Rare Cancer Clinical Trials

Clinical trials are research studies that test how well new medical approaches work in people. All clinical trial participants are volunteers.

Clinical trials are not possible without people who volunteer to participate. People participate in clinical trials for a variety of reasons, including to:

• access treatments in development that have the potential to be more effective than currently available options.
• access therapies that are not approved for their specific cancer, but are approved by the U.S. Food and Drug Administration (FDA) for other cancers.
• help future patients by contributing data to research that will help drive discoveries.

To me, clinical trials equal hope – and it’s something all cancer patients cling to.

Katie Doble, patient advocate

The safety of clinical trial participants is the top priority of medical researchers. To protect patients and ensure reliable study results, clinical trials follow strict safety procedures. Volunteers who participate in clinical trials are protected by the careful review and approval of the clinical trial protocol by an independent review panel, the informed consent process, and ongoing monitoring. Monitoring of participants on clinical trials is often more frequent and more specialized than would occur with treatments delivered outside of a trial.

Informed consent is the process through which a doctor or research team explains the trial to potential participants before they decide whether or not to participate. This includes the trial’s purpose, procedure, and possible risks and benefits. It exists to inform participants about their rights and ensure they have all the information they need to voluntarily enroll. It is also worth noting that participants can choose to exit a clinical trial at any time.

No. A common myth about clinical trials is that they are a last resort, to be explored only after other forms of treatment have failed. But today, many people with rare cancers choose to participate in a clinical trial early in their treatment, and sometimes even as a first treatment.

While clinical trials may be suggested in cases where the standard of care treatment options have not been successful, that is not the only time they are applicable. In fact, sometimes, patients who have been treated with insufficient standard of care therapies are ineligible for clinical trials so it is important to consider a trial early in your treatment journey.

Clinical trials can be a critical treatment option for people with rare cancers where no standard of care exists. Having access to clinical trials means that patients have access to the latest science through some of the top medical experts in the world specializing in rare cancers.

Yes. You can take part in clinical trials that you are eligible for, regardless of whether or not your primary doctor or medical center is involved.

My experience is that oncologists are enthusiastically supportive of patients who are interested in enrolling in clinical trials at other institutions. I strongly recommend talking to your oncologist about your interest in trials as your doctor can help guide you through the process.

James Cleary, MD, Dana-Farber Cancer Institute

Let your doctor know that you are interested in joining a clinical trial. They can help determine what studies you may be eligible for and also help provide copies of your medical record for enrollment.

Rare cancer-specific patient advocacy foundations are another good way to locate clinical trials that may be right for you. Search for a foundation dedicated to your specific type of rare cancer and ask if they have a list of relevant trials or a patient navigator who can help you find trials dedicated to your cancer type.

Additionally, ClinicalTrials.gov is an online database of clinical research studies where you can learn about trials and results around the world.

Before enrolling in a trial for which travel is required, ask the research team if travel costs — such as flights or housing expenses — will be reimbursed. If not, reach out to the patient advocacy foundation focused on your type of cancer to get connected to patient assistance services and programs that may be able to help.

Clinical trials are divided into different phases, each with a different purpose.