Frequently Asked Questions

A clinical research study tests the effectiveness of an experimental drug. Studies may also test medical devices or new procedures. For example, a research study could test whether a new drug lowers blood pressure. Some studies compare treatments that have already been approved by the FDA or another regulatory agency. For example, a research study could compare two different medications to see which works faster. Clinical Research Studies are also called "Clinical Trials".

Medical science is always looking for better solutions to treat diseases. Before a new procedure, device, or drug can be used as a treatment it must be tested for effectiveness and safety. Most likely, every doctor-prescribed medication that you use was first tested in a clinical research study. Clinical trials are key to improving medicine for all patients.

Ideas for clinical research studies usually come from researchers. First researchers test new drugs or procedures in the laboratory and in animals. Those with the most promising results are moved into clinical research studies. The studies evaluate a new drug, device, or procedure, its risks, and how well it may or may not work.

All clinical research studies have guidelines about who can participate. Using set rules to include or exclude a person is important to produce reliable results. The factors that allow someone to be in a study are called "inclusion criteria". Factors that disallow someone from participating are called "exclusion criteria". These criteria could be based on age, gender, the type of disease, previous treatment history, and other medical conditions. Before joining a study, a person must qualify for the study. Some research studies seek participants with illnesses. Others need healthy persons. The criteria are used to find appropriate participants. This ensures that researchers will be able to answer the questions they plan to study.

Participants in clinical research studies can gain access to new research procedures, drugs, or devices before they are widely available. They help others by contributing to medical research.

When thinking about joining a study, remember that it is being done to learn what works, what doesn't work, or which thing works best. Unlike standard medical treatment, a clinical research study may involve a drug that may not work. For example, a new drug may not lower blood pressure in people as well as it did in animals. Or the new drug may not work as well as one already on the market. By joining the study you help answer questions about what works and how it works.

Patients considering participating in clinical research should talk about it with their doctors and medical caregivers. They also should ask about the experience of the study team.

Other questions to ask include:

• What is the main purpose of the research study?
• How long will the research study last?
• Who will be in charge of my care?
• Where is the research study being conducted?
• What procedures, drugs, or devices will be used and how?
• How might this research study affect my daily life?
• Will hospitalization be required?
• How will safety be monitored?
• Are there any risks involved?
• What are the possible benefits?
• What are the alternative treatments besides what is being tested in the research study?
• Who is paying for the research study?
• Do I have to pay for any part of the research study?
• Will I be reimbursed for other expenses?
• What happens if I am harmed by the research study?
• Can I choose to receive the procedure, drug, or device, even after the research study ends?

Clinical research studies are all different, but typically,

• You must be willing to follow the directions and instructions of the research team
• You must be willing to attend all the study visits. The number of visits and the length of the visits depend on the type of trial.
• You must take any study medications or complete procedures exactly as directed
• You must always inform the researchers of any symptoms you may be having and any other medications you may be taken. If you are asked about your medical history, you must give full and complete information. This information is important to ensure your safety.

Every study is different. For example, some studies pay for all medications, doctor visits, tests, and procedures. Other studies will only provide some of these things. The cost of other procedures and tests may be billed to you or your insurance company. You should freely discuss what costs are involved with the researchers prior to joining in the study.

Some funding for clinical research comes from federal government agencies such as the National Institutes of Health. Some funding comes from private industry like pharmaceutical and biotech companies. The funder is called the “sponsor”. If research is sponsored by the government, physicians are usually given a sum of money to help cover the costs of conducting the trial.

Sometimes physicians may be paid on a per-patient basis. In either case, the medical care is often provided free to the participant. Participants may also be paid a small fee to participate in a clinical trial.

The clinical research study process depends on the kind of research being done. See What are the different types of clinical research studies? The clinical research study team may include 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 research study, give specific instructions for participating in the research study, monitor the participant carefully during the research study, and may stay in touch with study participants after the research study is completed. Before you agree to participate, the researchers must fully explain what will happen during the trial. If you are considering participating in a trial, you should always be sure you understand all the trial procedures before making your decision.

It depends on the research study. They can vary from days to a number of months, or even years. You will be told the expected length of the research study before you start. This information is discussed with you during the informed consent process.

This will depend on the type of research study. You will always be informed of results that might influence your decision to remain in the research study. If the study is blinded (see glossary), medical information may not be shared until the research study is completed. Because clinical trials can take many years, you may not learn the final results of the study. What you can expect to learn from being in the study will be discussed in the informed consent process.

Just as people can refuse to participate in a study, they may choose to stop participation at any time. Leaving a clinical trial before it is over will never result in any penalty to you. You will have the same rights and benefits that you had before the trial began. However, sometimes leaving a study early can have risks. This should be discussed with you during the informed consent process.

The physician can also withdraw you from the study. This may happen if new risks are found or if you are no longer able to complete study visits.

Just as people can refuse to participate in a study, they may choose to stop participation at any time. Leaving a clinical trial before it is over will never result in any penalty to you. You will have the same rights and benefits that you had before the trial began. However, sometimes leaving a study early can have risks. This should be discussed with you during the informed consent process.

