Introduction to Rare Disease Grants
Americans are known to be the world’s most generous people, with 1.44% of the GDP donated to various charities.(1) There are 1.5 million charitable organizations in the US. Of these, some 81,000 are in the category of “health service-related”. Many of these charities support common diseases such as heart disease, cancer, diabetes and Alzheimer’s just to name a few.
Donations to these organizations fund the care of those affected by the target disease/disorder. In addition, they fund hope for those yet to be affected through research leading to early detection, prevention and the ultimate goal of a cure.
With large segments of the population being either directly or indirectly affected by common diseases, funding of research grants is highly attainable. Family members, friends, co-workers as well as corporate donors create an enormous pool of potential donors. Aggressive marketing further augments the ability of organizations supporting common diseases to fund grants leading to the eradication of the disease.
What are the Rare Diseases / Disorders?
There are 7000 known disorders classified as “rare” affecting 25 – 30 million Americans (National Organization of Rare Disorders). To be classified as a rare disease/disorder, it must affect less than 200,000 persons.
The most common rare disorders are:
- Ehlers Danlos syndrome
- Sickle Cell
- Cystic Fibrosis
- Duchenne Muscular Dystrophy
- Progressive Multifocal Leukoencephalopathy
- Paraneoplastic Neurological Syndrome
- Dercums Disease
Despite the relatively small numbers of people affected by rare disorders, many are life-threatening and debilitating and dramatically affect the quality of life of their victims. The small number of affected persons creates confounding challenges to victims and their caregivers, including:
- Raising awareness of the general population.
- Funding research to develop care and treatment options.
- Obtaining resources, including pharmaceuticals, services, and devices.
- Incentivizing and funding research leading to a cure.
What Resources are Available to Treat and Battle Rare Diseases
This article will discuss three valuable resources available to victims and those interested in engaging in or supporting research targeting these disorders:
- National Organization for Rare Disorders (NORD)
- National Institutes for Health (NIH)
- Food and Drug Administration (FDA)
In addition, though their work is not focused on rare diseases/disorders, some corporate entities and associations offer some tangential support. An example would be the Muscular Dystrophy Association, which serves as a resource for persons affected by a wide variety of neuromuscular disorders, including Duchenne Muscular Dystrophy. March of Dimes is yet another organization that offers assistance and engages in research related to the prevention and detection of rare genetic disorders and birth defects.
1. National Organization for Rare Disorders (NORD)
Established in 1983 as a 501(C)(3) NORD serves as a “hub” resource for driving advances in care, research, policy and creation of community for researchers and victims of rare disorders. In addition, NORD serves to enhance public awareness of these disorders through campaigns, including National Rare Disease Day, held on the last day of February each year.
NORD supports research through seed grants to scientists whose studies advance the development of potential new diagnostics and treatment of rare diseases. In 2022, NORD awarded 200 grants totaling $9 million in funding.
The organization maintains several databases related to the incidence and treatment of rare diseases. Through these databases, NORD serves researchers, patients, and caregivers as a trusted resource. Patients can look to NORD for disease-specific information, including centers of excellence when exploring treatment options.
2. Food and Drug Administration (FDA)
Government-funded research is pivotal to advances in the management and eradication of rare diseases. The FDA is one of the agencies that offers researchers in the field funding sources through four programs. These programs yielded 19 new grants and two contracts totaling more than $38 million in funding in 2022.
The Clinical Trials Grants Program, though not specific to rare disease research, serves as a potential funding source for scientists. The program seeks proposals for studies related to unmet needs for persons with rare diseases/disorders.
The Office of Orphan Products Development (OOPD) is a valuable funding source for scientists and pharmaceutical companies that support rare diseases. Orphan Products are drugs or products targeting conditions affecting < 200,000 persons. Orphan designation results from these products having low profitability given the few potential consumers. This program serves as a source of funding to offset the low profitability of current products or those products which will require at least 7 years to become profitable following approval. Hence, this program gets products benefiting rare disease patients into the market more expeditiously.
The Natural History Studies Grant Program focuses on the unmet needs of those suffering from rare disorders. The program funds innovative natural history research that advances medical product development.
Another FDA program, the Rare Neurodegenerative Disease Grant Program, targets ALS (Amyotrophic Lateral Sclerosis) and similar diseases. The funds support research and development of interventions intended to prevent, diagnose, mitigate or treat qualifying rare diseases.
Finally, the Pediatric Device Consortia Grant Program, seeks to create a non-profit cooperative that facilitates the development of medical devices for pediatric patients with rare disorders.
3. National Institutes of Health (NIH)
In 2022, the NIH awarded $6.5 billion to research related to rare diseases and disorders. Projected awards for 2024 total $7.07 billion. The majority of this funding arises from the Rare Disease Clinical Research Network of the NIH. This program serves to foster collaboration between researchers to enhance understanding of how rare diseases progress. In addition, the program funds research designed to improve approaches to diagnosis and treatment of rare disorders.
Though collectively, rare diseases and disorders affect a large segment of the population, individually, they affect few. Notwithstanding, victims of these diseases experience debilitation, pain, suffering, and death. They require treatment, pharmaceuticals, devices and care assistance.
The rarity of these disorders frequently leads to misdiagnosis and treatment. In addition, once diagnosed, navigating the healthcare system to obtain treatment is, at best, a challenge.
Research and development of diagnostics, pharmaceuticals, devices and treatment protocols for disorders that affect so few is also challenged by lack of awareness and questionable profitability.
This article has identified several entities whose purpose and mission are focused on bringing hope to those who suffer from these disorders and those who care for them.