10 Common Questions About Cancer Clinical Trials

We are in an era of remarkable progress in understanding why and how cancers start and grow. Researchers are developing new ways to treat many cancers. Targeted therapies and immunotherapy have opened the doors to real hope for people whose cancers could not be treated successfully before.

Clinical trials are the engine that drive progress in cancer treatment. Patients who join cancer clinical trials have access to the newest therapies and a high level of care. By joining a clinical trial, they give themselves hope for longer and better lives. They also contribute to a better future in cancer care and treatment for others facing cancer.

Not everyone can or should participate in a cancer clinical trial. But for some patients, clinical trials can be lifesaving.

Here’s what to know:

1. What are clinical trials?

Cancer clinical trials are research studies to determine whether a new approach to cancer prevention or treatment works better than the best-known available treatment. Clinical trials compare the new drug or approach with the existing standard of care to make sure the drug is safe and effective.

There is no guarantee that any individual will benefit from a clinical trial. But every drug or agent that reaches a clinical trial has already been thoroughly tested and shown to have significant promise. In many cases, trials are the only way to get the newest cancer treatments.

2. How do clinical trials work?

Anyone who joins a clinical trial must go through the informed consent process first. During this process, the patient's doctor explains the trial, procedures it involves, and potential benefits and risks. This is also an opportunity for the patient to ask any questions they have about the trial. Questions might include:

• Will the trial involve hospital stays? Extra medical appointments?
• What are the costs associated with the trial, and how are they covered?
• Are there any out-of-pocket costs to consider?

During a clinical trial, researchers track all of the side effects participants in the study may have. Participants are often asked to describe their experience. This is called patient-reported outcomes.

3. Should I consider joining a clinical trial?

Everyone facing a cancer diagnosis of any kind should be aware of clinical trials as a treatment option — but not everyone can or should participate in a trial. Deciding whether to join a clinical trial is a choice you make with your healthcare team.

It may be a good option if you answer yes to any of the following questions:

• Are you interested in getting access to the newest cancer treatments?
• Was your cancer diagnosed at an advanced stage?
• Are you no longer responding to other treatments?
• Is your cancer at high risk for recurring or spreading after primary treatment?
• Are you interested in helping other cancer patients by advancing cancer treatment?
• Do you have a rare, aggressive, or difficult-to-treat cancer?
   

Tip

The best way to know if you should consider joining a clinical trial is to talk with your doctor and treatment team. If your doctor is not involved in doing clinical research or does not explain your options for a clinical trial, consider getting a second opinion.

4. If I join a clinical trial, who covers the costs?

In many cases, costs associated with a clinical trial — including any drugs, study-related tests, and medical visits — are covered by the trial's sponsor or by the research institution. A patient's health insurance may also cover some costs. Insurance coverage for clinical trial participation varies based on an individual’s insurance company and policy. 

Before committing to a clinical trial, be sure to ask about the costs involved, including any indirect costs (such as childcare or lost job time) for additional doctor’s appointments, clinic visits, or procedures.

Transportation and lodging may also be indirect costs. Some patients need to travel out of town to take part in a trial. Some but not all trials will offer financial assistance for travel and lodging.

5. How do I find a clinical trial that is right for me?

Finding a trial that is right for you and your cancer can be a challenge. The best way is to discuss your situation with your healthcare team or to get an opinion from a medical center that is actively involved in clinical trials for your cancer.

If you choose to look for trials online:

• Stick with established sites or with organizations that help match patients to trials.
• Be sure you know your exact cancer type, any previous cancer treatments you have had, and any medical problems that could cause you to be ineligible for a trial.
• Print out anything you find and take it to your doctor to discuss.

6. How are the results of a clinical trial measured?

Every trial measures whether the new approach is safe and effective. Safety is assessed by recording the side effects each patient experiences and the severity of these problems. This information will come from the treatment team and — more and more often today — from the patients themselves.

Clinical trials also measure what happens to the tumor as a result of the treatment. The researchers look at what happens in individual patients and in the whole group of patients being treated. Many trials now focus on reducing the risk of recurrence in people whose cancers have factors that make them likely to come back or spread.

7. Will I get a placebo if I participate in a trial?

It is very rare for people with cancer in clinical trials to get a placebo (an inactive substance, sometimes called a sugar pill). In a cancer clinical trial, patients will get at least the standard of care. This means getting the best-known available treatment or practice for any type of cancer.

Most trials compare adding a new treatment, versus adding a placebo, to the standard of care.

8. What kind of care will I receive on a trial?

If you participate in a clinical trial, you will be part of a research study. But you are not a guinea pig. People who participate in clinical trials get very high levels of care. Their cancers and their overall condition are monitored and cared for very closely.

9. Will I have to leave my current doctor?

That depends on what kind of cancer you have, where you are being treated, and for what kind of trial you enroll. If there is no trial available in your treatment facility, your doctor should be willing to refer you to another cancer center.

10. Is a clinical trial an option only if I don't have any other options?

No. There are trials for every type and stage of cancer. For many cancers, a trial can even be the first option. As you consider your options, take time to think about what is most important to you. It’s also helpful to have an open and honest conversation with your doctor about the goals and expected treatment outcomes for a clinical trial. This will help you decide whether a clinical trial might be right for you.

Editor's Note: This blog post was published in 2023 and has been updated.