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How Clinical Trials Work

Medical progress starts with keeping you safe

A clinical trial (also called clinical study) is scientific research designed to test if a medicine or medical device is effective and well tolerated for use by people.

From idea to medicine cabinet: the drug discovery process

Most ideas for a new medicine or treatment start in a laboratory. After researchers test the product in the laboratory and in animal studies, the most successful treatments move on to clinical research, where they are tested in people who volunteer to be study participants. 

Throughout the study, information is collected to answer specific research questions.

Is it safe?

Participant safety is a top priority. Researchers evaluate the drug/device to see if it is safe and how well it is tolerated, while documenting any reported side effects.

Does it work?

The drug/device is evaluated to see how much is needed and how well it works compared to either a placebo or the current standard treatment, if one exists.

Who's it for?

 The trial is used to define categories of patients the investigational product is most appropriate for treating, including type of disease, severity and other criteria.

What can we learn?

Trials can help researchers gain a better understanding of how a disease affects not only the human body, but also people’s lives.

Clinical trial phases

Every prescription medicine available in the U.S. must go through a clinical trial process. The process includes different phases of research. The studies may take place at a hospital, doctors’ office or clinical research center and are conducted by doctors, nurses and other health care team members.

PhaseI
Researchers test the study drug on a group of healthy volunteers for the first time to evaluate its safety and identify side effects. In some disease states, such as cancer, patients will be studied in place of healthy volunteers.
PhaseII
The study drug is given to a larger group of patient volunteers with a specific disease or condition to determine an optimal dose, identify short-term side effects, and test its safety and effectiveness compared to placebo or other treatment.
PhaseIII
The study drug is given to patient volunteers to monitor side effects and confirm its effectiveness compared to other possible treatments.
FDA
Submission
Once the drug successfully completes Phases 1-3, laboratory and animal research data and the trial results are submitted to the U.S. Food and Drug Administration (FDA) for review to ensure that the drug or device works and does no unexpected harm. Only after FDA approval can the medicine be prescribed by doctors for use by their patients.
PhaseIV
After the medicine is approved by the FDA and made available for treatment by a doctor’s prescription, real-world studies continue for an extended time. The drug is studied in large groups of patients to monitor for rare, long-term side effects, evaluate its use in combination with other treatments and explore new categories of patients that may benefit from the medicine.

Participant health is a priority

The safety and well-being of study participants are top priorities for the sponsors, doctors, nurses and research professionals who conduct clinical trials. If you choose to join a study, your health will be closely monitored and your care will be adjusted if the medical team thinks it’s in your best interest. If you decide at any point that you wish to end your study participation, you may do so without providing a reason.

Independent groups help ensure quality standards and safety

Throughout the entire clinical trial experience, many people provide trial oversight to safeguard study participants.  These include the sponsoring organization that develops the study plan and funds the research, the doctors and nurses who provide care during the study and an independent committee called the Institutional Review Board (IRB). 

Study Sponsor

Medical institutions, pharmaceutical companies, federal agencies or others that fund the research study to see if the investigational product is safe and effective.

Research Facility

Doctors’ offices, hospitals, university medical centers and clinical research centers that enroll study participants and conduct the clinical trial on behalf of the study sponsor.

Institutional Review Board (IRB)

Independent committee of doctors, statisticians, and members of the community who ensure the clinical trial is ethical and the rights of the study participants are protected.

Learn all you can before you enroll

If you’re thinking about joining a clinical trial, you have the right to ask the study staff any questions you have before you ever enroll. The study team wants you to understand your role and what you can expect as a participant.

What to Expect