Clinical research is research conducted with human subjects, or material of human origin, in which the researcher directly interacts with human subjects. Clinical research helps doctors and researchers to find new and better ways to understand, detect, control, and treat illness. A clinical research study is a way to find answers to difficult scientific or health questions. For example, the study might explore the best ways to treat people with colon cancer. By studying cancer cells from patients, researchers may be able to determine the specific genetic mutations (changes in gene sequence) that caused the normal, healthy cells to become cancerous, and may help doctors decide on the best drugs to prescribe or surgeries to perform. Clinical research today may help other doctors in the future screen their healthy patients before they ever develop cancer.
All clinical studies are based on a set of rules or directions called a protocol. A protocol describes what types of people are eligible to participate in the study; determines the schedule of tests, procedures, medications, and dosages; and sets the length of the study.
A clinical Trial is a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.
Clinical Trials are at the heart of all medical advances. They're conducted with the help of human volunteers and aim to find out if a new drug, surgical procedure, medical device or new way of using an existing treatment is safe and effective.
Clinical Trials follow strict ethical and legal codes and regulations to protect the safety and rights of volunteers. These rules are set and enforced by individuals and groups at the sites where the clinical Trials take place. Such groups include an Institutional Review Board and government agencies such as the Food and Drug Administration and the Office of Human Research Protections.
Clinical Trials of experimental drugs proceed through four phases:
In many clinical Trials, one group of patients will be given an experimental drug or treatment, while a control group is given either a standard treatment for the illness, or a placebo (a harmless "fake" drug), or no treatment at all.
In a "blinded" or "masked" study, participants do not know whether they are getting the drug being tested, or whether they are in the control group. The goal is to prevent the so-called "placebo effect" from influencing the results of the experiment. The placebo effect is the phenomenon of patients feeling better simply because they think they are receiving a helpful drug or treatment.
Sometimes, clinical Trials are "double-blind" or "double-masked." That means that neither the participants, nor the study staff members, know who is receiving the experimental drug and who is in the control group. Studies are performed in this way so that neither the patients' nor the doctors' expectations about the experimental drug can influence the observations and results.
Clinical research is a vital part of finding new treatments and cures for diseases. Carefully conducted clinical studies are the fastest way to find treatments that are safe and effective. By volunteering for a clinical study, you would be participating in research that may result in a new treatment for a deadly or debilitating disease.
Without volunteers, we wouldn't have many of the medicines and scientific discoveries we enjoy the benefits of today. Clinical Trials offer you an opportunity to help scientists find better ways of preventing and treating diseases, improving health for all. By volunteering in a clinical Trial, you or someone you love could:
Before you agree to participate in a study, you must be given complete information about the study, known as "informed consent." Informed consent involves two essential components: a document and a process. The informed consent document gives a summary of the research project (including the study's purpose, research procedures, potential benefits and risks, etc.) and explains the individual's rights as a research participant. This document is part of an informed consent process, which consists of conversations between the research team and the participant, and may include other supporting material such as study brochures. The informed consent process provides research participants with ongoing explanations that will help them make informed decisions about whether to begin or continue participating in the research project (See: Informed Consent for Genomics Research). The Food and Drug Administration (FDA) provides details about informed consent with the information page: Informed Consent for Clinical Trials [fda.gov].
Because participating in a clinical study is an important decision, there are many questions that you should consider before agreeing to participate. The Centers for Disease Control and Prevention (CDC) have prepared a list of questions to help you get the information you need to make a decision about participating: Taking Part in Research Studies: What Questions Should You Ask? [cdc.gov].
All clinical Trials have guidelines about who can participate, called inclusion and exclusion criteria. Both sets of criteria are based on factors such as age, gender, the type and stage of a disease, previous treatment history and other medical conditions.
Before joining a clinical Trial, a participant must qualify for the study. Some studies look for participants with illnesses or conditions to be studied in the clinical Trial, while others need healthy volunteers. Some studies need both types of volunteers.
Inclusion and exclusion criteria are not used to reject people personally; rather, the criteria are used to see if the study is a good fit for participants, keep them safe and help ensure scientists can find the information they need.
