Clinical trials are carefully designed research studies that depend on human volunteers to improve medical care over time.
In cancer research, pre-clinical studies first test new treatments that show promise in the lab or in animals. These treatments may ultimately be tested in clinical trials involving people.
Clinical trials are conducted to:
- ensure a new cancer treatment is safe to use in people, and to see if it works better than the best therapy that is currently available (known as the standard of care)
- study new ways of diagnosing cancer
- reduce the risk of cancer developing or returning
- manage treatment side effects
- help people feel better during or after cancer treatment
Learn more about:
- Who can consider a clinical trial?
- Types of clinical trials
- How clinical trials are conducted
- How clinical trial participants' safety is protected
- How to find out if you’re eligible for a clinical trial
- Covering the costs of clinical trials
- Benefits and risks of clinical trial participation
- Questions to ask before enrolling in a clinical trial
- How to find a clinical trial
- Glossary: Common terms used to talk about clinical trials
- Special Report: Increasing diversity in clinical trials
Who can consider a clinical trial?
You might think that clinical trials are only an option for people with cancer that does not respond to standard treatments. But that’s not the case. You can consider a clinical trial at any point in your experience with breast cancer, including if:
- you’ve been told you’re at higher-than-average risk for developing breast cancer
- you’ve just been diagnosed with breast cancer
- you’re in the middle of treatment
- you’ve finished treatment, whether recently or long ago
- you’re facing a recurrence of breast cancer
- you have metastatic breast cancer that requires ongoing treatment
Although trial participation is a personal decision, some people with breast cancer choose to take part because they want access to the newest treatments being studied by researchers that may not be available outside of a trial. Many people like knowing that they will be followed closely by a research team, and that they may be contributing to future advances in breast cancer treatment.
Even in early-stage breast cancer, researchers are looking for better ways to prevent future recurrences and improve short- and long-term treatment side effects. Right now, only about 5 percent of adults with cancer take part in clinical trials, so there is always a need for more candidates.
Types of clinical trials
Depending on your individual situation, there are different types of clinical trials to consider.
Therapeutic clinical trials
In therapeutic clinical trials, people agree to try new therapies under careful supervision to help doctors identify the best treatments with the fewest side effects. These studies help improve the standard of care for breast cancer and other diseases. Before a new treatment can be approved by the U.S. Food and Drug Administration (FDA) and similar government agencies in other countries, it must be tested in clinical trials.
A therapeutic clinical trial may study a new medication that has shown promise in lab research, animal studies, or previous clinical trials. It also might test new combinations of existing medicines, new types of surgery or radiation therapy, or new ways of giving treatments, such as the dosage, timing, or delivery method (by mouth vs. through an IV, for example). Therapeutic trials can study first-line treatments, which are given after an initial cancer diagnosis, or treatments for cancer recurrence or metastatic disease.
Therapeutic trials are just one type of research that's done before a new treatment becomes widely available to people. New medicines must first be discovered, purified, produced, and then tested in pre-clinical studies that are done on cells in petri dishes or on animals. Clinical trials in people are started only after several years of pre-clinical trials suggest that a new treatment will be safe and beneficial for people.
Therapeutic trials are designed to answer two major questions:
- Is the new treatment safe for people? All breast cancer treatments have side effects. In clinical trials, researchers try to find out if the benefits of a new treatment or procedure outweigh potential side effects.
- Does the new treatment work in people, and if so, how effective is it? Once a new treatment is proven to be safe, more clinical trials are needed to answer additional questions: Is the new treatment better than standard treatments? Does it cause more or fewer side effects? Does it work in a group of people who don't benefit from standard treatments?
Researchers don't know what the results of a therapeutic clinical trial will be. Sometimes participants have no benefit from the study treatment. And study treatments may have unknown side effects, especially if they are completely new. In rare cases, clinical trial volunteers have been hurt by the treatment or procedure being tested.
On the other hand, many people receive effective treatments through clinical trials. And hundreds of thousands of others are helped because those volunteers chose to participate in a trial that resulted in a new, more effective treatment. Virtually every standard treatment for breast cancer available today was first studied in clinical trials.
Basket and umbrella trials
Basket and umbrella trials are newer types of therapeutic trials designed to study new treatments in people with cancers that share specific gene mutations or biomarkers. A mutation is a change in a gene related to the cancer’s development. A biomarker is a substance found in the blood or tissue that can be a signal or “marker” of cancer development.
When cancers in different people share the same gene mutations or biomarkers, it suggests there may be similar processes that cause the cancer to grow and affect how the cancer cells behave. Therefore, these groups of people may respond to similar types of treatment.
Basket and umbrella trials are designed to test and potentially approve new treatments more quickly than traditional clinical trials.
Basket trials — also called bucket trials — enroll people with different types of cancers that have the same gene mutation or biomarker. Participants receive a treatment that targets the specific molecular process that is believed to play a role in the development of these cancers.
