Patient Safety in Clinical Trials

Approved by the Cancer.Net Editorial Board, 09/2019

Watch the Cancer.Net Video: Clinical Trials and Safety with Eric Singer, MD, MA, adapted from this content.

Keeping volunteers safe is the main concern in clinical trials about cancer. So every clinical trial has to go through a strict review process. Experts also supervise the doctors, research staff, and procedures in a specific clinical trial.

Who makes the rules for clinical trials?

Several groups of experts make the rules for clinical trials. Doctors and research staff must follow these rules exactly. This keeps you and other volunteers as safe as possible in the clinical trial. Below are the 3 main groups that make rules for clinical trials in the United States:

  • Institutional Review Board (IRB). An IRB is a committee of experts that makes sure a clinical trial is designed correctly, safely, and fairly. It protects the rights and well-being of clinical trial volunteers. An IRB must approve and supervise any clinical trial that involves people. The IRB does most of its work before a clinical trial starts. When it has all the information it needs, it approves the clinical trial so doctors can start it.

  • Government organizations. In the United States, the U.S. Department of Health and Human Services provides clarification and guidance on the clinical trial process, develops educational programs and materials, and provides advice on ethical and regulatory issues. The Food and Drug Administration (FDA) makes rules for clinical trials that involve experimental drugs or medical devices. The National Institutes of Health also checks clinical trials.

  • Data and Safety Monitoring Board (DSMB). A DSMB is made up of experts who review data and check on how the clinical trial is going. They also make sure the clinical trial is safe for volunteers and is giving doctors helpful information. They can also report on whether they think a clinical trial should change, stop, or keep going. A DSMB sometimes also visits the place where a clinical trial is happening, called a site visit. Site visits may also be done by the FDA or other organizations.

Who can help me understand a clinical trial before I join?

The clinical trial's research staff can help you understand a clinical trial before you decide to join it. They will give you printed information about what will happen in the clinical trial. This is called the informed consent form.

Your doctor, the research staff, or both will talk with you about the informed consent form and answer questions. They will talk with you about:

  • All your treatment options. This includes your choices if you decide not to join the clinical trial.

  • The treatment given in the clinical trial. This includes how it is different from the standard treatment and why doctors are studying it.

  • The possible risks and benefits of the clinical trial's treatment. These might be different from the possible risks and benefits of the standard treatment. Or they might be similar.

  • What you need to do if you want to join the clinical trial.

You will also learn:

  • How many doctor visits and medical tests you will need

  • When and how often you need them

  • How much it might cost you to be in the study

  • How the clinical trial staff protect your privacy and personal information

  • How to contact the research staff

  • How to leave the study if you want to

You might want to talk about this information with family members or friends. If you join the clinical trial, you will get a signed copy of your consent form. You may want to keep this with your personal medical records.

The research staff wants to hear your questions and concerns any time you have them. They also want to give you new information about the clinical trial or its treatment.

Can I leave a clinical trial?

Yes. You can leave at any time, for any reason. You will still get standard medical care and treatment for your cancer.

What if the cancer gets worse?

Just like the standard treatment, clinical trials do not always help. If your cancer gets worse, you may choose to leave the clinical trial or stay in it. You can have standard treatment or stop treatment if you want. You will probably want to talk with your doctor, family members, and friends. You might also want to talk with the research staff about your choice.

What if I have severe side effects?

Tell the research staff right away. You can get treatment to relieve side effects, and learning about side effects is an important part of clinical trials. Also, tell your doctor and other members of your health care team. You may choose to leave the clinical trial if the side effects are too severe. If the treatment causes severe side effects, the DSMB might stop the clinical trial.

What if the research staff puts me in a different group?

Sometimes, the clinical trial's treatment is obviously better than the standard treatment. If so, the DSMB might want everyone in the clinical trial to have it. The research staff will talk with you before putting you in a different group. Ask them any questions, including about your group and treatment.

Will I get a “sugar pill” or placebo?

A placebo is the medical word for something that looks like a real treatment, but is not. For example, a pill with no active ingredients is a placebo.

Doctors rarely use placebos in cancer clinical trials. They usually compare the new treatment to standard treatments instead.

If you might get a placebo, the informed consent form you receive when you agree to be part of a clinical trial will tell you. If you do get one, you will also get another treatment. This is so every person who has cancer gets a real treatment. You will never get only the placebo. Ask the clinical trial's staff any questions you have about this.

Learn more with free videos

PRE-ACT, Preparatory Education About Clinical Trials

You can watch a free series of educational videos on Cancer.Net. The series is called Preparatory Education About Clinical Trials, or PRE-ACT.

For a personalized video series, answer questions about your own situation and create an account. You may also watch the whole series. With your account, you can start and stop watching any time.

Related Resources

Cancer.Net Video: What is Informed Consent? with Carolyn Runowicz, MD

Phases of Clinical Trials

The Importance of Clinical Trials in Treating People With Cancer

How Are Eligibility Criteria for Clinical Trials Changing to Help People With Cancer?

ASCO Answers: Cancer Clinical TrialsDownload a free fact sheet on Cancer Clinical Trials (PDF).  This 1-page (front and back) fact sheet provides an introduction to cancer clinical trials, including a description what a clinical trial is, why clinical trials are important, patient safety, common concerns, words to know, and questions to ask the health care team and clinical trial staff.