Below is a list of frequently asked questions by patients that are considering participation in a research study. The list below is not exhaustive. If you have a question that is not answered here, please talk with your doctor to see if he or she can help you find the answer.
What is clinical research?
Clinical research is research conducted with human subjects, or material of human origin, in which the researcher directly interacts with human subjects. Clinical research helps doctors and researchers to find new and better ways to understand, detect, control, and treat illness. A clinical research study is a way to find answers to difficult scientific or health questions. For example, the study might explore the best ways to treat people with colon cancer. By studying cancer cells from patients, researchers may be able to determine the specific genetic mutations (changes in gene sequence) that caused the normal, healthy cells to become cancerous, and may help doctors decide on the best drugs to prescribe or surgeries to perform. Clinical research today may help other doctors in the future screen their healthy patients before they ever develop cancer.
What is a protocol?
All clinical studies are based on a set of procedures or directions called a protocol. A protocol describes what types of people are eligible to participate in the study; determines the schedule of tests, procedures, medications, and dosages; and sets the length of the study.
What is a clinical trial?
If a clinical research study involves testing or studying a drug or medical device to see if it is a safe and effective treatment for people, it is called a “trial.” For example, a clinical trial may test the effectiveness of a new drug for treating Parkinson’s disease.
Many new medicines and drugs are found to work in the researcher’s lab, and to be safe and effective in animal tests. But drugs and devices must be proven to be safe and effective for people before the Food and Drug Administration (FDA) can approve them and doctors can prescribe them to patients. The FDA has strict rules that govern how clinical trials are conducted. These rules are designed to ensure the safety of those who participate.
What are clinical trial "phases?"
Clinical trials of experimental drugs proceed through four phases:
In Phase I clinical trials, researchers test a new drug or treatment for the first time in a small group of normal, healthy volunteers (about 20 to 80) to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase II clinical trials, the study drug or treatment is given to a larger group of people (about 100 to 300), including patients with the particular disease, to see if the drug or treatment is effective, and to further evaluate its safety.
In Phase III clinical trials, the study drug or treatment is given to large groups of people (from 1,000 to 3,000), including patients, to confirm its effectiveness, monitor side effects, compare it to other commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase IV clinical trials are done after the drug or treatment has been approved by the FDA and marketed for public use. These studies continue testing the drug or treatment to collect information about its effect in various populations and gather data on any side effects associated with long-term use.
What are "blind" or "masked" studies?
In many clinical trials, one group of patients will be given an experimental drug or treatment, while a control group is given either a standard treatment for the illness, or a placebo (a harmless “fake” drug), or no treatment at all.
In a “blinded” or “masked” study, participants do not know whether they are getting the drug being tested, or whether they are in the control group. The goal is to prevent the so-called “placebo effect” from influencing the results of the experiment. The placebo effect is the phenomenon of patients feeling better simply because they think they are receiving a helpful drug or treatment.
Sometimes, clinical trials are “double-blind” or “double-masked.” That means that neither the participants, nor the study staff members, know who is receiving the experimental drug and who is in the control group. Studies are performed in this way so that neither the patients’ nor the doctors’ expectations about the experimental drug can influence the observations and results.
Should I volunteer for clinical research?
Clinical research is a vital part of finding new treatments and cures for diseases. Carefully conducted clinical studies are the fastest way to find treatments that are safe and effective. By volunteering for a clinical study, you would be participating in research that may result in a new treatment for a deadly or debilitating disease.
Before you agree to participate in a study, you must be given complete information about the study through a process known as “informed consent.” Informed consent involves two essential components: a document and a process. The informed consent document gives a summary of the research project (including the study’s purpose, research procedures, potential benefits and risks, etc.) and explains the individual’s rights as a research participant. This document is part of an informed consent process, which consists of conversations between the research team and the participant, and may include other supporting material such as study brochures. The Food and Drug Administration (FDA) provides details about informed consent with the information page: Informed Consent for Clinical Trials [fda.gov].
Because participating in a clinical study is an important decision, there are many questions that you should consider before agreeing to participate. The Centers for Disease Control and Prevention (CDC) have prepared a list of questions to help you get the information you need to make a decision about participating: Taking Part in Research Studies: What Questions Should You Ask? [cdc.gov].
Will I be compensated?
A research study may provide compensation to study participants for their time and, in some instances, for the inconvenience of a procedure. Importantly, research compensation in considered to be taxable income by the Internal Revenue Service, and when research compensation totals more than $600 in a year you will receive an IRS “Form 1099-Other Income” at the end of the year to file with your tax return.
What is a "healthy volunteer"?
A volunteer subject with no known significant health problems who participates in research to test a new drug, device, or intervention is known as a “healthy volunteer” or “Clinical Research Volunteer.” Research procedures with healthy volunteers are designed to develop new knowledge, not to provide direct benefit to study participants. Healthy volunteers have always played a vital role in medical research. We need to study healthy volunteers for several reasons: for example, when developing a new technique such as a blood test or imaging device, we need healthy volunteers to help us define the limits of “normal.”
What is a placebo?
Placebos are harmless, inactive substances made to look like the real medicine used in the clinical trial. Placebos allow the investigators to learn whether the medicine being given works better or no better than ordinary treatment. In many studies, there are successive time periods, with either the placebo or the real medicine. In order not to introduce bias, the patient, and sometimes the staff, are not told when or what the changes are. If a placebo is part of a study, you will always be informed in the consent form given to you before you agree to take part in the study. When you read the consent form, be sure that you understand what research approach is being used in the study you are entering.
What is randomization?
Randomization is when two or more alternative treatments are selected by chance, not by choice. The treatment chosen is given with the highest level of professional care and expertise, and the results of each treatment are compared. Analyses are done at intervals during a trial, which may last years. As soon as one treatment is found to be definitely superior, the trial is stopped. In this way, the fewest number of patients receive a potentially less beneficial treatment.
What are single-blind and double-blind studies?
In single- or double-blind studies, the participants don’t know which medicine is being used, and they can describe what happens without bias. Blind studies are designed to prevent anyone (doctors, nurses, or patients) from influencing the results. This allows scientifically accurate conclusions. In single-blind (“single-masked”) studies, only the patient is not told what is being given. In a double-blind study, only the pharmacist knows; the doctors, nurses, patients, and other health care staff are not informed. If medically necessary, however, it is always possible to find out what the patient is taking.
Are there risks involved in participating in clinical research?
Risks are involved in clinical research. In thinking about the risks of research, it is helpful to focus on two things: the degree of harm that could result from participating in the study, and the chance of any harm occurring. Most clinical studies pose risks of minor discomfort, lasting only a short time. Some volunteer subjects, however, experience complications that require medical attention. The specific risks associated with any research protocol are described in detail in the informed consent document, which you are asked to sign before taking part in research. The major risks of participating in a study will be explained to you by a member of the research team. Before deciding to participate, you should carefully weigh these risks.
What is an Investigator-Initiated Study (IIS) ?
An Investigator-Initiated Study (IIS) is a research project in which the investigator designs, implements and sponsors the study, rather than a large company such as a medical device or drug manufacturer. Many large industry-sponsored clinical trials are not, and cannot be, designed to determine all the potential uses for a medication. IITs expand product knowledge, including safety. Physician researchers often identify new ways of using existing treatments, thus improving the health of numerous other patients.