For example, there could be a basket trial with multiple groups that include people with breast, lung, colorectal, or other types of cancer that are activated by the same molecular trait. This is a different approach from more traditional therapeutic trials, which usually enroll patients with the same type of cancer, based on where the cancer is found in the body.
Umbrella trials enroll people with the same type of cancer — such as breast cancer — but with different gene mutations or biomarkers that may play a role in the cancer’s development. This type of trial is designed to look at how new therapies work in people with different molecular subtypes of the same cancer. Participants are grouped by the specific mutation or biomarker found in their cancer. The study treatments may change during an umbrella trial as researchers learn more about the different subtypes of cancer and how people respond to therapies.
Non-therapeutic clinical trials
Not all clinical trials test treatments for cancer or other diseases. Non-therapeutic clinical trials study ways to improve the detection or diagnosis of cancer, prevent it from developing or returning, reduce side effects, or improve quality of life for people affected by cancer.
Prevention trials investigate new ways of reducing the risk of breast cancer or its recurrence. Examples could include medicines or lifestyle changes like diet or exercise. For people with a higher risk of breast cancer, prevention trials may study cancer risk reduction with strategies like medications or surgery.
Screening and diagnosis trials look for new and better ways to detect breast cancer, such as imaging techniques and tissue sampling (biopsy) approaches.
Supportive care and symptom management trials — also called quality-of-life trials — study ways to reduce treatment side effects and improve peoples’ overall functioning during and after cancer treatment. These strategies may include medications or lifestyle changes such as exercise or diet.
How clinical trials are conducted
Researchers conduct clinical trials in different settings. Many clinical trials are done at major cancer centers and academic research institutions.
Community-based hospitals, clinics, and doctors’ offices that take part in research also can enroll people in clinical trials. In fact, the National Cancer Institute (NCI) launched the NCI Community Oncology Research Program (NCORP) to bring more clinical trials to community-based practices, where most people receive their cancer care. As a result, some major NCI-funded studies may be available in these settings.
Every clinical trial is led by a head researcher called a principal investigator, who is often a medical doctor. The research team may include research scientists, doctors, nurses, social workers, dietitians, other healthcare professionals, and research coordinators.
Clinical trials can be sponsored (funded) by:
- Government agencies, such as the National Cancer Institute, the Department of Defense, and the Department of Veterans Affairs
- Pharmaceutical, biotechnology, or medical device companies
- Healthcare institutions, such as academic medical centers or health maintenance organizations (HMOs)
- Non-profit organizations or private individuals
The sponsoring organization or company may start the clinical trial and help to oversee and manage it.
The researchers write a detailed plan for how the clinical trial will be done and what they hope to find out. This plan is known as a protocol and is written in fairly technical language. For a therapeutic trial, the protocol includes details about the dose of each treatment and how it will be given, as well as how often and for how long. For other types of trials, the protocol describes the type and timing of interventions that will be studied. Examples might include cancer screening tests, an exercise program, or a medication aimed at reducing treatment side effects. The protocol also lists all the lab work, tests, and other evaluations that will be done to figure out how each person in the trial is reacting to the treatment.
The trial protocol also describes:
- which patients are, or are not, eligible to take part
- how many participants there will be
- how long the trial is expected to last
- how patients will be protected and kept safe
After the protocol is approved, the researchers can begin asking people to be part of the trial.
Randomization, blinding, and placebos
Researchers design clinical trials in different ways to help ensure that the results will be as reliable as possible. The following are examples of three important strategies.
Randomization: Clinical trials usually group participants in one to four different treatment or intervention plans, called “arms.” In a trial with more than one treatment arm, participants are often assigned randomly to one of the arms (meaning by chance, rather than by choice). This is called “randomization” or a “randomized clinical trial.” By randomly assigning participants into treatment arms, researchers can reduce bias, ensure the treatment arms are balanced, and more accurately compare the effectiveness of treatments or other interventions as they follow groups over time.
The simplest form of randomization occurs when there are two study arms. In a therapeutic trial, for example, one group of patients is assigned to receive the new therapy as part of the intervention group, or investigational arm. The other group, called the control group, receives the standard treatment currently in use for their particular diagnosis. However, some trials can have as many as three or four different study arms.
Blinding: Many clinical trials are “double-blinded.” This means that neither the participant nor the researcher knows which treatment plan the participant is following. Results from a randomized, double-blinded trial are often considered more credible than results from a trial that isn't randomized or double-blinded.
In a double-blinded trial, the treatment that you receive is labeled with a code that is known only by a small group of people not involved in your treatment. The code is revealed at the end of the study when the results are evaluated.
Double-blinded clinical trials help researchers see the actual benefits and side effects of a treatment without bias or outside influence. For example, if you and your research team knew that you were receiving a new therapy for breast cancer, there might be a tendency to overestimate the impact it is having. This could influence the study results. Whenever possible, clinical trials are both randomized and double-blinded.
