When you’re in cancer treatment and your doctor suggests a clinical trial, you might think the main goal is to understand your type of cancer better to help others who will have it in the future.
That’s part of it, sure, but the primary purpose of a clinical trial is to help you—right now.
“The benefits to participating in a clinical trial are huge,” says UNC Health oncologist Megan McNamara, MD. “You can get access to medications or treatments you wouldn’t otherwise have access to, possibly years before they’re available to everyone.”
As researchers and doctors continue to learn more about the best, most precise way to treat cancer, the options available in clinical trials could become the standard of care for your type of cancer in the future.
“Clinical trials are the only way to develop new cancer medications, which will help patients live longer, have a higher likelihood of cure and have the least negative effects on quality of life,” says UNC Health oncologist Carrie Lee, MD, MPH. “All of the drugs we take, from Tylenol to novel therapies for cancer, went through the clinical trial process, and that research process helps us learn how to make cancer patients’ lives better.”
Drs. McNamara and Lee explain what you should know about clinical trials in cancer care.
The Clinical Trial Process for Cancer Treatment
Clinical trials can evaluate a variety of things, including new medications, devices and treatment interventions; methods of screening for, diagnosing and measuring disease; and possible preventive measures. While your type and stage of cancer might have a typical treatment or standard of care such as surgery and radiation, a clinical trial could evaluate whether a new medication improves upon that treatment or could potentially replace some part of the treatment.
“Typically, what a patient with cancer might be offered is a treatment study where you’re either receiving the standard treatment—what we know works, a new treatment that has already shown promise either in the lab or in patients, or a combination of the standard with the new treatment,” Dr. McNamara says. “If you take a new drug prior to surgery, does it help to shrink the cancer? If you take a new drug after a surgery, could it prevent or delay the cancer from recurring?”
The specific questions that researchers are trying to answer are all documented and carefully reviewed in the process of getting the trial approved. These reviews are done to protect patient safety and minimize risk.
“Clinical trials are designed in a very regimented way to be sure we fully evaluate the benefit of the treatment being developed and any risks or side effects, and they’re reviewed to ensure that patients are as safe as possible when participating in clinical research,” Dr. Lee says. “Before anyone participates in any clinical trial, the researcher obtains informed consent, which is an explanation of what is being asked of the patient and gives the patient an opportunity to ask any and all questions they may have about the research.”
Clinical trials are typically conducted in phases; a patient might be asked to participate in any of them. Let’s say researchers have a new drug that has shown promise in shrinking a certain type of cancer tumor on cells in a laboratory. In Phase I, that drug might be given to a small number of people (typically, fewer than 50), so researchers can learn more about the appropriate dose, side effects and whether results seen in the lab will also be seen in people.
After learning what dose is safe and tolerable in terms of side effects, the medication might go into Phase II, where a larger group of people (about 100) take it to ensure that the treatment is working the way it was intended.
Phase III introduces it to still more people, typically hundreds, to compare the new drug to the standard of care. The information from all phases of research is provided to the Food and Drug Administration (FDA), which decides whether to approve the drug as a treatment.
Following that approval, a Phase IV clinical trial monitors continued safety of the drug once more people are taking it.
The timeline of a clinical trial for cancer treatment can vary.
“If a drug is being given to decrease the risk of cancer coming back or spreading after a surgery, and that cancer typically doesn’t come back for five years, then you have to follow the patient for at least five years and beyond to see if it was effective,” Dr. McNamara says. “It will depend on what the study is trying to show, but trials can follow people for multiple years.”
Common Misconceptions about Clinical Trials in Cancer Care
In some clinical trials, a group of people receiving a medication or treatment is compared to a group of people receiving a placebo, something that has no active properties and is not designed to treat the condition. That is very rarely the case in cancer care because it would be unethical to give cancer patients no active treatment for their cancer.
“The most common misconception people have is that they’ll get a placebo, but the vast majority of trials are not designed that way,” Dr. McNamara says. “They are designed to compare a new treatment to the standard treatment, so they only way you’d do nothing is if the current standard is to do nothing.”
“We have an obligation to be sure that cancer patients receive active therapy for their cancer,” Dr. Lee adds, noting that these trials don’t compare cancer patients to healthy patients, the way a trial for a different disease might. “For new cancer therapies, we just enroll cancer patients.”
Drs. McNamara and Lee say patients also worry that they’re only being offered a clinical trial if there are no other remaining options for their treatment, but that’s usually not the case.
