Article
Paying It Forward: Advocating for Others in Clinical Research
By paigel|Jan 13, 2022
CISCRP hosted a 3-part webinar series titled “Navigating Rare Disease and Clinical Research: Every Patient Matters”. This article brief is based on the third episode, “Rare Disease Clinical Trials: After Participation, Paying It Forward”. Moderated by Britta Dornan, Senior Director of Communications and Marketing at EveryLife Foundation for Rare Diseases, panelists included Ryan Colburn, Rare Disease Patient Advocate, Pompe Disease, Dr. Kim Stephens, PhD, President of Project Alive and Marc Yale, Advocacy and Research Coordinator at the International Pemphigus and Pemphigoid Foundation. You can access the webinar recording here.
To “pay it forward” is a philosophy popularized by author Catherine Ryan Hyde’s novel of the same name, where acts of kindness received are not reciprocated to their originator, but forward through the world, creating a ripple effect of good deeds, both big and small. This sentiment is shared by many members of the clinical research community, via the avenues of clinical trial participation, advocacy, and awareness.
“I am a big proponent of paying it forward, sharing your story and experiences for others to benefit. That’s why I created a blog for my rare disease, lymphemadiary.com,” says Britta Dornan, a rare disease advocate, communications professional with EveryLife Foundation for Rare Diseases, as well as a rare disease patient for over three decades. Lymphedema (LE) is a chronic lymphatic disease that can be hereditary, or can occur after a surgical procedure, infection, radiation, or other physical trauma. (1) Despite wearing compression garments 24 hours a day, Britta skis, is a runner and has climbed the Great Wall of China. “I am determined to challenge my limits, rather than let LE limit my challenges,” says Britta.
Marc Yale views becoming an advocate and choosing to participate in a clinical trial as being intertwined concepts. It took six months for Marc to be correctly diagnosed with Cicatricial Pemphigoid, a rare autoimmune skin blistering disease. (2)
“I was kind of angry, I was like ‘How come nobody knows about this disease?’ I understand it’s rare, but shouldn’t doctors know about this? As a patient, you go to the doctor and expect them to be able to answer your questions,” says Marc. In order for more doctors to understand and know about this disease, Marc realized he had to become an advocate for himself and others and create awareness about it.
While seeking a diagnosis, Marc lost the vision in his left eye to the disease. “I was in sort of a desperate situation. I had to take aggressive steps and so I thought, ‘What about a clinical trial?’” recalls Marc. He applied for a clinical trial in France, but he did not meet the inclusion criteria. The experience spurred him on to advocacy efforts.
“What else can I do to advocate for people with my disease so that they don’t have to go through what I was going through? That was the “aha” moment for me, where I decided that I am going to crank disease awareness. I decided that I wanted to become an expert about it, so that when I get into a room with researchers, I know what I am talking about,” says Marc.
He initially volunteered with the International Pemphigus and Pemphigoid Foundation and helped create a peer health coach program, before joining as staff. “I found a community of people that was experiencing what I was experiencing, and it really gave me a lot of hope,” says Marc. He later focused his efforts on the legislative and policy side of advocacy. “Policy and regulation and changing those things is really my calling,” says Marc.
Britta adds, “Working to impact and change policy is a long game. It can sometimes feel like Sisyphus, you’re rolling and rolling and pushing that boulder up the hill, and it keeps rolling back over you down the mountain.” Maintaining focus on the end game of effecting change, is key.
“My son Cole was diagnosed with Hunter Syndrome when he was 2 and a half. Immediately, I was thrown into the world of rare disease, not having any experience in that area at all, but knowing that I needed to find our next steps. We jumped into the clinical trials world,” says Dr. Kim Stephens, PhD. Hunter Syndrome is a rare progressive disease almost always occurring in males, where the body does not break down sugar molecules. Cases range from mild to severe, and life expectancy may be shortened. (3) Cole qualified for the trial, but it was placed on hold for two years. Cole entered the trial at age four and a half and has been participating in the clinical trial for over six years.