In some cases, clinical trials are single-blinded, meaning that only the researcher knows which treatment you are receiving until the study is over. This also ensures that participants won’t be biased in reporting how they are feeling or what side effects they may be experiencing.
Even if a trial is blinded, you would be able to find out which study arm you are in if there is an urgent medical reason, such as having serious side effects or developing a new health issue that could make it unsafe for you to continue. Also, if it becomes very clear in a study that a new treatment is superior to standard treatment, the trial may be stopped early and unblinded so that everyone can benefit from it.
It's not always possible to conduct a blinded clinical trial, which means that the trial is non-blinded. For example, if participants are on different treatment schedules, such as for radiation or chemotherapy, or if a trial is comparing surgery to another form of cancer treatment, it would be obvious who is receiving which treatment. If researchers are conducting a trial of an intervention such as dietary counseling or physical therapy, participants would also know which group they are in.
Placebos: Many patients are understandably concerned about receiving a placebo, or inactive treatment that resembles the real thing, instead of real treatment. However, it is very rare for placebos to be used alone in cancer clinical trials. It is unethical to withhold treatment from a participant if there is a known effective therapy for the cancer — so you would always at least receive standard treatment. Placebos would only be used if:
- there is no effective treatment for your form of cancer, and researchers are evaluating whether an investigational therapy is better than no therapy at all
- researchers are trying to determine if adding a new medication or other substance to standard treatment can improve outcomes. If that’s an oral medication, you could receive an inactive pill that looks like the real thing. However, you would still be getting the current standard treatment for your cancer.
Understanding the phases of clinical trials
Clinical trials of a new medication, therapy, or medical device are often conducted in a series of four steps, or phases. If the treatment has already been proven safe for humans, then all four phases are not always necessary.
The phases of clinical trials are not related to the stages of breast cancer (I, II, III, IV). A clinical trial's phase tells you how far along the therapy is in its testing and what the goal of the trial is. People with all stages of breast cancer can be candidates for different clinical trial phases.
Phase I trials: Is it safe?
Phase I trials, sometimes called first-in-human trials, are the first clinical trials to test a new treatment in people. Preclinical studies in petri dishes and animals have usually been done before phase I trials begin. In the United States, before a phase I trial can begin, the FDA must approve an investigational new drug application (IND), which includes results from previous studies and detailed information about the new treatment, such as its ingredients and how it is made. Phase I trials are relatively small, with about 15 to 50 people participating.
In phase I trials, researchers are figuring out:
- the best way to give a new treatment (such as by injection or as a pill)
- the highest dose that can be given safely, without serious side effects
Researchers closely monitor the participants and adjust the dose a little at time until they find the amount that works the best with acceptable side effects. This dose is usually the one used for further testing.
Everyone in a phase I trial receives the study treatment; no placebos (inactive treatments) are used. Although participants may benefit from the therapy, the main goal of a phase I trial is to find the safest dose, not to figure out if the treatment is effective.
Generally, phase I trials involve the most risk because so little is known about how the therapy works in humans. People who have a form of breast cancer that is very difficult to treat, or who have tried other treatments that weren’t successful, may decide to take part in phase I trials. These are usually offered at major cancer centers that do large amounts of research.
Phase II trials: Does it work?
Phase II trials look at how effective a new treatment is. Phase II trials are slightly larger than phase I trials and usually involve 25 to 200 people. Researchers start with the dose and method of giving the new treatment that were found to be best in phase I trials. The phase II participants are given the new treatment and the researchers watch to see if the treatment has benefits. Depending on the goals of the research, benefits the researchers look for may include:
- the tumor(s) get smaller
- the cancer stops growing
- longer time before the cancer comes back
- longer survival time
- better quality of life
Some phase II trials randomly assign people into different groups that get different doses of the treatment or receive the treatment in different ways. If a certain percentage of participants benefit from the treatment and the side effects are still acceptable, the new treatment may go on to a phase III trial. In some cases, positive results from a randomized phase II trial may be enough for a new treatment to be approved.
Phase III trials: Is it better than what we have now?
Phase III trials compare the safety and effectiveness of a new treatment to the current standard of care. Phase III trials are usually large — some involve thousands of participants — and are done at many places in the United States and around the world, including cancer centers, community hospitals, and doctors’ offices.
A phase III trial is the last step a new treatment goes through before the FDA considers approving it for general use.
In a phase III trial, participants are usually randomly assigned to receive the current standard treatment or the new treatment. If possible, the trial is double-blinded so that neither the researchers nor the participants know who's getting which treatment.
In some cases, a phase III trial may look at whether adding a new medication to the current standard of care is better than using the standard treatment alone. In that case, one group may receive a placebo in addition to standard treatment, while the others would receive the new treatment combination. A placebo is never used alone if there is an effective treatment available for your cancer. In some cases, patients in the placebo group may be switched over to the other group if the new treatment combination is proving more effective.
