Rare Patients Learn to Make Their Voices Heard

Rare Disease Week on Capitol Hill brings together rare disease community members from across the country to learn about federal legislative issues, meet other advocates, and share their stories with legislators. Because of ongoing concerns about the pandemic, this year’s event will be conducted virtually. We spoke to Britta Dornan, senior director of communications and marketing for the EveryLife Foundation and Sarah Tompkins, advocacy chair of Rare Disease Week on Capitol Hill 2022, about this year’s event, why rare disease patients should consider getting involved in legislative advocacy, and how rare disease patients and caregivers can best tell their stories to lawmakers.

Daniel Levine: Britta, Sarah, thanks for joining us.

Britta Dornan: Thanks for having us, Danny. Yes. Thank you. Excited to be here.

Daniel Levine: We’re going to talk about legislative advocacy and how rare disease patients and caregivers can add their voices to bring about change. Britta, let’s start with Rare Disease Week on Capitol Hill. How did it come about and how long has this been going on?

Britta Dornan: Great question. Rare Disease Week on Capitol Hill was started just over a decade ago by the EveryLife Foundation for Rare Diseases under its RDLA program, which stands for Rare Disease Legislative Advocates. So, for your listeners who might be getting ready for this weekend’s game, let me put it this way. Out of all of our events year-round, and we have many, Rare Disease Week is our Super Bowl. It’s the biggie, it’s the event that brings the entire community together. The event is

Daniel Levine: Lots of chips and beer?

Britta Dornan: You know, it is virtual. So, whatever you’re doing on the other end is fine by us. I don’t think that made it into the care packages going to people, though maybe next year. It’s all about the community raising their voices up together so that our policy makers in Washington DC understand that rare disease is not somebody else’s problem. It’s a public health issue that we should all care about and prioritize. And so, as a patient myself living with a rare disease called primary lymphedema, I love Rare Disease Week because everyone touched by Rare Disease Week, no matter their experience level—they’re welcome because we know that every voice matters and Rare Disease Week on Capitol hill is a huge way to make that voice heard and make an impact for the entire community.

Daniel Levine: My guess is that this began as Rare Disease Day on Capitol Hill, but give me some sense of how this has grown over the years.

Britta Dornan: You would be right. In fact, it started 11 years ago when just about 70 people came from all around the country and they met up in a tiny restaurant before heading over to Capitol Hill for what was a one day of advocacy, as you said. And at that time they advocated in support of provisions that were later included in the Food and Drug Administration Safety and Innovation Act of 2012. That’s known as FDASIA. And that bill was signed into law later that year. But yeah, over the years, the day involved into a series of events called Rare Disease Week on Capitol Hill. And it’s really grown so much that before COVID we were hosting the event for nearly a thousand people at the Ronald Reagan building in DC, which is a massive event space. But this year it’s going to be held virtually from February 22nd to March 2nd. And we’re still expecting hundreds of advocates representing hundreds of diseases and patient organizations to join us.

Daniel Levine: Well, before we talk about this year’s event in general, what takes place during Rare Disease Week on Capitol Hill?

Britta Dornan: Okay. Let me give you a quick rundown of the week. The meat of the week is the meetings that we set up at between advocates and their members of Congress. These meetings happen at the end of the week and they’re held via zoom. Everything else leading up to that are the potatoes and the gravy. And these meetings are the opportunity for advocates to make their voices heard to policy makers. But we kick off the week with a rare disease congressional caucus briefing, and that’s really focused to educating the members of Congress and their staff on issues impacting the rare disease community. This year we’re focusing on accelerated approval during that briefing. And then that evening is when we have our screening of a rare disease documentary, so they can grab their popcorn, maybe put down the chips and beer for that one and enjoy the film. The week continues with two half days of training. That’s when we prepare advocates online via zoom by teaching them about the legislative process, what legislation is current under consideration, and various tools and techniques that they can use to build the relationships with the members of Congress. And sprinkled in throughout the week, there’s a lot of fun stuff. We’ve got an online rare art gallery and it’s kind of like a video game. You go in and you click on artwork and on videos where you meet the artists who made these wonderful, pieces of art. It’s really cool. And then there’s things for young adults who are really craving some social contact. There’s a meetup for them and there’s different networking receptions throughout the week. So, that’s what makes up the week—we got a lot packed in.

Daniel Levine: Due to ongoing concerns about COVID this year, the week will be conducted virtually. Does that allow more people to participate? Does it create challenges in having the type of impact you seek to have that can happen when you meet with a congressional representative in person?

