in the rare disease space it's important that you realize you are joining a community not just marketing to consumers
Siren had the great opportunity to attend the World Orphan Drug Congress on April 13-15. For three days, we were immersed in amazing content that included presentations from more than 50 contributors addressing the challenges and opportunities for the commercialization of drugs to treat rare diseases. We heard from individual patients, advocacy groups, leaders from biotech and pharmaceutical companies, and government agencies.
The theme throughout the Congress was the importance of engaging patient communities and caregivers. It was inspiring to hear the deep level of commitment companies like Shire, Genzyme, Lundbeck, BioMarin, Prosensa, and Amicus have to the patients they serve. It was clear that there is a true partnership between industry and patient groups when it comes to developing life-critical treatments.
Powerful Content Throughout
You can tell how compelling the content was by the number of people who packed the room each day and most presenters didn’t scurry out after they spoke. Siren’s Founder & President, Wendy White, spoke about the power of social media when it comes to engaging patients and caregivers. The key take-away was that when you work in the rare disease space it’s important that you realize you are joining a community not just marketing to consumers.
Wendy and Bill Cusick, Siren’s VP of Creative Services, kicked off the event with a pre-conference workshop “The Voice of the Caregiver: Building Relationships and Brand Advocacy through Relationship Marketing Programs.” Attendees learned the value of including caregivers in their companies’ marketing efforts since the majority of patients with rare diseases are children. Pat Furlong shared her moving story: after both of her sons were diagnosis with Duchenne muscular dystrophy, she was empowered to start Parent Project Muscular Dystrophy which is now the largest nonprofit organization in the United States focused entirely on Duchenne.
Dr. Timothy Coté, Director of the Office of Orphan Product Development at the FDA encouraged companies to take part in an orphan drug workshop series – an opportunity for academics, biotechnology companies and pharmaceutical firms to spend two days in creation of applications for orphan status designation. There are five scheduled for 2011 including two international ones.
Several leaders suggested reaching out to the National Organization for Rare Disorders (NORD) when it comes to forming a patient advocacy group. And, as an advocacy group, it was clearly communicated that you are a partner with the industry – do not consider yourself a “second-class citizen”.
Biggest “AHA” Moment
For me – and many others I spoke to afterward – the most poignant story about the importance of treating rare diseases came on the last day. Wayne, a patient with Gaucher Disease, shared his story that morning. He had suffered greatly until he began treatment in 1993, then he was able to live a normal life. His journey was similar to others that we had heard throughout the conference – until he mentioned what he does for a living.
Wayne practices psychology and works in an emergency psychiatric ward. A week prior to the conference, he worked with a mother who was admitted for attempted suicide. He helped prevent what would have been a devastating event. After the incident, he got a call from a member of the woman’s family to thank him.
In this industry, we hear many inspiring stories of patients who are alive because of the treatment available to them. But it isn’t often we think about the ripple effect. Had Wayne not been able to go to work that night because of his disease, the story of the mother who was going to take her own life may have had a different ending.