Just as in phase I and phase II trials, phase III trial participants are watched closely for any serious side effects. Treatment is stopped if side effects are too dangerous or difficult to manage. The trial also may be stopped early if the new treatment or treatment being tested is clearly better than the standard of care.
In the United States, when phase III clinical trials (or sometimes phase II trials) show a new treatment is more effective or safer than the current standard of care, a new drug application (NDA) is submitted to the Food and Drug Administration (FDA) for approval. The FDA reviews the results from the clinical trials and other relevant information.
Based on the review, the FDA decides whether to approve the treatment for use in patients with the illness the drug was tested on. If approved, the new treatment often becomes a standard of care option, and newer drugs may be tested against it before they can subsequently be approved.
If the FDA decides that more evidence is needed to show that the new treatment's benefits outweigh its risks, it may ask for more information or require that more studies be done.
Phase IV trials: Are there any other uses or benefits?
Phase IV trials may be done after a treatment has been approved for use by the FDA. They can involve hundreds or thousands of people. The goal is to track people over time to find out if the treatment offers benefits or produces long-term side effects that weren't studied or seen in the phase II or phase III trials.
How long clinical trials take
Clinical trials can vary widely in how long they take. A phase I trial can last several months to a year, while a phase II trial can last for a couple of years. Phase III trials can enroll and then follow patients for many years. Even if the new treatment is shown to work better than the standard treatment, researchers often continue to follow participants. It can take up to 10 years or more for a new treatment to progress from a phase I trial to widespread use in patients.
Clinical trials of new approaches to reduce the risk of cancer or improve cancer screening can also take many years, as researchers must follow participants to figure out if an intervention lowered their cancer risk or increased the odds of earlier detection.
Supportive care and symptom management trials tend to move more quickly, as they look at whether interventions such as medication, dietary changes, or exercise improve quality of life or reduce side effects for people with cancer.
Faster approvals for promising treatments
The U.S. FDA has a number of programs to speed up review and approval for new treatments that show clear promise in clinical trials or are clearly superior to standard therapies. Examples include:
- Fast track, which speeds up the FDA review process for therapies that show advantages over standard treatments (such as better outcomes and fewer side effects)
- Breakthrough therapy designation, which enables researchers with a promising therapy in early-phase clinical trials to work closely with the FDA to speed up the process of drug development
- Accelerated approval, which could make a treatment available before phase III trials are completed if it shows promise based on lab tests, imaging, symptom improvement, or some other marker
- Priority review, which can help speed up the approval process for a new treatment after the research is completed and has proven its effectiveness or superiority to standard treatments
How clinical trial participants’ safety is protected
Any research team conducting a trial of a new medication must first submit an investigational new drug (IND) application with the FDA. The FDA reviews the IND application and decides whether there is enough evidence to support moving ahead with the clinical trial. This helps protect participants because the FDA looks at previous study results, the study plan (or protocol), and the qualifications of the research team that will conduct the trial.
In the United States, most clinical trial protocols — whether for therapeutic or non-therapeutic trials — have to be reviewed by an Institutional Review Board, or IRB. An IRB is a committee that reviews research to make sure people’s rights and safety are protected, both before the study starts and as it proceeds. IRB members may include physicians, Ph.D.-level scientists, nurses, statisticians, or pharmacists who are not involved in the study. There also has to be a “non-scientist” member of the IRB, such as a lawyer, clergy member, patient advocate, or ethicist. In Europe, Ethics Committees play a similar role to IRBs.
The IRB considers several questions when making its decision about whether or not to approve the trial:
- Is it ethical to ask people to volunteer for this investigational treatment, intervention, or other approach?
- Has the study been designed to make sure the people involved will be safe?
- In a therapeutic trial, will the participants receive a treatment that is at least as good as or better than what they'd get if they weren't in the study?
If the clinical trial is approved, the IRB monitors the trial and formally reviews it each year.
Some trials also may be reviewed by a data monitoring committee, also called a data and safety monitoring board, which is an independent group of experts who conduct ongoing reviews of data from the trial to make sure it is valid and that participants are protected. Federal organizations such as the FDA and the Office of Human Subjects Research Protection also play a role in making sure that trial sponsors have protections in place for participants.
If you find a trial that you’re interested in joining, and for which you appear to be eligible, you would then go through a process called “informed consent.” Members of the research team explain the study in ways you can understand and give you and your family members an opportunity to ask questions. They also will give you an informed consent document that provides information about the study, such as:
- the purpose
- who is eligible to take part
- what is already known about the treatment or intervention being studied
- any tests or procedures that will be needed, and how often
- how the study is designed
- how long it’s expected to last
- possible risks and benefits
- alternatives to taking part in the trial
- the costs of the trial and who is responsible for them
- a contact person for further information or questions
The document also will state that participating is voluntary and you can leave the trial at any time for any reason. You can take the document home with you to discuss it with family or friends and figure out what additional questions you have. You also can work through the document with your doctor and the trial team to make sure your questions are answered. If you decide to participate, you would then sign the informed consent document.
Giving informed consent does not automatically mean you’ll be accepted into the trial. You may need to have medical tests to make sure you’re eligible to participate.
The informed consent process should continue throughout the trial. If there are changes to the study or the treatments being used, or new information about risks and benefits, you would go through informed consent again and sign a new document. Even if there aren’t any changes in the trial, there should be a process in place for updating your informed consent over time.
How to find out if you’re eligible for a clinical trial
Researchers want to make sure the results of a clinical trial are due to the study treatment and not just chance. So eligible participants are usually limited to a group with very specific characteristics.
These characteristics are known as “inclusion criteria,” and they may include your age, type and stage of the cancer, your treatment history, your current level of daily function, and other factors. A trial might be designed to answer questions about treating patients with a particular stage of breast cancer, or who have not had any treatment yet, or who already received a certain type of chemotherapy.
For example, a study comparing the effectiveness of two types of hormonal therapy — an aromatase inhibitor vs. tamoxifen, for example — might be limited to participants with the following characteristics:
- post-menopausal women
- diagnosed with early-stage hormone-receptor-positive breast cancer
- have had surgery to remove the cancer
- may or may not have had chemotherapy
- have not taken tamoxifen or any other hormonal therapy
It’s important for researchers to enroll patients who are alike in certain ways, so that the results are meaningful at the end of the study.
Trials also specify certain “exclusion criteria,” or characteristics that would prevent your participation. These may include past treatments you’ve had or other medical conditions that could make it unsafe for you to participate. For example, a new medicine may be safe only in people with normal kidney or liver function. So people with kidney or liver problems would not be accepted into the study.
If you’re interested in enrolling in a specific clinical trial, you or your doctor can reach out to the principal investigator or research coordinator to find out if you meet the eligibility requirements. You can also read about those requirements by looking up the trial on Clinicaltrials.gov or another listing service. Every trial has different requirements, so if one isn’t a good match, another is likely to be.
If you meet the requirements and give your consent to join, you’ll likely need additional lab and imaging tests to confirm your eligibility and that the trial is safe for you.
If you’re interested in a new treatment that is showing promising results in a phase III trial, but you’re not eligible for the trial, you may still be able to access it through the FDA’s Expanded Access program. Your doctor can help you decide if this makes sense for your situation.
Covering the costs of clinical trials
Generally, clinical trials involve three types of costs:
- The costs of routine care, which is the care that anyone with your type of cancer would normally receive, such as doctors’ visits, lab tests, medications to help with symptoms, and other routine procedures. These should be covered by your health insurance plan. However, if the members of the trial team are outside your plan’s network, your plan may not cover the costs, unless it includes coverage for care from out-of-network providers.
- Costs associated with the trial itself, such as the new treatment being studied and any tests or procedures needed to follow your progress or collect other data for the study. In most cases, the study sponsor would cover these costs.
- Costs of participating in the trial, such as transportation, parking, missed time at work, childcare, and possibly lodging if the trial is located far from home. Although a sponsor may cover some of the transportation costs, typically you would need to cover these costs on your own.
The Affordable Care Act requires Medicare and commercial insurers to cover routine care costs for clinical trial participants. A new coverage requirement for Medicaid is expected to be enacted as of January 2022. Generally, the trial has to be:
- approved or funded by a federal organization, such as the National Cancer Institute (NCI), the Centers for Disease Control and Prevention (CDC), or some other government department, or
- have an investigational new drug application (IND) approved by the FDA. However, some trials of existing drugs are not required to file an IND.
Insurance plans that existed on or before March 23, 2010, when the Affordable Care Act took effect, are not required to cover routine care costs in clinical trials.
As part of the informed consent process, you and the research team can talk about the costs of participating. Ask about other patients’ experience with getting coverage for routine care costs, and make sure you understand what costs you will be expected to cover. Ask who’s available to help if you encounter any issues with your insurance plan.
Benefits and risks of participating in a clinical trial
Each clinical trial has its own benefits and risks, depending on the type of trial and what its goals are. Still, some common risks and benefits are generally associated with clinical trial participation. Potential benefits
- Access to a treatment that isn't widely available yet. Through a therapeutic trial, you can gain access to a new therapy that may be better than currently available treatments. Researchers are testing it because they have reason to believe it may be more effective, or have fewer or less serious side effects than standard treatments. Also, if your cancer is no longer responding to standard treatment options, or there are no effective treatments available, a clinical trial can offer a way to access treatment.
- Regular and careful attention from some of the best cancer doctors. A clinical trial team typically includes top doctors and scientists from around the United States and the world who understand the latest advances in cancer treatment. You’ll likely have more office visits and be watched more closely than you would during standard treatment — and some people find this reassuring. Because of such close monitoring, any side effects you experience are likely to be noticed and dealt with immediately.
- Treatment that may be free or low cost. The sponsor of the clinical trial will often pay for the non-routine costs of your care related to the study, such as the study treatment, procedures, and tests. You or your health insurance are expected to cover any “routine care,” which means any cancer care you would have received even if you were not on the trial. If you need to travel to participate in a trial, costs such as gas, parking, tolls, taxis, airfare, meals, and overnight lodging may or may not be reimbursed by the trial. Make sure you know exactly what you'll have to pay for before you agree to be part of a clinical trial.
- Access to an intervention that may improve your quality of life, help reduce your cancer risk, or help to detect cancer earlier. If you participate in a supportive care trial, you may benefit from interventions such as diagnostics, dietary changes, exercise, counseling, or medications aimed at reducing treatment side effects. Prevention and screening trials can give you access to new tests and other strategies that could help reduce your risk of cancer or cancer recurrence, or increase your chances of cancer being detected earlier.
- Contributing to research that may save or improve lives in the future. The breast cancer treatments we have today are available because large numbers of women chose to participate in clinical trials. Regardless of how much you individually benefit from a trial, you’ll know that you helped researchers gain information about whether or not a new treatment is effective or better than standard treatments.
- The feeling that you're playing an active role in your care. Deciding to participate in a clinical trial can make some people feel as if they have more control over their situation, which can lead to a more positive outlook and better quality of life.
- Not being able to choose which treatment you get. In randomized trials, you’re assigned at random (by a computer) to get a specific treatment, and you could receive standard therapy rather than the newer treatment. Some people find this uncertainty quite difficult. Some cancer clinical trials may use a placebo, an inactive treatment that resembles the real treatment. However, if there is an effective standard treatment available for your cancer, you will always receive at least that in addition to the placebo. And before you enroll in a trial, you’ll always be able to find out if it is randomized, blinded, or uses a placebo.
- The risk of getting a treatment or intervention that doesn’t work for you, even if it benefits other people in the trial. It also may turn out that the new treatment isn't as effective as what's currently available.
- The risk of more severe side effects than current treatments. Though you will be closely monitored in a clinical trial, there is a chance you could experience serious side effects from an experimental therapy.
- Possible difficulty with getting your insurance to cover all the costs of routine care during the trial. Although routine care costs should be covered by your health insurance plan, since you'd need this care even if you weren’t in a clinical trial, there may be issues with coverage. Talk to your insurance company and the trial's research coordinator before you agree to participate so you'll understand all of the costs involved and who is responsible for covering them.
- More frequent testing and doctor visits, and possibly travel. Because you'll be closely monitored, you may have to undergo testing more often than you would if you weren't in a trial. This could mean more travel and time in the doctor's office or hospital. Also, if you’re in a trial at a medical center that is not located near your home, you may need time off from work or family caregiving responsibilities, and there may be travel-related expenses.
- The possibility that the trial ends early. A clinical trial may stop earlier than planned if the treatment has unexpected serious adverse events, or if it becomes clear that it is not as safe or effective as standard treatment. This can be difficult emotionally for you and other participants invested in the success of the trial.
Questions to ask before enrolling in a clinical trial
Deciding to be part of a clinical trial takes time and is different for everyone. As you're considering options, it can be helpful to ask the research team some questions. Bring along a trusted family member or friend, take notes, and consider asking if you can record any meetings when you discuss the trial.
Questions about the trial itself:
- Why are you doing this trial?
- Why do you think this new treatment/intervention will be effective?
- What phase is this trial?
- Do I meet the eligibility criteria to enroll in this trial?
- Has this treatment/intervention been tested before?
- What were the results of any previous trials?
- Can I talk to someone who's already in the trial?
- What are the possible treatments I can get? How often are they given?
- What side effects am I likely to have and how are they different from side effects with standard treatment?
- Will I be randomly assigned to a specific treatment or intervention within the trial? Will I know what group I am in, and if not, will I be able to find out after the trial is over? Will the researchers know?
- Where is the study site and how often do participants have to go there? Can I participate at a site close to home?
- What are my alternatives if I do not take part in this trial (such as standard treatment, other trials, etc.)?
- When will the results of this clinical trial be available?
Questions about safety:
- Who reviewed and approved the trial? Will that review process continue?
- How is patient safety being monitored? Have there been any unexpected or serious side effects?
- What are the potential benefits and risks of taking part? How do these compare with standard treatment for my cancer?
- What if my health gets worse during the clinical trial? Whom should I contact?
Questions about time commitment, costs, and other practical concerns:
- What types of tests will I need to have and how often will I have to have them? How different are they from the tests I would have outside of a clinical trial?
- How will being part of this study affect my daily routine?
- Who is paying for the trial?
