Why Consider a Clinical Trial?

For many women, a clinical trial may represent the best treatment option available for their breast cancer — yet most patients never consider it. Understanding why begins with addressing the fears that hold people back.

“Will I receive a placebo — the sugar pill?”

No. Breast cancer trials do not give inactive placebos when an established treatment exists. Every trial has two treatment arms: the first offers the best currently available standard therapy; the second offers that same standard therapy plus a promising new treatment being evaluated. You will always receive real, active treatment.

“Will I be stuck in the trial — treated like a guinea pig?”

No. Participation is entirely voluntary and you may withdraw at any time. Beyond that, the physicians overseeing your care are ethically bound to act in your best interest at every stage. If the experimental arm is not working for you, your doctor is obligated to remove you from it. You and your health care team retain decision-making power throughout.

“What if I’m randomized into the wrong group?”

Randomization — the process by which a computer impartially assigns patients to a treatment arm — is the scientific cornerstone of reliable clinical research. It is important to understand that if one treatment arm were clearly superior to the other, the trial would not need to be conducted at all. Randomization exists precisely because genuine uncertainty remains about which approach is better — and resolving that uncertainty is how medicine advances. Many trials also include provisions allowing patients to switch to the experimental arm under certain conditions.

Every advance in breast cancer treatment over the past four decades — every drug, every surgical refinement, every radiation protocol now considered standard of care — became available because patients chose to participate in randomized clinical trials. Participating in a trial is not gambling with your health. It is taking an active, informed role in your own care while contributing to a body of knowledge that will benefit every person diagnosed after you.

Read on to learn what clinical trials involve, what questions to ask, and how to find one that may be right for you.

Clinical Trials: Discover Your Options

A breast cancer diagnosis can feel overwhelming — but it also opens doors to options that many people never consider. A clinical trial is not a last resort. It is one of the most forward-looking, proactive choices a patient can make. Through a clinical trial, you may gain access to the most advanced treatments available anywhere in the world, often before they reach standard clinical practice.

This page is designed to help you understand what clinical trials are, what to expect, what questions to ask, and how to find a trial that may be right for you.

“Without the heroic efforts of those who participated in breast cancer clinical trials, new medicines, procedures and other treatment options may not have been obtainable. These people took an active role in their treatment — and may have not only helped themselves, but also the millions of other breast cancer survivors who have benefited from their efforts.”

— Bev Parker, Breast Cancer Survivor and Y-ME Hotline Director

What Is a Clinical Trial?

A clinical trial is a carefully designed research study that evaluates new approaches to cancer prevention, diagnosis, treatment, or survivorship care in human participants. Clinical trials are the gold standard through which promising new therapies move from laboratory discovery into approved medical practice.

When you join a clinical trial, you will receive either a new treatment being evaluated or the current standard of care — and in some cases, a combination of both. Researchers then compare outcomes to determine whether the new approach is more effective, safer, or better tolerated than existing options.

Every treatment currently used to treat breast cancer — every chemotherapy regimen, targeted therapy, immunotherapy, surgical technique, and radiation protocol — was validated through clinical trials conducted by patients who chose to participate. Clinical trials are how progress happens.

Despite this, fewer than 5% of adult cancer patients in the United States currently enroll in clinical trials. This gap slows the pace of discovery and delays the availability of potentially life-saving treatments. Your participation matters — not just for you, but for every person diagnosed after you.

Types of Clinical Trials

Not all clinical trials test new drugs. Breast cancer trials span a wide range of research questions:

• Treatment trials — Test new drugs, drug combinations, surgical techniques, radiation approaches, or targeted therapies. The majority of breast cancer trials fall into this category.
• Prevention trials — Study ways to reduce the risk of developing breast cancer in people who are at elevated risk but have not yet been diagnosed.
• Screening and detection trials — Evaluate new methods for detecting breast cancer earlier or more accurately, such as novel imaging technologies or blood-based biomarker tests.
• Quality of life and supportive care trials — Examine ways to improve comfort, manage side effects, and support the emotional and physical wellbeing of patients during and after treatment.
• Biomarker and genomic trials — Investigate the role of specific genes, proteins, or molecular markers in predicting breast cancer behavior or response to treatment.

