What is a clinical trial?
Clinical trials help the medical community discover possible ways to help treat disease.
They help us answer important questions about investigational medicines, such as:
- How does this medicine work?
- How will it affect certain disease or condition?
- Is this medicine safe?
To find the answers to questions like these, we need people to take part in the trials.
Thousands of volunteers all around the world participate in clinical trials every year; they decide to do it for many different reasons.
Phases of a clinical trial
There are several steps to the clinical trial process, called “phases.” Each phase is designed to answer specific questions that help the researchers decide whether or not the investigational medicine should continue in development.
Phase 1 is the first step, and the primary goal of this phase is to determine the safety of the investigational medicine being tested. Phase 1 is usually conducted in a small group of healthy volunteers but can also include patients with the disease for which the investigational medicine is being tested.
If Phase 1 trials are successful, the investigational medicine moves to Phase 2. Phase 2 trials are designed to evaluate the efficacy of an investigational medicine in the patients with the disease for which the investigational medicine is being tested. In addition, the Phase 2 trial will continue to assess the safety of the investigational medicine. These trials are often slightly larger than Phase 1 trials.
If the Phase 2 trials are successful, the investigational medicine moves to Phase 3. The objective of Phase 3 is to confirm the efficacy and safety findings of Phase 2 in an even larger group of patients with the disease for which the investigational medicine is being tested. If there is a standard of care treatment for the disease being studied, the new investigational medicine will often be compared to the efficacy and safety of that standard of care treatment. Regulatory agencies use the findings from all of these phases to decide whether the investigational medicine should be approved for use in a specific disease indication outside of clinical trials.