National Organization for Rare Disorders: Strengthening Rare Cancer Advocacy

Rare cancers account for 25% to 30% of all cancer diagnoses and approximately 25% of all cancer deaths, thereby posing a significant public health burden.¹ Recognizing the need for action to address this health crisis, the National Organization for Rare Disorders (NORD) established the Rare Cancer Coalition in 2017 to alleviate the challenges faced by people living with rare cancers. Since its inception, the Rare Cancer Coalition has reached millions of people through patient and caregiver education sessions, healthcare provider education, and public awareness campaigns. Since its inception, the Rare Cancer Coalition has reached millions of people through patient and caregiver education sessions, healthcare provider education, and public awareness campaigns. The Coalition Members have had an impact on other rare cancer advocacy groups, contributed to medical publications, and provided collaborative networking opportunities among patients, advocates and researchers.

Thanks to successful advocacy by the Rare Cancer Coalition, the United States Congress established “Rare Cancer Day.” This event takes place annually on September 30 and brings global awareness to rare cancers through mass media and public events. In recognition of Rare Cancer Day 2024, NORD focused its public education on the importance of patient participation in rare cancer research. 

NORD and the Rare Cancer Coalition are deeply committed to addressing the unique challenges faced by the rare cancer community. Moving forward, we will focus on promoting the development of innovative and effective treatments, enhancing access to diagnostic testing, advancing new technologies, and fostering research that leads to improved medical approaches for patients with rare cancers.

To that end, we are pleased to present the 2024 Rare Disease Report: Hematology and Oncology in collaboration with our partners at MDedge. This issue will highlight some of the latest advances in rare cancer research, diagnosis, and treatments that are providing new hope for improved outcomes. In this issue, you will find articles that cover recent discoveries on specific rare cancers, including:

• The promise of mTOR inhibitors in improving malignant PEComas
• How novel immunotherapies are demonstrating the potential for improved outcomes for large cell neuroendocrine carcinoma of the lung
• Potential paradigm shifts in the treatment of glioblastoma leveraging CAR T-cell therapies and targeted inhibitors
• Future directions in the treatment of gallbladder cancer with molecular profiling, immunotherapies, and targeted treatments
• The benefits of a multidisciplinary approach in addressing cutaneous T-cell lymphomas
• The evolving role of JAK inhibitors in managing symptoms of myelofibrosis
• Advancements in staging and tailored treatments for hepatoblastoma
• And more!

We hope these articles will enhance your knowledge and enrich your day-to-day clinical practices. We invite you to explore NORD resources including digital CME sessions and disease-specific reports written in accessible language for patients and families. Additionally, you can sign up for our quarterly Caring for Rare newsletter, for timely updates on rare diseases. 

Thank you for your commitment to advancing care for rare cancer patients. Your dedication to staying informed is vital for improving patient outcomes.

Read more from the 2024 Rare Diseases Report: Hematology and Oncology.

Rare cancers account for 25% to 30% of all cancer diagnoses and approximately 25% of all cancer deaths, thereby posing a significant public health burden.¹ Recognizing the need for action to address this health crisis, the National Organization for Rare Disorders (NORD) established the Rare Cancer Coalition in 2017 to alleviate the challenges faced by people living with rare cancers. Since its inception, the Rare Cancer Coalition has reached millions of people through patient and caregiver education sessions, healthcare provider education, and public awareness campaigns. Since its inception, the Rare Cancer Coalition has reached millions of people through patient and caregiver education sessions, healthcare provider education, and public awareness campaigns. The Coalition Members have had an impact on other rare cancer advocacy groups, contributed to medical publications, and provided collaborative networking opportunities among patients, advocates and researchers.

Thanks to successful advocacy by the Rare Cancer Coalition, the United States Congress established “Rare Cancer Day.” This event takes place annually on September 30 and brings global awareness to rare cancers through mass media and public events. In recognition of Rare Cancer Day 2024, NORD focused its public education on the importance of patient participation in rare cancer research. 

NORD and the Rare Cancer Coalition are deeply committed to addressing the unique challenges faced by the rare cancer community. Moving forward, we will focus on promoting the development of innovative and effective treatments, enhancing access to diagnostic testing, advancing new technologies, and fostering research that leads to improved medical approaches for patients with rare cancers.

