National Organization for Rare Disorders

CPAN Advocacy Chats typically begin with COA and CPAN news updates. This month’s announcements featured two major events affecting advocacy and patient care.

Save the Date – September 28, 2022

September 28 is an important date for advocates. It is the 2022 Community Oncology Alliance (COA) and COA Patient Advocacy Network (CPAN) virtual Advocacy Summit. The Summit will feature presentations by leading health care experts on the latest issues affecting patient care. For additional information and to register, go to the CPAN Advocacy Summit 2022 Website.

Advocacy Chat listeners should also review the Breaking News section of the COA website for the latest cancer care news. An explanation of the newly announced Enhanced Oncology Care Model (EOM) is available in this section.

Rare Diseases Are Not So Rare

This month’s Advocacy Chat guest speaker was Jill L. Pollander, RN, MSN, vice president of patient services for the National Organization for Rare Disorders (NORD). NORD offers hope, support, and resources to patients with rare disorders, including rare types of cancers.

When assessing a patient, the conventional wisdom says, “When you hear hoofbeats, think horses, not zebras,” meaning think of the common diagnoses. Sometimes what a provider is seeing is not common, not usual, unconventional, and rare. As a reminder, the zebra has become the official mascot of rare disorders.

For many, the term “rare” means something they will likely not encounter or something that will not affect them. With over 7,000 rare diseases affecting 25-30 million people, “rare” is not particularly rare and touches many.

The History of Rare

As recently as the 1970s, families suffering from rare diseases felt alone and forgotten. Little was done to diagnose, study, or develop drugs and treatments for rare disorders. Leaders of various rare disease organizations formed a coalition to focus on this problem. This coalition eventually became the non-profit organization NORD and was instrumental in the Orphan Drug Act of 1983 which created financial incentives and stimulation for the development of treatments for rare diseases. Since that time, NORD has served as a hub for the rare disease community leading efforts to connect patients, patient organizations, and other stakeholders to drive progress for the rare disease community. NORD believes that all people living with rare disorders have a fundamental right to their best health and well-being. With that belief as its guide, the organization advocates for solutions to address complex issues and needs.

Patient Services and Support

NORD has a dedicated Patient Services team that understands the needs that accompany a rare diagnosis and provide:

  • Patient Assistance Programs
  • Information and Resource Services
  • Clinical Trial Support

With a 40-year focus on serving and supporting the rare disease community, their programs and services have assisted in overcoming obstacles and access to care.

Some of the most common patient needs are:

  • Resources and information
  • Inquiries for financial assistance
  • Diagnosis information
  • Finding specialists and Centers of Excellence
  • Navigating insurance
  • General guidance and direction

NORD’s dedicated communications center enables seamless communication via phone, email, and fax, providing patient and information services with high-touch, caring, concierge-level service.

Demonstrating that “rare is not so rare,” in 2021, NORD assisted nearly 10,000 individuals, provided $36 million in financial support, and answered almost 135,000 calls and emails.

NORD’s Rare Cancer Coalition

One in five people in the United States living with cancer is diagnosed with a rare cancer. All pediatric cancers are classified as rare cancers. Officially launched in 2018, NORD’s Rare Cancer Coalition (RCC) is comprised of more than two dozen rare cancer patient advocacy organizations that are collaborating on issues facing the rare cancer community. RCC collaborates with many organizations including:

  • S. Department of Defense (DoD)
  • Peer Reviewed Cancer Research Program (PRCRP)
  • National Institutes of Health (NIH)
  • Global Drug Development (World Orphan Drug Congress)
  • Rare Cancer Day
  • Food and Drug Administration
  • Oncology Center of Excellence (OCE)
  • Rare Cancer Congress

The RCC has been a powerful advocate for the rare cancer community through collaborations with the U.S. Department of Defense and congressionally directed medical research programs. In 2019, for the first time, a panel dedicated to rare cancers was held at Rare Disease Day at NIH with the RCC included on that panel, and RCC also spoke on several panels at the World Orphan Drug Congress. Additionally, NORD’s RCC launched the first Rare Cancer Day which is observed annually on September 30, focusing on raising awareness of all rare cancers and the challenges and emerging trends in the rare cancer space. The day is designated not only to those with rare cancers but also as a day of support for those caring for them.

