BIO2023, a huge international biotech convention organized by the Biotechnology Innovation Organization, was held in Boston, MA in early June, 2023. When I say huge, it means this: 19,000 participants from 85 countries attended. There were 140 sessions and a trade show floor with 1300 exhibitors. Big! I was happy to represent SynGAP Research Fund as an attendee.
Program content spanned the industry and the globe. There were sessions on cell and gene therapies, regulatory innovation, next generation biotherapeutics, vaccines, global markets, reimbursement and so much more. I’m also happy to say that there was an Orphan & Rare Disease track.
The Power of the Patient Voice
My first stop was the session The Power of the Patient Voice. This was a panel discussion that included some familiar faces even though we’d never met. Some of you may know the podcast Two Disabled Dudes. Well, the two dudes themselves were there, Sean and Kyle. They were engaging and witty as they moderated the discussion. The panelists included Daniel DeFabio, cofounder of The DISORDER Channel, and two biotech industry representatives,
Panelists talked about some of the ways to tell stories, such as podcasts, movies, and through social media. They talked about why it’s important to keep patient stories front and center. A company can’t be truly patient-centric without the patient voice. Without this, it could end up going down the wrong path, being less impactful in their therapeutic development or with a very expensive mistake. And because of this, patient stories are no longer an afterthought. The model is changing, with patients being included from the start.
In addition, stories create connections. Daniel shared some of his experience, which demonstrated so well that having your story “out there” is a way for you to find people and for them to find you. It has led to many great collaborations for him. We heard about #RareIs, a social media campaign launched by Horizon Therapeutics to elevate the voices, faces and experiences of the rare disease community.
Another important part of the patient voice is to have stories from all walks of life. Diversity means including people of different races, yes, but also of different geographies, cultures, socioeconomic status, religions, and gender identity/expressions.
After the session, we were treated to the BIO Film Festival, featuring short films from a number of companies, including DISORDER. We watched six films from the rare community, two of which featured SYNGAP1. I proudly watched Celebrating Caren, featuring Caren Leib. And next up was a very moving interview with caregivers of various rare patients, Bad Days, Good Days, Rare Days. This featured SRF’s co-founders Mike Graglia & Ashley Evans, whose son, Tony, was up there on the big screen.
Then & Now: 10-Year Regulatory Reflections
Another session in the Regulatory Innovation track called Enhancing Meaningful Engagement on Patient Experience Data in Drug Development continued the theme of patient engagement. Representatives from the FDA, National Health Council, and the biotech industry looked back ten years, when no one was asking the patient what outcomes were important to them. Fast forward to the present day, the culture is changing to include the patient voice from the start. The FDA presented its FDA-Sponsor Engagement Framework. Together, the FDA and drug developers aim to identify and enhance opportunities for patients to inform them of the clinical outcomes that matter to them.
From these sessions and others, I came away encouraged that the patient voice is more and more becoming part of the drug development process from the start. And who can best be the patient voice? Patient advocacy groups (PAGs) like SRF.
Let’s continue to tell our stories. Rare diseases sometimes need to yell a bit louder to be heard, so every voice helps. Every tweet, every social media post, every interview, every blog, every article, every #RareIs or Wednesday Warrior story shared–these all strengthen our voice. Keep it up, SYNGAP1 community!