“Clinical trials should always be considered if they’re available and appropriate from the first diagnosis of cancer through all stages and junctures,” Dr. Lee says. “In cancer care, clinical research is an integrated part of cancer care, and it’s usually delivered in the same space as the patients receiving the standard of care.”
If you participate in a clinical trial, you typically maintain the same care team, which includes your doctor and your nurse navigator, but add on someone from the research team. This can be a helpful additional layer of support.
Questions to Ask Your Doctor about a Clinical Trial
If your doctor mentions the possibility of a clinical trial, take the time you need to decide about enrolling.
“Don’t make on-the-spot decisions about treatment,” Dr. Lee says. “Take any paperwork home to read and review, and make a list of questions for your provider and the research team.”
Here are some common questions you might ask:
Why am I a good candidate for this trial? What do we know about this treatment so far?
It’s important to understand your doctor’s rationale for considering a clinical trial: Maybe you have a certain biomarker in your tumor that makes it more likely to respond to this medication, or maybe researchers are investigating treatment intended to be given after surgical removal of cancer in a situation similar to yours.
“You should ask how the treatment is expected to work, and why it might work for you,” Dr. McNamara says. “You can ask what data they already have on this treatment. If it’s Phase II or III, there are prior studies showing if the medication is effective for certain tumor types or stages of the disease.”
Your doctor can help you understand the possible benefits and risks of the trial, the timeline, and how any new medications or interventions would be integrated into other treatment.
Will there be extra tests or visits?
Some clinical trials may require you to have additional visits or monitoring in the form of more biopsies, imaging scans or blood draws. These additional steps help researchers understand how a new drug is working while also ensuring that patients are safe and not experiencing adverse side effects. It’s important to understand the extent of any additional monitoring and the length of the treatment.
“There might be overnight stays, more visits or longer visits,” Dr. Lee says. “There may be compensation or support for those extra steps. The sponsor of the study will likely pay for biopsies or scans that are directly related to the research (and not part of the standard treatment), and there may be resources to cover transportation, meals or accommodation if a patient is traveling a long distance.”
Be sure to ask where extra visits or treatment related to the trial will be located. For some trials, you may get all of your care in your doctor’s office or in the same hospital building with your established care team; for others, you may have your regular appointments with your doctor but have additional visits in an entirely different location, even potentially out of town if your local care team does not have the clinical trial open where you typically receive care.
What are the risks or expected side effects? How do I communicate those?
It’s important to understand both the benefits and the risks of any cancer treatment. While the point of the clinical trial may be to better understand the possible side effects, your doctor should still be able to help you understand the risks, especially compared with the current standard of care, and they should provide guidance on how to handle side effects.
“The risks of participating will depend on the specific trial, but it will be spelled out on the consent form,” Dr. McNamara says. “The risks can be really varied depending on the medications.”
Ask your doctor how to report those adverse effects. Sometimes, you might work directly with them to address concerns; other times, you may need to notify someone from the research team. The possible next steps—adjusting the medication or adding another treatment to address the side effect—will depend on the goals and protocols of the clinical trial.
“It’s also important to know that you can withdraw from a clinical trial if you’re not comfortable or don’t want to continue at any point,” Dr. Lee says. “You can always change your mind.”
Finding a Clinical Trial for Cancer Care
If your provider doesn’t bring up clinical trials, ask them if they know of any that are relevant to your specific situation.
“Physicians try to know the portfolio of trials available at their site and which ones are relevant for certain cancers or tumor types, but they can’t know all the trials in the world,” Dr. McNamara says. “If there aren’t relevant trials at your location, the website clinicaltrials.gov has the inclusion and exclusion data for all open trials.”
Your doctor can help you assess your eligibility for a trial based on that inclusion and exclusion data; some trials are only available for certain stages of cancer or tumor types or may require you to have tried other treatments previously.
“Be proactive about asking, because we need patients of all different backgrounds to participate,” Dr. Lee says. “We need all gender identities, all ages, all races. Otherwise, the information we get may not be applicable to everyone.”
In addition to potential benefit in your own treatment, Drs. Lee and McNamara say people feel satisfaction at helping future generations.
“There’s a shared desire to make cancer treatment better among people who have it,” Dr. McNamara says.
If you’re interested in participating in clinical research, talk to your doctor. If you need a doctor, find one near you.