- What Are Clinical Trials?
- How Do Clinical Trials Work?
- Access to Clinical Trials
- When Should I Think About a Clinical Trial?
“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
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.
Cancer Clinical trials are:
- 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
Clinical Trials for Cancer - Phases
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.
PHASE II: 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.
PHASE III: 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.
PHASE IV: 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.
“When you are on a trial you will get at least the standard of care, and you will be watched more closely and get better are than you have gotten or will get.” Mary Scroggins, survivor and advocate
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.
A treatment is effective if patients:
- 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.
“To be honest, no one ever mentioned a clinical trial to me personally. I had to ask and I realized in talking to the women that I worked—they were mostly underserved African-American women and Latinas, that many of us have never been told there was the possibility of a clinical trial, ” says Mary Scroggins, an advocate and ovarian cancer survivor.
Barriers still exist that keep certain groups--people of color, those from lower social and economic groups or older patients from enrolling on clinical trials. There are many reasons why this happens, but the fact is that all of these groups have lower rates of clinical trial participation. In some instances, doctors do not discuss clinical trials with certain populations as often as they do with others. The conversation never occurs because they make certain assumptions about whether these patients will be interested, or they will be able to tolerate the therapies, or handle the extra doctor visits or procedures or adhere to the treatment schedule. They don’t participate because they are not asked.
In other instances, some patients may have more distrust of a participating in a clinical trial, due to a history of medical procedures being forced upon, or done to their community without their knowledge or denied care because of their race, ethnicity or gender. Older patients may be less open to trials they believe will result in more side effects. Individuals who have less income or jobs that do not allow them flexibility may be reluctant to become involved in a treatment program that requires more time commitment or adds out of pocket costs to their treatment.
“I was diagnosed with a rare and pretty nasty sarcoma. My doctor suggested that I go on a trial to see if chemotherapy before the surgery and radiation helped shrink the tumor and reduce the risk it would come back. I knew that meant I would have more aggressive treatment and more side effects—with no guarantee that it would make any difference. But I also knew the odds if the cancer came back. I chose the trial.” Doug, sarcoma patient
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.
A clinical trial can be particularly important if:
- 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