Amid all the high-powered speakers at the Democratic National Convention, two lesser known voices were particularly captivating. Those of Anastasia Somoza and Ryan Moore.
Anastasia, an outspoken advocate for Americans with intellectual and developmental disabilities, suffers from the rare disease cerebral palsy and spastic quadriplegia. Ryan Moore has a rare form of dwarfism, spondyloepiphyseal dysplasia.
In closing her talk with the words, “Live boldly with a courageous heart,” Anastasia brought the house to its feet.
Those of us passionate about being involved with rare disease communities were thrilled to see the additional attention people like Anastasia and Ryan are bringing to rare diseases. Of the 56 million Americans living with disabilities, simple things like having access to classrooms and buildings, living independently, and being enabled to contribute to society are often a daily battle.
We salute our many clients who work with rare disease communities and go to great lengths to get to know their patients on a more personal level, such as holding patient-celebration days. And those clients who invest in meaningful resources, like an illustrated storybook to help children understand their disease and why they have to see so many doctors. The outpouring of appreciation from the communities in response to these initiatives is a reflection of how badly they are needed.
At this past year’s World Orphan Drug Congress USA, we heard some terrific ideas on how best to educate on rare diseases, how to reach appropriate patients, and what types of resources are most needed. Additional topics included updates on patient-reported outcomes, biomarkers, the patient journey, government incentives, success metrics, and new ways to partner with patients and advocacy groups.
Nothing about rare diseases is easy. But improvements happen every day, particularly when, thanks to advocates like Anastasia and Ryan, people start paying attention.
Our in-depth report on the World Orphan Drug Congress, including 12 Marketing Takeaways from the 2016 WODC, can be downloaded by filling out the form below.