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 just 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 and of those with rare cancers, about 1 out of 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 one and 14. What is surprising is that rare cancers actually represent more than 20% of all cancer diagnoses, making them as a category, the most common.  The reason for this is that there are many types of rare cancer, but few per specific type are afflicted.

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

History of Rare Cancer Clinical Trials

Researchers have discovered through learning about the genetics of individual diseases, that there are certain rare forms of any particular disease.   As there is limited experience or clinical data on successful treatments for these newly discovered cancers, most treatment decisions are based on the limited or anecdotal information of what is known about that cancer or cancers that are similar. Learn More from Healio

It is very challenging to conduct clinical studies in each rare cancer type because there are so few patients to conduct large clinically significant studies and current regulations do not allow for studies in such small populations due to statisitical significance (also called powering).  Additionally, the costs of supporting such studies are much higher than in cancers with larger populations because patients are harder to find and studies would be lengthy.  Due to these challenges, the Rare Diseases Act of 2002 was enacted to create more support, research, funding  and awareness of individual cancers that are classified as rare. Learn more from the Sloan Kettering Cancer Center

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Current Rare Cancer Research

More recently, the International Rare Cancers Initiative – IRCI – was formed in 2011 as a partnership between the National Institute of Health Research Cancer Research Network (NCRN) in England, Cancer Research UK, the Europe an Organisation for Research and Treatment of Cancer (EORTC) and the United States of America (USA) National Cancer Institute Cancer Therapy Evaluation Program (CTEP), and was joined in 2013 by the French National Institute of Cancer (INCa) to foster the more rapid development of international clinical trials for rare cancers.

This group is working to develop harmonized  rare cancer clinical trial designs that will allow for smaller populations and still prove efficacy and safety. 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 a specific tumor mutation (or mutations) that may also be present in other more common cancer types, and can potentially result in the identification of therapies targeted to that mutation rather than tumor type.  Rare cancer patients with the mutation may qualify for clinical trial studies, called “basket studies” Learn more about basket trials clinical that test a therapy (or combination of therapies) targeted to the mutation in multiple tumor types.  These studies are an opportunity for patients with rare cancers to join a larger study and help in 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.

Through our Clinical Trial Exchange, Cure Forward collects data on clinical trials and studies from multiple sources making it easier to find all the options, including basket trials, that are available for you or your loved one. We work directly with clinical trial recruiters to help bring current, active studies directly to you.

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