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May. 4, 2018

TOPIC: Advocacy, Featured News, Patient Stories, Patients & Members

35 Ways and Growing: NORD’s Service to the Rare Disease Community

Posted by Laura Mullen

For 35 years, NORD has been leading the fight to improve the lives of patients and families with rare diseases. We do this by supporting patients and organizations, accelerating research, providing education, disseminating information, raising public awareness, and driving public policy. The following are 35 examples of how NORD provides service to all of the rare disease community, including the testimonials of some patients and advocates regarding their involvement with NORD.

PATIENTS & FAMILIES

1. Patients and families turn to NORD for help finding a physician, getting an accurate diagnosis, receiving financial assistance, addressing issues of access, or any other rare disease related questions
2. NORD serves as the key educational resource for the 1 million+ individuals who visit www.rarediseases.org each month and the 110,000+ who reach out via phone for information or guidance
3. NORD helps families and organization navigate the complex payor landscape through educational programs and webinars
4. NORD developed the patient assistance concept and today provides financial assistance through its various assistance programs to those in need
5. “Thank you so much for renewing coverage for the VSL#3 DS for my daughter, Hagan Hill. Her doctor has seen marked clinical improvement and Hagan has seen significant at home improvements since being on this medical grade probiotic. We are so very grateful for this NORD program. I feel that my daughter would not be able to achieve these results as successfully as she has without this vital probiotic and NORD making it possible.” –Jeanie M. Hill
6. A 2nd-year medical resident with a rare disease (autoimmune hepatitis) contacted NORD because there was no report on her condition on the NORD website. From her unique perspective as both a patient and a physician, she felt it was very important for NORD to provide patient-friendly information on this topic. She offered to help develop such a report, working with a leading medical expert. With her help, and the guidance of the medical expert, a report on autoimmune hepatitis was developed for NORD’s Rare Disease Database and now is available free to all who visit the NORD website.
7. “NORD’s Eileen Mayo has been a tremendous help to me. She has patiently listened to my long, drawn out explanations and guided me through each individual step required for initiating the process. She is very good at hearing what I have to say, knowing all the rules and processes, and answering my endless questions. She is good at returning my calls in a timely manner and explaining things in a detailed, easy to understand, and cheerful way. Thank you, Eileen.” –Mike Dunn, patient

PATIENT ORGANIZATIONS

8. NORD serves as the umbrella organization for rare disease patient organizations, and mobilizes a network of 280 patient advocacy, support and research organizations around legislative and regulatory priorities
9. NORD provides programs to help patient organizations grow and develop through mentorship and peer-to-peer support and will also provide tailored solutions to meet an organization’s specific needs
10. NORD offers grants to aid organizations in developing natural history studies to further research into their disease using NORD’s registry platform
11. “NORD has been a beacon for us at PSC Partners. From being an organization that was growing alone, we are now an organization that is using all the cues, guidance and opportunities offered to us by NORD to create networks with stakeholders we couldn’t even dream of connecting with before. We hope we can give to other rare disease organizations all that we are learning from NORD.” –Rachel Gomel, PSC Partners
12. “For both ATOF and myself, attendance at last year’s NORD Summit changed our nonprofit and put us years ahead in research and collaborations with our medical partners. It is imperative we are able to participate again this year. We are saving lives because of you.” –Andrea Taylor, A Twist of Fate
13. “The Morgan Leary Vaughan Fund (Morgan’s Fund) emerged from our family’s recognition of the need to establish a mechanism for disseminating credible and timely information about Necrotizing Enterocolitis (NEC) to parents whose infants are most at risk for developing NEC. NEC is a rare, inflammatory disease that leads to necrosis (death) of the intestine. NEC is the second leading cause of death in premature infants.

In April 2015, we became the first patient organization to advocate for NEC as a rare disease when we joined NORD and gained NEC official recognition as a rare disease and a listing in
their Rare Disease Database. On January 9, 2017, we launched the Natural History Registry for Necrotizing Enterocolitis (NEC Registry) in collaboration with NORD and supported in part
by a cooperative agreement with the US Food and Drug Administration. In the first 48 hours following our press release announcement, 1,404 people were reached on Facebook, 1,067
impressions and were generated on Twitter, and 3 participants registered. Currently, 50 participants are registered from Canada, The United Kingdom, and the United States including:
Arizona, Colorado, Connecticut, Indiana, Iowa, Massachusetts, Ohio and Texas.