- Will the sponsors of the trial cover the costs of the treatment I receive through the trial? Will my insurance cover any of the costs? What will I have to pay for?
- Who can help me work with my insurance plan if I face issues with coverage?
- Will I be reimbursed for any out-of-pocket expenses, such as transportation?
- How long will the trial last?
- Will I still be seen by my usual care team? If so, how often?
- Where will I receive trial-related care?
- If traveling to access the trial: Can I receive any lab tests or imaging closer to home? Are telemedicine visits an option?
- Who will oversee my medical care while I am in the trial?
- Is long-term follow-up care part of the trial? What does it involve?
- If the treatment or intervention benefits me, can I continue to receive it after the trial is done?
- How do I get the results of the trial?
- If I take medications for other medical conditions, can I continue to take them during the trial?
- If I withdraw from the trial, how will this affect my care?
How to find a clinical trial
Many people find clinical trials through their doctors, but there are online resources available to help.
Before you start searching, gather as much information as possible about your current cancer diagnosis, including your pathology report and any tumor testing results. You should also gather information on any treatments you’ve had, medications you’re on, and any history of other health conditions. This will be helpful as you try to determine your eligibility for various trials.
- If you don’t have this information handy, you can read about How to Get and Organize Your Medical Records here on Breastcancer.org.
- The National Cancer Institute offers this Cancer Details Checklist (PDF) to help you organize the information you’ll need about your cancer diagnosis when looking for a trial.
Think about how far you would be able to travel to take part in a clinical trial. If you find a trial that is right for you but is far away, organizations such as the Lazarex Cancer Foundation, the American Cancer Society, and the Cancer Support Community might be able to assist with travel costs.
Helpful tips for finding a clinical trial:
- Ask your doctors (particularly oncology providers such as your medical oncologist, surgical oncologist, or radiation oncologist) if they know of any clinical trials that would be suitable for you. If your doctors aren’t knowledgeable about clinical trials, ask if they have a colleague in the area who might be willing to assist you.
- Either set up a telehealth appointment or travel to meet in person with an oncologist at a major cancer center that is actively involved in clinical trials. Many cancer centers based at academic medical centers have searchable databases of clinical trials, which can be a good starting point.
- Search for trials on Clinicaltrials.gov, a database of privately and publicly funded clinical studies conducted around the world. You can reach out to a specific trial’s contact person to ask for more information or verify whether you meet the eligibility requirements. Contact information for the principal investigator or research coordinator is often listed towards the bottom of the page for trials listed on Clinicaltrials.gov. (If not, you can often find their contact information through the medical center where they work.) If a trial has multiple sites that are enrolling patients, you may want to reach out to the principal investigator or research coordinator at the site closest to you.
- Contact the following organizations that can help you find clinical trials:
Breastcancertrials.org has an online matching tool to help you find breast cancer clinical trials that may be right for you. The descriptions of the trials are written in patient-friendly language. You can also contact the help desk for assistance in English, Spanish, or Mandarin with finding trials over the phone or by email: 888-282-7099; firstname.lastname@example.org. If you’re looking for a clinical trial for metastatic breast cancer, you can use the Metastatic Breast Cancer Trial Search tool, which is run by the same team.
Lazarex Cancer Foundation has patient navigators who can help people with cancer find clinical trial options over the phone or by email. The Foundation can also provide support with expenses related to clinical trial participation.
The National Cancer Institute has a database of clinical trials that are sponsored or financially supported by the NCI. You also can call the NCI Cancer Information Service at 1-800-4-CANCER to get help with a clinical trial search tailored to your situation. Clinical trial-related live chats are also available.
Susan G. Komen Breast Care Helpline provides information over the phone or by email about breast cancer clinical trials, including assistance with finding a trial, knowing what to expect, and making decisions about trial participation.
Beyond these resources, there are also clinical trial listings and matching services such as CenterWatch, EmergingMed, Trialjectory, and Antidote. You can search for clinical trials according to your diagnosis and geographic location. Given that there are so many listing services, you may wish to enlist the help of a trusted family member or friend to help research what's available.
You can also read about clinical trial opportunities from Breastcancer.org sponsors on our Sponsored Clinical Trials page.
If you find any clinical trials that interest you, and for which you think you meet the eligibility criteria, print out or save the information you find. You can contact the principal investigator or research coordinator for more details and then discuss options with your doctor and care team.
Glossary: Common terms used to talk about clinical trials
As you read about clinical trials, you might see the following terms used:
- Arm: A term used to describe each group in a clinical trial that is receiving a specific treatment or intervention (or no intervention), according to the trial plan (protocol).
- Basket trial: Also called a bucket trial, it enrolls patients with different types of cancer that share a key characteristic, such as a genetic mutation or a molecular pathway that is driving the cancer. Participants receive the same treatment, targeted to that particular molecular trait, so that researchers can evaluate its effectiveness.
