What Are Clinical Trials?

February 10, 2014
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This podcast explains what clinical trials are, and how they are conducted.


You’re listening to a podcast from Cancer.Net. This cancer information website is produced by the American Society of Clinical Oncology, known as ASCO, the world’s leading professional organization for doctors that care for people with cancer.

Today we’ll discuss the basics of cancer clinical trials, including patient safety, phases of clinical trials, and the costs.

Let’s begin by talking about what a clinical trial is and why they are important in finding new medical approaches that work against cancer. A clinical trial is a research study involving volunteers to help find new ways to treat or prevent disease. Many cancer clinical trials test new treatments to find out whether they are safe, effective, and possibly better than the current treatment—called the standard treatment. There are also clinical trials that study new ways to ease the symptoms and side effects of cancer and its treatment.

Some people with cancer join clinical trials in order to gain access to new treatment or cancer care options. This is because patients who participate in clinical trials are often among the first to receive new treatments before they are widely available. However, it’s important to remember that there is no guarantee that the new treatment will be safe, effective, or better than a standard treatment. Other patients volunteer for clinical trials because they know that these studies are the only way to make progress in treating cancer. Even if they don’t benefit directly from the clinical trial, their participation may benefit other people with cancer.

Almost all cancer prevention and treatment information is a result of the scientific advances made through clinical trials. The knowledge from clinical trials has also helped doctors make progress in diagnosing cancer and finding cancers that have come back after treatment. Because clinical trials may involve hundreds or even thousands of people, it often takes a long time to find out the results. But as new information is learned and studied, new treatment or prevention options replace old standards. Clinical trials are the most reliable and only accepted scientific method to find out if a new treatment works.  

Despite the promise offered by clinical trials, less than five percent of adults with cancer enroll in clinical trials. This low rate of participation slows the development of new and better cancer treatments. In contrast, more than sixty percent of children with cancer participate in clinical trials. And, these studies have significantly improved the survival rate for children with cancer.

Next, let’s discuss the priority of patient safety in clinical trials. In each clinical trial, there are requirements about who can join based on a person’s age, type of disease, medical history, and current health. This is called inclusion and exclusion criteria. Inclusion criteria ensures that all the people in the clinical trial are medically similar so that doctors can interpret the results correctly. Exclusion criteria helps keep people safe by excluding those who might have potential risks to their overall health by participating.

Each clinical trial follows a specific set of rules, called a protocol. A protocol decides who can participate, the treatment, and the length of the study. Before the clinical trial begins, the protocol is approved by an institutional review board—called an IRB—to ensure patient safety. An IRB is an independent committee of doctors, statisticians, advocates, clergy, lawyers, and others who ensure that rights and welfare of study participants are protected. Then, another independent committee—called the Data Safety Monitoring Board—reviews the research data during the study as another patient safety measure.

For each clinical trial, researchers must obtain the informed consent of each participant before starting. Informed consent is a process to educate patients about the clinical trial, other treatment options, the risks and benefits of participating in the study, and their rights as a volunteer.

Sometimes people have concerns that, by participating in a clinical trial, they may receive no treatment by being given an inactive drug called a placebo, or sometimes called a “sugar pill.”  However, the use of placebos in cancer clinical trials is rare. When a placebo is used in a study, it’s done with the full knowledge of the participants.

As part of the clinical trial, the research team keeps close track of each volunteer’s health to see whether the treatment is safe and effective. The research team includes doctors, nurses, social workers, and other health care professionals. A person’s participation will be most successful if they carefully follow the instructions given to them by the research team and stay in contact with the research staff after the study has ended. It’s also important to know that a participant can leave a study at any time and for any reason.

Now, let’s talk about the three main phases of clinical trials. Phase One clinical trials are used to show that a new treatment is safe for a small group of people. It also helps find the best dose and treatment schedule. Phase Two clinical trials give researchers more information about the safety of the treatment and how well it works. And, a Phase Three clinical trial compares the new treatment with a standard treatment to determine how it will be used.

Many people may have concerns about the costs of participating in a clinical trial compared with the costs of standard treatment. The Patient Protection and Affordable Care Act requires insurers to cover the routine costs of an approved clinical trial. These are the costs that would be covered even if a person is not participating in a clinical trial. Coverage of research costs, such as the treatment being studied and tests or procedures only needed for the study, is not required. However, these costs are often covered by the study’s sponsor, which may be a government organization, pharmaceutical company, health care center, or other organization. Talk with both your insurance provider and study sponsor about which costs would be covered if you participate.

Before making cancer treatment or care decisions, talk with your doctor about all your options, including clinical trials. Each treatment option will have benefits and risks to consider. Based on these factors, you and your doctor can talk about whether a clinical trial is right for you.

For more information on clinical trials, talk with your doctor or visit www.cancer.net. Cancer.Net is supported by the Conquer Cancer Foundation, which is working to create a world free from the fear of cancer by funding breakthrough research, sharing knowledge with physicians and patients worldwide, and supporting initiatives to ensure that all people have access to high-quality cancer care. Thank you for listening to this Cancer.Net podcast.