What Are Clinical Trials?

Table of Contents

Clinical trials for cancer are research studies that compare the most effective known treatment for a specific type or stage of cancer with a new approach. This can be a new drug, or combination of drugs or a different way of using established therapies. There are trials that involve new approaches to surgery and radiation therapy. There are clinical trials for every type of cancer. While many trials focus on late stage disease, there are also trials to prevent cancer, improve early diagnosis, stop the cancer from coming back, reduce side effects or improve quality of life.

What is a clinical trial?

  • The engine that drives progress in cancer treatment
  • The only way to find out if a new drug or treatment is better than the existing standard of care
  • In many instances, the only way for a patient to get a new or experimental approach before it is approved for general use
  • A proven way for people facing cancer to get high quality treatment and care
  • A chance to benefit from a new therapy or approach
  • An opportunity to help future cancer patients and contribute to research
  • A way to be involved in the frontlines of research to advance cancer care
Duration: 2 min

What Are Clinical Trials?

This video explains the purpose of clinical trials, how they work, and how they can expand your treatment options. Have a concern of your own? Please call our Cancer Support Helpline to talk with an experienced counselor or navigator: 888…

The treatments we use today were discovered, tested and first made available to patients in clinical trials — and the drugs that are the future of cancer treatment are in trials today. I want to emphasize that being in a clinical trial is how you get access to the next generation of cancer treatment.

David Carbone, MD Ohio State University

Cancer Clinical Trial Phases

There are different types of phases of clinical trials. The different phases of clinical trials are marked by Roman numerals I, II, III, and IV, which stand for the numbers 1, 2, 3, and 4. All trials are done to compare new treatments to established therapies, but each of these phases has a somewhat different goal. Trials are usually done in order, beginning with Phase I.

Once a drug is approved for human studies, it is tested in a small trial to determine the optimal safe dose. Phase I studies often involve patients with different kinds of cancer, or more recently, a single genetic change.

If a drug can be given safely to people on the Phase I trial, it is tested in a Phase II study. These are larger studies, usually for one or more specific types and stages of cancer. The goal of Phase II studies is to both determine the optimal dosing and provide an early assessment of whether the drug works.

These trials take place after a drug has shown good results in earlier studies. They are large studies, often involving hundreds, or even thousands of patients, in multiple centers in the United States and/or abroad. Patients on Phase III trials have specific types and stages of cancer. Many Phase III trials are randomized — meaning that patients are randomly assigned to receive either the new treatment or the established standard of care. These trials are designed to provide definitive evidence to support FDA approval of the drug or agent for use in the public.

These trials take place after a drug is approved and are often called post-marketing trials. The goal is to make sure that no safety or other concerns come up after a drug is approved that may not have been seen in the pre-approval trials. It is important to follow patients for a number of years to determine if there are any long-term side effects or other issues that affect the way the treatment is used.

How Do Clinical Trials Work?

A clinical trial is a research study to find out if a new treatment provides better outcomes than the existing standard of care — the best available known treatment. Clinical trials establish the effectiveness and safety of new cancer treatments.

What makes a treatment effective?

  • Live longer (overall survival)
  • Have longer periods of time when their cancers shrink or do not grow or spread to new places in the body (disease free or progression free survival)
  • Have better quality of life with fewer side effects or symptoms of their disease
  • Have lower rates of recurrence after their primary treatment — meaning the cancers do not come back

The safety of a new drug or treatment is also very important in deciding if, how and when to use a new treatment. During any clinical trial, researchers track all of the side effects, or adverse events, that happen to the people in the study. Today, participants are often asked to describe their experience, which is known as patient reported outcomes.

Before any drug is used with human beings, there is a great amount of work done in the laboratory to know how the drug works and understand the likely side effects. This is called preclinical work. Once this is done, doctors, researchers, nurses and other health care team members are ready to work together with patients in a clinical trial.

