For Patients and Community

The idea for a clinical research study – also known as a clinical trial – often starts in the laboratory. Researchers test new therapies or procedures in the laboratory and in animal studies. Then the most promising experimental treatments are moved into clinical trials, which are conducted in phases. During a trial, more information is gained about an experimental treatment, its risks and benefits, and how well it works.

Clinical trials are sponsored or funded by various organizations or individuals, including physicians, foundations, medical institutions, disease-specific patient support organizations, and pharmaceutical companies, as well as federal agencies such as the National Institute of Health and the Department of Veterans Affairs.

Many different types of people participate in clinical trials. Some are healthy, while others may have illnesses. 

Research procedures with healthy volunteers are designed to develop new knowledge, not to provide direct benefit to study participants. Healthy volunteers have always played an important role in research.

Healthy volunteers receive the same test, procedure, or drug the patient group receives. Then researchers compare the outcomes of the healthy volunteers and the patient volunteers.  The differences provide important information about how the treatment being studied works. 

A patient volunteer has a known health problem and participates in research to better understand, diagnose, treat, or cure that disease or condition. Research procedures with a patient volunteer help develop new knowledge. These procedures may or may not benefit the study participants.

Patient volunteers may be involved in studies similar to those in which healthy volunteers participate. These studies involve drugs, devices, or interventions designed to prevent, treat, or cure disease. Although these studies may provide direct benefit to patient volunteers, the main aim is to prove, by scientific means, the effects and limitations of the experimental treatment. Some patients serve as controls by not taking the test drug, or by receiving test doses of the drug large enough only to show that it is present, but not at a level that can treat the condition. A study’s benefits may be indirect for the volunteers but may help others.

All clinical trials have guidelines about who can participate, called Inclusion/Exclusion Criteria. Factors that allow someone to participate in a clinical trial are “inclusion criteria.” Those that exclude or do not allow participation are “exclusion criteria.” Criteria are the trial’s rules about who can take part. They are based on factors such as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. 

There are many factors to consider when deciding to participate in a clinical trial. You will want to get answers to all of your questions.

A clinical study is led by a principal investigator (PI), who is often a doctor. Usually there is a clinical research coordinator who helps the PI. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the patient carefully during the entire trial, and stay in touch after the trial is completed. These are the people who will explain the clinical trial to you and answer your questions.

Below are some items you should discuss with the PI and clinical research coordinator. It is also helpful to talk to your doctor.

• Protocol: Clinical trials are conducted according to an action plan known as a protocol. The protocol is carefully designed to protect the participants’ health and answer specific research questions. A protocol describes who is eligible to participate in the trial, explains what tests, procedures, medications, and dosages will be used, and lists the length of the study and what information will be gathered. You should find out how much of your time will be needed to take part in the trial, any discomfort you may feel, or extra tests that will be required. While some trials require minimal amounts of time and effort, other trials may require a large commitment in time and effort on behalf of the volunteer. Some trials may compensate volunteers for time and travel expenses.
• Institutional Review Board: Each clinical trial in the United States must be approved and monitored by an Institutional Review Board (IRB) to ensure that the risks to patients are minimal and are worth any 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. Federal regulation requires all institutions in the United States that conduct or support biomedical research involving people to have an IRB initially approve and then periodically review the research.
• Informed Consent is the process of providing potential participants with the key facts about a clinical trial before they decide whether to participate. To help someone decide whether or not to participate, members of the research team explain the details of the study. If a participant’s native language is not English, translation or interpretive assistance can be provided. The research team provides an informed consent document that includes details about the study, such as its purpose, length of time, required procedures, and who to contact for further information. The informed consent document also explains risks and potential benefits. The participant then decides whether to sign the document. Informed consent is not a contract. Volunteers are free to withdraw from the study completely or to refuse particular treatments or tests at any time. Sometimes, however, this will make them ineligible to continue to participate in the study.

If you are invited to participate in a clinical trial, feel free to ask any questions or bring up any issues concerning the trial at any time. The following suggestions may give you some ideas as you think about your own questions:

• What is the purpose of the study?
• What is required of me?
• What is my role in the study – am I a healthy volunteer or a patient volunteer?
• Will the study directly benefit me?
• Will the study benefit others?
• Are there risks? If so, what are they and what are the chances that they will occur?
• What discomforts are involved?
• What is the total time involved?
• Are there other inconveniences?
• Have I discussed participation in the study with those who are important to me, such as family and friends?
• Do I wish to participate in this study?

Clinical trials involve risks, just as routine medical care and the activities of daily living. When weighing the risks of research, you can consider two important factors: the degree of harm that could result from participating in the study and the chance of any harm occurring.

Most clinical studies pose the risk of minor discomfort, which lasts only a short time. However, some study participants experience complications that require medical attention. In rare cases, participants have been seriously injured or have died of complications resulting from their participation in trials of experimental therapies. The specific risks associated with a research protocol are described in detail in the informed consent document, which participants are asked to sign before participating in research. Also, a member of the research team will explain the major risks of participating in a study and will answer any questions you have about the study. Before deciding to participate, carefully consider possible risks and benefits.

Well-designed and well-executed clinical trials provide the best approach for participants to:

• Play an active role in their health care.
• Gain access to investigational research treatments before they are widely available.
• Receive regular and careful medical attention from a research team that includes doctors and other health professionals.
• Help others by contributing to medical research.

Risks to participating in clinical trials include the following:

• There may be unpleasant, serious, or even life-threatening side effects to experimental treatment.
• The study may require more time and attention than standard treatment would, including visits to the study site, more blood tests, more treatments, hospital stays, or complex dosage requirements.  

After a clinical trial is completed, the researchers carefully examine information collected during the study before making decisions about the meaning of the findings and about further testing. Individual participant names will remain secret and will not be mentioned in any reports about the trial.

Results from clinical trials are often published in peer-reviewed scientific journals. Peer review is a process by which experts review the report before it is published to ensure that the analysis and conclusions are sound. If the results are particularly important, they may be featured in news media and discussed at scientific meetings and by patient advocacy groups before they are published. Once a new approach has been proven safe and effective in a clinical trial, it may become the standard of medical practice.