Phases of Clinical Trials

Clinical trials progress through four phases, each designed to answer different questions:

• Phase I — Tests a new treatment in a small group of patients (typically 20–80) for the first time. The primary goal is to assess safety, determine a safe dosage range, and identify side effects. Phase I trials are often open to patients who have exhausted standard options.
• Phase II — Expands the study to a larger group (typically 100–300 patients) to evaluate whether the treatment works for a specific cancer type and to continue assessing safety.
• Phase III — Compares the new treatment against the current standard of care in a large, often randomized group of patients (hundreds to thousands). This phase determines whether the new treatment is more effective or has fewer side effects. FDA approval is typically based on Phase III results.
• Phase IV — Conducted after a treatment has received FDA approval to monitor its long-term safety, effectiveness, and optimal use in real-world populations.

Are Clinical Trials Safe?

All clinical trials conducted in the United States must be reviewed and approved before they begin, and are subject to ongoing oversight throughout. The safeguards in place include:

• Institutional Review Board (IRB) approval — Every trial must be reviewed and approved by an independent IRB, which evaluates the study’s scientific merit, ethical design, and protections for participants.
• FDA oversight — Trials of new drugs or devices are regulated by the U.S. Food and Drug Administration.
• Informed consent — Before enrolling, you must be fully informed of the study’s purpose, procedures, potential benefits, known risks, and your rights. You may take as much time as you need before deciding, and you may ask questions at any point.
• Data Safety Monitoring Boards (DSMBs) — Independent committees review ongoing trial data at regular intervals. If a new treatment is found to be clearly superior — or clearly harmful — the trial can be modified or stopped early to protect participants.
• NCI Common Terminology Criteria for Adverse Events (CTCAE) — A standardized system for reporting and grading side effects ensures consistent safety monitoring across all trials.

As with any medical treatment, there are inherent risks. These may include unexpected side effects, the possibility that the experimental treatment proves no more effective than the standard, or additional time and visits required by the study protocol. These risks are thoroughly disclosed during the informed consent process.

You will never receive a placebo (inactive treatment) in a breast cancer trial if an established standard of care exists. All participants receive at minimum the current accepted standard of treatment. Placebos are used only in addition to standard treatment, or when no proven treatment exists for the condition being studied.

Potential Benefits of Participating

• Access to cutting-edge therapies not yet available outside of a research setting
• Care provided by specialists and leading researchers in breast cancer
• Closer monitoring of your health, with more frequent assessments than standard care
• Many trial-related costs — including the investigational drug itself, additional imaging, and extra lab work — are paid for by the trial sponsor
• An opportunity to contribute to science and help future patients
• A more active role in your own care and treatment decisions

Will My Insurance Cover a Clinical Trial?

Insurance coverage for clinical trials has improved significantly over the past decade:

• The Affordable Care Act (ACA) requires most health insurance plans to cover the routine patient care costs associated with approved clinical trials — including standard tests, doctor visits, and hospitalizations that would be covered outside of a trial.
• Medicare covers routine costs for Medicare beneficiaries enrolled in qualifying clinical trials.
• Medicaid coverage varies by state, but many states now cover routine trial costs.
• Costs for the investigational treatment itself, extra tests required only by the trial protocol, and research-related procedures are typically covered by the trial sponsor — whether a pharmaceutical company, the NCI, or another institution.
• Some trials also provide reimbursement for travel, lodging, and time — ask the trial coordinator about this when you inquire.

Always verify coverage details with your insurer before enrolling. Your trial coordinator and hospital social worker can help you navigate coverage questions.

Can I Leave a Trial Early?

Yes — at any time, for any reason, without penalty. Participation in a clinical trial is entirely voluntary. You may withdraw at any point, and doing so will not affect your access to other medical care or your relationship with your health care team. Your decision will always be respected.

Who Conducts Breast Cancer Clinical Trials?

Clinical trials are sponsored and conducted by a wide range of organizations:

• The National Cancer Institute (NCI) — the largest public funder of cancer research in the world, sponsoring trials through a national network of cancer centers and cooperative groups
• NCI-designated Cancer Centers — comprehensive research hospitals throughout the U.S. that conduct trials across all cancer types
• Pharmaceutical and biotechnology companies — testing new drugs and combinations they have developed
• Academic medical centers and universities
• Nonprofit research organizations and patient advocacy groups

Major cooperative groups currently conducting breast cancer research include:

• ECOG-ACRIN Cancer Research Group (formerly ECOG and ACRIN, now merged)
• NRG Oncology (formed by the merger of NSABP, RTOG, and GOG)
• SWOG Cancer Research Network
• Alliance for Clinical Trials in Oncology (formerly CALGB and NCCTG)
• Canadian Cancer Trials Group (CCTG)

Current Areas of Active Breast Cancer Trial Research (2024–2025)

