It’s time to let the world get to know our amazing rare advocates! Each week I will be highlighting an advocate here in my blog as well as social media. If you would like to be featured in the spotlight, please click here.
Parent Advocate | Hunter Syndrome
- Founder/President with Saving Case & Friends
- Thompson’s Station, TN
- Contact info: Twitter | Website | Facebook | Linked-In
- Hunter Syndrome affects approximately 2,000 people worldwide
- Passionate about: Research
Why did you become an advocate for your rare disease community? What was happening in that community that made you want to take action?
My training as an attorney naturally made me start advocating once my son was diagnosed, initially for him, but then for others because I was passionate about families not having to “recreate the wheel” in helping their own child. Later on, my son enrolling in a clinical trial that needlessly excluded many patients and took longer to enroll and complete than it should have made me ask the question why? And what could I do about it? I saw deficiencies on both the industry side and the regulatory side, both of which could be impacted by patient/caregiver involvement – so I sought to make an impact in both areas.
What was your first success or defining moment as a rare disease advocate?
My first success was in meeting with Dr. Janet Woodcock about a clinical hold placed on a Phase II/III trial for a groundbreaking treatment in Hunter Syndrome. That meeting led to a partial removal of the hold and later, to a meeting with DGIEP, the division that reviews drugs for Hunter Syndrome, to talk more about symptoms, clinical trial considerations, and issues important to patients.
Share some of your biggest accomplishments as a rare advocate?
Some of my biggest accomplishments are actually behind the scenes – when industry/regulatory decisionmakers tell me they’ve made different decisions based on perspectives I’ve shared with them about our Hunter Syndrome community or clinical trials. Publicly, my biggest accomplishment was the launch of the ProjectAlive.org campaign that generated significant research funds and celebrity backing.
Share some of the hard realities you faced advocating for your rare disease.
Advocating at the same time as caring for a child with that rare disease is hard. I often clinically describe the symptoms of Hunter Syndrome in my speeches at conferences. I have to function, make my points and the connections we need as an organization, and maintain composure and credibility. But more often than not, that crumbles on the plane ride home as I cry looking out the window.
What project(s) are you working on now for your community?
We are working on a new fundraising and awareness campaign, building upon our success with Project Alive. Families have really stepped up to the plate and are generating campaigns that I believe will be transformative for Hunter Syndrome awareness and research funding. We also continue to assist industry partners with clinical trial design and are working on a clinical trial impact framework.
What advice would you give to an advocate just starting out?
Prioritize the objectives for your work or your organization. Select the top three priorities and become excellent in those areas by attending conferences targeted to those areas, learning from other advocates, and just plain hard work. Find your tribe. Every advocate needs close friends in your disease area who are both supportive and think differently than you. Perspectives are a good thing.
Why do you find working with advocates from different disease groups beneficial?
I firmly believe in identifying best practices and not having to recreate the wheel. In rare disease, more than many other areas of study, disease areas, or professions, people genuinely want to help others succeed and usually don’t mind sharing their tricks of the trade. When we support one another, we are able to succeed on a broader agenda for the entire rare disease community as well.