National Organization for Rare Disorders, Inc.
Alone we are rare. Together we are strong.
Programs and results
What we aim to solve
For many years, people with rare diseases walked alone. Patients and their families coped with daunting medical and financial issues with few resources and no one to guide the way. Then, a small group of patient advocates formed a coalition to unify this community and mobilize support to pass the Orphan Drug Act. In 1983, the coalition became NORD, the National Organization for Rare Disorders. For more than 35 years, NORD has led the way in voicing the needs of the rare disease community, driving supportive policies, advancing medical research, and providing patient and family services for those who need them most.
Our programs
What are the organization's current programs, how do they measure success, and who do the programs serve?
Patient Services
NORD works closely with humanitarian-minded pharmaceutical and biotechnology companies to ensure that certain vital medications are available to those individuals whose income is too high to qualify for Medicaid but too low to pay for their prescribed medications. NORD’s programs have set the standards
for fairness, equity, and unbiased eligibility. The NORD programs have gained respect from the patient communities, pharmaceutical companies, healthcare professionals, government officials, and the public.
Participants have the assurance that NORD protects their confidentiality. There is no conflict of interest with respect to NORD’s eligibility decisions.
Education
NORD has been a trusted source for providing critical educational resources to the rare disease community. NORD’s educational initiatives include innovative partnerships and programs to empower patients and their families, inform students of all ages, and support the vitally important work of physicians and other healthcare professionals.
Some of the educational programs and resources we offer for patients, students, and medical professionals include:
Rare Disease Database:
The Rare Disease Database provides reports on more than 1,200 rare diseases for patients and their families.
Continuing Medical Education:
This program will raise diagnostic awareness of typical patient symptoms and treatments that are actually atypical and continue to manifest over time. Diagnostic resources, tools and detailed infrastructure of resources for both providers and patients will be demonstrated in this program.
Students for Rare:
Graduate and medical students can launch a student chapter program at their university to get students informed and interested in rare disease research and awareness. Additionally, high school students and undergraduate medical students have the opportunity to launch a NORD student club at their school to promote rare disease education and awareness.
Joint Publications with NORD:
Through its partnerships with Frontline Medical Communications and Medscape, NORD provides useful medical publications and video resources for clinicians as well as researchers.
Research
NORD’s Research Grant Program provides seed grants to academic scientists for translational or clinical studies related to the development of potential new diagnostics or treatments for rare disease. In at least two cases, NORD grants have resulted ultimately in FDA-approved treatments for patients.
The studies funded with NORD research grants provide preliminary data, on drugs, devices or medical foods that researchers may then use to attract funding from government or industry sponsors to pursue further clinical studies.
Policy & Advocacy
Starting with the Orphan Drug Act in 1983, NORD’s mission has always included ensuring that the laws, regulations, and incentives surrounding the drug development process encourage orphan therapy development. For over 35 years, NORD has worked to strengthen the orphan drug development process. NORD works with patients, patient organizations, Congress, the FDA, and the biopharmaceutical industry to strengthen the orphan drug development landscape.
NORD’s Rare Action Network® (RAN) is the nation’s rare disease advocacy network working to improve the lives of the 30 million Americans impacted by rare disease. The mission of RAN is to connect and empower a unified network of individuals and organizations with tools, training, and resources to become effective advocates for rare diseases through national and state-based initiatives across the United States.
Where we work
External reviews
Our results
How does this organization measure their results? It's a hard question but an important one.
Evaluation documents
Download evaluation reportsNumber of policies formally established
This metric is no longer tracked.Totals By Year
Related Program
Policy & Advocacy
Type of Metric
Outcome - describing the effects on people or issues
Direction of Success
Increasing
Average number of dollars given by new donors
This metric is no longer tracked.Totals By Year
Type of Metric
Other - describing something else
Direction of Success
Increasing
Number of coalition members
This metric is no longer tracked.Totals By Year
Related Program
Policy & Advocacy
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Our Sustainable Development Goals
Learn more about Sustainable Development Goals.
Goals & Strategy
Learn about the organization's key goals, strategies, capabilities, and progress.
Charting impact
Four powerful questions that require reflection about what really matters - results.
What is the organization aiming to accomplish?
For rare disease patients fortunate to have a treatment for their disease, there are still many barriers to accessing affordable and accessible care. From high drug costs, to insurance difficulties, to trouble finding a specialist, rare disease patients face unique hurdles to finding appropriate treatment. NORD is committed to breaking down these barriers and ensuring access to affordable treatment for every rare disease patient.
What are the organization's key strategies for making this happen?
• Bring a national awareness and recognition of the challenges endured by people living with rare diseases;
• A culture of innovation that supports basic and translational research to create diagnostic tests and therapies for all rare diseases;
• Access for all patients to the diagnostics and therapies that will extend and improve their lives.
• A regulatory environment that encourages development and timely approval of safe, effective diagnostics and treatments.
What are the organization's capabilities for doing this?
NORD's Rare Action Network℠ (RAN) is the nation's rare disease advocacy network working to improve the lives of the 25-30 million Americans impacted by rare disease. The mission of RAN is to connect and empower a unified network of individuals and organizations with tools, training, and resources to become effective advocates for rare diseases through national and state-based initiatives across the United States.
