Clinical Studies FAQ

Here is a list of Frequently Asked Questions related to Clinical Research and Clinical Studies… click on the hot links to read more about each topic…

What is a clinical study?

A clinical study is a research study designed to answer specific questions about a disease and its treatment. Clinical studies (also called medical research and research studies) are used to determine whether new drugs or treatments are both safe and effective. Clinical studies are the fastest and safest way to find treatments that work.

Ideas for clinical studies usually come from medical researchers. Once researchers test new therapies or procedures in the laboratory and observe promising results, they begin planning the next step in the testing of the new therapy; this is the clinical study. New therapies are tested on humans only after laboratory and animal studies show encouraging results. Because of the differences between animal and human physiology, these results cannot be confirmed until they are observed successfully at work in human study participants.

What is a clinical protocol?

All clinical studies are based on a set of rules called a protocol. A protocol describes what types of people may participate in the study; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical study, participants are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

What are clinical study phases?

Clinical studies of experimental drugs proceed through four phases:

In Phase I clinical studies, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In Phase II clinical studies, the study drug or treatment 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 studies, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments and collect information that will allow the drug or treatment to be used safely.

Phase IV studies are done after the drug or treatment has been marketed. These studies continue testing the study drug or treatment to collect information about their effect in various populations and any side effects associated with long-term use.

Will I be paid for participating in a clinical study?

Some clinical studies will pay you for joining the study, while others will not. In some programs, researchers will reimburse you for expenses associated with participating in the research. Such expenses may include transportation costs, meals and accommodations.

Can I leave a clinical study after it has begun?

Yes. You can leave a clinical study at any time. If you plan to stop participating, let the research team know why you are leaving the study.

Should I continue working with my primary health care provider if I participate in a study?

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

What questions should I ask?

Some questions you might ask about the research include: Why is this research being done? What is the purpose of the study? Who is sponsoring the study? Who has reviewed and approved this study? Why does the research team think the treatment, drug or medical device will work? Some questions about your participation in the study include: Where is the study site? What kinds of therapies, procedures and/or tests will I have during the study? Will they hurt? If so, for how long? How will the tests in the study compare to tests I would have outside the study? How long will the study last? How often will I have to go to the study site? Who will provide my medical care after the study ends? Will I be able to take my regular medications during the study? What medications, procedures or treatments must I avoid while in the study? What are my responsibilities during the study? Will I have to be in the hospital during the study? Will the study researchers work with my doctor while I am in the study? Can anyone find out that I am participating in a study? Can I talk to other people in the study? Will I be able to find out the results of the study?

How should I prepare for a meeting with a research coordinator or with a doctor?

Plan ahead and write down the questions you want to ask. Ask a friend or relative to come with you for support and to hear the responses to your questions

What should I know before I join a clinical study?

You should know as much as possible about the research study. It is important for you to feel very comfortable asking questions and the staff should answer them in a way you can understand.

What are the risks associated with clinical studies?

The risks associated with clinical study may include: There may be side effects or adverse reactions to medications or treatments. The treatment may not be effective for you. The protocol may require a lot of your time for trips to the study site, treatments, hospital stays or complex dosage requirements.

What are the benefits associated with clinical studies?

Some of the benefits in participating in a clinical study are: Take an active role in your own health care. Gain access to new treatments that are not available to the public. Obtain expert medical care at leading health care facilities during the study. Help others by contributing to medical research. If you qualify you may receive compensation for participating.

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, skin irritation or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

What is a blinded or masked study?

The clinical study process depends on the kind of study you participate in. The team will include doctors and nurses as well as social workers and other health care professionals. They will check your health at the beginning of the study, give you specific instructions for participating in the study, monitor you carefully during the study and stay in touch with you after the study. A blinded or masked study is one in which participants do not know whether they are in the experimental or control group in a research study. Those in the experimental group get the medications or treatments being tested, while those in the control group get a standard treatment or no treatment.

What protections exist for people who participate in clinical studies?

The government has strict guidelines and safeguards to protect people who choose to participate in clinical studies. Every clinical study in the U.S. 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 study 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.

What is a control or control group?

A control is the standard by which experimental observations are evaluated. In many clinical studies, 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.

What is a placebo?

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

What happens during a clinical study?

The clinical study process depends on the kind of study you participate in. The team will include doctors and nurses as well as medical assistants and other health care professionals. They will check your health at the beginning of the study, give you specific instructions for participating in the study, monitor you carefully during the study and stay in touch with you after the study. Some clinical studies involve more tests and doctor visits than you would normally have for your illness or condition. For all types of studies, you will work with a research team. Your participation will be most successful if you follow the protocol carefully and stay in contact with the research staff.

Who sponsors clinical studies?

Clinical studies are sponsored by government agencies: such as the National Institutes of Health (NIH), pharmaceutical companies, individual physician- investigators, health care institutions such as health maintenance organizations (HMOs) and organizations that develop medical devices or equipment. Studies can take place in a variety of locations, such as hospitals, universities, doctors’ offices or community clinics.

Who can participate in a clinical study?

All clinical studies have guidelines about who can get into the program. Guidelines are based on such factors as age, type of disease, medical history and current medical condition. Before you join a clinical study, you must qualify for the study. Some research studies seek volunteers with illnesses or conditions to be studied in the clinical study, while others need healthy volunteers. Healthy volunteers participate in Phase I studies, vaccine studies and studies on research on preventive care for children or adults. The factors that allow you to participate in a clinical study are called inclusion criteria and the factors that keep you from participating are called exclusion criteria. 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.

What is informed consent?

Informed consent is the process of learning the key facts about a clinical study before you decide whether or not to participate. These facts include: Why the research is being done. What the researchers want to accomplish. What will be done during the study and for how long. What risks are involved in the study. What benefits can be expected from the study. What other treatments are available. The fact that you have the right to leave the study at any time. If you are considering joining a clinical study, the research staff will give you informed consent documents that include the details about the study. If English is not your native language, you can ask for the consent documents in languages other than English. Since joining a clinical study is an important decision, you should ask the research team any questions you may have about the study and the consent forms before you make a decision. It is also a good idea to take the consent documents home and discuss them with family members or friends. Talking about your options can help you to feel comfortable with your decision. If you decide to join the clinical study, be sure to ask for a copy of the informed consent documents so you can review them at any time. Remember informed consent is more than signing a form. It is a process that continues through the study. You should feel free to ask the research team questions before, during and after the study. Informed consent continues as long as you are in the study.

What is an Institutional Review Board (IRB)?

An IRB is an independent group or committee whose primary purpose is to review and assure the protection of the safety, rights and welfare of the human subjects in clinical research studies. The people that serve on IRB’s come from diverse backgrounds including physicians, scientists, clergy, nurses, social workers, patient advocates and regular healthcare consumers.