Clinical research studies are done to test investigational medications for diseases and conditions. Studies help determine if investigational medications are safe to use, if they work to improve the health of people, and which doses work best. Before any medication can be approved and made available to the general public, it has to go through several phases of clinical research.

Clinical research studies on people are only started after significant research has already been done on an investigational medication or device. The study medication is considered investigational until it has passed all phases of clinical research successfully and has been approved by the Food and Drug Administration (FDA):

  • Phase I: Researchers test a new drug or treatment in a small group of people for the first time to evaluate safety, effective doses, and identify side effects.
  • Phase II: The drug or treatment is given to a larger group of people to further evaluate its safety and effectiveness.
  • Phase III: The drug or treatment is given to large groups of people to confirm its effectiveness, evaluate side effects, and compare it to standard of care.
  • Phase IV: Studies are done after the investigational medication has been marketed to gather information on the drug's effect in the general population after long-term use.

Why do people participate in clinical research studies?

People participate in clinical research studies for a variety of reasons. Some volunteers may participate in studies because they want to learn more about their disease and have access to an investigational therapy that may be a potential new treatment option. Others volunteer to participate because they want to help researchers learn more about a disease so that it may help them and others in the future.

Are clinical research studies safe?

Clinical research studies follow a specific set of standards and are closely regulated to ensure the safety of all participants. Safety precautions are put in place to protect people who participate in clinical research. In addition, studies follow a written plan that is called a protocol. The protocol is designed to make sure all study procedures are conducted correctly and safely.

The risks of a clinical research study vary depending on the particular study. Some investigational medications being studied can have unpleasant or even serious side effects. Before a potential new treatment is tested in a clinical research study with people, it will have already been studied in laboratories in great detail. However, no treatments are entirely without risk, including those treatments already used by doctors. Clinical research studies help researchers and doctors decide whether the benefits of a potential new treatment option outweigh its potential side effects.

Before you participate in the study, all of the known study risks will be fully explained to you by the study team during the informed consent process. If additional risks are discovered later during the study, the study staff will inform you of those risks as well.

How does a clinical research study work?

Clinical research studies follow a specific set of standards and are closely regulated. In addition, studies follow a written plan that is called a protocol. The protocol is designed to make sure all study procedures are conducted correctly and safely. At the beginning of the study, participants go through the informed consent process to make sure they understand the study requirements and potential risks before agreeing to participate. Each participant's health history and current status will be checked by the study doctor and staff at the beginning of the clinical research study. Throughout the study, each participant will be monitored carefully by the study doctor and staff. What data is collected during a clinical research study? In a clinical research study every effort is made to protect your private information. Your results are assigned a random number that is used by the study team to analyze the data collected from your participation in the study.

What data is collected during a clinical research study?

In a clinical research study every effort is made to protect your private information. Your results are assigned a random number that is used by the study team to analyze the data collected from your participation in the study.

It is critical for all study participants to remain in the study throughout the study duration in order to have complete data. Accurate analysis about the investigational medication requires the availability of complete data on each participant. Every effort is also made to select a number of participants that is large enough to generate an amount of data that can be said to be representative or predictive of results in the general population.

Before any investigational medication can be marketed, regulatory bodies analyze whether the investigational medication is safe and effective. Complete and accurate data is a factor in this analysis of the investigational medication.

What is the informed consent process?

Informed consent is a process through which people learn the important facts about the clinical research study to help them decide whether to participate. Because many clinical trials involve new medications or test medications in new ways, there are risks associated with participation. It is important that people understand these risks before agreeing to participate in a study. The possible benefits and risks of participating in a particular study should be discussed with a doctor. Potential participants are also encouraged to talk to a doctor about any study-related questions or concerns they may have. If a person decides to become a study participant, he or she will be asked to sign an informed consent form. Participants will each receive a copy of the informed consent form to take home with them and refer to throughout the study.