Frequently Asked Questions about Clinical Trials

What are Clinical Trials

Clinical trials test the safety and effectiveness of new treatments for cancer in select patients before they can be made widely available. Clinical trials may also evaluate new procedures or new approaches for cancer screening or prevention. Clinical trials involve very detailed and standard procedures for administering the new treatment or procedure and measuring its effects (both good and bad) on patients, compared to existing approaches. Clinical trials enable researchers and clinicians to test promising therapies to improve existing treatment options for patients.

What are the potential benefits and risks of participating in a clinical trial?

Each clinical trial offers potential benefits and risks. Benefits include earlier access to new and novel treatments, the potential for an improved outcome in terms of recurrence and survival and quality of your care, and the chance to participate and add to cancer research. Potential risks include previously unrecognized side effects, and the new intervention may not be as effective and possibly are less effective than the standard approaches.

How are the University of Hawaiʻi Cancer Center Participants Protected?

All University of Hawaiʻi Cancer Center clinical trials are conducted according to strict scientific and ethical principles. Each trial is carefully designed to show how a particular anti-cancer treatment will affect the people who choose to participate. Before they are allowed to begin, all trials are closely reviewed by independent investigators, who ensure that the trials are scientifically sound and that they protect the safety of those who participate. Such reviews are required for all trials whether they are funded by the government, the institution, or a pharmaceutical company. At the University of Hawaiʻi Cancer Center, clinical trials are carefully examined by members of its Protocol Review and Monitoring Committee (PRMC), which focus on the science basis for the study. In addition, clinical trials are reviewed by an Institutional Review Board (IRB), which focuses on patient safety and ethical considerations. Every study must be approved by both the IRB and the PRMC to be implemented and made available to participants.

Several federal government agencies also help ensure that all research is conducted with patient safety in mind. The Office of Human Research Protections (OHRP) is the government's main guardian of patient safety and welfare in clinical trials. It enforces the rules regarding the informed consent process (see below), institutional review boards (IRBs), and the participation of people with special needs in clinical trials, such as children and those with mental disabilities. The National Cancer Institute (NCI), part of the National Institutes of Health (NIH), sponsors many of the cancer clinical trials going on at any one time and conducts its own review of these studies before they are approved. The final authority on clinical trials is the Food and Drug Administration (FDA). The FDA reviews clinical trial information and decides whether the results of a study are safe and effective enough to be approved as a standard treatment.

Each person who enrolls in a clinical trial is informed about all aspects of a study's treatments and tests, including why each portion of the study is necessary, as well as its possible benefits and risks, prior to deciding to participate. The principal investigator (PI) of a study is required to obtain the informed consent (generally written) from all clinical trial participants prior to the start of the trial. During the informed consent process, the researchers (doctors or nurses) will explain the details of the study to participants and respond to any questions and concerns.

Who is Eligible to Participate in a Clinical Trial?

Each study has its own guidelines outlining who is eligible to participate. These are frequently referred to as inclusion and exclusion criteria. The University of Hawaiʻi Cancer Center clinical trials are designed to benefit a broad scope of participants, with a variety of different types of cancer and having received a variety of past treatments.

What Happens Before a Clinical Trial begins?

Clinical trials are initiated only after a number of "pre-clinical" studies are performed and found to suggest that a proposed treatment is likely to be safe and effective. Pre-clinical studies include experiments performed on cancer cells and laboratory animals. Although pre-clinical studies provide a lot of useful information, they do not provide the essential information needed to treat humans. For example, humans and animals may have different reactions to the way a new drug is absorbed and processed. A treatment that works against cancer in a mouse, for instance, may not work in people. If pre-clinical studies are completed and the treatment seems promising, the FDA must give permission to test the new treatment in humans.

How Can I Find Out if There is a Clinical Trial that is Right for Me?

