What are clinical studies?
Clinical studies are research studies in which real people participate as volunteers. Clinical research studies (sometimes called trials or protocols) are a means of developing new treatments and medications for diseases and conditions. There are strict rules for clinical trials, which are monitored by the National Institutes of Health and the U.S. Food and Drug Administration. Some of the research studies at the Clinical Center involve promising new treatments that may directly benefit patients. Research is vital to help us understand these factors and their complex interactions. Because of the answers research can provide, it is a powerful source of hope for people experiencing mental health conditions and their families.
Why should I participate?
The health of millions has been improved because of advances in science and technology, and the willingness of thousands of individuals like you to take part in clinical research. The role of volunteer subjects as partners in clinical research is crucial in the quest for knowledge that will improve the health of future generations. With your help, we can pave the path for future cures.
Will I be compensated?
Many clinical trials offer compensation for volunteering. Please ask if compensation is available for you.
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.” The clinical research volunteer may be a member of the community, an NIH investigator or other employee, or family members of a patient volunteer. Research procedures with these volunteers are designed to develop new knowledge, not to provide direct benefit to study participants. Clinical research volunteers have always played a vital role in medical research. We need to study healthy volunteers for several reasons: When developing a new technique such as a blood test or imaging device, we need clinical research volunteers to help us define the limits of “normal.” These volunteers are recruited to serve as controls for patient groups. They are often matched to patients on such characteristics as age, gender, or family relationship. They are then given the same test, procedure, or drug the patient group receives. Investigators learn about the disease process by comparing the patient group to the clinical research volunteers.
What are Phase I, Phase II and Phase III studies?
The phase 1 study is used to learn the “maximum tolerated dose” of a drug that does not produce unacceptable side effects. Patient volunteers are followed primarily for side effects, and not for how the drug affects their disease. The first few volunteer subjects receive low doses of the trial drug to see how the drug is tolerated and to learn how it acts in the body. The next group of volunteer subjects receives larger amounts. Phase 1 studies typically offer little or no benefit to the volunteer subjects.
The phase 2 study involves a drug whose dose and side effects are well known. Many more volunteer subjects are tested, to define side effects, learn how it is used in the body, and learn how it helps the condition under study. Some volunteer subjects may benefit from a phase 2 study.
The phase 3 study compares the new drug against a commonly used drug. Some volunteer subjects will be given the new drug and some the commonly used drug. The trial is designed to find where the new drug fits in managing a particular condition. Determining the true benefit of a drug in a clinical trial is difficult.
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 the placebo effect?
Medical research is dogged by the placebo effect – the real or apparent improvement in a patient’s condition due to wishful thinking by the investigator or the patient. Medical techniques use three ways to rid clinical trials of this problem. These methods have helped discredit some previously accepted treatments and validate new ones. Methods used are the following: randomization, single-blind or double-blind studies, and the use of a placebo.
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 the 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, as in routine medical care and activities of daily living. In thinking about the risks of research, it is helpful to focus on two things: the degree of harm that could result from taking part 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 consent document, which you are asked to sign before taking part in research. In addition, the major risks of participating in a study will be explained to you by a member of the research team, who will answer your questions about the study. Before deciding to participate, you should carefully weigh these risks. Although you may not receive any direct benefit as a result of participating in research, the knowledge developed may help others.
Are clinical trials safe?
There are many processes in place to ensure the highest safety standards for those entering a clinical trial. The following are areas of protection for the volunteer.
Ethical guidelines: The goal of clinical research is to develop knowledge that improves human health or increases understanding of human biology. People who take part in clinical research make it possible for this to occur. The path to finding out if a new drug is safe or effective is to test it on patients in clinical trials. The purpose of ethical guidelines is both to protect patients and healthy volunteers, and to preserve the integrity of the science.
Protocol review: As in any medical research facility, all new protocols must be approved by an institutional review board (IRB) before they can begin. The IRB, which consists of medical specialists, statisticians, nurses, social workers, and medical ethicists, is the advocate of the volunteer subject. The IRB will only approve protocols that address medically important questions in a scientific and responsible manner.
Informed consent: Informed consent is the process of learning the key facts about a clinical trial before deciding whether to participate. The process of providing information to participants continues throughout the study. To help you decide whether to take part, members of the research team explain the study. The research team provides an informed consent document, which includes such details about the study as its purpose, duration, required procedures, and who to contact for various purposes. The informed consent document also explains risks and potential benefits.
