Clinical Research: What Is It?
Your doctor may have said that you are eligible for a clinical trial, or you may have seen an ad for a clinical research study. What is clinical research, and is it right for you?
Clinical research is the comprehensive study of the safety and effectiveness of the most promising advances in patient care. Clinical research is different than laboratory research. It involves people who volunteer to help us better understand medicine and health. Lab research generally does not involve people — although it helps us learn which new ideas may help people.
Every drug, device, tool, diagnostic test, technique and technology used in medicine today was once tested in volunteers who took part in clinical research studies.
At Johns Hopkins Medicine, we believe that clinical research is key to improve care for people in our community and around the world. Once you understand more about clinical research, you may appreciate why it’s important to participate — for yourself and the community.
What Are the Types of Clinical Research?
There are two main kinds of clinical research:
Observational studies are studies that aim to identify and analyze patterns in medical data or in biological samples, such as tissue or blood provided by study participants.
Clinical trials, which are also called interventional studies, test the safety and effectiveness of medical interventions — such as medications, procedures and tools — in living people.
Clinical research studies need people of every age, health status, race, gender, ethnicity and cultural background to participate. This will increase the chances that scientists and clinicians will develop treatments and procedures that are likely to be safe and work well in all people. Potential volunteers are carefully screened to ensure that they meet all of the requirements for any study before they begin. Most of the reasons people are not included in studies is because of concerns about safety.
Both healthy people and those with diagnosed medical conditions can take part in clinical research. Participation is always completely voluntary, and participants can leave a study at any time for any reason.
“The only way medical advancements can be made is if people volunteer to participate in clinical research. The research participant is just as necessary as the researcher in this partnership to advance health care.”Liz Martinez, Johns Hopkins Medicine Research Participant Advocate
Types of Research Studies
Within the two main kinds of clinical research, there are many types of studies. They vary based on the study goals, participants and other factors.
A biorepository is a facility that collects, catalogs and stores specimens of biological material such as blood, tissue, or cells to be used for laboratory studies. A person’s medical information and/or written consent to use the samples in research may go along with the samples to be studied.
People with no known major health issues — also called normal volunteers — are needed for some studies. For new medications, it is safer and easier to start with healthy individuals. These studies look at how the study drug affects people and how it is absorbed and removed from the body.
Surveys are commonly used to study health behaviors, practices and opinions.
Clinical trials study the safety and effectiveness of interventions and procedures on people’s health. Interventions may include medications, radiation, foods or behaviors, such as exercise. Usually, the treatments in clinical trials are studied in a laboratory and sometimes in animals before they are studied in humans. The goal of clinical trials is to find new and better ways of preventing, diagnosing and treating disease. They are used to test:
Drugs or medicines
New types of surgery
New ways of using current treatments
New ways of changing health behaviors
New ways to improve quality of life for sick patients
Because every clinical trial is designed to answer one or more medical questions, different trials have different goals. Those goals include:
Treatment trials test the safety and effectiveness of new treatments, such as medications, surgical procedures, and new ways to combine existing treatments.
Prevention trials study health interventions such as vaccines, medications and lifestyle practices, and habits that physicians think may affect risk of disease. These studies look for the best ways to prevent illness or a return of illness, often in people who are at high risk for developing the condition.
Screening trials look for the most effective ways to detect, diagnose and monitor disease. For example, studies of mammography show that women of a certain age who had mammograms were less likely to die of breast cancer. These trials are important for developing ways to detect a disease or condition in its earliest stage, when it is most treatable.
In general, a new drug needs to go through a series of four types of clinical trials. This helps researchers show that the medication is safe and effective. As a study moves through each phase, researchers learn more about a medication, including its risks and benefits.
Is the medication safe and what is the right dose? Phase one trials involve small numbers of participants, often normal volunteers.
Does the new medication work and what are the side effects? Phase two trials test the treatment or procedure on a larger number of participants. These participants usually have the condition or disease that the treatment is intended to remedy.
Is the new medication more effective than existing treatments? Phase three trials have even more people enrolled. Some may get a placebo (a substance that has no medical effect) or an already approved treatment, so that the new medication can be compared to that treatment.
Is the new medication effective and safe over the long term? Phase four happens after the treatment or procedure has been approved. Information about patients who are receiving the treatment is gathered and studied to see if any new information is seen when given to a large number of patients.
“Johns Hopkins has a comprehensive system overseeing research that is audited by the FDA and the Association for Accreditation of Human Research Protection Programs to make certain all research participants voluntarily agreed to join a study and their safety was maximized.”Gail Daumit, M.D., M.H.S., Vice Dean for Clinical Investigation, Johns Hopkins University School of Medicine
Is It Safe to Participate in Clinical Research?
There are several steps in place to protect volunteers who take part in clinical research studies. Clinical Research is regulated by the federal government. In addition, the institutional review board (IRB) and Human Subjects Research Protection Program at each study location have many safeguards built in to each study to protect the safety and privacy of participants.
Clinical researchers are required by law to follow the safety rules outlined by each study's protocol. A protocol is a detailed plan of what researchers will do in during the study.
In the U.S., every study site's IRB — which is made up of both medical experts and members of the general public — must approve all clinical research. IRB members also review plans for all clinical studies. And, they make sure that research participants are protected from as much risk as possible.
Earning Your Trust
This was not always the case. Many people of color are wary of joining clinical research because of previous poor treatment of underrepresented minorities throughout the U.S. This includes medical research performed on enslaved people without their consent, or not giving treatment to Black men who participated in the Tuskegee Study of Untreated Syphilis in the Negro Male. Since the 1970s, numerous regulations have been in place to protect the rights of study participants.
Many clinical research studies are also supervised by a data and safety monitoring committee. This is a group made up of experts in the area being studied. These biomedical professionals regularly monitor clinical studies as they progress. If they discover or suspect any problems with a study, they immediately stop the trial. In addition, Johns Hopkins Medicine’s Research Participant Advocacy Group focuses on improving the experience of people who participate in clinical research.
Clinical research participants with concerns about anything related to the study they are taking part in should contact Johns Hopkins Medicine’s IRB or our Research Participant Advocacy Group.