If there is one thing clear at #RDDNIH, or Rare Disease Day at NIH, it’s that there are amazing, brilliant people working hard for our community.
The rare disease community often feels neglected, especially given the realization that big pharma is a business… and they get very little return-on-investment if they invest resources in curing relatively few patients.
But there are people on our side. Not just people, but TEAMS of people.
Among the list of impressive speakers today at NIH are Christopher Austin, M.D., Director of the NCATS, NIH and Chair of IRDiRC (International Rare Diseases Research Consortium), and other NIH Clinical Center Leadership. In listening to them, I have taken away some very important points about IRDiRC and NIH as a whole, that fills me with motivation to keep going and to keep fighting.
- The NIH is celebrating the strides they are making. NIH and it’s sub-centers have been, and continue to be, making headway in the rare disease sector.
- They want us to gain knowledge about what is going on not just at NIH, but around the world, so we can celebrate with them. IRDiRC is one of the centers coordinating research throughout the world. It’s mission, Transformation, not Incrementalism, heavily involves sharing knowledge, data, infrastructure, expertise and viewpoints to incite radical improvements for the rare disease community.
- They want to empower patients and advocates. IRDiRC strives to enable all people living with a rare disease to receive diagnosis, care, therapy within one year of coming to sub-specialty medical attention. They want to arm us with research and knowledge regarding our rare diseases, from natural history to available therapies, and treatments coming down the pike.
Four other less technical and more inspiring take-aways from IRDiRC, NCATS, and NIH’s Clinical Center are:
- Remember the important of persistence and not giving up.
- Have hope.
- Maintain hope through action.
- Remember you are not alone.