Britta Dornan: I’ll take the first part of your question first. In general, we find that our events are engaging more people virtually than when they were held in person. And we think this is because of two reasons. One, that virtual events are more accessible. Patients and caregivers can join us for events that they otherwise would not have been able to because of travel costs, time off work to travel, or simply managing the complications of travel with their rare disease. These are no longer barriers. Secondly, we’re finding that virtual events have been attractive to people who are new to the advocacy space. The in-person events can seem a little daunting to first time attendees. And it’s a little easier to dip your toe into the world of advocacy through zoom and through your computer at home. So, in this virtual atmosphere, it feels more accessible just to be able to pop into presentations or review recordings on your own and still be able to network if you would like to. These same advocates who have joined us and never had before during virtual events, they say that they’re planning to come back again regardless of whether it’s held in person or virtual, because they’ve been able to dip their toe in. They’ve been able to see what a life changing experience it can be. But, to your second question about having the same impact going virtual, well there’s nothing quite like meeting someone in person and forming that bond, but virtual meetings do allow members of Congress and their staff to see into the window of the advocate’s real life to peek inside. They can see where they are and it allows them to look outside and experience what’s going on outside of what can sometimes be the bubble of Capitol Hill. And that creates an intimacy if you don’t blur your background on Zoom that is. I had a virtual meeting with my member of Congress last year and they were taking the meeting from their home. I could see their kitchen, and that’s a unique experience and creates a kind of intimacy. So again, I’ll say that virtual meetings also allow for children to join their parents in these meetings when they might not have been able to travel to DC with their parents. But I would be curious about Sarah’s experience with that as well, about whether she’s been able to have the same kind of impact cause she’s been doing this for quite some time.

Daniel Levine: Well, Sarah, let’s talk about your experience. You’ve been an advocate for Ehlers Danlos and other conditions. You’ve been outspoken on issues of chronic pain. You began your advocacy work in 2015. What happened? What made you want to share your story publicly? And why have you worked at both the state and national level to bring about legislative change?

Sarah Tompkins: Thank you for that question, Danny. Well, really my patient advocacy journey began explaining my symptoms to get that diagnosis, which took nine years from the onset of symptoms. But really, my advocacy torch was picked up after the unexpected passing of my best friend Kelly Seltzer Doyle, who shared my diagnosis of Ehlers Danlos syndrome. She had been on her way to the NIH to participate in a study when she became ill in her family’s home state and upon her passing that study ended. And it really ignited in me the passion to pick up the torch for patient advocacy that she imparted with me in 2015. I was proud to advocate for the Washington State proclamation of Rare Disease Day, February 28th in Olympia with other rare disease advocates and my journey sharing my story and patient advocacy only continued. I believe that it’s so important to advocate on state and federal levels because with healthcare, each state has such a different structure for how they manage healthcare and legislation and policy. One of the blessings of our disease week is that it gives us that opportunity to connect with other advocates in your state and share information, issues, upcoming events, and really build that advocacy’s team as a state so that you’ve got your state advocacy and federal advocacy teams really put together. And I’ve been so fortunate to have found the EveryLife Foundation and the RDLA to build that foundation for my advocacy to inform and empower it to be at its best.

Daniel Levine: What’s been your experience meeting with legislators and their staffs?

Sarah Tompkins: I have been so blessed to have great experiences meeting with my legislators and their staffs. My best friend Taylor Hiddle actually introduced me to the RDLA and Rare Disease Week as a healthcare staff for representative Markwayne Mullin of Oklahoma, and she was the one who shared with me how I could share my story to Washington legislators, and hearing how dedicated she and their office and Representative Mullin were, working for their constituents and supporting rare disease legislation. And policy really taught me how invaluable it was to share your story to staff and legislators, and began my first Rare Disease Week in 2017. Everyone that participates is advocating and sharing their story. They’re doing something so meaningful for not only themselves, but for their patient communities and for all patients and rare disease communities.

Daniel Levine: One of the things that I imagine can surprise advocates, and I’ll say this politely, is that when they meet with a legislator the level of understanding they have can be quite varied and their knowledge of issues can be quite varied. How do you gauge that and how do you level-set what you’re going to say?

