Who is most affected when we talk about creating solutions for rare diseases? Those who live with them and their families. Patient communities know what they need and feel the impact of policies. They won’t forget about it when life gets busy.
So, who’s going to advocate for your cause if not you? On February 25th to 28th, EveryLife Foundation for Rare Diseases (ELF) will bring hundreds from the patient communities from all 50 states to become their own advocates in the annual ‘Rare Disease Week.’ Patients don’t need to be familiar with policy and congress to be an advocate, ELF will get them up to speed. By the way, you’re invited.
Who Are The Advocates?
The event brings in patients like Aidan, a 14-year-old with ectodermal dysplasia. He was nervous, a novice in advocacy; now testifying before his congressional staffer. His lack of advocacy experience didn’t take away from his powerful story. After thanking the audience, Senator Baldwin kept him and his family; they had listened and were going to help. So began their years-long quest to enact the Ensuring Lasting Smiles Act. The senators from both parties gathered a hundred co-sponsors to provide health plans to cover the comprehensive medically necessary services for patients born with defects in order to bring them to medical stability without paying enormous out of pocket fees.
Why Rare Disease Week?
ELF recognizes the patient’s story is so powerful because it shows the human behind the policy. It’s not just the numbers and returns of the Ensuring Lasting Smiles Act that convince senators to act, it’s the boy telling them how much the act could impact his life. The non-profit organization doesn’t tell patients what to say or what to advocate for, instead they teach patients how to use advocacy to express what they need. Rare Disease Week makes the process easy: ELF will demystify the bureaucracy by providing rare disease advocacy bootcamp, book congressional appointments, plan community art events. In sum, they provide the platform for patients to express themselves in a way congress can understand and respond to. Patients just need to show up and share their story.
I spoke with Julia Jenkins, executive director of EveryLife Foundation, who came into the role through her focus on grassroots advocacy. She is a strong believer in good policy and public involvement. She explained what patients can expect from this event, why advocacy for policy is so important, and the current policy challenges for the rare disease world.
What Goes on at Rare Disease Week?
The week in Washington is built to bring patients into the world of advocacy and to build a community to encourage collaboration to benefit the entire rare disease community.
It begins with a full training day teaching a bit of public policy 101. In this ‘Legislative Conference’, patients learn to frame their policy objective most effectively. as Jenkins explained, “Members of congress must understand both the problem and the solution on one page, and that the ask is fixable by congress. [For example], not all problems are on the federal level, some are on the state level. We don’t go in and ask for a billion dollars, we are really looking for achievable policy solutions.” Then patients connect the policy to their own story to express how congresses decisions change their lives.
The event has a series of events to form multi-dimensional relationships between the congressional and rare disease communities. There’s a RD Congressional Caucus briefing in which experts brief congressional staff on the RD community. Then the actual lobbying, in which ELF sets up meetings between patients and their Members of Congress. There is a Rare Artist reception that displays art that expresses the rare disease experience, and events that bring patients together: a shared breakfast, documentary screening, a cocktail night. All of these create community to share that these patient experiences are not so obscure, they are imbedded in the lives of many around us.
The Importance of Advocacy
Jenkins and her team’s work is so valuable because while Washington is full of requests, they are low on time and resources to meet all of them. Without the political will to remind congress that these policies need protecting, they fall onto the backburner, needs are left unmet.
Accessing New Technologies
Jenkins explained public policy is the bridge between what scientists are capable of creating, and whether that actually gets done. She said, “What is really incredible about the time that we are in right now is that we actually have the technology and capability to likely treat and cure the majority of the cases in the world, but we lack the political will to really make that happen. Without good policies that foster investment, these treatments are not going to become reality.”
The rare disease community is buzzing with talk of gene therapies, CRISPR, faster technology and more data. It feels like the future of technology is here, and finally the solutions are within grasp. But as Jenkins explained, “I think these things are so exciting that people think that they will happen in their lifetime very quickly without them having to do anything, but that’s the misconception. Because we are going to have to fight to protect these new technologies.”
Preventing Current Policies from Expiring
Jenkins discussed how many policies that fund rare disease needs are expiring. This means the need now isn’t just to implement new policies, it’s to protect existing ones. When there are -reforms, rare diseases are often the first to be affected. Jenkins described them as the canary in the coal mine, meaning rare disease funding is the first to get cut when congress is shrinking the budget. In 2017, tax reforms halved the Orphan Drug Tax Credit, a critical incentive for industry to develop drugs. Drug prices are a crucial challenge for rare diseases; keeping them profitable for companies to invest and also then affordable to patients is of utmost importance. Investors became scared to contribute. This is the type of policy they are fighting to simply keep, let alone advance.
Getting Through to Washington
Achieving policy approval is difficult right now in part because Washington is currently swamped. Jenkins explained, the current issue is that “The elections and impeachment are sucking the air out of the room. There’s not a lot of space in congress to get good policies done.” She conveyed how Rare Diseases are not partisans. Rather it’s exciting that rare diseases are one of the lucky rare issues that goes beyond party lines. However, while the federal government is quite occupied, she mentioned it’s a good time to look to state governments too, which enact their own set of policies.
Advocacy Off Capitol Hill
ELF has their own goals they are advocating for: improving access to medicines that have been approved by the FDA, protecting incentives, closing the innovation gap for rare diseases, and improving the FDA regulatory process. They are pushing for policy on newborn screening, and making accessing treatments more accessible through the many interested stakeholders that become hurdlers to patients actually receiving treatment. They focus on advocating for these issues all year long. Rare Disease Week though isn’t exclusively focused on these topics, but also a chance to hear the many varied needs that the patients with different rare disease uniquely need.
Of course, rare disease advocacy isn’t contained to this conference. If you can’t make this event, there are always opportunities to get involved. She discussed how engaged patients are throughout the year, often calling and emailing members of congress. Many make sure to check in through the year to remind congress people of the concerns they’ve listened to. ELF visits other cities throughout the year to build grassroots organizations there too and keep the patients involved.
Still, this week will be a powerful motivator and create real progress, and Jenkins wanted to remind patients if they read this, they should consider coming themselves. When it comes to rare disease advocacy, there’s so much work to do that there’s enough to go around.
What are your thoughts on the best advocacy methods? Share your stories, thoughts, and hopes with the Patient Worthy community!
