How Do Breast Cancer Clinical Trials Work?

No matter your breast cancer stage, participating in a clinical trial can not only improve your level of care, but can make a difference in the lives of future patients, often at little or no extra cost. Yet despite these benefits, only a small minority of patients (approximately 5%) will enroll in a clinical trial as part of their cancer care [1-3]. Here are some of the most common questions I get from patients about participating in clinical trials.

What Are Clinical Trials?

Clinical trials are performed to evaluate whether a specific medical treatment, diagnostic test, or lifestyle intervention is superior to the traditional (or “standard-of-care”) therapy.

Some clinical trials investigate drugs that are at the earliest stage of development and haven’t previously been provided to human patients. The majority of studies, however, are evaluating drugs which have been studied either in smaller groups of patients (“early phase studies”) or in patients with other tumor types.

The most common clinical trial type investigates a new drug or new drug combination. For patients felt to be at increased risk, these trials often include a combination of an existing therapy with the trial drug. For patients perceived to be at lower risk, trials can evaluate whether we can safely reduce therapy. Due to the complexity of clinical trials it is important that patients discuss the details of any specific trial with their oncologist to determine whether a specific study is appropriate based on their medical history.

Why Are Clinical Trials Important?

Trials are critical to the overall progress in cancer care and are responsible for the improvement in cancer survival rates. Well-designed clinical trials have lead to the approval of highly effective new treatments and identified potential drugs that were ineffective or even harmful.

In addition to benefiting the overall patient population, a trial can be important in an individual patient’s management by providing access to a therapy that would otherwise be unavailable.

Are Clinical Trials Safe?

Because of the critical importance of prioritizing patient safety, clinical trials in the U.S. undergo significant internal and external review prior to being permitted to enroll patients. Trials often include more frequent office visits and imaging studies than would otherwise be performed as part of the standard-of-care management. They also have mechanisms in place to promptly identify unexpected side effects of the treatment under investigation. If the therapy the patient is receiving is not working, the patient can transition to an alternative therapy in a timely manner.

Who Can Participate in a Clinical Trial?

Frequently the initial conversation with a patient regarding participation in a clinical trial is met with concern or confusion. For patients with active cancer, there is often fear that participation indicates that they have exhausted all standard treatment options. For cancer-free patients, the suggestion of participation is often met with the concern that their oncologist’s recommendation indicates that their cancer is set to return.

While clinical trials are an important treatment option for patients with advanced cancer (stage IV or metastatic cancer), clinical trial participation can be appropriate for patients with any stage of breast cancer (Including stage 0!). At the end of 2019, 217 therapeutic breast cancer studies were actively enrolling in New York on clinicaltrials.gov (a repository of clinical trials in the US). Notably, nearly half were intended for early-stage cancer, cancer that has not spread beyond the breast.

How Do Clinical Trials Work With Insurance?

Given the increased monitoring, patients are often concerned about the costs of studies performed on the trial. Trials are often funded by a pharmaceutical company or a medical organization or group. While each trial has an individual payment structure, usually this funding organization will cover the costs of the drug or test being studied as well as any additional monitoring above and beyond what would be considered as standard-of-care. Standard-of-care costs, the treatments and monitoring a patient would normally receive as part of their cancer care, are then covered by a patient’s insurance.

In general, federal law protects patients. Patients should not experience any increase in out-of-pocket cost from participating in an approved clinical trial. If any concerns exist regarding the patient’s coverage this should be explained in advance to the patient by the study team.

The Bottom Line

In summary, I would encourage all patients with breast cancer to discuss clinical trial participation with their oncologist. The majority of cancer physicians have been involved at some point in their career with a clinical trial and I routinely enroll patients in trials, when appropriate. Clinical trials allow me to both provide innovative care as well as partner with my patient in defining improved treatment strategies, which will inform patient care in the future.