Questions About Clinical Trial Eligibility, Safety, Preparation, and Others


What Are Clinical Trials?

Clinical trials or research studies are organized tests of medicines and new treatment options. These studies rely on patient volunteers who try the new or changed treatment. Sometimes they are new 'experimental' medicines that have not been approved by the FDA for sale in the United States. Sometimes they are drugs that are currently available for sale but are being tested for a new disease or new doses of the same medicine. Not all doctors choose to do clinical trials. You may enter a trial with your own doctor or with another local doctor who is running a trial you are interested in. If you go to a doctor other than your own, you are always free to go back to your own doctor for your routine medical care. Questions relevant to the trial should always be addressed to the trial doctor for the duration of your trial participation. Any clinical trial participation is always voluntary. In most clinical trials the treatment, office visits, and all tests are free. You may also be given financial compensation for participating in some cases. Your doctor or the study coordinator at your doctor's office should share information regarding the compensation package with you during your first visit.

Q: Where do the ideas for trials come from?

Ideas for clinical trials usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.

Q: Who sponsors clinical trials?

Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.

Q: Are Clinical Trials safe?

Clinical trials are conducted in a healthcare setting (a hospital or clinic) and are typically monitored by a trained healthcare professional. An Institutional Review Board or IRB approves all clinical trials. This is a committee made up of doctors, ethicists, and members of the general public and administrators. This group is held responsible for agreeing that a trial does not represent an unreasonable risk to patients who are participating. Trials are also reviewed by the FDA. Your doctor is responsible for your well being and is the best person to decide whether or not you are eligible to participate in a trial. Whenever you agree to enter a trial you are given the name and telephone number of a contact in your local physician's office who will answer your questions as well as a member of the IRB whom you can contact if you have any doubts.

Q: Why should I participate in a Clinical Trial?

There are many reasons people take part in research studies. Often it gives you a chance to access a medicine that is not available on the market for prescription. If this is the case you should remember that the study is being performed to find out if the drug works and if it is safe. This means that there is some information that is unknown about the drug and it is considered experimental. Often the process of collecting information in the study will allow your doctor to find out more about your disease and the effects it has on you. This may allow you to benefit from better treatment after you have completed a trial. A trial may not benefit you directly, but the information gathered may be of help to other patients with the same condition. Many patients derive satisfaction from knowing that they are part of the effort to potentially reduce the suffering of other people.

Q: How will I know if I am eligible for a Clinical Trial?

All clinical trials have guidelines about who can participate. Using inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. The factors that allow someone to participate in a clinical trial are called "inclusion criteria" and those that disallow someone from participating are called "exclusion criteria". These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

Q: How do patients find a clinical trial at University of Utah Health?

Review all of the clinical trials being conducted at University of Utah Health to see if we are currently enrolling in a trial that might be right for you. At the end of each trial description, you will find information about who to contact if you are interested in enrolling or in receiving more information.

Q: How do patients pay for clinical trials?

The clinical trial sponsor (whether it is the government or a company) may pay for the experimental treatment, special testing, extra doctor visits, travel time, and travel expenses. Some health insurance companies also will cover routine costs in clinical trials. Some health insurance companies will cover the costs of procedures that the patients would have even if they were not in a clinical trial (routine costs). For Medicare patients, routine costs are covered in all Medicare-qualified clinical trials. You will be responsible for any costs not covered by the clinical trial sponsor or your health insurance company. Therefore, be sure to ask questions before deciding to participate, especially to confirm what costs your health insurance company will cover.

Q: What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?

-Plan ahead Ask a friend or relative to come along for support and to hear the responses to the questions -Write down possible questions to ask

Q: What should people consider before participating in a trial?

People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document. -What is the purpose of the study? -Who is going to be in the study? -Why do researchers believe the new treatment being tested may be effective? -Has it been tested before? -What kinds of tests and treatments are involved? -How do the possible risks, side effects, and benefits in the study compare with my current treatment? -How might this trial affect my daily life? -How long will the trial last? -Will hospitalization be required? -Who will pay for the treatment? -Will I be reimbursed for other expenses? -What type of long-term follow up care is part of this study? -How will I know that the treatment is working? Will results of the trials be provided to me? -Who will be in charge of my care?

Q: Does a participant continue to work with a primary health care provider while in a trial?

Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.

Q: What are the different types of clinical trials?

Treatment trials test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy. Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes. Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition. Screening trials test the best way to detect certain diseases or health conditions. Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.

Q: What is a phase in a clinical trial?

Most clinical trials are carried out in steps called phases. Each phase is designed to find different information. Patients may be eligible for studies in different phases depending on their general condition, the type and stage of their condition, and what therapy, if any, they have already had. Patients are seen regularly to determine the effect of the treatment, and treatment is always stopped if side effects become too severe. (Please continue for a description of each of the four phases involved in a clinical trial.)