Since each clinical trial has different enrollment criteria, we recommend that you and your physician look over the available clinical trials and contact the Principal Investigator(PI) for further information. Your physician and PI will be able to help you identify if a trial is suitable for you, help explain the trial, and answer any other questions you may have.

Could I receive a placebo?

In treatment trials involving people with cancer, placebos ("dummy" pills or injections that contain no active ingredient) are sometimes used. Many phase III treatment trials are designed to compare a new cancer treatment with the current standard treatment. Sometimes patients are randomly assigned to one group or another. In general, these studies would not use a placebo in place of active treatment. However, it is possible that a placebo could be used in addition to active treatment if the study was evaluating the addition of a new drug with unproven benefit. When no standard treatment exists for a cancer, a study may compare a new treatment with a placebo. In any study that involves a placebo, you will be told about this possibility during informed consent, before you decide whether or not to volunteer for the clinical trial.

What happens during informed consent?

Informed consent is a process in which people learn the key facts about a clinical trial to help them decide whether to participate. This information includes the purpose of the study, the tests and other procedures used in the study, expected benefits and possible risks. Informed consent continues as long as you are in the study. You can change your mind and leave a clinical trial at any time without penalty, even after signing the consent forms.

What is randomization and why is it important?

Randomization is a process that assigns clinical trial participants by chance (like a flip of a coin), rather than by choice, to one of two (or more) groups. In large phase III trials, the "investigational group" receives the new cancer treatment under study. The "control group" or "reference group" receives the standard or currently accepted therapy. In smaller phase II trials, both groups are generally "investigational," but may involve a different dose and/or schedule of the new cancer treatment. Participants are randomly assigned so that bias does not weaken the study results. The goal is to have comparable groups with similar characteristics, such as age, gender, cancer type and cancer stage. In a well-balanced randomized trial, if one group has a better outcome than the other, the investigators may conclude with some confidence that one cancer treatment is better than the other.

Who conducts a clinical trial?

Every clinical trial has a person in charge, usually a doctor, who is called the protocol chair or principal investigator (PI). A clinical trial participant works with a research team led by the principal investigator. Team members may include doctors, nurses, research support staff, and other health professionals. This team monitors participants' care while on the study assuring all aspects of the study are complied with for the benefit of the participant. It is important that everyone work together to assure each individual participant receives the full benefit from their participation in the clinical trial.

Where do the ideas for clinical trials come from?

The idea for a specific clinical trial often originates in the laboratory. Researchers develop a clinical trial protocol (the plan for a trial) after laboratory studies show promise for a new drug or intervention. The first clinical trials of a particular drug or procedure focus on safety (phase I). Later trials focus on whether the drug or procedure is effective (phase II or phase III). See Phases of a Clinical Trial.

Who pays for the costs of a clinical trial?

Before taking part in a clinical trial, it's important to ask what your costs will be.

The study sponsor (such as the government, drug makers or technology companies) often pays for the cost of the new treatment, any special testing or extra doctor visits and research costs, such as data management.
Your medical insurance may be willing to pay for some or all of the costs of your treatment, depending on the type of clinical trial. Check with your provider and treatment team prior to participation in a trial.
Medicare will frequently pay for the routine costs associated with many clinical trials, especially those sponsored by the National Cancer Institute. It would be good to ask your doctor or call your local Medicare provider to find out what costs Medicare will cover.

What happens when a clinical trial is over?

The researchers look carefully at the data collected from the trial before making decisions about the meaning of the findings and further testing. Clinical trial results 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. Once a new approach has been proven safe and effective in a clinical trial, it may be applied as standard practice.

Before you decide to take part in a clinical trial, you should know more about it.

Protocol
Each study has a plan called a protocol. It explains all the facts about the clinical trial, such as:

The reason for doing the study
Who can join the clinical trial
How many people can take part in the trial
Any drugs someone in the trial will be given, the amount they are given, how often they will receive them and how it will be given to them
What medical tests are given and how often
What information will be gathered from the study

Informed Consent
Once you've decided to participate, an Informed Consent form is required.

