Post Rare Disease Day 2016 the FDA’s Office of Special Medical Programs announced a new program that would work to provide funding to patient advocacy groups and other organizations who were studying natural history studies.
In February the FDA released funding to study over time how specific rare diseases progress in individuals. Over $2 million in research grants was made available to organizations conducting research about the “natural history” of diseases.
It is no secret that what we really know about rare diseases isn’t much.
In regards to most rare diseases, what we actually understand about the course of the disease with or without treatment is very little. Studying this is referred to as natural history. Any kind of research is really expensive, but it is so necessary. For rare diseases, the more we know, the closer we can get to treatments and the possibility of cures. This kind of research can influence and speed up the drug development process; and the FDA has been working to create more and more programs and grants for acceleration throughout each phase of the process.
You can read the entire Press Release here. The director of the FDA’s Office of Orphan Products Development, specifically of the Office of Special Medical Programs was quoted in an FDA press release saying the following,
“Not understanding how a rare disease progresses is often a major obstacle in the development of life-saving medical products. Information about a disease’s natural history can aid in clinical trial design, identify study end points and lead to faster, better trials – hopefully leading to new and effective diagnostics and treatments.”
– Gayatri Rao, M.D., J.D.
This was a HUGE milestone for rare disease advocates as this was the first time the FDA provided such funding (all thanks to the FDA’s Orphan Products Grants Program- read more here).
If you have ever felt like you know more about your rare disease than your doctor or any health care professional you should Read Why Patient Perspective is Worthy of Industry Ears here and Contributor Lisa’s The Good, the Bad, and the Ridiculous Part‘s 1 and 2. Your voice matters and the FDA’s grant of $2 million for rare disease natural history research is a perfect example of why you should get involved and keep talking!
For more information on the Orphan Products Grants Program (OOPD) click here to go to FDA.gov or the resources below for general information:
General Information:
