Before an investigational medication, device or procedure is approved or cleared by the FDA/Regulatory Authority for general use, it must be shown to be both safe and effective.
This is accomplished through clinical research studies, also called clinical trials. Clinical research studies are carefully designed and monitored research studies intended to test and evaluate these investigational medications, devices or procedures. They answer important questions, such as:
Clinical research studies are regulated by government agencies to ensure the highest standards are being met. All potential new medications must be tested in a series of clinical research studies before they can be prescribed by doctors.
People who participate in clinical research studies volunteer for many reasons. Some of these are:
Clinical research studies are divided into different phases. Each phase is designed to collect specific information about the investigational medication or treatment.
The first human tests of investigational medications or treatments are in Phase 1 studies. Phase 1 studies are designed to determine the most appropriate dose of the investigational medication and to check for any potential side effects. These studies usually involve a small number of participants. Because Phase 1 studies use investigational medications that have never been tested in humans, they may involve significant risks.
Phase 2 studies test the investigational medication in a larger group of patients to evaluate the effectiveness of the medication on the disease being studied and to confirm the safety data established in the Phase 1 study.
In Phase 3 clinical research studies, basic information is known about the investigational medication from results collected in earlier studies. Phase 3 studies test the safety and how well the investigational medication works in hundreds or even thousands of participants. Phase 3 studies also often compare the investigational medication to a placebo (no active medication) or an existing standard treatment.
Learn more about the BTKi-SLE Study