The efficacy of the drug on the target population is the focus of this phase. The goal is to establish safety and tolerance, including gaining evidence of efficacy and determining kinetics. Several hundred volunteers may be used during this phase of study. Each study may last several months to 2 years.
Several thousand volunteers from the target population are required for testing at this phase. These studies are designed to establish the safety of the drug in a larger population and to further establish efficacy. Phase III trials are usually large, multi-center, randomized trials. One group of patients will be randomly assigned the experimental drug, while a second “control” group will be assigned to receive a standard treatment or placebo. Often these studies are “double-blinded”, meaning neither the patients nor the researchers know who is receiving the experimental drug. In this manner, the study can provide the pharmaceutical company and the FDA comparative information about the relative safety and efficacy of the drug under study. Phase III studies may last several years.
Phase III ½
This phase uses Phase III procedures to acquire additional safety and efficacy data and to anticipate and answer FDA inquiries during the approval process.
Patient selection Process
Once our site is selected by the sponsor, we begin to tell patients about our new protocol to see if they qualify and are interested. Each patient is carefully informed of the risks and benefits of the program.
Detailed information is provided that they can review at their leisure either in our office or at home. Questions are strongly encouraged, both from the patient themselves and from family members. No research program is ever a contract.
Patients are free to discontinue participation at any time for any reason. The only thing we usually ask is one last visit to make sure that the patient is healthy before ending the program, but even that is optional. If a patient decides that they no longer want to continue, it does not impact their relationship with our physicians.