Sarah Tompkins: You are so right. It is absolutely important to do your homework and prep for Rare Disease Week and RDLA has some amazing resources online through rare advocates.org/rdw to really look at what your Congressperson’s history is in supporting rare disease legislation and policy, knowing what committees they serve on, and keeping an eye up for anything you might share in common like schools or communities. For example, I had a wonderful first experience meeting with Representative Adam Smith, who adamantly stated his lack of awareness and knowledge for healthcare committees and rare disease needs. But he was so glad for my time and to learn more about my life with Ehlers Danlos syndrome and other conditions, and in explaining my genetic connective tissue disease to him and how I needed frequent joint surgeries to repair my stretchy tissue. We discovered that we had actually shared a hip surgeon of all things. Since then, he and his staff have been tremendous supporters of all of the legislation, and the policies that I’ve brought to them, and they joined the rare disease caucus right after our meeting, which really meant the world to me.

Daniel Levine: The other thing that may surprise advocates is how little time they actually get to communicate what they want to say. How much time do you typically get and how focused is the message you need to be able to deliver?

Sarah Tompkins: Yes. We ask advocates at the beginning of the meeting just to ask how much time they have so you can gauge, because it’s a good idea to have a three minute, two minute and a 30 second elevator pitch. So, you have three versions of sharing your story and your ask that you can go to that can be very brief and to the point, or can be more detailed if there are less advocates with you in your meeting and you have time for. If the legislator only has 10 minutes, we really want to try to prioritize our newer advocates who haven’t spoken before and knowing that sometimes not all our advocates are going to be able to speak. This is actually an opportunity. You are always able to contact them and follow up through email and we suggest you do so to send a thank you. But this is a really great way to say, “Hey, thank you so much for our conversation for rare disease advocacy. Here was the legislation I was referring to,” and maybe you want to invite them to a support group or a local event for your patient community, or maybe there’s a rare disease event place nearby that you can invite them to, and really start to establish that relationship with them because that’s what you want to have already so that when you have some plan of action, you already have the relationship and connection to go.

Daniel Levine: Advocates who have not done this before, I imagine, can feel quite intimidated about speaking to an elected representative. What kind of training does the Rare Disease Legislative Advocates offer and how much preparation does someone need to go through?

Sarah Tompkins: Well, luckily the Rare Disease Legislative Advocates offer an incredible amount of training, and participating in their virtual events and really going through their resources online are a thorough preparation for your meetings. What’s so incredible is that not only do we offer the two legislative conference days, which as Britta said are the educational foundation of the legislation and policies that we are focused on for this year’s Rare Disease Week, but we also have a virtual share your story event for you to practice sharing your story, as well as offering virtual office hours open for all to attend. And again, with that wealth of resources online at raredadvocates.org/rdw to help with that homework with preparing for meetings.

Daniel Levine: And why is it important for patients and their families to advocate? What’s the case you’d make for doing this?

Sarah Tompkins: You know, rare disease affects more than just the patient. We know that one in 10 Americans has a rare disease, but that’s not considering the parents, families, friends, and communities of these patients who are also impacted by the wellbeing of the rare disease patients. When my husband was able to attend Rare Disease Week with me in 2020, we realized that he had a whole perspective as a caregiver husband advocate in sharing the sacrifice he makes to ensure that I have healthcare insurance, transportation, caregiving for me, and just everything. And it’s important that we advocate for the rare disease community, including caregivers, including friends and family and patients, because it’s more than just patients. It’s the entire community that’s impacted.

Daniel Levine: As someone who’s done this, what would you tell others about your experience? What what would you encourage them to get out of this?

Sarah Tompkins: Personally, being able to just share my story and support legislation that I know will better my rare disease communities has brought me so much confidence, self-esteem, and purpose. I’m a disabled full-time patient, and being able to create awareness and advocacy for my patient community and their needs is not just an honor and a privilege, but it really helps my own self-care and helps me practice being my own best advocate and being my own best advocate for others as well. I’m certainly a better and more effective advocate because of the foundation of advocacy. I’ve grown infinitely advocating with RDLA and EveryLife Foundation during Rare Disease Weeks.

Daniel Levine: Given that an advocate has such a short time to communicate to a representative, would you advise them to speak in very personalized terms? Should they make a formal ask?

Sarah Tompkins: Great question. It’s so hard to balance that personal story with a formal ask, but it’s so important to tie the two together. And even if you have to shorten your personal story along with your ask, remember that it’s a great chance for you to contact them and follow up through email, to share more about your personal story or say, “Oh, I wanted you to also know this.” And as long as you’re sharing your experience, I spoke about the loss of my best friend Kelly, who also had EDS. And when I shared that with Senator Cantwell, I became so tearful that I really struggled through my ask, but that gave me the opportunity to follow up an email with the specifics of my legislative ask for 21st Century Cures Act and invite Senator Cantwell and her office and staff to our local support group. So, remember that if you’re being honest, and if you don’t know something, use it as an opportunity to provide the answer through follow up and remember whatever you aren’t able to say is a great opportunity to just continue that relationship through conversation and follow up.

