NORD: Making Progress Through Collaboration



Katie Kowalski, MPH

For nearly 40 years, the National Organization for Rare Disorders (NORD) has worked to drive meaningful and enduring impact so that people living with rare diseases, including rare cancers, can live their best lives. We are proud to collaborate with MDedge to deliver timely information about rare cancers to healthcare professionals. Rare cancers are those that affect fewer than 40,000 people per year in the United States. While the incidence of each rare cancer may be low, collectively, they make up a significant proportion (27%) of all cancers.Moreover, rare cancers present unique challenges: they are difficult to identify and often diagnosed at later stages when they are harder to treat. Patients often have trouble finding specialists who are familiar with their rare cancer. Additionally, the availability of effective drugs to treat rare cancers is limited and enrollment in rare cancer clinical trials is challenging due to small, and often not diverse, study populations. Currently, the 5-year survival rate for rare cancers in adults (48.5%) is worse than for common cancers (63.4%).2

While people living with rare cancers continue to face daunting obstacles, progress is being made, and there are reasons to hope for a better future. Advances in genomic testing and precision medicine provide increasing evidence that rare cancers can be more efficiently and effectively diagnosed and treated. Genomic tests examine tumor DNA to identify mutations that are unique to an individual’s cancer. This genetic information enables a more precise diagnosis and targeted treatment approach. Jim Palma, Co-Lead of the NORD Rare Cancer Coalition, said “There is promise for rare cancer patients due to increased legislative efforts to cover the costs of genomic testing coupled by an increase in FDA approvals for targeted and tissue agnostic therapies.”

In 2019, the National Cancer Institute established MyPART, a vast pediatric and adult rare tumor network that aims to bolster patient involvement in research and develop effective therapies through tumor sample collection, shared data, shared samples, new methods to test treatments, and new trial designs. In 2022, MyPART welcomed NORD’s Rare Cancer Coalition as an advocacy partner.

Meanwhile, advocacy organizations are giving rare cancer a rising voice. NORD’s Rare Cancer Coalition unites rare cancer patient advocacy organizations and helps them drive progress together. The coalition promotes research and awareness through its annual Rare Cancer Day (September 30) campaign. Additionally, NORD has produced over 22 continuing medical education modules on rare cancers in collaboration with PlatformQ Health, providing updates on new therapies and treatment approaches. NORD also offers rare disease reports and educational videos on rare cancers, sessions inclusive of rare cancer topics at the annual NORD Summit, and a quarterly e-newsletter, “Caring for Rare” for healthcare professionals. Please visit us at to access these resources.

Much work on rare cancers remains to be done, but the progress over recent years points to better outcomes moving forward. We are grateful for the work you do and your dedication to your patients, including those with rare cancers and other rare conditions. We hope you will find the information in this special issue useful for your clinical practice.

– Katie Kowalski, MPH
Associate Director of Education
National Organization for Rare Disorders


Click to read more from 2023 Rare Diseases Report: Cancers

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