In 2020, the coalition secured representation at a special stakeholder meeting at the DoD regarding its congressional-directed medical research funding. This meeting worked to direct increased funding for medical research for rare cancers. The RCC continually plays a critical role in advancing rare cancer concerns.

Patients with rare cancers and their caregivers can access additional information, resources, and support by contacting NORD’s Rare Cancer Coalition directly.

NORD’s Centers of Excellence Program

In 2021, NORD launched its Centers of Excellence (CoE) program by recognizing 31 Centers of Excellence throughout the United States. In partnership with these centers, NORD works to advance the needs and cares of the rare cancer community by bringing together teams of clinical experts in a nationwide network of cutting-edge facilities to provide standards of specialized care for people living with a rare cancer diagnosis.

The most frequent questions about rare disease treatment are often, “How do I find treatment?” and “How do I find a medical home for my disease?” NORD’s CoE providers work in partnership to share expertise, advance education, and define standards and protocols to shape what rare disease care can look like so patients can build complex care plans for their complex diseases.

The Importance of Caregivers

NORD provides support for caregivers because behind every patient with a rare disease there is a caregiver who provides daily, non-stop support that is critical to the patient’s well-being. From the rare disease respite program to educational support programs, NORD recognizes that to support the caregiver is to support the patient, and when a caregiver falters, a patient can falter. The NORD website has complete information about what is available for caregivers.

NORD’s Policy and Advocacy Team

NORD’s Policy and Advocacy Team focuses on what is needed to advance the care of patients with a rare cancer or other rare disease. They work to identify, define, and advocate for critical legislation and regulation at the federal and state levels to address the unique needs of the almost 30 million Americans living with rare diseases.

The team’s activities also encompass educational initiatives that include:

  • Living Rare, Living Stronger Patient and Family Forum
  • Rare Diseases and Orphan Products Breakthrough Summit
  • Providing a clear, vetted source of medical information which is differentiated from social information
  • Partnerships and programs to empower patients and their families to take an active role in managing their health
  • Informing and engaging the next generation of providers at every professional level
  • Dedicated research department that provides support, tools, expertise, and grants
  • Supporting patient advocacy groups and their collection of natural history data about many rare diseases
  • Participating in FDA grants to develop clinical outcome assessments and to promote data standardization
  • Developing innovative study design that takes into consideration the smaller numbers of participants as compared to more common diseases, such as diabetes or heart disease

Finding a clinical trial for patients with rare diseases can be difficult. NORD’s advocacy team will help those patients navigate the very robust website which is incrementally more complex for those with rare diseases. While NORD will not recommend a particular clinical trial, it can prepare patients to have a clinical trial conversation with their physician.

NORD Is an Advocacy Partner for Patients with a Rare Cancer

At NORD there is a saying: “Alone we are rare; together we are strong.” For those who would like additional information or to participate in Rare Disease Day on September 30, contact NORD.

August Advocacy Chat

CPAN Advocacy Chats are regular virtual events with a guest speaker to discuss issues important to advocacy and advocates in a 30-minute webinar. Do not miss the next Advocacy Chat which will be on –

Wednesday, August 10, 2022
12:00 PM – 12:30 PM ET

The guest speaker will be –
Lauren Chatalian, MSW, LCSW
Director of Advocacy

This event will be part of our virtual CPAN Advocacy Chats series – short, monthly educational webinars on key cancer issues and the policies that impact them. Each month will feature a new topic and special guests joining us. Patients, survivors, caregivers, and other cancer care advocates are all invited to join us for these free, interactive, virtual events!

CPAN Advocacy Chats are regular virtual 30-minute educational conversations about cancer advocacy and policy with a guest speaker invited to discuss issues important to patients and advocates. Summaries of previous Advocacy Chats are available on the CPAN website.

  • Upcoming Advocacy Chat
    Cancer Wellness – A Focus on the Importance of Supportive Care

    February 14, 2024 12:00 pm

    The Community Oncology Alliance Patient Advocacy Network (CPAN) held a virtual Advocacy Chat on Wednesday, February 14, 2024 at 12 p.m. ET.