To that end, we are pleased to present the 2024 Rare Disease Report: Hematology and Oncology in collaboration with our partners at MDedge. This issue will highlight some of the latest advances in rare cancer research, diagnosis, and treatments that are providing new hope for improved outcomes. In this issue, you will find articles that cover recent discoveries on specific rare cancers, including:

• The promise of mTOR inhibitors in improving malignant PEComas
• How novel immunotherapies are demonstrating the potential for improved outcomes for large cell neuroendocrine carcinoma of the lung
• Potential paradigm shifts in the treatment of glioblastoma leveraging CAR T-cell therapies and targeted inhibitors
• Future directions in the treatment of gallbladder cancer with molecular profiling, immunotherapies, and targeted treatments
• The benefits of a multidisciplinary approach in addressing cutaneous T-cell lymphomas
• The evolving role of JAK inhibitors in managing symptoms of myelofibrosis
• Advancements in staging and tailored treatments for hepatoblastoma
• And more!

We hope these articles will enhance your knowledge and enrich your day-to-day clinical practices. We invite you to explore NORD resources including digital CME sessions and disease-specific reports written in accessible language for patients and families. Additionally, you can sign up for our quarterly Caring for Rare newsletter, for timely updates on rare diseases. 

Thank you for your commitment to advancing care for rare cancer patients. Your dedication to staying informed is vital for improving patient outcomes.

Read more from the 2024 Rare Diseases Report: Hematology and Oncology.

Rare cancers account for 25% to 30% of all cancer diagnoses and approximately 25% of all cancer deaths, thereby posing a significant public health burden.¹ Recognizing the need for action to address this health crisis, the National Organization for Rare Disorders (NORD) established the Rare Cancer Coalition in 2017 to alleviate the challenges faced by people living with rare cancers. Since its inception, the Rare Cancer Coalition has reached millions of people through patient and caregiver education sessions, healthcare provider education, and public awareness campaigns. Since its inception, the Rare Cancer Coalition has reached millions of people through patient and caregiver education sessions, healthcare provider education, and public awareness campaigns. The Coalition Members have had an impact on other rare cancer advocacy groups, contributed to medical publications, and provided collaborative networking opportunities among patients, advocates and researchers.

Thanks to successful advocacy by the Rare Cancer Coalition, the United States Congress established “Rare Cancer Day.” This event takes place annually on September 30 and brings global awareness to rare cancers through mass media and public events. In recognition of Rare Cancer Day 2024, NORD focused its public education on the importance of patient participation in rare cancer research. 

NORD and the Rare Cancer Coalition are deeply committed to addressing the unique challenges faced by the rare cancer community. Moving forward, we will focus on promoting the development of innovative and effective treatments, enhancing access to diagnostic testing, advancing new technologies, and fostering research that leads to improved medical approaches for patients with rare cancers.

To that end, we are pleased to present the 2024 Rare Disease Report: Hematology and Oncology in collaboration with our partners at MDedge. This issue will highlight some of the latest advances in rare cancer research, diagnosis, and treatments that are providing new hope for improved outcomes. In this issue, you will find articles that cover recent discoveries on specific rare cancers, including:

• The promise of mTOR inhibitors in improving malignant PEComas
• How novel immunotherapies are demonstrating the potential for improved outcomes for large cell neuroendocrine carcinoma of the lung
• Potential paradigm shifts in the treatment of glioblastoma leveraging CAR T-cell therapies and targeted inhibitors
• Future directions in the treatment of gallbladder cancer with molecular profiling, immunotherapies, and targeted treatments
• The benefits of a multidisciplinary approach in addressing cutaneous T-cell lymphomas
• The evolving role of JAK inhibitors in managing symptoms of myelofibrosis
• Advancements in staging and tailored treatments for hepatoblastoma
• And more!

We hope these articles will enhance your knowledge and enrich your day-to-day clinical practices. We invite you to explore NORD resources including digital CME sessions and disease-specific reports written in accessible language for patients and families. Additionally, you can sign up for our quarterly Caring for Rare newsletter, for timely updates on rare diseases. 

Thank you for your commitment to advancing care for rare cancer patients. Your dedication to staying informed is vital for improving patient outcomes.

Read more from the 2024 Rare Diseases Report: Hematology and Oncology.