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 and observational studies.

Clinical Trials
In a clinical trial (also called an interventional study), 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, for example, diet.

Clinical trials may compare a new medical approach to a standard one that is already available or to a placebo that contains no active ingredients or 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 not usually known whether it will be helpful, harmful or the same as 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).

Note: Some people who are not eligible to participate in a clinical trial may be able to get experimental drugs or devices outside of a clinical trial through an expanded access program.

Observational Studies
In an observational study, investigators assess health outcomes in groups of participants according to a protocol or research plan. Participants may receive interventions, which can include medical products, such as drugs or devices, or procedures as part of their routine medical care, but participants are not assigned to specific interventions by the investigator (as in a clinical trial). For example, investigators may observe a group of older adults to learn more about the effects of different lifestyles on cardiac health.

In general, clinical studies are designed to add to medical knowledge related to the treatment, diagnosis and prevention of diseases or conditions. Some common reasons for conducting clinical studies include:

• Evaluating one or more interventions (for example, drugs, medical devices, approaches to surgery or radiation therapy) for treating a disease, syndrome or condition
• Finding ways to prevent the initial development or recurrence of a disease or condition. These can include medicines, vaccines or lifestyle changes, among other approaches.
• Evaluating one or more interventions aimed at identifying or diagnosing a particular disease or condition
• Examining methods for identifying a condition or risk factors for that condition
• Exploring and measuring ways to improve the comfort and quality of life of people with a chronic illness through supportive care

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. Most trials involve some risk of harm or injury to the participant, although it may not be more than the risks related to routine medical care or disease progression. (For trials approved by institutional review boards (IRB), the IRB has decided that the risks of participation have been minimized and are reasonable in relation to anticipated benefits.)

Many trials require participants to undergo additional procedures, tests and assessments based on the study protocol. These will be described in the informed consent document for a particular trial. A potential participant should also discuss these issues with members of the research team and with his or her usual health care provider.

A clinical study is conducted according to a research plan known as the protocol. The protocol is designed to answer specific research questions as well as safeguard the health of participants. It contains the following information:

• The reason for conducting the study
• Who may participate in the study (the eligibility criteria)
• The number of participants needed
• The schedule of tests, procedures or drugs and their dosages
• The length of the study
• What information will be gathered about the participants

Clinical studies have standards outlining who can participate, called eligibility criteria, which 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.

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. These are based on variables such as age, gender, the type and stage of a disease, previous treatment history and other medical conditions.

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 might be helpful during such a discussion. Answers to some of these questions are provided in the informed consent document. Many of these questions are specific to clinical trials, but some also apply to observational studies.

• What is being studied?
• Why do researchers believe the intervention being tested might be effective? Why might it not be effective? Has it been tested before?
• What are the possible interventions that I might receive during the trial?
• How will it be determined which interventions I receive (for example, by chance)?
• Who will know which intervention I receive during the trial? Will I know? Will members of the research team know?
• How do the possible risks, side effects and benefits of this trial compare with those of my current treatment?
• What will I have to do?
• What tests and procedures are involved?
• How often will I have to visit the hospital or clinic?
• Will hospitalization be required?
• How long will the study last?
• Who will pay for my participation?
• Will I be reimbursed for other expenses?
• What type of long-term follow-up care is part of this trial?
• If I benefit from the intervention, will I be allowed to continue receiving it after the trial ends?
• Will results of the study be provided to me?
• Who will oversee my medical care while I am in the trial?
• What are my options if I am injured during the study?