Rare Cancer Clinical Trials

The true definition of “rare cancer” is challenging because it can be defined in different ways- it can be a cancer that is in an unusual location (such as inner ear); a cancer that is a subtype of a more common cancer but rarely diagnosed (for example male breast cancer or acute myeloid leukemia); or a type that is simply not very common (such as GIST-gastrointestinal stromal tumor). Rare cancer is defined in the US as a cancer that occurs in less than 15 people out of 100,000 each year. Of those who are diagnosed with rare cancers, about 1 in 3 have a very rare type (1 out of 100,000). All pediatric cancers are considered rare, despite the fact that cancer is the leading cause of death by disease in children between the ages of 1 and 14.

What is surprising to many is that rare cancers actually represent more than 20% of all cancer diagnoses, making them in aggregate, the most common category of cancer. The reason for this is that there are many types of rare cancer, but very few people affected by each individual type of cancer.

Rare cancers represent more than 20% of all cancer diagnosis, making them as a category the most common.

History of Rare Cancer Clinical Trials

There is a large body of research in the area of rare cancers; however, because there is limited clinical treatment data, as well as very little experience successfully treating these newly discovered cancers, most treatment decisions are based on limited or anecdotal information. Learn More from Healio

It is very challenging to conduct clinical studies in each rare cancer type, because there are so few patients that it is difficult to recruit enough patients to conduct large clinically significant studies. Current regulations make it difficult to gain approval based on studies in small populations, because of concerns that they are not well powered, and therefore not likely to produce statistically significant results.  Additionally, the costs of such studies are much higher than cancer trials involving larger populations, because patients are harder to find and trials need to be longer term in order to generate sufficient data. In response to these challenges, the Rare Diseases Act of 2002 was enacted to create more support, research, funding and awareness around rare cancers.  Learn more from the Sloan Kettering Cancer Center

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Current Rare Cancer Research

In 2011, the International Rare Cancers Initiative (IRCI)was formed in as a partnership between the National Institute for Health Research Cancer Research Network (NCRN) in England, Cancer Research UK, the European Organization for Research and Treatment of Cancer (EORTC), the United States of America (USA) National Cancer Institute Cancer Therapy Evaluation Program (CTEP), and the French National Institute of Cancer (INCa), with the goal of facilitating more rapid development of therapies for rare cancers by conducting international clinical trials that can combine patients from various countries to create a larger study population .

This group is working to develop harmonized (meaning that the standards are the same) rare cancer clinical trial designs that will allow for smaller populations, while still being able to prove that an intervention is safe, effective, and possibly ready for regulatory approval.  Learn More from the European Journal of Cancer

The advent of molecular profiling has also made a significant impact on rare cancer research. It allows for identification of specific tumor mutations that may also be present in other more common cancer types, potentially resulting in identification of therapies targeted to that mutation rather than tumor type. Rare cancer patients who have a specific mutation may qualify for clinical trial studies, called “basket studies.” Basket studies are designed to test a therapy (or combination of therapies) targeted to the mutation in multiple tumor types. These studies represent an opportunity for patients with rare cancers to join a larger study, while being part of the search for a cure.

Learn more from the Sloan Kettering Cancer Center

Molecular profiling has made a significant impact on Rare Cancer research.

Why Cure Forward?

When you’re seeking rare cancer clinical trials, it can be hard to find trials that might be a good match.

When you get started with Cure Forward, your personal Clinical Trial Navigator will help you and your care team build a robust profile inclusive of your full medical history, personal preferences and molecular profile (when applicable), all at no cost. We are able to provide this free service, because we focus on building robust profiles so we can match patients with relevant and active clinical trial options, opening the door to advanced treatments and accelerating medical innovation. We work directly with clinical trial recruiters to help bring current, active studies directly to you.