Ron Bartek and Brigid Brennan joined rare disease advocates from 48 states for the annual Rare Disease Week in Washington DC. The theme this year was research - a mission FARA has been supporting for almost 20 years. Here are some highlights:
Monday - Rare Disease Day at the National Institute of Health (NIH)
It was standing room only as doctors and advocates shared updates from their respective fields. Over 700 people were present and, for the first time, many more livestreamed from home. Everyone was in agreement that research is needed in the rare disease community with less than 500 treatments for over 7000 diseases. However, we are not alone in benefitting from goal. Rare disease are often the "window" into common disease treatments as well. Rep Leonard Lance (R-NJ) spoke about his continuing support for the rare disease community. The crowded erupted in applause to learn that Dr Francis Collins, a champion for our community, was asked to stay on as head of the NIH.
That evening Rare Disease Legislative Advocates (RDLA) hosted a cocktail reception and documentary screening of Up for Air. Senator Ed Markey (D-MA) and Representatives Jim McGovern (D-MA) made brief remarks of support. Our own Ron Bartek moderated the panel discussion of the movie where everyone left in awe by Jerry Cahill's inspirational journey with Cystic Fibrosis.
Approximately 350 advocates gathered to learn a wealth of information on the new Congress, The Trump administration, top health policy issues and working with federal agencies. Most importantly, the experts shared tips on how to effectively meet with your legislator and continue building that relationship after you have left. With so many key pieces of health care legislation currently in debate, there has never been a better time for all of us to engage with our representatives. This conference was also livestreamed for the first time but if you missed it, video and presentations will be available on the Legislative Conference resource page. www.rareadvocates.org/207-legislative-conference-resources/
Wednesday - Lobby Day
The day started with the opportunity to gain some legislative insight from Representative Gus Bilirakis (R-FL) and former Representative Brian Baird (D-WA) over breakfast. Then Dr Janet Woodcock, Director of the Food and Drug Administration's Center for Drug Evaluation and Research, addressed the crowd. A resounding note of appreciation was heard in the room for Dr Woodcock's continued support in incorporating the patient perspective in research.
Then we were off to the hill! 328 advocates participated in 270 legislative meetings. Everyone was grouped by state and meetings ranged from 1 to over 20 people. The asks this year included:
· The Open Act - which would support repurposing already approved medication
· Affordable Care Act (ACA) - provides important coverage for the rare disease community including no pre-existing bans, no lifetime limits, coverage until 26
· Funding for NIH/FDA - without appropriate funding, neither agency can hire the needed doctors and scientists to maximize research potential
· Rare Disease Congressional Caucus
Most legislators were supportive of our general concerns and requests. However, with so much uncertainty on the hill around health care and funding, it is important for us to stay firm and persistent on the issues that matter the most to us.
A long but successful day was wrapped up with RDLA hosting a Rare Artist Reception in which advocates had another opportunity to talk to attending legislators and staff members while everyone enjoyed seeing the winning art entries from the rare disease community.
Thursday - Rare Disease Congressional Caucus briefing
Caucus co-chair Senator Amy Klobuchar (D-MN) welcomed the advocates and Congressional staff to the final event of the week. A review of the 21st Century Cures Act, the PDUFA Reauthorization Process as well as the ACA were reviewed. Video of the meeting will be posted on the Caucus webpage within a few weeks. www.rareadvocates.org/rare caucus/
The week provided a great resource for information but also a great opportunity to share strategies and form coalitions. Together our voices were a roar in DC.
Written by Brigid T Brennan, Esq