Anyone who enrolls in a clinical trial, of any kind, goes through informed consent. This means that your doctor explains the trial, its potential benefits and risks and the procedures it involves. Informed consent is a formal process that involves a lot of paperwork. You can ask your doctor or the protocol coordinator any questions you have about the treatment, side effects and potential outcomes. You can also ask about what being in the trial means in terms of extra doctor appointments, hospital stays and procedures and any costs that you might not be covered.

Deciding whether or not to be on a clinical trial is more than just signing the forms. It is a choice you make with your doctor and health care team. You should always feel free to ask any questions you have before you enroll in a trial, and while you are participating in the trial.

Watch: Patients Share Their Stories

Duration: 2 min

Glen's Prostate Cancer Clinical Trial

Glen lives with prostate and Merkel cell cancer. He joined a clinical trial for prostate cancer when he was first diagnosed to give himself a chance at less side effects than the standard treatment.

Finding a Clinical Trial

Many people say they have never been told about clinical trials as treatment options. That’s because not all providers who treat cancer offer clinical trials. And not all cancer centers offer clinical trials.

The best way to find a clinical trial that is right for you is to talk to your healthcare team. If your provider does not talk to you about clinical trials, you should ask if they offer clinical trials or can refer you to one. If the answer is no, you may want to get a second opinion from a specialist who does. Getting a second opinion from a specialist who focuses on your specific cancer type — even if it is over the phone or online — can help you learn more and feel confident about your treatment decisions. Advocacy groups that help people with your type of cancer can help you find a specialist.

Tips for finding a clinical trial:

  • Ask your provider what clinical trials are being offered at their site for your cancer type.
  • Ask your provider if they know about a clinical trial they think would be right for you.
  • If they don’t know about any trials, ask for information about how to find a clinical trial.
  • Ask an oncology nurse, social worker, or nurse navigator working with your doctor if they can help you find a clinical trial.
  • Get a second opinion from a specialist at a cancer center that offers clinical trials, such as an NCI (National Cancer Institute)-designated cancer center. Find a cancer center that offers clinical trials.
  • Look at the website of a cancer center near you or call them to see what trials they have open.
  • Call the Cancer Support Community Helpline at 888-793-9355. Or call a patient advocacy group for your type of cancer and ask about clinical trials.


When Should I Think About a Clinical Trial?

A cancer clinical trial may be a treatment option for anyone who has received a cancer diagnosis. The best way to learn more about your specific options is to talk to your doctor, nurse or other members on your health care 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.


When would a clinical trial be important?

  • You are diagnosed with cancer that has spread or your cancer recurs or comes back after your primary treatment
  • You are interested in getting access to the newest cancer treatments
  • You have a rare or difficult to treat cancer
  • Your treatment that was working has stopped working
  • Your cancer requires that you remain on treatment for a long period of time or the rest of your life
  • You are interested in helping other cancer patients today and tomorrow by advancing cancer treatment
  • Your treatment involves significant side effects and the trial is designed to reduce the impact of these on your life

Clinical trials are so important for people with cancer because it provides access to state-of-the-art care. Clinical trials should be offered to patients at any time-point during their cancer care. Historically we thought of cancer clinical trials as being the last option, but for many people it should really be thought of as the first option.

Dr. Patricia Robinson Hematology/Oncology

Questions to Ask Your Healthcare Team

You will want to think about your goals and values as you make treatment decisions. Your age, overall health, and treatments you have received can all influence your goals and values. Ask your healthcare provider all of your questions and then take time to think about the answers. Take someone with you to your doctor’s visits to listen and take notes. Then he or she can help you think about what you’ve learned. This will help you decide what is right for you.

Here are some questions that can help you talk to your healthcare team about clinical trials:

  • What type of cancer do I have?
  • What stage of cancer do I have?
  • Are there any biomarkers I should be tested for? Could that help me qualify for a clinical trial or for other therapies?
  • What trials are available for the type of cancer I have?
  • What are the possible benefits of this trial?
  • What side effects might I experience?
  • Have any other trials been done with this drug/treatment in people with the type of cancer that I have?
  • Will I know if I’m getting the experimental treatment?

