Before agreeing to participate in a clinical trial, you should have a very good understanding of the benefits and risks of doing so. The following FAQ might be helpful to you in making your decision. The US Government also has a good website that might help answer your clinical research questions regarding medical studies (http://clinicaltrials.gov/ct2/info/understand).
You may also want to talk to your own health care provider, the investigator or a study coordinator of the particular study. You may call RMCR directly at (720) 776-0092 if you have any questions or concerns, or would like to request medical study information.
WHAT IS CLINICAL RESEARCH?
Clinical research involves testing an investigational drug or device in humans. Each clinical trial must use a protocol that has been written according to government regulations and that is also approved by the company sponsoring the study. An investigational drug may be:
- a drug that has not yet been approved for marketing (i.e., a drug that is not allowed to be sold)
- a drug that is currently on the market but that is being studied for a non-approved indication or is being studied in a new route of administration, strength, or formulation;
- a drug that is being sold on the market by one company but that is being considered for in-license or acquisition by another company;
a drug that contains known and established ingredients but has not yet been marketed by the company;
- a drug that is currently on the market but is being studied in a population for which the drug has not yet been approved, such as in children or in elderly patients.
Before an investigational drug or device can be studied in humans, its safety and effectiveness is carefully studied in the laboratory. However, this early research cannot predict how safe and effective the drug will be in humans. With any new treatment there may be benefits as well as known and unknown risks. Clinical research trials are conducted to determine whether promising new treatments will be safe and effective for patients.
WHAT ARE THE PHASES OF CLINICAL RESEARCH?
Phase I studies involve testing the investigational drug in a small number of healthy adult volunteers. The sole objective of these studies is safety. These studies are designed to determine how the drug is metabolized by the body and what pharmacologic actions the drug has. One objective of these studies is to determine the best way to administer the drugs (i.e., the preferred route).
Phase II studies help determine how safe and effective a drug is, common short-term side effects of the drug, pharmacokinetic effects of the drug (i.e., the amount of drug that gets into the bloodstream, how quickly it gets there, and how long it stays there) and pharmacodynamic effects of the drug (i.e., the effects of the drug on the body). These studies involve a larger group of patients (e.g., 100 to 200 patients) and are usually conducted in patients who have the medical condition for which the drug is being studied. These studies are used to identify the dose range of the drug that will be tested in subsequent larger studies (i.e., Phase III studies).
Phase III studies provide additional information about safety and efficacy when the drug is used in the way it is intended to be marketed. These studies are initiated only after earlier studies have demonstrated that there is an acceptable risk to benefit profile (i.e., the risk of taking the drug is outweighed by the potential benefit of the drug to the patient). These large studies are usually conducted at many different study centers; up to 10 to 30 study centers and between 500 and 5,000 patients may be included. These studies are used to confirm the indication and best dosage for the drug.
Phase IV studies are conducted after the drug is available for sale (i.e., post-marketing studies) and they evaluate how safe and effective the drug is when exposed to the “real” population. These studies evaluate parameters such as different formulations, dosages, treatment durations, and drug interactions. The Food and Drug Administration (FDA) may require a company to conduct certain Phase IV studies as a provision for product approval. These studies may include new age groups, various races and other types of patients. These studies are useful for detecting and defining previously unknown or inadequately quantified adverse events and related risk factors. The size of these studies is broad and can range from less than 100 to over 20,000 patients.
WHO SPONSORS CLINICAL TRIALS AND WHERE DO THEY TAKE PLACE?
WHAT IS A PRINCIPAL INVESTIGATOR?
WHAT IS A STUDY COORDINATOR?
WHY SHOULD I PARTICIPATE IN A CLINICAL TRIAL?
People choose to take part in clinical trials for many reasons. Usually, they hope for benefits for themselves such as hope for a cure of disease, a longer time to live, an increased quality of life, or a way to decrease or eliminate pain. Some people choose to participate because of the financial compensation they receive. Others may want to contribute to a research effort that may help others.
People who participate in clinical trials are among the first to receive new research treatments before they become widely available. Sometimes these people have the first chance to benefit from a cutting-edge treatment. However, there is no way to know whether an investigational treatment will be effective for a patient. (Even standard treatments, although effective in many patients, do not guarantee benefits for everyone.) Some people in a study may not receive the active treatment and instead will receive a placebo (i.e., a sugar pill). Although all patients in clinical trials are carefully monitored both during and after conclusion of the trial, there are always known and unknown risks associated with new treatments. Patients should decide whether or not to participate in a study only after they understand both the potential risks and benefits.
