When I walked in to Rare Disease Day 2016 at NIH, the atmosphere was full of hope, on a scientific level, legislative level and patient level. And I’m not just saying that to sound positive!
As someone that suffers from chronic Lyme, I approached this day with a healthy amount of caution and skepticism. But I had an open mind given the newest legislation that has passed in my state surrounding my disease.
Challenges were not without mention, such as global competition (i.e. foreign governments dedicating more money for research), flat funding for young and new researchers making it harder for them to work, and immigration reform (i.e. Mayo Clinic or St. Jude can’t get the best of the best doctors or researchers due to immigration processes).
However, it was made clear that despite the ugly political environment, there are still people willing to go to work everyday to do the right thing.
For example, one of my favorite pieces of potential legislation was the OPEN Act which would “provide an additional six months of exclusivity to the patent life of the major market drug being re-purposed for rare disease treatment so long as the sponsor company establishes that the therapy: (1) is designated to treat a rare disease, (2) obtains a rare disease indication from FDA on the drug label.”
Hearing about this proposed legislation made me hopeful that new treatment options would soon be made available for rare disease patients who otherwise couldn’t access therapies for their condition.
