This week, I joined 800 people from all 50 states for Rare Disease Week at Capitol Hill (https://everylifefoundation.org/rare.../rare-disease-week/) to meet with our representatives in Congress and advocate for legislation to improve access to treatments for rare diseases.
Before today, I had never met with my Congressional representatives or their staff. Part of my motivation comes from what Scott and I went through when we realized how few effective treatments there were for pheochromocytoma/paraganglioma (pheo/para), his rare neuroendocrine cancer. Facing that reality, and seeing Scott realize it, is something that I can never forget.
This is a common issue for people battling rare diseases. About 10% of the U.S. population has a rare disease, and less than 5% of rare diseases have an FDA-approved treatment. The only FDA-approved treatment for metastatic pheo/para, Azedra, was developed under the Orphan Drug designation but was recently discontinued by its manufacturer Lantheus due to a lack of profitability, a devastating blow to the pheo/para community.
Of course, I'm doing this for Scott and rare disease patients. But I'm also doing this for myself and the caregivers. Despite all that he went through, I don't think Scott would have felt particularly strongly about me doing this. He just wanted me to be happy and to live a full life. But the feeling of helplessness as I watched his strength and health fade away, and to see him in pain, was agonizing. Being a voice for the patients and caregivers who must devote all their energy to fighting disease and the obstacles to health care access is something that makes me feel a bit less helpless.
Today I met with staff members for Senator Ben Cardin, Senator Chris Van Hollen, Representative Andy Harris, and Representative John Sarbanes, all from Maryland. I was part of a team of rare disease advocates who were constituents, and we introduced ourselves, shared our stories, and made our legislative asks. At least half of Americans with rare diseases are children, and it was inspiring and moving to hear the stories from parents, and in some cases, the children, fighting for their lives.
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Meeting in Rep. Andy Harris' office
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View of the U.S. Capitol from Rep. Andy Harris' office
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Meeting with Rep. John Sarbanes' office
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| Senator Chris Van Hollen,
a strong supporter of rare disease legislation, stopped by and shared
some remarks. He observed that while it is important to find cures for
common diseases, it is also important to fight rare diseases because of
the significant impact they have on patients and their families. For
this reason, he is a member of the Rare Disease Congressional Caucus and
a co-sponsor of the Safe Step Act (https://www.congress.gov/bill/118th-congress/senate-bill/652). |
While the current outlook on whether Congress can get anything done feels daunting, there is hope in that the bills that we advocated for have bipartisan support and co-sponsors in Congress. One of the speakers at the legislative conference on Monday noted that these efforts take patience and persistence. Forty years after the Orphan Drug Act was signed into law by President Reagan, this is still true. I'm grateful that I could advocate on behalf of patients and caregivers, make some connections within the rare disease community, and spend time with the amazing people representing the Pheo Para Alliance.
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| Maryland delegation in front of the plaster model of the Statue of Freedom, which stands on the dome of the U.S. Capitol |
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Patience and persistence
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