Rare cancers, collectively, are not so rare: 1 in 10 Americans, or 30 million individuals in the United States,1and more than 4 million Europeans2 are diagnosed with a rare form of cancer. Rare cancers—186 of which have been identified—comprise at least 20% of all cancer diagnoses, more than any single commonly occurring cancer, including breast (16%), lung (13%), colorectal (13%), and prostate (12%).3
“Rare cancers pose significant challenges to patients, their families, and all parties involved in cancer care,” according to Rare Cancers Europe. These include “late or incorrect diagnosis, lack of access to appropriate therapies and clinical expertise, very limited number of clinical studies due to the small number of patients, lack of interest in developing new therapies due to limitations in the market, and few available registries and tissue banks.”4The International Rare Cancers Initiative (IRCI) notes, “unfortunately, the average outcome for patients with a rare cancer is inferior to those with more common cancers.” Established in 2011, the IRCI is a joint initiative among the National Institute for Health Research Cancer Research Network and Cancer Research UK in the United Kingdom, the US National Cancer Institute (NCI), and the European Organisation for Research and Treatment of Cancer (EORTC).3
In the United States, the Rare Disease Act of 2002 defines a rare disease as one that affects small patient populations, that is, fewer than 200,000 persons.5 By this definition, “pediatric cancer as a whole is a rare disease,” noted Alberto S. Pappo, MD, of St. Jude Children’s Research Hospital in Memphis, TN, and colleagues writing in the Journal of Clinical Oncology in 2010.6 Approximately 13,500 children and adolescents are diagnosed with cancer annually in the United States, more than 90% of whom are cared for at a Children’s Oncology Group (COG) member institution.7
The COG, an NCI-supported clinical trials group, “is the world’s largest organization devoted exclusively to childhood and adolescent cancer research,” with 8,000 experts in childhood cancer at more than 200 institutions in North America, Europe, and New Zealand.
In 2002, the COG developed a Rare Tumor Initiative, which subsequently became the Rare Tumors Committee in 2008. By 2012, a Working Group of the infrequent tumor subcommittee developed recommendations for clinicians caring for eight rare tumors: thyroid carcinomas, colorectal carcinoma, melanoma, gastrointestinal stromal tumor (GIST), pancreatoblastoma, desmoplastic small round cell tumor, gonadal stromal tumors, and carcinoid.8 Published in the May 2012 issue of the Journal of Pediatric Hematology/Oncology, “the authors of each rare tumor guideline also form a network of experts available to treating clinicians to help with the management of these rare tumors.”
Recently, the Rare Tumors Committee “completed accrual for the first time for two trials for pediatric rare cancers: nasopharyngeal carcinoma and adrenocortical carcinoma,” Dr. Pappo told ChemotherapyAdvisor.com. “The results of these two trials will allow better therapies and better understanding of these diseases in children.”