Every single over the counter medication, prescription medication, surgical device, cancer therapy, diagnostic test – basically anything that can be ingested by, or performed on, a human patient – has resulted from clinical research. 

Clinical research leads to new and better ways of caring for patients that aim to improve outcomes, lead to less pain or discomfort, involve shorter recovery times, and much more..

Studies that are designed to test potential new therapies are called clinical trials. These trials are overseen by medical doctors (called investigators) and support staff working in the field of clinical research. By understanding and comparing the experiences of trial participants, clinical researchers can demonstrate if and how a potential new therapy is effective in meeting stated objectives (called endpoints).

Clinical trials enable the medical and scientific communities to study the treatment, diagnosis, and prevention of illness, under clearly defined rules and regulations to protect trial participants. Clinical trials have many different objectives. The evaluation of drugs, devices, or procedures for the treatment of a medical condition is a primary reason trials are conducted. Clinical trials also look at approaches to prevent illness (such as a vaccine or lifestyle change), tools to diagnose illness (such as a new blood test), and approaches to identify personal risk factors for illness (such as genetic causes).

Each clinical trial uses a comprehensive research plan that outlines and guides every aspect of the trial; this is called a protocol. A trial protocol outlines the reason for the trial, the criteria that participants must match, the number of participants, the schedule of visits and events over a defined timeframe, the length of the trial, the information or data to be gathered, and the safeguards in place to protect participants.

Besides the primary investigator (typically a medical doctor), there are a number of supporting clinical and professional staff that help manage clinical trials. These teams might look very much like those you interact with at your regular physician’s office: nurses, lab technicians, administrative support members, and social workers.

The group funding a clinical trial is commonly referred to as a sponsor. Sponsors may be pharmaceutical companies, though academic medical centers, patient advocacy groups and federal agencies such as the National Institutes of Health or National Cancer Institute may also be involved in directing clinical trials.

Clinical trials are usually held at research sites. The sites may be independent, or they may be part of a physician practice, hospital, university, or community-based clinic. Some trials may be held in a virtual or at-home setting depending on the protocol requirements.

Each clinical trial is described in terms of what “phase” of therapeutic discovered it represents. Phase I trial: A therapy is evaluated in a small group of participants to learn about safety and possible side effects. Phase II trial: A therapy is evaluated in a larger group of participants to learn about its effectiveness and more about its safety profile. Phase III trial: The effectiveness of a therapy, along with evaluation of side effects and comparison to existing treatments or approaches, is looked at in a very large universe of participants. Phase IV trial: This happens after a therapy is approved and made available on a widespread basis; the purpose is to learn more about its safety and benefit.

Clinical trials must follow strict rules and undergo rigorous review. In terms of oversight, clinical trials are carefully monitored by various regulatory agencies such as the Food and Drug Administration (FDA) in the United States. Additionally, every clinical trial protocol must be assessed and approved by an “Institutional Review Board” or IRB, prior to participant recruitment. An IRB, which may also be referred to as an Ethics Committee, is a panel of scientists, doctors, community members and other experts that are charged with ensuring that the rights, safety, and well-being of participants are at the forefront of every clinical trial. This group seeks to independently ensure that trial protocols minimize overall risk to participants.

In a trial that is evaluating a potential new therapy against a standard therapy, the research team will want to know how it compares to the existing approach – better, worse or same outcomes. In order to do this, a placebo may be utilized. This is an inactive product that offers no therapeutic value despite looking like the product being investigated. A placebo will not be used in the event it puts a participant at risk (such as in the case of serious illness). You will be notified if placebos are part of a trial design.