The Importance of Diversity in Clinical Trials

People of color, Hispanics, women, and older adults are underrepresented in clinical trials. This is a problem because a person’s age, gender, race, or ethnicity may affect how their cancer responds to treatment. It is important to increase the diversity of participants in clinical trials. If diverse people take part, clinical trial results can better show how new treatments work with all patients. The results could also better show if some groups are more likely than other groups to develop side effects.

If all groups are not represented in clinical trials, they will not have the chance to benefit equally from the newest treatments. This can widen the differences we see in cancer outcomes in the United States. For example:

  • ­African Americans have the highest cancer death rate.­
  • Cancer is the leading cause of death for Asian Americans.­
  • Hispanic men and women are twice as likely to die from liver cancer than non-Hispanic whites.­
  • American Indian/Alaskan Natives have the highest rate of colorectal cancer.­
  • African American women have a higher risk of dying from breast cancer than white women.­
  • African American men have higher rates of lung cancer than white men. ­
  • Asian Americans with liver cancer have a higher death rate than whites.
Duration: 2 min

What Are Clinical Trials

Dr. Patricia Robinson, an oncologist, explains cancer clinical trials and their benefits to patients.

Two women smiling at each other

Peer Clinical Trials Support Program

If you are a Black or African American cancer patient and want one-on-one support to learn more about clinical trials, this program is for you.

Get More Details & Apply Today

Watch & Learn More About Clinical Trials

Watch Duration: 2 min

Why Is Diversity Important in Clinical Trials?

This video shares the importance of diversity in clinical trials and how a person’s age, race, gender, or ethnicity may affect how their cancer responds to treatment.

Have a concern of your own? Please call our Cancer Support Helpline to talk with an experienced counselor or navigator: 888-793-9355. 

Watch Duration: 49 min

Clinical Trials & Immunotherapy

Watch the webinar, Frankly Speaking About Cancer: Clinical Trials and Immunotherapy, that took place on October 24, 2016, for what you need to know about immunotherapy treatment and finding clinical trials that might be right for you. Immunotherapy works by boosting the body’s natural ability to fight cancer. 

Watch Duration: 92 min

Who Is Missing from Cancer Clinical Trials?

Watch the webinar, Frankly Speaking About Cancer: Who Is Missing From Cancer Clinical Trials that took place on November 20, 2017. Three advocates will discuss which groups are missing from cancer clinical trials and how we can address this.

Barriers Keep Patients Out

There are hundreds of clinical trials looking for cancer patients. Yet, less than 10% of all cancer patients take part in a clinical trial. And most of those who do are white men. People of color, Hispanics, women, and older adults often do not participate in trials in large enough numbers. Some people in these groups might stay out of trials because they:

  • ­Do not trust medical research­.
  • Were given poor medical care in the past­.
  • Speak a first language other than English­.
  • Have experienced discrimination when seeking healthcare.
  • ­Have low reading levels that make it hard to learn about trials­.
  • Have health problems that may not allow them to take part in the study­.
  • Have no sick leave or no ability to take time off of work.
  • ­Must care for parents, grandparents, or grandchildren­.
  • Cannot afford clinical trial costs related to transportation and housing. This may be a large factor if the trial is in another city or state.

If you have concerns about any issue related to being on a trial, bring it up directly with your healthcare provider and your treatment team. Getting the best cancer treatment means knowing about and having access to clinical trials. Don’t settle for anything less.

For my patients who have participated in clinical trials, the benefits have been access to quality care, equal access to care, in many respects individualized care. They have a team providing care, including the physician, the research nurse, the office nurse. It just builds a team — and a village — for them.

Dr. Patricia Robinson Hematology/Oncology

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