One of the big takeaways from our forthcoming State of American Philanthropy report on funding for neurological research is the sprawling footprint of patient advocacy organizations focusing on a broad swath of activities like research, education, prevention, patient care and advocacy.
Consider the Parkinson’s Foundation. For the fiscal year ending June 2021, it had $42 million in revenues and awarded $20 million in grants. Of that amount, it earmarked 42% for medical and clinical research grants, with the balance flowing to community engagement, patient education and professional training. Other prominent patient advocacy organizations in the neurological research field include the Amyotrophic Lateral Sclerosis Association, the Alzheimer’s Association and Autism Speaks.
The Quincy, Massachusetts-based National Organization for Rare Disorders (NORD) is a particularly interesting grantmaker in this space. The organization is committed to the identification, treatment and cure of rare disorders and “orphan diseases” — which describes any condition that affects fewer than 200,000 people nationwide — through education, advocacy, research and patient services. Diseases listed on NORD’s rare disease database include Refsum disease, acanthocheilonemiasis, and Galloway-Mowat syndrome.
Funders understandably tend to direct support toward more prevalent health conditions, if for no other reason than the fact that donors are often moved to give by a personal or family experience. Researchers expect the number of people living with Alzheimer’s disease to triple to 153 million people by 2025, so it should come as no surprise to know that the Alzheimer’s Association has over $400 million in total revenue.
But there’s also a crucial role in science philanthropy for funders backing more niche or otherwise underfunded medical causes. It’s also important to remember that the word “rare” is a relative term, especially when viewed in aggregate.
“There are over 25 million Americans — 1 in 10 people — who are living with a rare disease today,” noted NORD President and CEO Peter L. Saltonstall in the organization’s 2021 Annual Report, “but despite incredible advancements in research and technology over the last 40 years, more than 90% of the estimated 7,000 known rare diseases do not yet have an FDA-approved treatment.”
A focus on patient assistance
The organization’s roots date back to the late 1970s and early 1980s when support groups began lobbying Congress for more federal funding to treat rare diseases. The coalition eventually became NORD, and went on to pass the Orphan Drug Act of 1983, which created financial incentives for the development of treatments for rare diseases.
NORD is made up of 330 patient advocacy organizations that pay annual dues on a sliding scale based on the organization’s annual gross revenue. Thirty percent of member organizations have revenues of less than $75,000.
According to its Form 990 for the 2021 calendar year, NORD had $43 million in total revenues, $1.8 million of which came from membership dues, with the balance courtesy of grants and other contributions. It disbursed $44 million in grants that year, of which 93% flowed to “patient assistance programs,” which it defines as providing “access to the services and support the community needs to obtain the best care and treatment, and to help patients live their best lives.”
In 2019, NORD launched the first-of-its-kind respite caregiver program, which provides financial assistance to caregivers — parents, spouses, family members — of those living with a rare disorder. Caregivers can use the funding to cover the costs of a certified nursing assistant or home health aid. It has provided 682 respite grants to rare disorder caregivers to date. In 2021, NORD created the Hispanohablantes Advisory Committee, which works to engage diverse populations within the greater rare disease community.
Advancing research
NORD’s Jayne Holtzer Research Grants Program provides seed grants to academic scientists for translational or clinical studies related to development of potential new diagnostics or treatments for rare diseases.
Since its launch in 1989, the program has awarded more than 200 grants and over $9 million. Grants have led to the development of two FDA-approved treatments and numerous peer-reviewed publications. One treatment, Northera (droxidopa), was approved by the FDA in 2014 to treat neurogenic orthostatic hypotension. Researchers can check out open grant opportunities here.
In 2014, NORD and community partners launched the IAMRARE registry program, which supports data collection efforts for over 40 rare conditions and has more than 11,000 participants. During the early days of the pandemic, it awarded nearly $200,000 in mini-grants to rare disease nonprofit organizations, with individual awards up to $15,000.
Zooming in on the neuroscience field, NORD, along with the National Institutes of Health, the Brain Research Foundation and National Ataxia Foundation, funded research into what could become the first effective treatment for a debilitating and fatal disease of the central nervous system called SCA1. It partners with Neurology Reviews to publish an annual Rare Neurological Diseases Special Report, which promotes awareness of rare diseases and advances in diagnosis and treatment among healthcare professionals.
NORD also works with medical experts to create reports on rare neurological diseases for its Rare Disease Database and videos for its Rare Disease Video Library. “In each case, funding is provided by donors and the content is developed by NORD in collaboration with medical experts at leading hospitals and medical schools,” said Debbie Drell, NORD’s director of membership, via email.
A wealth of resources for members
Drell’s comment speaks to another big takeaway from our upcoming paper on funding for neurological research — the constant and often grueling work of raising money in a space where many donors want to see immediate breakthroughs. This challenge is acute in relatively well-funded fields like Alzheimer’s and Parkinson’s research, and can be all the more difficult for rare disease organizations, and especially the roughly 100 NORD members with less than $75,000 in annual revenues.
“During this time of uncertainty related to the coronavirus and the evolving public health and economic impacts of COVID-19, it’s critically important for NORD’s member organizations to be prepared to build their case for funding opportunities,” Drell said. “As a result, we regularly convene expert speakers to discuss guidelines for relationship building and engaging industry and foundations to secure funding for programs and research.” For example, NORD reps invited an individual who received a grant from the Chan Zuckerberg Initiative’s Rare As One Program, which funds research partnerships focused on rare diseases, to share insights with members about how to build a winning proposal.
NORD members also have access to a slew of resources, including capacity building tools for organizations interested in coordinating and funding research, governance and policy templates, webinars, and guidance for individuals looking to start a foundation focused on tackling a rare disease. Click here for information on applying for membership.
