What Are Clinical Trials, and Why Are They Important?

Clinical trials are a type of research study that tests a new treatment, medical procedure or device, collectively known as "interventions." These trials are designed to determine if a new intervention is safe, effective, and beneficial compared to existing care.

The United States Food and Drug Administration (FDA) reviews each intervention for safety and effectiveness, weighing the benefits against the risks.1 Every FDA-approved drug, medical device, or therapy available today has gone through this rigorous process. As a result, the language "FDA-Approved" means something (in the U.S.).

Clinical trials and the people who choose to participate make this possible.

How Are Participants Protected During Trials?

Safety and fairness are of the utmost importance in trials. For this reason, this type of study is carefully reviewed and monitored by oversight groups.

In the United States, these include the Food and Drug Administration (FDA) and Institutional Review Boards (IRBs).2 These groups evaluate the design of a clinical trial and it's risks, benefits, and ethics before a study begins.

While researchers cannot eliminate all risk from clinical trials, they are expected to run their studies as safely as possible while closely monitoring the health of participants.

How Are Clinical Trials Different from Standard Medical Care?

While the goal of general medical care is to help an individual patient feel better, clinical trials advance science.

For this reason, standard medical care is built around an individual's needs, risk factors, and treatment preferences. However, clinical trials are built around protocols that researchers establish to help them compare results at the end of each study. A protocol is a detailed set of rules that outlines exactly how the study will run and what participants will do.

While in a clinical trial, you may be required to keep logs that allow researchers to objectively compare participant experiences. Participants may also interact with the researchers more frequently than they would with their regular doctor as a patient.

The study's researchers must stay aware of your experiences during their trial to maintain safety and consistency. The strength of this standardization allows them to determine with relative confidence how a treatment will impact the general population.

Even while enrolled, participants are encouraged to keep seeing their regular doctors for non-study-related care. This ensures your overall health is continuously monitored outside the scope of the trial.

The General Process of a Clinical Trial

While details vary from study to study, most follow the same general process. Researchers design each step of the clinical trial process to protect your health while ensuring researchers can gather reliable information.

Recruitment

To find participants, researchers may share information about the trial through medical centers, online databases, or referrals from doctors.

For example, you might find out about a trial that interests you on clinicaltrials.gov.

Interested individuals learn what the study is about, what kind of participants are needed, and what the commitment will look like. Some trials are performed in labs or a central location. Others are managed through remote methods such as video chat or phone.

Informed Consent

Before joining, participants receive a detailed explanation of the trial. This includes the purpose, what will happen during the study, potential risks and benefits, and any other important details. You’ll have time to ask questions before deciding whether to sign the consent form.

While commitments vary, researchers are looking for people who are willing to stick with them for the duration if at all possible. It's important to understand what's expected of you and for how long. Signing up doesn’t mean you give up your rights—you can leave the trial at any point.

Screening

Every trial has inclusion criteria that determines if an individual is a fit for the trial. These are factors that make you eligible. For example, they may want people with a specific condition or those who are generally healthy.

There are also exclusion criteria. These are factors that prevent participation, like certain medications or health risks that could make the study unsafe for you. 3

Screening may involve medical exams, lab tests, or a review of your health history. You may also be asked to undergo a wash-out period. This means you would safely reduce or stop certain medications for a time before the study starts.

Baseline Visit

If you qualify, you’ll attend a baseline appointment. This visit helps researchers understand your starting point before any treatment begins. They may:

• Take vital signs
• Perform physical exams
• Run lab work to establish a "baseline" for comparison later

Trial Participation

Once the study begins, participants follow the steps laid out in the protocol. You’ll also be asked to report symptoms, side effects, or concerns as they come up. Reporting even minor adverse changes is critical for helping them understand potential side effects that might occur in others.

Data Collection and Monitoring

Throughout the trial, participants may be required to go for regular visits, some of which may be remote. These may include:

• Physical exams
• Imaging
• Blood tests
• Interviews

Researchers use these check-ins to track both how well the treatment works and how safe it is. Independent safety boards often review the data at intervals to ensure participants are not being exposed to unnecessary risk.

How Long Do Clinical Trials Take?

Some studies last only a few months; others continue for several years. The timeline depends on the type of treatment being tested, the size of the participant group, and the specific research goals.

What Is a Placebo, and Why Are Placebos Used?

A placebo is a treatment that appears to be the real thing but contains no active ingredients. It could be a sugar pill, saline injection, or other harmless substitute designed to mimic the investigational treatment.

Placebos exist to help researchers rule out effects attributable to chance or the placebo effect. The placebo effect is a natural human bias in which a participant or researcher believes someone is getting better or experiencing side effects because that's what they expect the intervention to do.

In a placebo-controlled study, participants are divided into a control group (the placebo group) and a treatment or investigational group (those who get the real treatment).

With that said, not all clinical trials use a placebo. If withholding the treatment from a placebo group would put them at undue risk, researchers find other ways to control their study.

During the informed consent step, it's essential to ask about the possibility of receiving a placebo. You have the right to know.

What Does Random Assignment Mean, and Why Is It Important?

Random assignment is the process of placing participants into different groups within a trial by chance, rather than by choice. Special computer programs are often used to ensure assignments can produce reliable results.

From your perspective, this randomization can feel challenging. You don't know which group you'll be in when you sign up. Therefore, you must be comfortable with the fact that you may not receive the study drug. It's important to recognize that, regardless of the assignment, you play a crucial role in the success of a clinical trial.

What Does Blinding Mean in Clinical Trials?

Blinding is a method that allows researchers, participants, or both to remain unaware of whether a participant is in the control group or the intervention group. By not knowing, researchers can review data objectively regardless of their desired outcome. At the same time, the participant can report their experiences without knowing whether those experiences are due to a placebo effect or intervention.

Are Clinical Trials Right for You?

Clinical trials are one of the most important tools in modern medicine. They give researchers the necessary evidence to decide whether new treatments are safe and effective, while providing opportunities for participants to contribute directly to medical progress. To determine if clinical trials are right for you, ask questions. Be sure you understand what's expected and what to expect.

Sources

1. U.S. Food and Drug Administration. About FDA (Nov. 2024). Retrieved from https://www.fda.gov/media/151975/download
2. Grady, C. (2015). Institutional Review Boards: Purpose and Challenges. Chest, 148(5), 1148. Retrieved from https://pmc.ncbi.nlm.nih.gov/articles/PMC4631034/
3. U.S. Food and Drug Administration. Screening Tests Prior to Study Enrollment: Guidance for Institutional Review Boards and Clinical Investigators (Jan. 1998). Retrieved from https://www.fda.gov/regulatory-information/search-fda-guidance-documents/screening-tests-prior-study-enrollment