The Role of Clinical Trials in the Treatment of Rare Cancers

Clinical trials are important treatment options for anyone with a rare cancer to consider. Because standard therapies for incurable rare cancers are often very limited, a clinical trial may be the best way to access investigational treatments that are more effective than current treatment options.

While some clinical trials are specific to one type of rare cancer, other trials, like our TCF-001 TRACK clinical trial, enroll people with different rare cancer types.

All modern advances in cancer treatment — from drugs to vaccines to immunotherapies and beyond — are founded on progress made through clinical trials. However, the decision to participate in a clinical trial can feel overwhelming and confusing. Here’s what you need to know.

haley ellis

Clinical trials are absolutely critical for advancing treatment options, particularly in rare cancers where standard of care often is just insufficient in giving us the responses we hope for.

Haley Ellis, MD

Massachusetts General Hospital

Rare Cancer Clinical Trials Q&A

What are clinical trials?

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

Why consider participating in a rare cancer clinical trial?

Today, people with cancer are having better health outcomes thanks to the results of clinical trials. Clinical trials test new approaches to diagnosing, treating, and managing rare cancers and help doctors improve the quality of life for people living with them. They also allow researchers to gather data on new treatments and procedures using safe, controlled methods before they are approved by the FDA and available to the general public.

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
Katie Ortman Doble

Are rare cancer clinical trials safe?

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.

Are clinical trials a last resort for individuals with rare cancer?

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.

Can I participate in a rare cancer clinical trial that my doctor is not involved in?

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

Dr James Cleary 1

How do I find a rare cancer clinical trial that is right for me?

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

Will I have to travel to participate in a rare cancer clinical trial?

Not necessarily. Some clinical trial programs require eligible participants to come to the main investigational site, while a small but growing number offer the option to participate remotely (like TCF-001 TRACK). Some hybrid trial models may only require participants to travel once or twice and then collect data using electronic devices like phone or computer apps, mobile health devices, or telehealth videoconferences between participants and clinical trial doctors.

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.

What do I need to understand about the phases of rare cancer clinical trials?

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

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Phase 1: Safety

Phase 1 trials test if a new treatment is safe, examine possible side effects and, if the trial is testing a new drug, establish recommended dosage. It may also begin looking for signs that the cancer is responding to the new treatment. It is worth nothing that response rates in Phase 1 trials have nearly doubled over the last 20 years.

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Phase II: Efficacy

Phase II trials test whether or not the cancer responds to the new treatment. It looks for evidence of this efficacy, which may include data such as whether or not the treatment being tested shrinks or stops the growth of a tumor.

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Phase III: Benefit over other treatments

Phase III trials test if a new treatment is better than the standard treatment for the cancer in question. This could include testing the new treatment against either a placebo or the current standard treatment. A clinical trial will never use a placebo without first explicitly telling the participants. It must be disclosed on the consent form. In cancer clinical trials, placebos by themselves are used very infrequently. It is more common that, if a placebo is used, it is used in conjunction with the current standard of care. In addition, many trials using placebo also offer a “crossover” option, meaning that patients on placebo will be moved to the clinical trial drug if their cancer progresses.

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Phase IV: Long-term benefits and use

Phase IV trials test the long-term benefits and side effects of a new treatment after it has been approved for use in patients. Phase IV trials are less common than the other phases.

Watch TCF Webinar: Clearing the Air About Clinical Trials

Watch Speaker Katie Doble discuss the importance of clinical trials in her care

Key questions for your doctor about rare cancer clinical trials

If you are thinking about taking part in a clinical trial, you should feel empowered to ask any questions you have before, during, and after the trial. Possible questions may include:

  • What is the purpose of this clinical trial?
  • Why do researchers think the therapy/approach being tested will be effective?
  • What will my responsibilities be if I take part?
  • How long will this clinical trial last?
  • How could being in this study affect my daily life?
  • Where will I have my medical care?
  • Who has reviewed and approved the clinical trial protocol?
  • How is the safety of participants being monitored?
  • How will I learn about the results of this clinical trial?
  • What are my possible benefits and risks?
  • What other options are available?
  • How do the possible benefits and risks of this trial compare with other treatment options?
  • What side effects can I expect? And who do I speak with to help manage any side effects?
  • Will I be able to take my regular medicines while taking part in the clinical trial?
  • Is there a cost to participate in this clinical trial?
  • If so, what will the charges be and what is my health insurance likely to cover?

TCF-001 TRACK Clinical Trial

TCF-001 TRACK is an innovative rare cancer clinical trial enrolling 400 patients with rare cancers or cancer of unknown primary. Through a remote consenting process, patients can fully enroll in the TRACK study from their homes, without traveling to a clinical trial site. Enrolled patients and their physicians receive biomarker (genomic) testing of both tissue and blood, as well as interpretation and treatment recommendations from an expert panel of rare cancer clinicians and scientists.