The physician can also withdraw you from the study. This may happen if new risks are found or if you are no longer able to complete study visits.

There are possible risks to consider when joining any clinical research study. You will be informed of known risks or side effects before starting the research study. Risks also are explained in the informed consent document. Make sure you fully understand them. There is always the possibility of unknown risk. If any new side effects or risks are discovered during the research study you will be informed. For instance, you may be given a new informed consent document to consider.

Side effects are any undesired actions or effects of drug or treatment. Side effects are sometimes called adverse effects or adverse reactions. For instance, effects could include a headache, nausea, hair loss, skin irritation, or other physical problems caused by the study procedure, drug, or device. Clinical research studies may help researchers learn about immediate and long-term side effects.

Informed consent is the process of learning the key facts about a clinical research study before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help you decide whether or not to participate, the doctors and nurses involved in the research study explain the details of the study. If your native language is not English, translation assistance should be provided.

Then the research team usually provides an informed consent document that includes details about the study, such as its purpose, how long it lasts, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. Informed consent is not a contract. The only purpose of the informed consent document is to make sure that participants have all the facts before making a decision. Even after you sign an informed consent document, you may withdraw from the trial at any time.

Although you might have talked to the study doctor or study nurse, you might read the consent form and find out something about the clinical research study that you didn't expect. Perhaps it never occurred to you to ask about the risks of a certain procedure. Maybe you didn't know that there would be an overnight study visit. Maybe any one of these details is enough for you to decide that you don't want to join after all. You can decide not to join the study or leave it. There will be no hard feelings. Your medical care at the University of Michigan will not be affected by choosing not to be in a research study.

The research study follows a study plan called a protocol. This plan details what researchers will do in the study. This includes the tests and procedures that will be done to watch your health and well being. For example, you may receive blood tests or receive phone calls from the study nurse to ask how you are feeling.

The federal government regulates most clinical research. An Institutional Review Board also reviews all clinical research at the University of Michigan. The Institutional Review Board is a group that includes doctors, ethicists, and community members. If the Institutional Review Board things the planned study is not safe, they will not allow it to start.

Who monitors a study depends upon the type of research being done. For example, once a clinical research study involving a new drug or device begins, the Food and Drug Administration (FDA) closely monitors its progress.

Every study requires approval and monitoring by a committee called the Institutional Review Board (IRB). The IRB includes doctors, nurses, other scientists and members of the local community. The purpose of this committee is to monitor the study and protect the rights of participants. The IRB continually reviews clinical research study activities, risks, and benefits. If you have concerns about how research is being conducted, call the University of Michigan's IRB office.

The study doctor is directly responsible for your safety. He or she will be watching for any problems you may have.

Intervention research studies develop new drugs, devices, or procedures. They may test new approaches to surgery or radiation therapy.

Pvention research studies look for better ways to prevent disease. These studies may include medicines, vitamins, vaccines, minerals, or lifestyle changes.

Diagnostic research studies are conducted to find better tests or procedures for diagnosing a particular disease or condition.

Screening research studies test the best way to detect certain diseases or health conditions.

Quality of Life research studies (or Supportive Care research studies) explore ways to improve comfort and the quality of life for individuals with a chronic illness.

Clinical research studies are usually conducted in phases. The research studies at each phase have a different purpose and help scientists answer different questions.

In Phase I research studies, a drug or device is being tried in humans for the very first time. These studies usually involve a small group of participants (20-80). The purpose of the studies is usually just to see if the new drug or device is safe. The studies are usually too small to tell whether the drug or device actually works.

In Phase II research studies, the study procedure, drug, or device is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In Phase III research studies, the study procedure, drug, or device is given to a large group of people (1,000-3,000) to confirm its effectiveness, and further monitor side effects. If Phase III trials are successful, the drug may be approved for sale to the general public. Even so, researchers continue to study the drug.

Phase IV research studies collect additional information about the drug's risks, benefits, and optimal use.

To see if a new drug works, researchers must compare a group of people who took the drug with a group of people who did not. This group is called a “control group”. Control groups are used in studies of drugs, devices, and procedures. Usually, participants are assigned to be in the regular group or the control group randomly. Randomly means by chance, like flipping a coin.

A placebo is an inactive substance that is designed to look exactly like real medication or a real medical device. In many clinical research studies, one group of patients will receive the experimental drug, while a second "control" (see glossary) group will receive a placebo. Often the participant, the physician, and the study coordinator are "blind" (see glossary), which means they don't know which group is getting the actual drug and which is receiving the placebo. In a blinded study, the study volunteers are randomly assigned to receive the study drug or a placebo. The "blind" research study method helps ensure reliable results.

Well, you actually can't. The study group you are assigned to is decided randomly, like flipping a coin or drawing a card. Often, a computer program randomizes participants. The study doctor has no influence over which study group you will be in.

A protocol is a study plan on which all clinical research studies 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 research study; the schedule of tests, procedures, medications, and dosages; and the length of the study.