Q: What are Phase I trials?

Phase I studies are primarily concerned with assessing the drug's safety. This initial phase of testing in humans is done in a small number of healthy volunteers (20 to 100), who are usually paid for participating in the study. The study is designed to determine what happens to the drug in the human body--how it is absorbed, metabolized, and excreted. A phase I study will investigate side effects that occur as dosage levels are increased. This initial phase of testing typically takes several months. About 70 percent of experimental drugs pass this initial phase of testing.

Q: What are Phase II trials?

Once a drug has been shown to be safe, it must be tested for effectiveness. This second phase of testing may last from several months to two years and involve up to several hundred patients. Most phase II studies are randomized trials. One group of patients will receive the experimental drug while a second "control" group will receive a standard treatment or placebo. Often these studies are "blinded"--neither the patients nor the researchers know who is getting the experimental drug. In this manner, the study can provide the pharmaceutical company and the FDA comparative information about the relative safety of the new drug and its effectiveness. Only about one-third of experimental drugs successfully complete both phase I and phase II studies. Safety continues to be evaluated.

Q: What are Phase III trials?

In a phase III study, a drug is tested in several hundred to several thousand patients. This large-scale testing provides the pharmaceutical company and the FDA with a more thorough understanding of the drug's safety, effectiveness, benefits, and the range of possible adverse reactions. Most phase III studies are randomized and blinded trials. Phase III studies typically last several years. Seventy to 90 percent of drugs that enter phase III studies successfully complete this phase of testing. Once a phase III study is successfully completed, a pharmaceutical company can request FDA approval for marketing the drug.

Q: What are Phase IV trials?

In late phase III/phase IV studies, pharmaceutical companies have several objectives: (1) studies often compare a drug with other drugs already in the market; (2) studies are often designed to monitor a drug's long-term safety, effectiveness and impact on a patient's quality of life; and (3) many studies are designed to determine the cost-effectiveness of a drug therapy relative to other traditional and new therapies.

Q: How do drugs and procedures qualify for clinical trials?

Patients who participate in a clinical trial receive drugs or procedures that already have been research in successful laboratory and/or animal studies. Most clinical trials study new drugs or procedures, but some study drugs or procedures that have already received approval by the US Food and Drug Administration.

Q: What happens during a clinical trial?

The clinical trial process depends on the kind of trial being conducted (See What are the different types of clinical trials?) The clinical trial team includes doctors and nurses as well as social workers and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed. Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff.

Q: What are side effects and adverse reactions?

Side effects are any undesired actions or effects of drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

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

Benefits Clinical trials that are well-designed and well-executed are the best treatment approach for eligible participants to: -Play an active role in their own health care -Gain access to new research treatments before they are widely available -Obtain expert medical care at leading health care facilities during the trial -Help others by contributing to medical research Risks There are risks to participating in a clinical trial. There may be unpleasant, serious or even life-threatening side effects to treatment -The treatment may not be effective for the participant. -The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.

Q: How is the safety of the participant protected?

The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants' names will remain secret and will not be mentioned in these reports. Every clinical trial in the US must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.

Q: Can a participant leave a clinical trial after it has begun?

Yes. A participant can leave a clinical trial, at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study.

Q: After the clinical trial ends, what happens to the information and results?

As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Researchers give patients a number or code to protect their identities. When the clinical trial results are published, patients’ names are not used. Medical professionals around the world use information from these presentations and publications to refine their approach to treating clinical problems. In addition, results from clinical trials are used by regulatory agencies (in the US, the Food and Drug Administration) to evaluate whether to grant regulatory approval to a new drug or medical device.

Q: What is a protocol?

A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

Q: What is informed consent?

Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant's native language is not English, translation assistance can be provided. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

Q: What is the Institutional Review Board (IRB)?

A board consisting of health care professionals from the institution where the clinical trial takes place, as well as members of the local community. The board scrutinizes all trial activities including recruitment, advertising, and potential risks. The IRB also makes sure that FDA regulations are being followed in a particular trial.

Q: What is a Clinical Investigator?

A medical researcher in charge of carrying out a clinical trial's protocol. Researchers are usually doctors, nurses, pharmacists, or other health care professionals.

Q: What is the Food and Drug Administration?

A government agency that enforces laws on the manufacture testing and use of drugs and medical devices. All drugs and medical devices must be approved by the FDA before they can be used by the general public.

Q: What is a placebo?

A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the experimental treatment's effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or experimental treatment.

Q: What is a control or control group?

A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.