If you decide to enroll in the trial, you will need to sign the informed consent document. You are free to withdraw from the study at any time.
IRB review: Most, but not all, clinical trials in the United States are approved and monitored by an Institutional Review Board (IRB) to ensure that the risks are minimal when compared with potential benefits. An IRB is an independent committee that consists of physicians, statisticians, and members of the community who ensure that clinical trials are ethical and that the rights of participants are protected. You should ask the sponsor or research coordinator whether the research you are considering participating in was reviewed by an IRB.
For more information about research protections, see:
For more information on participants’ privacy and confidentiality, see:
- HIPAA Privacy Rule
- The Food and Drug Administration, FDA’s Drug Review Process:
Ensuring Drugs Are Safe and Effective
What safeguards are there to protect participants in clinical research?
Patient representative: The Patient Representative acts as a link between the patient and the hospital. The Patient Representative makes every effort to assure that patients are informed of their rights and responsibilities, and that they understand what the Clinical Center is, what it can offer, and how it operates. We realize that this setting is unique and may generate questions about the patient’s role in the research process. As in any large and complex system, communication can be a problem and misunderstandings can occur. If any patient has an unanswered question or feels there is a problem they would like to discuss, they can call the Patient Representative.
Patient Bill of Rights. Whether you are a clinical research or a patient volunteer subject, you are protected by the Clinical Center Patients’ Bill of Rights. This document is adapted from the one made by the American Hospital Association for use in all hospitals in the country. The bill of rights concerns the care you receive, privacy, confidentiality, and access to medical records.
Source: The Clinical Center at the National Institutes of Health.
Research is not always just about taking a new medication. You can make a difference in the advancement of scientific research in many ways. Different types of clinical research are used depending on what the researchers are studying. Below are descriptions of some different kinds of clinical research.
Treatment Research generally involves an intervention such as medication, psychotherapy, new devices, or new approaches to surgery or radiation therapy.
Prevention Research looks for better ways to prevent disorders from developing or returning. Different kinds of prevention research may study medicines, vitamins, vaccines, minerals, or lifestyle changes.
Diagnostic Research refers to the practice of looking for better ways to identify a particular disorder or condition.
Screening Research aims to find the best ways to detect certain disorders or health conditions.
Quality of Life Research explores ways to improve comfort and the quality of life for individuals with a chronic illness.
Genetic studies aim to improve the prediction of disorders by identifying and understanding how genes and illnesses may be related. Research in this area may explore ways in which a person’s genes make him or her more or less likely to develop a disorder. This may lead to development of tailor-made treatments based on a patient’s genetic make-up.
- Genetic Information Nondiscrimination Act (GINA) of 2008
- National Human Genome Research Institute
- The National Advisory Mental Health Council Workgroup on Genomics
Epidemiological studies seek to identify the patterns, causes, and control of disorders in groups of people.
An important note: some clinical research is “outpatient,” meaning that participants do not stay overnight at the hospital. Some is “inpatient,” meaning that participants will need to stay for at least one night in the hospital or research center. Be sure to ask the researchers what their study requires.
Phases of clinical trials: when clinical research is used to evaluate medications and devices
Clinical trials are a kind of clinical research designed to evaluate and test new interventions such as psychotherapy or medications. Clinical trials are often conducted in four phases. The trials at each phase have a different purpose and help scientists answer different questions.
- Phase I trials
Researchers test an experimental drug or treatment in a small group of people for the first time. The researchers evaluate the treatment’s safety, determine a safe dosage range, and identify side effects.
- Phase II trials
The experimental drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
- Phase III trials
The experimental study drug or treatment is given to large groups of people. Researchers confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.
- Phase IV trials
Post-marketing studies, which are conducted after a treatment is approved for use by the FDA, provide additional information including the treatment or drug’s risks, benefits, and best use.
Examples of other kinds of clinical research
Many people believe that all clinical research involves testing of new medications or devices. This is not true, however. Some studies do not involve testing medications and a person’s regular medications may not need to be changed. Healthy volunteers are also needed so that researchers can compare their results to results of people with the illness being studied. Some examples of other kinds of research include the following:
- A long-term study that involves psychological tests or brain scans
- A genetic study that involves blood tests but no changes in medication
- A study of family history that involves talking to family members to learn about people’s medical needs and history.
Source: FDA website.