Clinical trials are conducted in “phases.” Each phase has a different purpose and helps researchers answer different questions.
- Phase I trials: Researchers test an experimental drug or treatment in a small group of people (20–80) for the first time. The purpose is to evaluate its safety and identify side effects.
- Phase II trials: The experimental drug or treatment is administered to a larger group of people (100–300) to determine its effectiveness and to further evaluate its safety.
- Phase III trials: The experimental drug or treatment is administered to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, compare it with standard or equivalent treatments, and collect information that will allow the experimental drug or treatment to be used safely.
- Phase IV trials: After a drug is approved by the FDA and made available to the public, researchers track its safety, seeking more information about a drug or treatment’s risks, benefits, and optimal use.
Some Concepts to Understand
Typically, clinical trials compare a new product or therapy with another that already exists to determine if the new one is as successful as, or better than, the existing one. In some studies, participants may be assigned to receive a placebo (an inactive product that resembles the test product, but without its treatment value).
Comparing a new product with a placebo can be the fastest and most reliable way to demonstrate the new product’s therapeutic effectiveness. However, placebos are not used if a patient would be put at risk — particularly in the study of treatments for serious illnesses — by not having effective therapy. Most of these studies compare new products with an approved therapy. Potential participants are told if placebos will be used in the study before they enter a trial.
Randomization is the process by which two or more alternative treatments are assigned to volunteers by chance rather than by choice. This is done to avoid any bias with investigators assigning volunteers to one group or another. The results of each treatment are compared at specific points during a trial, which may last for years. When one treatment is found superior, the trial is stopped so that the fewest volunteers receive the less beneficial treatment.
In single-or double-blind studies, also called single- or double-masked studies, the participants do not know which medicine is being used, so they can describe what happens without bias. “Blind” (or “masked”) studies are designed to prevent members of the research team or study participants from influencing the results. This allows scientifically accurate conclusions. In single-blind (“single-masked”) studies, only the patient is not told what is being administered. In a double-blind study, only the pharmacist knows; members of the research team are not told which patients are getting which medication so that their observations will not be biased. If medically necessary, however, it is always possible to find out what the patient is taking.