Kim’s experience as an executive communications professional strengthened her skills as a patient advocate for her son, as well as others impacted by the disease. Her work includes serving as President of Project Alive, a non-profit organization dedicated to research and advocacy for Hunter syndrome. Her role includes meeting with clinical researchers to provide input about clinical trial design. “I want to make sure that all the clinical trials are successful in our space, because we can’t afford to have any boy in a clinical trial that is going to fail. There’s a real urgency for me to get out there,” says Kim.
“Sometimes, we look around for someone else to step up and then we’re like, ‘But what about me?’” says Kim. “A lot of our motivation is, if we can help someone else not go through what we’ve been going through, then that’s a big plus. As advocates, we’re always there saying ‘Well, what about this, or have you thought about this?’ and thinking more broadly. I’m urging companies now to treat boys that are older, not just doing treatments for newborns through five-year-olds – what about the boys who haven’t been able to be in a clinical trial? Really thinking about how can we include as many patients as possible in a clinical trial.”
“I don’t think I ever thought, even to this day, ‘Oh, I am going to be an advocate’” says Ryan, a patent-holding engineer who was diagnosed with Pompe disease in 2015. Pompe is a rare disease that disables the heart and skeletal muscles. (4) “When I got diagnosed, I tried to learn about the disease, for myself, just so I understood what it was. I continued to figure out that there were a lot more questions that I had that did not have answers, than the ones that did have answers. My mindset about clinical trials is that they’re about participation, and they are also one of the ways we can ask questions and learn something about our disease.” Ryan’s clinical trial participation is now in the double digits.
“Trials can be these long multi-year things, or they can be a day long. Some of them have been as simple as giving a blood sample, so that we can learn new techniques to do newborn screening. Some of them have been traveling regularly to get an experimental treatment injected into your veins. Participation has to make sense for you. It should not be like this sacrificial hero’s aspiration,” says Ryan.
There are tremendous benefits of paying it forward and making contributions that play to one’s individual strengths.
“It was cathartic to be able to do something, and for me, it was part of that healing process, in coming to terms with my condition. That power that I lost when I was diagnosed, I gained back by being able to do something to help others,” says Marc. “You have to ask yourself how can I take this lived experience and make it better for somebody else?”
“I am sort of a political science nerd, so I love going to The Capitol and talking with the Senators and our Representatives. I think of it as I am creating this broad network for myself and for my community, because I don’t know what we’re going to need in the future, and you never know who you are going to influence,” says Kim.
“You don’t have to go all in. It starts by doing something, and you do something a bunch of times, and all of a sudden, you’ve really done something. It doesn’t have to be this big, daunting thing in front of you,” says Ryan.
“Paying it forward is the idea of empowerment and taking back control. We may have these diseases, but they don’t have us,” says Britta.
You can view part-one of the series, “Rare Disease Clinical Trials: Being Informed About Clinical Research” here and part-two, “Rare Disease Clinical Trials: How to Prepare for When the Clinical Trial Ends” here.
Access the companion infographics:
- Clinical Research Participation Roadmap | Part-one here.
- A Garden of Questions to Ask Before the Clinical Trial Ends | Part-two here.
- Paying it Forward in Clinical Research | Part-three here.
To search for medical conditions in a specific location, visit our Search Clinical Trials page.
To stay informed about clinical trials, visit our resources page.
Sources:
- https://lymphaticnetwork.org/living-with-lymphedema/lymphedema/
- https://pubmed.ncbi.nlm.nih.gov/1869688/
- https://my.clevelandclinic.org/health/diseases/17932-hunter-syndrome
- https://www.ninds.nih.gov/Disorders/All-Disorders/Pompe-Disease-Information-Page
Written by Melissa E. Daley, Communications & Marketing Manager, CISCRP
