Tag Archives: Advocacy

Rare Disease Day is a day of unity for us patients with rare diseases. I can’t speak for you, but I felt very alone and isolated when I found out I had pemphigus vulgaris. Then I found community in the IPPF. It was very comforting to know there were others out there who had gone through what I was living through. Now imagine being with hundreds of others who have a variety of rare diseases.

Although there has been a huge public outcry over the cost of prescription drugs, there has been little public discussion about the need for innovation for patients who don’t yet have an effective therapy. It is estimated that 1 in 10 Americans suffer from a rare disease, and only 5% of rare diseases have a treatment approved by the Food and Drug Administration (FDA).

In November 2014, I used the chemotherapy drug Rituxan off-label for my rare disease, Immune Thrombocytopenia (ITP). The decision was made after careful consideration of all the possible outcomes. I was desperate for relief since ITP causes internal bleeding that can be fatal. This thought stayed in the back of my mind as I watched my platelets drop and my bleeding episodes increase in severity.