National Organization for Rare Disorders (NORD) Company Profile

Background

The National Organization for Rare Disorders (NORD) is a pioneering nonprofit organization dedicated to improving the health and well-being of individuals affected by rare diseases. Established in 1983 following the passage of the Orphan Drug Act, NORD has been instrumental in advocating for the rare disease community, providing support, education, and driving policy changes. The organization's mission is to advance care, research, and policy to ensure that people with rare diseases can live their fullest and best lives.

Key Strategic Focus

NORD's strategic objectives encompass:

Care: Facilitating access to world-class care and expertise for individuals seeking diagnoses, specialists, or clinical trials.

Research: Accelerating innovation in rare disease treatment through research grants, studies, patient registries, and partnerships.

Policy and Advocacy: Advocating for life-changing laws, policies, and programs that benefit the rare disease community.

Community Support: Strengthening a network of over 340 patient advocacy organizations to serve more than half a million rare disease patients.

Financials and Funding

As a 501(c)(3) nonprofit organization, NORD relies on contributions, program services, and investment income to fund its operations. In 2023, the organization reported:

Revenue: $66.4 million

Expenses: $52.8 million

Net Assets: $60.6 million

The majority of revenue (approximately 83.7%) was derived from contributions, underscoring the organization's strong support from donors and partners.

Pipeline Development

NORD actively engages in research initiatives to advance understanding and treatment of rare diseases. Notably, in 2020, the organization launched the Undiagnosed Rare Disease Registry, a study aimed at collecting de-identified information about challenging medical cases. This registry enables researchers to perform large-scale analyses, potentially leading to improved diagnostic methods and treatments for undiagnosed conditions.

Technological Platform and Innovation

NORD's commitment to innovation is evident through its IAMRARE® program, a patient-powered natural history study platform. This initiative empowers patients and caregivers to contribute data, facilitating research and enhancing the understanding of rare diseases. Additionally, NORD collaborates with partners like PlatformQ Health to develop digital education resources, improving clinical practice and patient outcomes in the rare disease community.

Leadership Team

NORD's leadership comprises experienced professionals dedicated to advancing the organization's mission:

Pamela Gavin: Chief Executive Officer

Kelly Esperias: Chief Strategy & Operations Officer

Edward Neilan, M.D., Ph.D.: Chief Medical and Scientific Officer

Prashant Goel: Vice President of Information Technology

Alexa Moore: Vice President of Development

Jill L. Pollander, RN, MSN: Vice President of Patient Services

Heidi Ross: Vice President of Policy and Regulatory Affairs

Tracey Sikora: Vice President of Research & Clinical Programs

Norine Schwarzchild: Vice President of Human Resources

Cheryl Herbert: Vice President of Marketing & Communications

Patrick Collins: Vice President of Community & Corporate Affairs

These leaders bring diverse expertise to NORD, driving initiatives in care, research, policy, and community support.

Leadership Changes

In October 2023, NORD hosted the Rare Diseases and Orphan Products Breakthrough Summit, bringing together over 800 advocates, pharmaceutical companies, regulators, and scientific and medical leaders to discuss pressing issues facing the rare disease community.

Competitor Profile

Market Insights and Dynamics

The rare disease sector encompasses over 7,000 known conditions affecting approximately 30 million Americans. Despite this significant impact, about 95% of these diseases lack FDA-approved treatments, highlighting a substantial unmet need and opportunities for organizations dedicated to rare disorders.

Competitor Analysis

While NORD is a leading organization in the rare disease advocacy space, other entities also contribute to this field:

Global Genes: A nonprofit organization that connects, empowers, and inspires the rare disease community through education, awareness, and advocacy.

EveryLife Foundation for Rare Diseases: Focuses on advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy.

Rare Disease Legislative Advocates (RDLA): A program of the EveryLife Foundation that provides a platform for rare disease patients and organizations to advocate for policy changes.

These organizations, along with NORD, play crucial roles in addressing the challenges faced by the rare disease community.

Strategic Collaborations and Partnerships

NORD collaborates with various partners to enhance its impact:

PlatformQ Health: Together, they have developed nearly 70 digital education sessions, reaching over 90,000 clinicians, patients, and caregivers, resulting in improved clinical practices and patient outcomes.

Mount Sinai Hospital: Designated as a Rare Disease Center of Excellence, this partnership aims to improve rare disease diagnosis, treatment, and research.

National Partnerships: NORD works with numerous national partners to advocate for better lives for individuals with rare diseases.

Operational Insights

NORD's strategic initiatives, such as the IAMRARE® program and the establishment of Rare Disease Centers of Excellence, position the organization as a leader in rare disease advocacy and research. These efforts, combined with strong financial health and a dedicated leadership team, provide NORD with a competitive advantage in addressing the needs of the rare disease community.

Strategic Opportunities and Future Directions

Looking ahead, NORD aims to:

Expand Research Initiatives: Continue developing patient registries and natural history studies to advance rare disease research.

Enhance Educational Programs: Strengthen partnerships to provide comprehensive education for clinicians, patients, and caregivers.

Advocate for Policy Changes: Drive legislative and policy initiatives that benefit the rare disease community.

Strengthen Community Support: Grow the network of patient advocacy organizations to provide robust support for individuals affected by rare diseases.

By leveraging its current strengths and strategic partnerships, NORD is well-positioned to achieve these objectives and further its mission.