Each study also has an informed consent process. This is how you learn all the facts about the trial before you decide to take part. You must read these facts in a language you can understand and ask your doctor questions about anything you don't understand. You will get a form that explains:

Why the study is being done
All the facts about the study
Possible risks and benefits
Alternative treatment choices
You have the right to leave the study at any time
Any costs that might not be paid for
How your medical records will be kept private

If you agree to take part in a study, you will sign a consent form. Signing the form means you understand all the facts about the trial. Even after you sign the consent form, you can still choose not to be in the study at any time.

Things To Think About

All Treatments Have Both Benefits And Risks
You may feel overwhelmed by the idea of a clinical trial. Here are some things to think about before you decide to take part.

Possible Benefits of a Clinical Trial

You may get a new treatment that is not yet open to people outside of the study
The research team will monitor your conditions closely
If the treatment being studied is better than the standard treatment, you may be first to benefit
The trial may help scientists learn more about cancer and help people in the future

Possible Risks of a Clinical Trial

The new treatment may not be better than, or even as good as, the standard treatment
New treatments may have side effects that your doctors do not expect
You may have to make more visits to the doctor than if you were getting standard treatment
There may be extra costs for these extra visits
You may need more tests. Some of the tests could be uncomfortable or take a lot of time
Even if a new treatment works in some patients, it may not work for you.
Insurance may not cover all patient care costs

Questions to Ask

It's normal to have a lot of questions. Here are some questions you may want to ask a member of your healthcare team before you decide.

About this trial

Why is this trial being done?
How long will I be in the trial?
What kinds of tests and treatments will I have?
What are the possible side effects or risks of the new treatment?
What are the possible benefits of the treatment?
How will we know when the treatment is working?
Who is my research team?
What information will be shared with me?
How will my information be kept private?
What are my rights?

Costs

Will I have to pay for any of the tests or treatments?
What costs will my health insurance cover?

Daily Life

How could the trial affect my daily life?
How often will I have to come to the hospital or clinic?
Will I have to travel far to take part in the trial?

Comparing Treatment Options

What are my other treatment choices, including standard treatments?
How does the treatment I would get in the trial compare with other treatment choices?

Why are clinical trials important?

Clinical trials have provided marked advances in cure, life extension, care, quality of life and prevention. Personalized care is becoming a reality to fit the specific care to the unique patient at the specific time. For example, a lung cancer trial examines all of the genes related to the cancer in hope of finding a unique gene change that can be attacked by new cancer drugs.

What kinds of cancer clinical trials are offered via the UH Cancer Center?

The UH Cancer Center provides clinical trials through a National Cancer Institute (NCI) Community Oncology Research Program. The Program is funded by the National Cancer Instute. Taking part in the Program has given people in Hawaiʻi, including those in underserved and minority populations, access to the best standard treatment and an opportunity for improved care.

The UH Cancer Center also conducts pharmaceutical-sponsored and investigator-initiated clinical trials. The trials are conducted at various clinical sites in our community. Innovative investigational new drugs under development are investigated in cancer types common to the local population.

Cancer clinical trials include research at three different phases:

Phase 1 clinical trials are studies aimed at determining the safety of an intervention and in the case of a medicine, the optimal dose.
Phase 2 clinical trials are aimed at determining the effectiveness and safety of an intervention.
Phase 3 clinical trials are aimed at determining whether a specific intervention is better, worse, or equally effective as the accepted standard of care. In many cases this type of trial is needed to determine FDA approval.

Does being in a clinical trial require extra of office visits and tests?

It depends on the type of clinical trial. It's true that most trial participants see their doctors more often, and have more blood draws, imaging scans and other tests than cancer patients on conventional treatment. Additional monitoring is important, because it assures the safety of participants enrolled in the study and may shows whether the experimental treatment is working.

