“The reason the Master Class is happening is because advocates like myself were not given a roadmap when our children were diagnosed with rare diseases,” states Jennifer McNary, Rare Disease Advocate. She’s flanked virtually, by her peers and fellow patient advocates for Reuter’s Events 2020 Pharma awards.
Jennifer, alongside three other nominees, is being honored for her work in furthering patient advocacy. Jennifer supports the Patients Rising Master Class she mentions as a faculty member, which is facilitated by fellow Reuters nominee, Lilly Stairs, and is simply a part of her everyday work in furthering the education that exists for patients who suddenly find themselves at the tail end of a rare disease diagnosis.
For Jennifer, and many advocates like her, proper education is the first step in creating a roadmap towards treatment. “Really, the key is that when you have success, instead of keeping that to yourself, share it.”
Thankfully, Jennifer has been instrumental in creating roadmaps for rare diseases for years, most notably with Duchenne Muscular Dystrophy (DMD). In fact, her work as the Director of Outreach and Advocacy at a Massachusetts based non-profit foundation is the reason for what was the largest FDA advisory committee hearing in history for DMD.
Jennifer’s end goal? Better access to treatment and greater speed to cures.
“Approved doesn’t mean accessible,” Jennifer says. “There are so many disparities around who can participate in a trial. When my son was in a trial we were flying back and forth across the country every week for blood work.”
Thankfully, Jennifer acknowledges that those days are starting to change. Companies across the globe, such as Medable, where Jennifer chairs the Patient Advisory Council for 2020, are helping to bridge the gap between patients and trials. Home measurements, remote assessments and other technologies greatly increase access to trials that were previously only available to a select few.
Recently, advancements in DMD treatment have given advocates like her hope. Yet, she acknowledges there is still much work to be done, not only in DMD, but in rare diseases with little to no treatment plans.
“My hope for all the rare disease community is that we continue enhance flexibility at companies like ours, enhance education, and be prepared to provide access and reimbursement for these kinds of therapies,” Jennifer notes, “So that when someone is born with a rare disease in five years there’s a pre-established treatment plan to help them.”
To learn more about the Master Class, please visit the Patients Rising Now website here.
Jenn McNary is a trusted voice in the rare disease community, as a mother, public speaker and fierce advocate. Her work in the rare disease space as a thought leader earned her the Ryan’s Quest Ryan’s Hero award in 2013, a nomination for the Global Genes Champion of Hope award in 2014, and the prestigious 2017 Meyer- Whalley instrument of change award. Formerly as the director of outreach and advocacy at a Massachusetts based non-profit foundation, she was responsible for the organization of the largest FDA advisory committee hearing in history, with over 1000 Duchenne Muscular Dystrophy (DMD) advocates, families, clinicians and researchers in attendance.