In the beginning of March, Patient Worthy joined many other groups from the rare disease community for Rare Disease Day at the NIH Clinical Center, an event which you can read more about here.
We were excited to see researchers, lawmakers, advocates, and patients working together to find solutions for the patient community. The NIH Clinical Center offered a great amount of insight into how they approach rare disease research and treatment. We were honored to see the behind-the-scenes facility at the forefront of rare disease research, and meet representatives from the organization that has changed many patient lives.
Following the event, we reached out to the NIH Clinical Center with a list of questions to help the rare disease community better navigate the options available. We wanted to know how the NIH prioritizes research funding, how advocacy groups can fund researchers at the NIH, how they support innovative tactics, and where we could read about initiatives where the NIH works with academia to advance the field of rare disease.
The NIH Clinical Center shared resources and shed light on the process. We break down the first part of their answers below.
First, we asked about how the NIH Clinical Center prioritizes funding. Funding, allocation, and rare disease prioritization is, of course, a very complex process, and we hope to promote better understanding around it. One thing we wanted to know was whether they take into account the number of people suffering from a disease, or rely on different factors.
The NIH explained the way it currently approaches rare diseases, which in the U.S., are defined as conditions affecting under 200,000 people. There are around 7,000 recognized disorders that meet this definition. The NIH told us that “many of NIH’s 27 Institutes and Centers (IC) fund research on or related to these conditions. Each NIH IC has its own specific research agenda or support function, often focusing on particular diseases or body systems. Each of the ICs have their own budget, and based on their funding priorities, fund grants for specific research, including research on rare diseases that fall under their respective purviews.”
While the NIH supplies different types of grants funding research, most NIH funding backs research project grants, especially investigator-initiated Research Project Grants (R01). This funding is given to organizations so that an individual principal investigator, also known as a PI, can research an objective in their area of expertise.
The NIH shared that, “For all areas of research, NIH funding decisions are based on the scientific merit of the proposed project, portfolio balance, budgetary considerations, and public health need. NIH ICs are interested in funding meritorious research applications, as determined by a rigorous peer review process. Increase in the number of meritorious applications submitted to the NIH for peer review will result in increased funding for rare disease research.”
To break that down, the NIH decides which grants to fund– rare disease related or not– based on a variety of scientific criteria, including need, expense, and quality of proposal. The more experts who submit rigorous proposals to the NIH focused on rare diseases, the better chance there is that rare research will receive funding.
The rare community also has a very influential friend at the NIH– NIH Director Dr. Francis Collins. Collins has focused on rare disease research in his own work, and has a background studying progeria. In his NIH Director’s Blog, he spotlights some of the great rare research that the NIH has funded. You can view some of his posts on the subject below:
- Creative Minds: Looking for Common Threads in Rare Diseases
- Clinical Trials Bring Hope to Kids with Spinal Muscular Atrophy
- Gene Editing: Gold Nanoparticle Delivery Shows Promise
- NIH Family Members Giving Back: Charlotte Phillips
- Another Milestone in the Cystic Fibrosis Journey
- Huntington’s Disease: Gene Editing Shows Promise in Mouse Studies
- Rare Disease Sleuths Uncover New Clues to Stroke