In some studies, volunteers who are in good health are needed to test a new drug or device that has never been given to people or to only a few people. In clinical Trials, these are often called "healthy volunteers." Though it can be hard to know what "healthy" means, a healthy volunteer is typically a person who is free from the disease or condition being studied or who has no major health problems.
While participating in a clinical Trial, a healthy volunteer is given a drug not to test if it will treat a disease, but rather to build new knowledge about the drug and to see if people can safely use it. Results from healthy volunteers are compared to results from patient volunteers who do have the disease or condition.
A patient volunteer has a known health problem and participates in a study to better understand, diagnose, treat or cure that disease or condition. Like healthy volunteers, patient volunteers may participate in studies testing drugs, devices or interventions designed to prevent or treat disease.
In some studies, patient volunteers may be put into a group to take an inactive drug called a placebo. Placement in placebo groups is random, so each volunteer has an equal chance of getting either the active drug or the placebo.
For most studies, neither you nor your doctor will know if you are getting the drug or the placebo. This is to prevent any bias in the study results.
Here are some questions to ask your doctor to help you decide if you want to take part in a clinical Trial:
Some treatments that are being tested may have side effects that can be unpleasant, serious or even life-threatening. Because the treatments being studied are new, doctors don't always know what the side effects will be. Many side effects last a short time and go away when the treatment is stopped. Others can be long-lasting or permanent. Some side effects appear during treatment, while others may not show up until after the treatment is over. The risks depend on the treatment being studied, and all known risks should be fully explained to you by the scientists in advance.
To help you decide if you want to be in a study, the study team is required to go through key facts about a clinical Trial with you. This process is called informed consent.
With the help of a document called the informed consent form, the study team will explain the details of the study to you and answer any questions you might have about the study and your participation in it. During informed consent, the study team should disclose:
This process also continues throughout a study, so this information is constantly available to participants.
If you don't understand the informed consent process or forms, be sure to ask the doctor or another medical person to explain it. Make sure you understand all of it before you agree to be in the study. It's okay to ask questions.
Before you can take part in the study, you must sign the informed consent form to indicate you have been given this information and understand it. The informed consent form is not a contract. You can leave the study at any time, for any reason.
Each clinical Trial in the United States must be approved and monitored by an Institutional Review Board (IRB) to ensure that the risks are minimal and are worth any potential benefits. An IRB is an independent committee that consists of physicians, statisticians and members of the community who ensure that clinical Trials are ethical and that the rights of participants are protected. Federal regulations require all institutions in the United States that conduct or support biomedical science involving people to have an IRB initially approve and periodically review the study.
These committees are used mainly when one treatment is being compared to another. These committees are particularly important in tests of treatments for serious or life-threatening disease. These experts review information from studies to make sure they are being done in a way that is safest for the volunteers. During a study, if the committee finds that the treatment is harmful or of no benefit, it will stop the study. If a study shows that one treatment works better than another, the committee stops the study and all volunteers are offered the better treatment.
Next steps vary from study to study. You might be allowed to continue taking the study medication if you had a good response to it during the study, or you might be given a chance to take the study medication if you didn't receive it. Most often, participation ends when the study ends because full safety information is not yet known.
Participants may also be given results from the study. Ask the study team members if the results have been or will be published. You can also find published study results by searching for the official name or protocol ID number in the National Library of Medicine's PubMed database. After a clinical Trial is over, scientists carefully look at the information collected during the study to determine the drug's effectiveness, if it is safe and if there are any side effects. Depending on the results, scientists then decide whether to stop or continue testing the new drug or treatment.
If scientists find the results are medically important, they may submit them to journals or scientific meetings for review by others in their field.
Results are also shared with government agencies in charge of the approval for new drugs, devices and treatments, such as the FDA. If a drug is approved, pharmaceutical companies may continue to conduct studies that compare the new drug's safety, effectiveness and cost to other drugs already on the market or assess its long-term effectiveness and impact on quality of life.
Once a new approach has been shown to be safe and effective in a clinical Trial, it may become the standard of medical practice.