- Blinding: A trial design approach to ensure that either the researchers, the participants, or both will not know which treatment or intervention each group is receiving.
- Control group: The group of people in a treatment trial who are getting therapy that is the current standard of care; a placebo in addition to the standard of care; or, in rare cases, a placebo alone. In non-treatment trials, the control group does not receive the intervention under investigation.
- Data monitoring committee: Also called a data and safety monitoring board, this independent group of experts conducts ongoing reviews of data from the trial to ensure its validity as well as patient safety.
- Double-blind or Double-blinded: This means that neither the researchers nor the participants in a clinical trial know which treatment any of the participants are getting. Double-blinding keeps the study objective and helps prevent bias.
- Exclusion criteria: Characteristics that would exclude people from participating in a certain clinical trial, such as medical conditions or test results.
- Inclusion criteria: Specific characteristics outlined in the trial protocol that make someone eligible to participate, such as age, cancer type, previous treatments, etc.
- Informed consent: The process through which a person decides whether or not to participate in a clinical trial. Researchers must explain the clinical trial's protocol, share any possible risks and benefits of taking part, describe how participants will be protected, and answer questions.
- Institutional review board: A committee that is charged with reviewing research to makes sure people’s rights and safety are protected, both before the study starts and as it proceeds.
- Intervention group (or investigational arm): The group of people in a trial who are getting the new treatment/ intervention that is being studied.
- Investigational new drug application: An application that researchers submit to the U.S. Food and Drug Administration for review before they can initiate a clinical trial of a new drug.
- Phase: A term used to describe the different steps in a clinical trial, from phase I through IV. With treatment trials, phase I is the earliest step to test whether a new drug or therapy is safe, while phases II and III evaluate its effectiveness against cancer and whether it is better than current treatments. Phase IV involves ongoing monitoring for effectiveness and side effects after a new treatment is approved.
- Placebo: A sugar pill or other inactive substance/ intervention that looks the same as an active drug or treatment and is given the same way. Placebos are rarely used alone in cancer clinical trials; in most cases, patients in the control group would receive both the current standard of care and a placebo.
- Principal investigator: The lead researcher on a clinical trial who prepares the protocol, carries out the trial, analyzes the data, and reports any results.
- Protocol: The plan that describes how the clinical trial will be done; why it’s being done; how many people will participate; who is (or is not) eligible; what treatments or interventions will be given; and any information that will be collected.
- Randomized or Randomization: The process of assigning clinical trial participants to different treatment or intervention groups by chance, rather than by choice. This helps ensure the reliability of the results.
- Research coordinator: A research professional who works under the direction of the principal investigator to coordinate and support the day-to-day operations of the clinical trial.
- Routine care costs: For clinical trial participants, these are the costs of cancer care they would need even if they were not part of a trial. They are typically covered by insurance.
- Single-blinded: A trial design in which the researchers know which groups are getting which treatment/intervention, but the participants do not.
- Sponsor: The person, organization, or group that initiates, manages, and/or pays for the clinical trial (or arranges for the trial to be funded).
- Standard of care: The best current therapy for a given medical condition, based on research to date. Phase III and some phase II clinical trials are often done to compare a new treatment regimen to the standard of care.
- Umbrella trial: A trial that enrolls patients with the same type of cancer who have different gene mutations or biomarkers associated with the cancer’s development. The trial is designed to look at how a new therapy or therapies work in people with different molecular subtypes of the same cancer.
Special Report: Increasing diversity in clinical trials
Disparities in clinical trial participation among Black women could be one of the reasons they have worse breast cancer outcomes than other racial and ethnic groups in the United States.
For this Breastcancer.org Special Report, we spoke with experts and clinical trial participants about why diversity in clinical research matters, and how you can overcome common barriers that may be preventing you from participating in a breast cancer clinical trial. Read the Special Report.
To connect with others who are talking about clinical trials, join the conversation in the Breastcancer.org Community Forum: Clinical Trials, Research News, Podcasts, and Study Results.
Written by: Kris Conner, contributing writer
This content was developed with contributions from the following experts:
Nadine Barrett, Ph.D. MS, MA, medical sociologist, assistant professor of family medicine and community health at Duke University School of Medicine, founding director of the Office of Health Equity and Disparities at the Duke Cancer Institute, director of the Center for Equity in Research within the Duke Clinical and Translational Science Institute, Durham, NC
Jenni Sheng, M.D., assistant professor of oncology, Johns Hopkins University School of Medicine, breast medical oncologist, and research member of GRASP (Guiding Research and Advocates to Scientific Partnerships) in Baltimore, MD
Chirag Shah, M.D., breast radiation oncologist, director of breast radiation oncology and clinical research in radiation oncology at the Cleveland Clinic in Cleveland, Ohio
Marisa Weiss, M.D., chief medical officer and founder of Breastcancer.org, director of breast radiation oncology at Lankenau Medical Center in Wynnewood, PA
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