Highlights of Rare Disease Week 2016

Highlights from Rare Disease Week 2016

I spent four days in DC for the Rare Disease Week on Capitol Hill. I highly recommend participating in these events, especially for anyone in the area. All events are free to the public and just require registration. There was the opportunity to apply for $600 travel stipends given to people who traveled from distant states. The days are filled with information that is valuable for being proactive with legislation and what you can do to make a difference with gaining the support of your state legislatures. I believe there were around 300 people in attendance.

The first day was held out at NIH, Rare Disease Day at NIH. There were several speakers lined up for an informationally packed day. It was the first time in four years I was at NIH for something other than a medical appointment! We heard from the NIH director, Dr. Francis S. Collins, the Office of Rare Diseases Research, and NORD to name a few. They offered tours of the clinical center at NIH and provided a small poster session. The highlight for me that day was hearing Senator Hatch (R-UT), and Senator Klobuchar (D-MN) speak about the Rare Disease Congressional Caucus. They both co-chair this caucus and emphasize that the Congressional Rare Disease Caucus provides a vital platform for discussing pressing policy issues and giving rare disease patients a voice in Washington, D.C.

That evening we were shuttled to the U.S. Naval Heritage Center for a cocktail reception and a screening of the documentary “Dusty’s Trail”. I was able to meet up with two other cholangiocarcinoma advocates, Doreen Darsh, and Brandon Leonard. (photos attached) Doreen’s son passed from cholangiocarcinoma at the age of 28, and Brandon’s father passed from cholangiocarcinoma after a short two month battle.

Tuesday was a long conference day filled with information on legislation. It prepared us for Wednesday, which was our day to visit with our legislators and ask for their support. We were briefed on what to ask for, how to tell our unique stories in a short time frame, and why it is important to build relationships with our senators and representatives. The conference day was meant to help you network, make new connections, and strengthen the rare disease community.

Wednesday started bright and early with a Lobby Day Breakfast where the newly confirmed FDA Commissioner Dr. Robert Califf spoke. I did not attend this breakfast because I was invited to a private Montana breakfast and was told this was a great opportunity for advocating. The Montana Coffee is held every Wednesday morning, and now that I know about it, I will be attending it when I am in the area. The breakfast provided a jumpstart on connecting with the senators and representative from my state. Quite an amazing opportunity, and really made me wish I had paid more attention to government classes in high school!! (photo attached)

We then had set appointments to meet with our legislatures privately or their aides. First stop was Senator Tester’s office where we met with his aide, Monica. We told our stories and I emphasized the importance of NIH funding and what it has done for me personally. We asked for support of the Orphan Drug Act, The Patient Focused Impact Assessment Act, and asked for him to join the Rare Disease Caucus.

Next stop was Representative Zinke’s office, we met with him personally and repeated our stories and asked for support in the Orphan Product Extensions Now, Accelerating Cures and Treatments, Rare Disease Fund Act, and to join the Rare Disease Caucus.

Final stop of the day turned into a bonus for us. We met with Senator Daines, told our stories and made our asks. He shared his personal story of losing his sister to PSC, she passed while waiting for a liver transplant and left four children behind. He was quite surprised when I told him that PSC is a risk factor to cholangiocarcinoma. He was very personal, kind and easy to talk with. He was on his way to vote in the Senate and asked if we would like an impromptu tour. We were overjoyed with excitement and felt like we had a little VIP treatment. We were able to look in on the senate floor and he showed us a room right next door to the senate called the Mansfield Room, which is named after the longest acting senator from Butte, Montana. Great day!!!

Thursday was a briefing on The Rare Disease Caucus. We again heard from the co-chairs of this caucus: Representative Leonard Lance (R-NJ), Representative Joe Crowley (D-NY), Senator Orrin Hatch (R-UT) and Senator Amy Klobuchar (D-MN). The day ended with a Rare Disease Artist Reception in the foyer of the Rayburn House Office Building.

All in all it was very informative and I learned so much. Please watch for upcoming legislation that will affect us and don’t be afraid to call or email your state legislatures and ask for their support.

“Never doubt that a small group of thoughtful, committed, citizens can change the world, indeed, it is the only thing that ever has.” Margaret Mead

Sincerely,
Melinda Bachini
Patient Advocate
The Cholangiocarcinoma Foundation