Daniel Levine: Well, you mentioned follow up. What advice would you have for advocates about following up and how much of an ongoing relationship should they try to build with representatives or their staffs?

Sarah Tompkins: Emails are a wonderful thank you note, but I am a big fan of handwritten thank you notes as well. Certainly, that’s not necessary for everyone. I think it’s to each advocate’s comfort and the level that they’re able to give to it. But being able to keep that conversation going by inviting your elected reps and their staff to local events for your patient community and the rare disease community is going to keep that contact going so that when you do need to email them about an urgent, rare disease action or legislation for your rare disease patient community, you’ll already have established that connection and conversation and relationship. There’s no wrong way to follow up. If it’s email, handwritten, phone call, there’s no wrong way to follow up.

Daniel Levine: Britta, each advocate may have their own policy agenda, but I’m wondering if you could touch on some of the major policy issues likely to be addressed during Rare Disease Week on Capitol Hill. What are some of the key issues you expect to be discussed?

Britta Dornan: Sure. I’d love to share that. Let me just first say: Sarah, you are an inspiration to me and I just know to everyone who is listening. When I listen to you, I always learn something new and I just think you’re amazing. So Danny, advocates who join us at Rare Disease Week are encouraged to speak to their members of Congress on whatever rare disease issue that they would like to discuss. It’s their time. And we honor that, but I will give you a quick rundown of just a few different bills that touch the full spectrum of rare disease that we educate advocates on, and that we expect to come up in these meetings. The first is the Speeding Therapy Access Today Act, that’s also known as the Stat Act. The average time it takes to develop a rare disease drug is 15 years—that’s way too long. Like its name, the Stat Act is all about speeding the development of therapies across the spectrum of rare diseases and speeding access to these therapies. It does this by enacting targeted policy reforms at the FDA. Second, we have the Newborn Screening Saves Lives Reauthorization Act. Our nation’s newborn screening program is one of the most successful disease prevention programs in the history of the United States. And yet the system is unsustainable and our babies are at risk. So, this act is focused on expanding and improving the program so that all babies born in the U.S. have access to early detection of disease and the delivery of lifesaving treatments. Next, the Better Empowerment Now to Enhance Framework and Improve Treatments Act, also called the Benefit Act. There has been much progress over the last decade in ensuring that the FDA is evaluating a drug for patients that the patient experience is being considered, a lot of progress, but we have a way to go. The Benefit Act helps to close this gap by codifying in law, that the FDA include patient experience data as part of what is called its risk benefit framework. And lastly, I’ll mention the Access to Genetic Counselor Services Act. Currently Medicare denies its beneficiaries direct access to genetic counselors. And as we know, genetic counseling is becoming an increasingly powerful tool in navigating complex factors affecting your health. So this bill would help close this gap for Medicare beneficiaries to gain access to genetic counselors. So that was a cliff notes version of the bills, but advocates can learn more about each of them during Rare Disease Week when they join us. Sarah, what are you going to be doing this Rare Disease Week?

Sarah Tompkins: I’ll be advocating for the Access to Genetic Counselor Services Act because Ehlers Danlos syndrome is a genetic connective tissue disease. I still don’t know the type of Ehlers Danlos syndrome I have because we don’t have that genetic information yet. And we need more patients, especially under Medicare, to be able to see the genetic counselors, to get these specific diagnoses. It’s important to me, it’s important to my patient community, and it’ll further genetic counseling service access for the entire rare disease community. I’m really excited to advocate for it and advocate with my Washington state team this year.

Daniel Levine: Britta, for people interested in learning more about how they can participate or get training, where can they go?

Britta Dornan: Well, anyone who would like to experience a week that will change their life and where they can help improve the lives of so many other people should go to rare advocates.org/rdw, but don’t delay because February 11th is the deadline to be able to register and participate in the Hill meetings that we’ve been talking about. So please join us and make your voice heard. We hope to see you there.

Daniel Levine: Britta Dornan, senior director of communications and marketing for the EveryLife Foundation and Sarah Tompkins, advocacy chair of Rare Disease Week 2022. Britta, Sarah, thanks so much for your time today.

Sarah Tompkins: Thank you so much, Danny. I’m so excited for a week that can change your life from home.

Britta Dornan: Thank you so much, Danny, for helping to spread the word about this amazing event.

This transcript has been edited for clarity and readability.