WHAT IS A CLINICAL TRIAL? 

A clinical trial is a research study that includes people like you. Scientists do these trials to find new or better ways to prevent, detect, or treat diseases. They want to make sure treatments are safe and work well. In the controlled circumstances of a clinical trial, the measure of how well the treatment works is called efficacy. Clinical trials are important because they help improve currently available treatments and discover new ones.

Without clinical trials, many of the medicines and cures we have today wouldn’t exist. These trials often begin in a lab. After scientists test new treatments on animals, they move on to clinical trials with people. As trials go through different stages, or phases, researchers learn more about the treatment, its risks, and its benefits

Types of Clinical Trials

There are different types of clinical trials. Each type has a specific goal:

Behavioral trials: Study ways to change behaviors to improve health.

Diagnostic trials: Test new methods to find and diagnose diseases.

Prevention trials: Look for ways to stop diseases before they happen or stop them from coming back. These trials might study medicines, vaccines, or lifestyle changes.

Quality-of-life trials: Find ways to make life more comfortable for people with illnesses.

Screening trials: Test new ways to detect diseases.

Treatment trials: Test new medicines, medicine combinations, or other treatments to find the best option. Most trials that involve patients with fungal disease are treatment trials.

Sometimes, scientists do observational studies instead of clinical trials. In these studies, they don’t give any specific treatment or change people’s care. Instead, they observe what happens naturally. There are no control or treatment groups in these studies.

Comparative Effectiveness Research. Effectiveness is a measure of how a treatment works in a real-world setting. Comparative effectiveness research looks at different treatment options to figure out which ones work best for different people. It helps find out which treatments give the most benefits and the least harm. The main question is: which treatment works best, for whom, and in what situation?

Clinical trials are how we find better ways to treat diseases. They are a critical part of medical progress and may be an option for people looking for the best care.

What Happens in the Phases of a Clinical Trial?

Clinical trials happen in steps, called phases. Each phase helps researchers learn something new about the treatment:

Phase I: Tests a new treatment on a small group of people to find the safest dose and check for side effects.

Phase II: Studies the treatment on a larger group to see if it works and to gather more safety information.

Phase III: Tests the treatment on an even bigger group to compare it to standard treatments, monitor side effects, and collect data to use the treatment safely.

Phase IV: After the U.S. Food and Drug Administration (FDA) approves a treatment, researchers keep studying it to learn more about its safety, benefits, and best use in the general population.

Science Sidebar:

The Science of Safety Measurement in Clinical Trials

Determining the side effects of new treatments is a difficult process that includes documenting every change in a patient’s health and determining if it may have been caused by the treatment. These side effects are called adverse events (AE) and are often graded on a scale of 0 to 5:

Grade 0: No problem or symptoms

Grade 1: Mild AEs that are generally not bothersome. For example, this could be a skin reaction such as a rash that is mild and involves a small area of skin.

Grade 2: Moderate AEs that interfere with some activities. An example is an itchy rash that burns and is uncomfortable enough to keep you from driving.

Grade 3: Serious AEs that interfere with basic activities. They often require medical treatment. For instance, this could be a rash that covers larger body surfaces, is very itchy, and keeps you from sleeping.

Grade 4: Life-threatening AE, often requiring hospitalization. This could be an extensive rash that is blistering. Such a skin condition could lead to dehydration with the loss of the protective skin barrier.

Grade 5: AEs that are fatal.

Studies are designed to have enough people to demonstrate the efficacy of the treatment. Sometimes, rarer side effects associated with the treatment do not show up when the treatment is studied in trials, which typically involves less than 1000 people (in mycology anyway). Then, when the treatment is used in large numbers of people in the real world, these rare side effects may show up. That’s why postmarketing surveillance programs are used to monitor the safety of treatments once they are approved and used in larger groups of people.