Should You Consider a Clinical Trial?


Sarah Lewis, PharmD

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A clinical trial is a research study that tests new drugs or devices in humans. Clinical trials start after laboratory and animal research demonstrates product safety and shows promise for the drug or device. People who take part in clinical trials volunteer to help test the drug or device. This process is vital for developing new and better ways to prevent, detect and treat cancer.

If you’re interested in a clinical trial related to cancer, consider the following information. 

What Are the Phases of Clinical Trials?

There are five clinical trial phases:

  • Phase 0 is the first phase in humans. It looks at how the body processes a drug. People with advanced cancer who have no other treatment options usually take part in this phase.

  • Phase 1 looks at the drug’s safety and maximum dosing. A small number of healthy volunteer subjects are studied to determine appropriate dose ranges.

  • Phase 2 tests a drug’s effectiveness at treating a cancer among a larger group of volunteers. This phase may or may not enroll people who have already received standard cancer treatments.

  • Phase 3 is the first phase to involve large numbers of people. This phase is usually the last phase before FDA (Food and Drug Administration) approval. It compares a drug’s effectiveness and side effects to standard cancer treatments. Like phase 2, people may or may not have already tried standard treatment. The vast majority of volunteer subjects are enrolled in Phase 3.

  • Phase 4—or post-marketing surveillance—looks at the long-term safety of a drug. Because it takes place after FDA approval, thousands of people may participate.

How Are Patients Protected in Clinical Trials?

Clinical trials involve risk, but there are patient protections in place. Every study must have institutional review board (IRB) approval before patients enroll. This independent board ensures the welfare of study subjects. It also ensures the study complies with the law and monitors the study as it progresses.

If you participate, you will read and sign an informed consent form. It explains the study design and possible risks. The IRB makes sure this form is accurate and easy to understand. It is important to determine if the treatment arms are randomized and "open label." That determines whether you will be receiving the trial treatment, standard therapy, or possibly a placebo. As a participant, you can contact the IRB if you have questions or concerns about safety during the trial.  

Clinical trials also have data safety monitoring boards (DSMB). The DSMB is an independent board that reviews data and statistics during the trial. The DSMB can stop a trial early if safety concerns arise. It can also halt a trial if the new treatment shows much better results than the standard therapy. This allows all subjects in the trial to get the better treatment. 

No one can force you to finish the trial. You can withdraw if you decide it is in your best interest.

What Are the Possible Benefits of a Clinical Trial?

Possible benefits of enrolling in a clinical trial include:

  • Gaining access to a treatment that isn’t otherwise available. If this treatment turns out to be more effective, you are the first to reap its benefits.

  • Having part or all of your treatment costs covered by the study sponsor. Make sure you understand the financial arrangements before enrolling.Helping others in the future with your type of cancer by advancing research

  • Increasing your treatment options if you haven’t yet received standard treatment

  • Seeing a healthcare provider more often and having more frequent follow-up and monitoring

What Are the Potential Risks of a Clinical Trial?

There are also potential risks of enrolling in a clinical trial:

  • For randomized trials, you don’t have a choice over which treatment you receive. You may or may not receive the study treatment. You will never be randomized to any treatment known to be less effective than current standard care.

  • For blinded trials, you won’t know which treatment you are receiving. Your doctor may not know either if the trial is double-blind.

  • The new treatment may not work as well as standard treatment or it may have harmful side effects that doctors didn’t anticipate.

  • The new treatment might be effective, but not for you.

  • Your health insurance may not cover all of your costs during the trial. Be sure you understand what costs the trial covers and what your costs will be.

  • Frequent appointments may be inconvenient, especially if travel is necessary.

  • You may need extra tests that you would not have otherwise undergone.

Questions to Discuss With Your Doctor

Deciding whether or not to enroll in a clinical trial is very personal and isn’t easy. Your doctor can help you weigh the benefits and risks as they relate to you. Here are some questions to discuss:

  • Why am I interested in a clinical trial?
  • What other treatment options, if any, do I have?
  • What are realistic expectations for the trial?
  • What options do I have if I participate and the treatment doesn’t help me?
  • What happens if I don’t participate?
  • Am I physically able to participate?
  • Can I commit to the time the trial will need from me?
  • What are my potential costs and can I afford them?
  • How will the trial affect my daily life?

Your doctor may not have concrete answers to these questions. And you may go back and forth over some of your own answers. The process helps you think about these issues so you are comfortable with your decision.

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Medical Reviewers: William C. Lloyd III, MD, FACS Last Review Date: May 26, 2016

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Medical References

  1. Clinical Trials Information for Patients and Caregivers. National Institutes of Health. National Cancer Institute.
  2. Clinical Trials: What You Need to Know. American Cancer Society.
  3. Deciding to Take Part in a Clinical Trial. National Institutes of Health. National Cancer Institute.
  4. Questions to Ask Your Doctor About Treatment Clinical Trials. National Institutes of Health. National Cancer Institute.
  5. What Are Clinical Trials? National Institutes of Health. National Cancer Institute.&nb