- What is a clinical research study (trial)?
- Why participate in a clinical research study?
- Who can participate in a clinical research study?
- What happens during a clinical research study?
- What is informed consent?
- What are the benefits and risks of participating in a clinical research study?
- What are side effects and adverse reactions?
- How is the safety of the participant protected?
- What should people consider before participating in a study?
- What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
- Does a participant continue to work with a primary health care provider while in a trial?
- Can a participant leave a clinical research study after it has begun?
- Where do the ideas for studies come from?
- Who sponsors clinical research studies?
- What is a protocol?
- What is a placebo?
- What is a control or control group?
- What are the different types of clinical research studies?
- What are the phases of clinical research studies?
- What is "expanded access"?
- Glossary terms
What is a clinical research study (trial)?
Although there are many definitions of clinical research studies (trials), they are generally considered to be biomedical or health-related research studies in human beings that follow a pre-defined protocol. Research Investigators at University of Kentucky conducts interventional and observational types of studies. Interventional studies are those in which the research participants are assigned by the investigator to a treatment or other intervention, and their outcomes are measured. Observational studies are those in which individuals are observed and their outcomes are measured by the investigators.
Why participate in a clinical research study?
Participants in clinical research studies can play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research.
Who can participate in a clinical research study?
All clinical research studies have guidelines about who can participate. Using inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. The factors that allow someone to participate in a clinical research study are called "inclusion criteria" and those that disallow someone from participating are called "exclusion criteria". These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical research study, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical research study, while others need healthy participants. 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 happens during a clinical research study?
The clinical research study process depends on the kind of study being conducted (See What are the different types of clinical research studies?) The clinical research study team includes 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 study, give specific instructions for participating in the study, monitor the participant carefully during the study, and stay in touch after the study is completed.
Some clinical research studies involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of studies, the participant works with a research team. Clinical research study participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff.
What is informed consent?
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 someone decide whether or not to participate, the doctors and nurses involved in the study explain the details of the study. If the participant's native language is not English, translation assistance can be provided. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the study at any time.
What are the benefits and risks of participating in a clinical research study?
Clinical research studies that are well-designed and well-executed are the best approach for eligible participants to:
- Play an active role in their own health care.
- Gain access to new research treatments before they are widely available.
- Obtain expert medical care at leading health care facilities during the study.
- Help others by contributing to medical research.
There are risks to clinical research studies.
What are side effects and adverse reactions?
- There may be unpleasant, serious or even life-threatening side effects to experimental treatment.
- The experimental treatment may not be effective for the participant.
- The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.
Side effects are any undesired actions or effects of the experimental drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.
How is the safety of the participant protected?
The ethical and legal codes that govern medical practice also apply to clinical research studies. In addition, most clinical research is federally regulated with built in safeguards to protect the participants. The study follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical research study progresses, researchers report the results of the study at scientific meetings, to medical journals, and to various government agencies. Individual participants' names will remain secret and will not be mentioned in
these reports (See Confidentiality Regarding Trial Participants
What should people consider before participating in a research study?
People should know as much as possible about the clinical research study and feel comfortable asking the members of the health care team questions about it, the care expected while in a study, and the cost of the study. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
- What kinds of tests and experimental treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this study affect my daily life?
- How long will the study last?
- Will hospitalization be required?
- Who will pay for the experimental treatment?
- Will I be reimbursed for other expenses?
- What type of long-term follow up care is part of this study?
- How will I know that the experimental treatment is working? Will results of the study be provided to me?
- Who will be in charge of my care?
What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
- Plan ahead and write down possible questions to ask.
- Ask a friend or relative to come along for support and to hear the responses to the questions.
- Bring a tape recorder to record the discussion to replay later.
Every clinical research study in the U.S. must be approved and monitored by anthese reports (See 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 research study is ethical and the rights of study participants are protected. All institutions that conduct or support biomedicalresearch involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
Does a participant continue to work with a primary health care provider while in a study?
Yes. Most clinical research studies provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.
Can a participant leave a clinical research study after it has begun?
Yes. A participant can leave a clinical research study, at any time. When withdrawing from the study, the participant should let the research team know about it, and the reasons for leaving the study.
Where do the ideas for study come from?
Ideas for clinical research studies usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical research studies. During a study, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.
Who sponsors clinical research studies?
Clinical research studies are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.
What is a protocol?
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 trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical research study, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.
What is a placebo?
A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical research studies, experimental treatments are often compared with placebos to assess the experimental treatment's effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or experimental treatment.
What is a control or control group?
A control is the standard by which experimental observations are evaluated. In many clinical research 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 are the different types of clinical research studies?
Treatment studies test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
Prevention studies look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vaccines, vitamins, minerals, or lifestyle changes.
Diagnostic studies are conducted to find better tests or procedures for diagnosing a particular disease or condition.
Screening studies test the best way to detect certain diseases or health conditions.
Quality of Life studies (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.
What are the phases of clinical research studies?
Clinical research studies are conducted in phases. The studies at each phase have a different purpose and help scientists answer different questions:
In Phase I studies, researchers test an experimental 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 studies, the experimental 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 experimental 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 experimental drug or treatment to be used safely.
In Phase IV studies, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
What is "expanded access"?
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical research study.
Most human use of investigational new drugs takes place in controlled clinical research studies conducted to assess the safety and efficacy of new drugs. Data from these studies are used to determine whether a drug is safe and effective, and serve as the basis for the drug marketing application. Sometimes, patients do not qualify for these controlled studies because of other health problems, age, or other factors, or are otherwise unable to enroll in such studies (e.g., a patient may not live sufficiently close to a clinical research study site).
For patients who cannot participate in a clinical research study of an investigational drug, but have a serious disease or condition that may benefit from treatment with the drug, FDA regulations enable manufacturers of such drugs to provide those patients access to the drug under certain situations, known as "expanded access." For example, the drug cannot expose patients to unreasonable risks given the severity of the disease to be treated and the patient does not have any other satisfactory therapeutic options (e.g., an approved drug that could be used to treat the patient's disease or condition). The manufacturer must be willing to make the drug available for expanded access use. The primary intent of expanded access is to provide treatment for a patient's disease or condition, rather than to collect data about the study drug.
Some investigational drugs are available for treatment use from pharmaceutical manufacturers through expanded access programs listed in ClinicalTrials.gov. If you or a loved one is interested in treatment with an investigational drug under an expanded access protocol listed in ClinicalTrials.gov, review the protocol eligibility criteria and inquire at the Contact Information number. If there is not an expanded access protocol listed in ClinicalTrials.gov, you or your health care provider may contact a manufacturer of an investigational drug directly to ask about expanded access programs.
For additional information on expanded access programs, please see the FDA website at Access to Investigational Drugs.
Expanded Access Studies can be found by in ClinicalTrials.gov:
- go to the Advanced Search page,
- select "Expanded Access Studies" from the "Study Type" pull-down menu, and
- press Search.
or use these prepared links:
All Expanded Access studies (available and no longer available)