Breast cancer clinical trial research is moving rapidly. Key areas with active enrollment include:

• Antibody-drug conjugates (ADCs) — including trastuzumab deruxtecan (T-DXd) and sacituzumab govitecan for HER2-positive, HER2-low, and triple-negative breast cancer
• PARP inhibitors — for patients with BRCA1/2 mutations, including combinations with immunotherapy
• Immunotherapy combinations — particularly pembrolizumab (Keytruda) in triple-negative and early-stage breast cancer
• CDK4/6 inhibitor combinations — extended use and novel combinations in hormone receptor-positive, HER2-negative metastatic breast cancer
• PI3K/AKT/mTOR pathway inhibitors — including capivasertib and inavolisib in PIK3CA-mutated metastatic breast cancer
• Neoadjuvant and adjuvant de-escalation trials — studying whether certain patients can safely receive less intensive treatment without compromising outcomes
• Liquid biopsy and ctDNA monitoring — using circulating tumor DNA to guide treatment decisions and detect recurrence earlier
• Metastatic breast cancer quality of life trials — studying interventions that improve physical and emotional wellbeing alongside treatment
• Prevention trials in high-risk populations — including BRCA mutation carriers and women with dense breasts or atypical hyperplasia

Questions to Ask Before Enrolling

Before joining any clinical trial, make sure you fully understand what is involved. Bring this list of questions to your conversation with your oncologist and the trial team:

About the Trial Itself

• What is the purpose of this study, and what phase is it?
• What are my other treatment options outside of this trial?
• What are the potential advantages and disadvantages of the trial treatment compared to the current standard of care?
• Has this treatment shown promising results in earlier phases?
• How many people are enrolled, and how long has the trial been running?

About Your Care

• What tests, procedures, and treatments does the study involve?
• How often will I need to come in for visits or tests?
• Will I still see my regular oncologist, or will my care transfer to the trial team?
• Who is responsible for my care if I experience side effects?
• What side effects should I expect, and how will they be managed?
• Is long-term follow-up care part of the study?

About Logistics and Cost

• Will this trial require extra travel or time away from work?
• What costs will my insurance cover, and what will I need to pay out-of-pocket?
• Does the sponsor cover the cost of the investigational treatment and extra tests?
• Is there reimbursement for travel, lodging, or childcare?
• If I am harmed as a result of the study, what treatment and compensation am I entitled to?

About Your Rights

• Can I withdraw from the trial at any time without affecting my other care?
• Will I be informed of the results when the trial concludes?
• Can I continue the treatment after the trial ends if it is working for me?
• Can I speak with other participants currently enrolled in the study?

How to Find a Clinical Trial

Finding the right trial begins with a conversation with your oncologist. You can also search independently using the following resources:

• ClinicalTrials.gov — The official U.S. federal registry of all clinical trials, both federally and privately funded. Search by diagnosis, location, phase, and eligibility criteria. Call 1-800-4-CANCER (1-800-422-6237) for assistance.
• National Cancer Institute (NCI) — Comprehensive clinical trial information including a patient-friendly search tool and guidance on how to talk to your doctor. Visit cancer.gov or call 1-800-4-CANCER.
• BreastCancerTrials.org — A specialized, patient-friendly matching service for breast cancer-specific trials. Enter your diagnosis details and location to receive a personalized list of open trials.
• Susan G. Komen Clinical Trials Support — Resources, guidance, and a helpline to assist with finding and understanding trials. Call 1-877-GO-KOMEN.
• CenterWatch — A comprehensive database of clinical trials searchable by condition, drug, and location, with news on recently approved therapies.
• Your treating institution — Ask your oncologist, breast surgeon, or breast center directly. Many NCI-designated cancer centers have a dedicated clinical trials navigator who can help identify options specific to your diagnosis.

Tip: When searching, have the following information available: your diagnosis (cancer type, stage, receptor status — ER, PR, HER2), prior treatments received, any relevant genetic test results (BRCA1/2, etc.), and your geographic location or willingness to travel.

We Are Here for You

Navigating the clinical trial process can feel daunting. You do not have to do it alone. If you have questions, want to talk through your options, or simply need to speak with someone who has been through a clinical trial herself, our Y-ME peer counselors are here for you — any hour of any day.

Y-ME National Breast Cancer Organization Hotline:
📞 1-800-221-2141 — English, 24 hours a day, 7 days a week
📞 1-800-986-9505 — Spanish, 24 hours a day, 7 days a week
Interpreters available in more than 150 languages.

All calls are confidential. You are not alone — and you never have to be.