This year, Cambridge BioMarketing is partnering with Akcea Therapeutics, the Boston Public Health Commission, and the Rare Disease United Foundation to host Rare Rights. Common Cause. The panel, moderated by Cambridge BioMarketing’s Director of Medical Strategy Dr Samuel Falsetti, will feature experts representing patients and caregivers, medical science, and biotechnology. All three will focus on the rare disease community from their different perspectives.
The primary topic of the panel is how healthcare stakeholders from all walks can “take the rare-disease-as-a-right challenge into their own hands” and become a voice in advocating for better recognition and care.
The three members of the panel are: Jenifer Jaeger, Interim Medical Director, Boston Public Health Commission; Paula Soteropolous, CEO, Akcea Therapeutics; and Patricia Weltin, CEO/Founder, Rare Disease United Foundation.
In an exclusive interview with Rare Disease Report, Weltin touched on why these topics are so important, why she was so honored to be a part of HUBWeek, and what the Rare Disease United Foundation is focusing on as they move forward.
RDR: What excites you most about participating in this event during HUBWeek?
Weltin: When (a representative from) Cambridge BioMarketing first called me about doing HUBWeek, I wasn’t sure really what HUBWeek was. It’s an event in Boston and it’s not just about science; it’s about the arts and technology. It’s happening all week, all over the city. I speak a lot, so I didn’t think much of it. After I was sent a link and I looked more closely, I can now honestly say that I’ve never been more honored to be involved in something in my entire life. You look at the website for the event, and we’re not called “speakers.” We’re called “change makers.” For me, that’s the highest honor – to know that I’ve made some kind of a difference in this space.
RDR: How different will this be from other talks that you’ve given or panels with which you’ve participated?
Weltin: The audience is going to be a little bit different from what I think our organization is used to seeing, because there is more of a platform to engage other people that might not be involved with rare diseases. It’s HUBWeek – It’s something bigger. I’m really excited about that, and that people will come in touch with subjects that they wouldn’t otherwise be around.
RDR: Will there be a primary message that you’re trying to convey at the event?
Weltin: One of our major concerns is diagnosis time. If there’s a treatment and you can get it somebody because they’ve been properly diagnosed, it makes such a difference. There’s so much benefit to early diagnosis, even if there’s not a cure. There are things that can be done without having to wait 18 years for a correct diagnosis, and that’s one of the issues I hope I can discuss. My big thing, too, is patient voice and how much we can offer to the community.
RDR: How important do you think organizations like yours are to the rare disease community?
Weltin: I’m not the only person (who knows somebody with a rare disease). This is not something that’s unique to me; it’s the families that are moving the needle, and we need to recognize that it’s not only data that’s important. It’s input and our insights that’s important. I personally believe that it’s as important as genetic testing and medical records. There’s just so much more to the conversation, and it needs to be more of a focus.
RDR: What future plans do you have for the Rare Disease United Foundation?
Weltin: We’re starting a program based around education. I know what it’s like to try to raise awareness for rare diseases, and my belief is that we need to start educating in middle and high school, so we have an entirely new generation of people who, when they hear the words “rare diseases,” their eyes won’t glaze over. We need to do it in a way that’s engaging to people, especially young people, and we’re going to do it through film. We’re going to make movies that give people a visual. We’ve seen what art exhibits and film festivals can do, and our next project is Rare Education Reel. It’s in the very early stages, but I know that there’s a huge difference between saying to somebody that there are 7,000 rare diseases and only 5% of them have treatment and showing somebody what those numbers look like. We want to create something that’s visually impactful.
The Rare Rights. Common Cause. panel is scheduled to take place at Cambridge BioMarketing at 4:45PM on October 11. For more news from the Rare Disease United Foundation and other advocacy groups within the rare disease community, follow Rare Disease Report on Facebook and Twitter.