For the past four years, I have joined IPPF patients and others in the rare disease community across the country in advocating to congressional members in my district. The IPPF is joining forces again this year with Rare Disease Legislative Advocates (RDLA) for In-District Lobby Days from August 7, 2017 through September 6, 2017.
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 addition to our general request for NIH funding, I stressed the importance of cutting edge research on rare diseases and emphasized the significance of the Open Act, which incentivizes pharmaceutical companies to make treatments available off-label.
During the summer recess, Congress returns to their home states to hear from constituents and attend community events. Meeting with them during this time is a critical opportunity for rare disease advocates to build relationships, educate them on key legislative needs, and follow-up on requests made during Rare Disease Week on Capitol Hill or phone calls or emails about legislative issues made since then.
When there was an opportunity to be an advocate, Marc suggested that I attend Rare Disease Week in Washington, DC. My first reaction was FEAR. The walking, my body aching, standing were just a few of my worries. EXCITEMENT was the next emotion. There would be stories that I could relate to and new people to meet, especially Marc Yale. Then I felt PROUD that I would represent Louisiana and get to meet the senators and representatives.
As we shared our stories on behalf of the entire P/P community, I could see that many of the legislators’ offices we visited began to understand the dilemma. However, it was clear this issue would take an effort by the entire P/P community for our voices to be heard.
The purpose of advocating in Washington, DC is to spread awareness and lobby for favorable legislation that affects the entire IPPF community. At Rare Disease Week on Capitol Hill 2016, we had 7 advocates from the IPPF community.