A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies. ClinicalTrials.gov includes both interventional and observational studies.
In a clinical trial, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants’ behavior, such as diet. Clinical trials may compare a new medical approach to a standard one that is already available, to a placebo that contains no active ingredients, or to no intervention. Some clinical trials compare interventions that are already available to each other. When a new product or approach is being studied, it is usually not known whether it will be helpful, harmful, or no different than available alternatives (including no intervention). The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants. For example, investigators may give a drug or treatment to participants who have high blood pressure to see whether their blood pressure decreases.
Clinical trials used in drug development are sometimes described by phase. These phases are defined by the Food and Drug Administration (FDA).
Every clinical study is led by a principal investigator, who is often a medical doctor. Clinical studies also have a research team that may include doctors, nurses, social workers, and other health care professionals.
Clinical studies can be sponsored, or funded, by pharmaceutical companies, academic medical centers, voluntary groups, and other organizations, in addition to Federal agencies such as the National Institutes of Health, the U.S. Department of Defense, and the U.S. Department of Veterans Affairs. Doctors, other health care providers, and other individuals can also sponsor clinical research.
Clinical studies can take place in many locations, including hospitals, universities, doctors’ offices, and community clinics. The location depends on who is conducting the study.
The length of a clinical study varies, depending on what is being studied. Participants are told how long the study will last before they enroll.
In general, clinical studies are designed to determine whether an investigational new drug is safe and effective when given under specific conditions, as well as to add to medical knowledge related to the treatment, diagnosis, and prevention of diseases or conditions. Some common reasons for conducting clinical studies include:
A clinical study is conducted according to a research plan known as the protocol. The protocol is designed to answer specific research questions and safeguard the health of participants. It contains the following information:
The FDA regulates the conduct of clinical studies and reviews protocols before a clinical study can begin.
Who Can Participate in a Clinical Study?
Clinical studies have standards outlining who can participate. These standards are called eligibility criteria and are listed in the protocol. Some research studies seek participants who have the illnesses or conditions that will be studied, other studies are looking for healthy participants, and some studies are limited to a predetermined group of people who are asked by researchers to enroll.
Eligibility. The factors that allow someone to participate in a clinical study are called inclusion criteria, and the factors that disqualify someone from participating are called exclusion criteria. They are based on characteristics such as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.
How Are Participants Protected?
Informed consent is a process used by researchers to provide potential and enrolled participants with information about the risks of participating in a clinical study, as well as to inform them of potential benefits and alternatives to the clinical study. This information helps people decide whether they want to enroll or continue to participate in the study. The informed consent process may involve question-and-answer sessions and activities to measure a person’s understanding of the risks and potential benefits of the investigational study. A person or a person’s parent or legal guardian must sign an informed consent document before a person may join a study to show that he or she was given information on the risks, potential benefits, and alternatives and that he or she understands it. Signing the document and providing consent does not create an obligation or a requirement to participate in the study. Participants may withdraw from a study at any time, even if the study is not over. See the Questions to Ask section on this page for questions to ask a health care provider or researcher about participating in a clinical study.
Institutional Review Boards
Every clinical study of an investigational drug, biological product, or medical device regulated by the FDA must be reviewed, approved, and monitored by an institutional review board (IRB). An IRB is made up of doctors, researchers, and members of the community. Its role is to make sure that the study is ethical and that the rights and welfare of participants are protected. This includes making sure that research risks are minimized and are reasonable in relation to any potential benefits, among other responsibilities. The IRB also reviews the protocol and the informed consent document before a study may begin recruiting participants.
In addition to being monitored by an IRB, some clinical studies are also monitored by data monitoring committees (also called data safety and monitoring boards).
Various federal agencies, including the Office for Human Research Protection and FDA, have the authority to determine whether research participants in federally funded or sponsored clinical studies are adequately protected.
Relationship to Usual Health Care
Typically, participants continue to see their usual health care providers while enrolled in a clinical study. While most clinical studies provide participants with medical products or interventions related to the illness or condition being studied, they do not provide extended or complete health care. By having his or her usual health care provider work with the research team, a participant can make sure that the study protocol will not conflict with other medications or treatments that he or she receives.
Considerations for Participation
Participating in a clinical study contributes to medical knowledge. The results of these studies can make a difference in the care of future patients by providing information about the benefits and risks of therapeutic, preventative, or diagnostic products or interventions.
Clinical trials provide the basis for the development and marketing of new drugs, biological products, and medical devices. Sometimes, the safety and the effectiveness of the experimental approach or use may not be fully known at the time of the trial. Some trials may provide participants with the prospect of receiving direct medical benefits, while others do not. All trials of experimental treatments involve some risk of harm or injury to the participant. (For trials approved by IRBs, the IRB has decided that known risks of participation have been minimized to the degree possible and that appropriate protections of potential participants have been put in place.) Many trials require participants to undergo additional procedures, tests, and assessments based on the study protocol. The risks and requirements for a clinical study are described in the informed consent document. A potential participant should also discuss these issues with members of the research team and with his or her usual health care provider.
Questions to Ask
Anyone interested in participating in a clinical study should know as much as possible about the study and feel comfortable asking the research team questions about the study, the related procedures, and any expenses. The following questions may be helpful during such a discussion. Answers to some of these questions are provided in the informed consent document. Many of the questions are specific to clinical trials, but some also apply to observational studies.
Source: National Library of Medicine, “Learn about Clinical Studies,” available at https://clinicaltrials.gov/ct2/about-studies/learn#Considerations, This page last reviewed in March 2019. Accessed December 20, 2020.