Let’s be honest, at the end of the day, what we want is a drug that works for our children.
We are honored to share this guest post from Melissa Hogan, founder of Saving Case. Melissa started the organization after her third son, Case, was diagnosed with the rare disease Hunter Syndrome or MPS II. She is also the author of Calmer: Medical Events with Cognitively Impaired Children.
During my recent time at the World Orphan Drug Congress, I had the pleasure of a nice dinner and conversation with some operations folks from a larger pharmaceutical company, otherwise affectionately known in our crowd as “big pharma.”
Interestingly enough, while we talk about big pharma (I’m going to keep using that term even though it is not precisely defined) entering the rare disease space for MPS/ML, some of the companies more established in our space are definitely not “small” by pharma standards – Shire and Genzyme/Sanofi.
But, sometimes it’s the case that you can slander your family, but you’re not going to let anyone else do it, you know what I mean? Our patient populations have grown up with these companies in many respects, from when they were small and testing that one compound that gave us hope.
harmonization across all rare disease stakeholders is essential for success
The World Orphan Drug Congress last week consisted of three days jam-packed with presentations and networking. While the event focused on rare diseases and orphan drugs, the scope was still wide and addressed issues ranging from pricing to patient registries to various country regulations.
The highlights for me were the patient/caregiver testimonies scattered throughout the conference which reminded everyone why we were in the room. The presentation by Yi-Ou Wang from the nonprofit organization China-Dolls Fund was especially powerful as she described the lack of social awareness and support for rare diseases in China.
I had the chance to talk with a few empowered caregivers: Melissa Hogan from Saving Case, Lori Sames from Hannah’s Hope Fund, and Kelli Foster from The Mastocytosis Society. I was also pleased to finally meet two legends within the rare disease industry: John Crowley from Amicus Therapeutics and Pat Furlong from Parent Project Muscular Dystrophy.
Harmonization
The theme that was woven through many of the presentations was the idea of harmony—the state of being in agreement. Kinnari Patel, Bristol-Myers Squibb, started the first day by noting that harmonization across all rare disease stakeholders is essential for success. There are a variety of levels of harmonization required, and many of the speakers are working on facilitating these partnerships.
I was part of the Siren team that attended the World Orphan Drug Congress last week in Washington, DC. On the first day we led an interactive workshop entitled, Engage with Rare Disease Patients Using MicroTargetingSM and Social Media.
We believe it’s essential to include the patient/caregiver point of view, so we invited Melissa Hogan, founder of Saving Case, to present with us. Melissa started the organization after her third son, Case, was diagnosed with the rare disease Hunter Syndrome or MPS II. She spends her time “writing, speaking, and advocating for children with MPS and other rare diseases in the health care, education, and social services contexts.”
At the workshop Melissa shared the journey to Case’s diagnosis and how she uses social media. Melissa calls the empowered moms in this space “pusher moms” because they are constantly pushing forward for their children. See the video below where she describes how she approached clinical trial recruitment.
Watch this space for more insights from the World Orphan Drug Congress.
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.