WHO CAN PARTICIPATE IN A CLINCIAL TRIAL?
WHAT HAPPENS DURING A STUDY?
The study procedures and process will vary depending on the type of clinical trial you participate in. Before you begin a study, the process and all procedures for that study will be explained to you. Throughout the study, the Study Coordinator and the Principal Investigator will assist you and can answer any questions you have. They will check your health at the beginning of the trial, give you specific instructions for participating in the trial, monitor you carefully during the trial, and stay in touch with you after the study.
Some clinical trials involve more tests and doctor visits than you would normally have for your illness or condition. Your participation will be most successful if you carefully follow all instructions you are given and stay in contact with the research staff.
WHAT ARE THE RISKS OF PARTICIPATING IN A CLINICAL TRIAL?
WHAT SAFEGUARDS EXIST TO MONITOR CLINICAL RESEARCH TRIALS?
Several safeguards have been put into place that all companies must follow when conducting clinical trials.
The Food and Drug Administration (FDA) is the regulatory agency that oversees all clinical research conducted in the United States. They are responsible for protecting the rights and, to the degree possible, the welfare of subjects participating in clinical trials. They ensure that data collected from clinical trials is of high quality and they are the agency that determines whether a new treatment should be sold in the US. Several organizations and individuals are governed by FDA regulations including clinical investigators, sponsors (i.e., the companies developing the treatments), Contract Research Organizations (i.e., companies that help conduct the studies), and Institutional Review Boards (groups that are responsible for ensuring the safety and welfare of study participants).
Good Clinical Practice (GCP) guidelines are another safeguard implemented to protect the safety of subjects. These guidelines encompass federal regulations and industry-accepted standards that govern clinical trials on humans. These regulations and standards apply to the conduct of the studies, record keeping, informed consent of subjects, collection of scientific data, and submission of information needed for the FDA to determine whether a new treatment should or should not be sold in the US. All companies must follow these guidelines when conducting clinical research.
An Institutional Review Board (IRB) is a committee that is formally tasked by an institution to review, approve, and monitor research involving human subjects. IRBs ensure that the risks to subjects are minimized; that subjects are adequately informed about the trial and the implications it will have on their treatment; that study protocols are modified, when necessary, to ensure safety; that risks are reasonable in relation to the anticipated benefits and the importance of the knowledge to be gained; that subject selection is equitable and that no classes of patients are discriminated against; that informed consent is obtained; that there is a provision for safety monitoring in the research plan; and that here are adequate provisions for the privacy and confidentiality for subjects and the data collected.
WHAT IS A PROTOCOL?
WHAT IS INFORMED CONSENT?
WHAT IS A PLACEBO?
A placebo is an inactive substance (pill, liquid, or powder) that has no treatment value. It may be referred to as a “blank” or “sugar pill.” In placebo-controlled clinical trials, experimental treatments are compared with placebos to assess the treatment’s effectiveness.
Some participants in these studies may receive placebo rather than active treatment. In pain studies, patients who receive placebo (as well as those who receive active treatment) will always have access to ‘rescue medication’, which is a standard pain medication given for the condition or procedure being studied. As soon as you feel that you need more pain medication, you will be given a rescue dose of the standard pain medication. Asking for rescue pain medication will not in any way have a negative impact on your participation in the study.
WHAT IS AN ADVERSE EVENT?
WHAT SHOULD I KNOW BEFORE PARTICIPATING IN A RESEARCH STUDY?
You should know as much as possible about the research study before you participate. It is important that you feel very comfortable asking questions and that the staff answers them in a way you can understand. When preparing for a meeting with the study coordinator or doctor you should plan ahead and write down the questions you want to ask. Review the study website to help you prepare questions to ask. You might ask a friend or relative to come with you for support and to hear the responses to your questions. You may want to ask the following questions when deciding whether to participate in a clinical trial:
- What questions should I ask?
- 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?
- What kinds of therapies, procedures, and/or tests will I have during the trial?
- 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?
- Will I be able to take my regular medications during the trial?
- 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 trial?
- Questions about risks and benefits include:
- How do the possible risks and benefits of the study compare with approved treatments for me?
- What are the possible immediate and long-term side effects?
- Will I have to pay anything to participate in the study?
- What are the charges likely to be?
- Is my insurance likely to cover those expenses?