What is a clinical trial?
A clinical trial is a research study that looks at how well a new treatment works in people. This is called “efficacy.” The treatment may be a drug, medical procedure, medical device, or even a lifestyle change. A clinical trial also makes sure the new treatment is safe, well tolerated, and does not cause harm or serious side effects in most people.
For example, a clinical trial may look at whether a new exercise routine helps people lose weight. Or it may look at whether a new drug helps lower blood pressure in people with high blood pressure.
In short, a clinical trial helps to answer two important questions:
- Does the new treatment work in humans? If so, how well does it work? Is it better or the same as treatments that are currently available? If it is the same, does it have fewer side effects than other treatments? Does it help people who have not responded to other treatments?
- Is the new treatment safe? No treatment is completely safe for everyone; however, a clinical trial helps make sure the benefits outweigh the possible risks for most people.
Who conducts clinical trials?
Each clinical trial is led by a principal investigator, who is usually a medical doctor. In addition, there is often a team of doctors, nurses, social workers, and other health professionals who will work directly with you and provide your medical care. To cover the costs involved, a study must also have financial support (funding). This funding may come from a drug company (pharmaceutical), university medical center, government agency, or other organization. The group that provides funding is usually referred to as the “sponsor.”
What is a study protocol?
Clinical studies in the United States are regulated by a government agency called the “Food and Drug Administration (FDA).” Each study is reviewed, approved, and watched over by the FDA. Before a clinical trial can begin, it must receive approval from the FDA. It must also have an action plan (called a “protocal”). The action plan describes what will be done in the study, how it will be conducted, and why each part of the study is needed. This helps make sure the study will answer certain questions about the treatment while trying to make sure the people taking part are kept as safe as possible. Among other things, a protocol describes:
- The reason for conducting the study
- Who may participate (“eligibility”)
- How many participants are needed
- What tests and procedures will be done during the study and how often
- Information about the drugs that will be used, including the name and dosage
- The phase of the study (phase I – phase IV)
- How long the study will last
Actual study protocols can be very long (over 100 pages) and very technical. They are not written with patients in mind, so they can be hard for most patients to understand. Clinical trials must also have guidelines about who can be in them, and this information can be helpful if you are trying to decide whether you want to participate.