This spring, we are hosting our inaugural “Speaking of NEC: Unplugged” event, a one-day regional conference focused on identifying practical solutions for reducing the devastating
effects of NEC on infants and their families, to be held in Hartford, Connecticut. Initial findings from the NEC Registry will be presented as part of the discussion about the
epidemiology of NEC. It will be the first time that a patient registry for NEC is used in a scientific report.

In the three years that we have been a member organization of NORD, we have seen a marked rise in the recognition within maternal infant health community that NEC is a rare disease
and, in turn, that many of the diseases of prematurity are rare diseases.” –Stephanie M. Vaughan, The Morgan Leary Vaughan Fund, Inc.

ADVOCATES

14. NORD facilitates collaboration on advocacy and policy campaigns at both the state and federal levels
15. NORD developed and manages the Rare Action Network, the largest national rare disease grass roots advocacy network of more than 3,600+ advocates who stand ready to mobilize and advocate on matters impacting the rare disease community
16. NORD believes that the corner stone of advocacy is good data, and invests in and provides verified data regarding orphan drug costs to serve as a foundation for discussions in the rare disease sector
17. “I chose to become Iowa Rare Action Network State Ambassador for my son who lost his battle with a rare disease. And to bring people together because alone we are one voice but together we are louder and stronger.” –Sharon Ponce, Iowa Rare Action Network State Ambassador
18. “We lost our daughter Chloe to a rare disease. We consider it a great honor to advocate for policies that will promote research and cures so that other families will be spared hearing ‘I’m sorry your child has a rare disease and there is no cure.’ ”–Erica Barnes, Minnesota Rare Action Network State Ambassador
19. “I know that voices are stronger and more effective when they are unified; especially after spending 2 1/2 months working on legislation for adrenal insufficiency. As a state ambassador I can work to bring even more of us together to join in creating change in Oregon.” — Jennifer Knapp, Oregon Rare Action Network State Ambassador
20. “I chose to become an ambassador to raise awareness and educate our elected officials of the many people in the Commonwealth of Virginia with rare diseases like two of my children and to put faces to these disorders. I want to ensure that this population in our state has appropriate access to medical expertise, treatment and services and economic support for costly expenses associated with rare disease or disorders” –Jana Monaco, Virginia Rare Action Network State Ambassador

LEGISLATORS

21. NORD is the voice of the rare disease community and is seen by Congress as a trusted partner in helping to help craft and shape rare disease health care policy
22. NORD birthed the Orphan Drug Act in 1983 and has advocated for and protected the Act successfully over the last 35 years
REGULATORS
23. NORD works closely with regulatory agencies as the independent voice of the rare disease patient community to ensure the viability of research, treatment and access.
24. NORD and FDA have worked together to develop and provide natural history studies to patient organizations in an attempt to capture longitudinal data with the goal of helping to de-risk the drug development process.
25. NORD recently signed with the FDA a one-of-a-kind MOU, designed to improve knowledge and understanding of disease burden, symptom management, impact on daily activities, and quality of life for various rare diseases within the agency

MEDICAL PROFESSIONALS

26. Physicians, nurses, pharmacists, and students look to NORD for rare disease education to support patients and families in need
27. NORD’s database of physician guides, rare disease reports and other general rare disease materials provides a compressive one stop resource center for those seeking information on how to better diagnose and treat rare diseases
28. Through medical rotations NORD open its doors to medical students to help provide them with exposure to the challenges and opportunities in rare diseases
29. NORD is establishing a network of certified rare disease centers of excellence across the U.S. with the goal of developing a virtual network of hospitals and clinicians with expertise to treat and manage rare diseases
30. NORD educates medical teams on rare diseases to assist with coverage decisions

RESEARCHERS

31. NORD supports basic and translational research with the goal of improving diagnostics and treatment for the 95% of diseases without FDA-approved therapies
32. NORD has awarded more than $7 million dollars in seed funding to researchers studying rare diseases; at least two FDA-approved products were a result of NORD’s funding
33. NORD’s IAMRARE registry platform is equipped to help researchers supplement their data for clinical trials and track patient outcomes after approval

MANUFACTURERS
34. NORD’s Corporate Council is a network of over 100 companies engaged in research and development of treatments for rare diseases, consultants and service providers
35. The Council provides an open forum where dialogue on legislative, regulatory and other issues concerning the rare disease community are discussed and shared
36. NORD serves as the independent bridge between the patient community and drug developers

One Response to “35 Ways and Growing: NORD’s Service to the Rare Disease Community”

  1. Laura McCloskey says:

    I was diagnosed with my rare disease/condition in 1985 and here it is 35 years ago, like you. Dermatitis Herpetiformis is awful and controlling it completely cannot be done. The moment stress hits, I break out again. Happy Friendversary!