How can I be sure I receive a new treatment when I participate in a therapeutic clinical study?

You can't. The purpose of a clinical trial is to find out whether an experimental drug works better than conventional treatment. To ensure valid results, most clinical trials are blinded so neither you nor your doctor will know which type of medicine you receive.

What if I decide I want out of the study?

You have the legal right to withdraw from a clinical trial at any time for any reason. If you decide to withdraw, you will continue to be treated at your clinic site with the best conventional therapies available for your type of cancer.

Who pays for a clinical trial?

Clinical trials are sponsored by federal agencies like the National Institutes of Health or by private pharmaceutical or biotechnology firms. The costs of the clinical trial and the experimental therapy are covered by the sponsor. You and your insurance company are responsible for the cost of tests and procedures that are part of standard medical care. Before enrolling in a clinical trial, it is important to know exactly what will be covered by the trial's sponsor. Ask the doctor or nurse-practitioner in charge of the trial and check with your insurance company before you agree to volunteer.

What factors affect whether a health insurance plan will pay for participation in a clinical trial?

Health insurance companies and managed care companies decide which health care services they will pay for by developing a coverage policy regarding the specific services. In general, the most important factor determining whether something is covered is a health plan's judgment as to whether the service is established or investigational. Health plans usually designate a service as established if there is a certain amount of scientific data to show that it is safe and effective. If the health plan does not think that such data exist in sufficient quantity, the plan may label the service as investigational.

Health care services delivered within the setting of a clinical trial are very often categorized as investigational and not covered. This is because the health plan thinks that the major reason to perform the clinical trial is that there is not enough data to establish the safety and effectiveness of the service being studied. Thus, for some health plans, any mention of the fact that the patient is involved in a clinical trial results in a denial of payment.

If I join a clinical trial, will I have to stop seeing my primary care doctor?

If you volunteer for a clinical trial, your clinical care must be provided by the University of Hawaiʻi Cancer Center doctor who is responsible for patients enrolled in the trial or by their associates. Chances are, your doctor is a UH Cancer Center doctor. If, however, you are traveling to Oahu to be seen, then we may be able to arrange for routine tests to be performed by your community doctor. When participating on a clinical trial, every participant should continue their regular checks with their primary care doctor.

Will I get paid for being a study volunteer?

That depends on the study's sponsor. Some clinical trials reimburse study volunteers for expenses involved in traveling to Oahu, especially if it requires an overnight visit. Some studies offer a small stipend for the time and inconvenience involved in participating in the study. Many clinical studies do not provide compensation for study participants.

If I join a clinical trial, what's in it for me?

Although no one can promise that an experimental treatment will cure your cancer, it may prolong your life and give you more quality time to spend with your family and friends. Even if you don't receive any direct personal benefit, you will have the satisfaction of knowing that you've made an invaluable contribution to research. Many cancer patients who volunteer for clinical trials say they do it to help their children, grandchildren and others who may be diagnosed with cancer in the future.

What kind of information will I get if I want to take part in a trial?

Before you join, a person on the research team will explain why the study is being done, and what will happen during the study. This would be a great time to ask any questions you may have about the study. You will also be given a consent form to read. This form will explain:

The plan for each step in the study
Possible side effects
How the study may affect your daily life

Make sure you ask questions about any part of the consent form you do not understand. If you decide to take part in the study, you will be asked to sign the consent form. Even if you sign the form, you can still change your mind and withdraw from the study at any time.

Where can I find information about University of Hawaiʻi Cancer Center clinical trials?

Start by asking your primary care or cancer care doctor about University of Hawaiʻi Cancer Center clinical trials for your type of cancer. There are many sources of information about clinical trials on the Internet:

The University of Hawaiʻi Cancer Center website includes a list of clinical trials for many diseases and conditions conducted locally in Hawaiʻi.
A federally funded website at clinicaltrials.gov, includes a list of clinical trials in Hawaiʻi as well as the other 49 states.