ears and to write down the answers to your questions. Come prepared with a list of questions, but do not hesitate to ask new questions if they arise. Here is a list of questions you may want to ask when deciding to participate in a clinical trial.

Questions about the trial:

• Why is this trial being done?
• Why do researchers think this trial is right for me?
• Who has reviewed and approved this trial?
• How are the trial results and safety of the participants being checked (does this mean regulated?)?
• Who is the sponsor for this trial?
• How many patients have participated so far?

Questions about the possible risks and benefits:

• What are the possible short-term and long-term benefits?
• What are the possible short-term and long-term risks?
• How is my safety protected during the trial?
• What other treatments could I get if I don’t participate in this trial?
• What will happen to my cancer with or without this treatment?
• How do the risks and side effects of this trial compare with the standard treatment?

Questions about participation and care:

• What kinds of tests and treatments are required for this trial?
• Will the treatment hurt and, if so, for how long?
• How do these treatments and tests compare to those I would have outside of this trial?
• Can I still take my regular medications while on this trial?
• How long does the trial last?
• Where will I receive my medical care?
• Who will be in charge of my medical care?
• Will I have to stay in the hospital during the trial? If so, how often and for how long?
• How will I be checked after the trial?

Personal Questions

• How will this trial affect my day to day life?
• Will I lose my hair?
• Can I speak with other people participating in this trial?
• Is there anything I’m not allowed to do while on the trial?
• Who will be allowed to see my medical records?
• How long do I have to make up my mind about joining this trial?

Financial Questions (see also “Who pays for the costs of patient care associated with cancer clinical trials?” above)

• Will my insurance cover my costs for being in the trial?
• Will I have to pay for the treatment drugs or trial procedures?
• Will I have extra costs because of the trial requirements?
• Who can I talk to about what my insurance will cover and the approximate remaining cost?