Since 1987, NORD has provided assistance programs to help patients obtain life-saving or life-sustaining medication they could not otherwise afford. These programs provide medication, financial assistance with insurance premiums and co-pays, diagnostic testing assistance, and travel assistance for clinical trials or consultation with disease specialists.
What have they accomplished so far and what's next?
For almost 40 years, we have been providing services for patients and their families, rare disease patient organizations, medical professionals, and those seeking to develop new diagnostics and treatments. We are here to support every member of the rare disease community with programs and services focused on one ultimate goal: to improve the lives of individuals and families affected by rare diseases. We hope to continue doing this through patient advocacy, patient and professional education, patient assistance programs, mentorship for patient organizations, research support, and international partnerships.
How we listen
Seeking feedback from people served makes programs more responsive and effective. Here’s how this organization is listening.
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How is your organization using feedback from the people you serve?
To identify and remedy poor client service experiences, To identify bright spots and enhance positive service experiences, To make fundamental changes to our programs and/or operations, To inform the development of new programs/projects, To identify where we are less inclusive or equitable across demographic groups, To strengthen relationships with the people we serve, To understand people's needs and how we can help them achieve their goals
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Which of the following feedback practices does your organization routinely carry out?
We collect feedback from the people we serve at least annually, We take steps to get feedback from marginalized or under-represented people, We aim to collect feedback from as many people we serve as possible, We take steps to ensure people feel comfortable being honest with us, We look for patterns in feedback based on people’s interactions with us (e.g., site, frequency of service, etc.), We engage the people who provide feedback in looking for ways we can improve in response, We act on the feedback we receive, We tell the people who gave us feedback how we acted on their feedback, We ask the people who gave us feedback how well they think we responded
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What challenges does the organization face when collecting feedback?
We don't have any major challenges to collecting feedback
Financials
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Operations
The people, governance practices, and partners that make the organization tick.
Connect with nonprofit leaders
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- Analyze a variety of pre-calculated financial metrics
- Access beautifully interactive analysis and comparison tools
- Compare nonprofit financials to similar organizations
Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.
Connect with nonprofit leaders
SubscribeBuild relationships with key people who manage and lead nonprofit organizations with GuideStar Pro. Try a low commitment monthly plan today.
- Analyze a variety of pre-calculated financial metrics
- Access beautifully interactive analysis and comparison tools
- Compare nonprofit financials to similar organizations
Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.
National Organization for Rare Disorders, Inc.
Board of directorsas of 02/01/2024
Kay Holcombe, MS
Milken Institute; Critical Path Institute
Frederick (Rick) E. Barr
Children's of Mississippi / Batson Children's Hospital
Leon Eidelman
JP Morgan Chase & Co
Dennis Jackman
Independent Board and Organizational Consultant
Jim Palma
TargetCancer Foundation
Mike Porath
The Mighty
Mark Skinner
Institute for Policy Advancement
Susan A. Berry, MD
University of Minnesota
Shafali Spurling Jeste, MD
Children's Hospital Los Angeles, USC Keck School of Medicine
Philip L. Pearl, MD
Boston Children's Hospital
Peter L. Saltonstall
National Organization for Rare Disorders
Lorna Weir
Elevate Healthcare
Tom Kelly
UnitedHealth Group (retired)
Sheldon Schuster PhD
Keck Graduate Institute of Applied Health Sciences
Board leadership practices
GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.
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Board orientation and education
Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations? Yes -
CEO oversight
Has the board conducted a formal, written assessment of the chief executive within the past year ? Yes -
Ethics and transparency
Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year? Yes -
Board composition
Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership? Yes -
Board performance
Has the board conducted a formal, written self-assessment of its performance within the past three years? Not applicable
Organizational demographics
Who works and leads organizations that serve our diverse communities? Candid partnered with CHANGE Philanthropy on this demographic section.
Leadership
The organization's leader identifies as:
Race & ethnicity
Gender identity
No data
Transgender Identity
No data
Sexual orientation
No data
Disability
No data
Equity strategies
Last updated: 09/30/2021GuideStar partnered with Equity in the Center - an organization that works to shift mindsets, practices, and systems to increase racial equity - to create this section. Learn more
- We disaggregate data to adjust programming goals to keep pace with changing needs of the communities we support.
- We employ non-traditional ways of gathering feedback on programs and trainings, which may include interviews, roundtables, and external reviews with/by community stakeholders.
- We disaggregate data by demographics, including race, in every policy and program measured.
- We have long-term strategic plans and measurable goals for creating a culture such that one’s race identity has no influence on how they fare within the organization.
- We have a promotion process that anticipates and mitigates implicit and explicit biases about people of color serving in leadership positions.
- We seek individuals from various race backgrounds for board and executive director/CEO positions within our organization.
- We help senior leadership understand how to be inclusive leaders with learning approaches that emphasize reflection, iteration, and adaptability.
- We measure and then disaggregate job satisfaction and retention data by race, function, level, and/or team.
- We engage everyone, from the board to staff levels of the organization, in race equity work and ensure that individuals understand their roles in creating culture such that one’s race identity has no influence on how they fare within the organization.