Five years. That’s how long, on average, people living with rare diseases wait between their first symptoms and receiving an accurate diagnosis. Five years of uncertainty. Five years of pressure on patients searching for answers, on caregivers managing the unknown, on clinicians navigating complexity, and on researchers racing to develop solutions.
In biotech and healthcare, pressure is part of daily life. It appears in the rush to bring new therapies to patients, and in the complexity of clinical trials. For patients and caregivers, it takes on a deeply personal form. More than half of people with rare diseases wait at least six months after their first medical contact before getting answers. Treatments can feel confusing, and speaking up in a system that does not always listen takes remarkable courage.
Even under that strain, something extraordinary happens. Purpose emerges. It is the reason we get up each morning, whether we are scientists in a lab, clinicians at the bedside, advocates in the community, or family members at home. Purpose transforms those years of uncertainty into a mission to create change for the next person facing a similar journey.
This belief is at the heart of MassBio’s 2025 Patient Advocacy Summit and its theme, Purpose, In Spite of Pressure. It reminds us that resilience is not about avoiding challenges, but about choosing to keep moving forward with meaning, even when the weight feels heavy. For industry professionals, this means keeping patients at the center when market forces shift. For advocates and researchers, it means transforming personal challenges into systemic change. For patients and families, it means finding strength in shared stories and collective action. For all of us, it means finding clarity of mission when the world demands speed, certainty, and results.
Patient advocacy leaders Yelak Biru and Fatima Scipione demonstrate every day how purpose-driven leadership can transform pressure into progress and empathy into impact. Yelak draws on a 30-year journey with multiple myeloma, while Fatima has redefined how patient voices influence R&D, access, and policy. “Joining lived experience with industry insight can light the way toward more compassionate and effective solutions for patients and families,” says Fatima.
One conversation at this year’s Summit will bring that purpose to life in a powerful way. The panel, Newborn Screening: Unlocking Lifelong Impact Through Early Detection, will explore how early testing can change the course of a life before symptoms ever appear. Right here in Massachusetts, newborn screening for spinal muscular atrophy has identified affected infants within days of birth, some of whom have remained asymptomatic and are meeting developmental milestones thanks to early treatment. For families, an early diagnosis can mean hope instead of heartbreak, or a manageable condition instead of a lifelong struggle. Our panelists will share how collaboration across advocacy, science, and policy is helping to modernize screening programs, close gaps in access, and ensure that every baby has the best possible start. It is a reminder that when purpose guides our work, innovation does more than advance science—it changes lives from the very first day.
Last year, we met parents Courtney and Joe Dion, who spoke candidly about two of their children, Peter and Maggie, and how their personal experience and devastating diagnoses have been harnessed to drive awareness and hope. “We are not doctors, we’re not biotech, we’re not professionals. We’re just two parents who are relentless in our pursuit to make a difference, said Courtney at last year’s event. “And I think that relentlessness goes a really long way.” This year, the Dions return to share what they’ve learned since: How has the clinical trial experience been for their family? What’s their take on the evolving landscape for rare disease research? Join us to hear their story firsthand.
From early detection in newborn screening to the lived experiences of rare disease communities, the discussions are united by one theme: when pressure is met with purpose, it can fuel breakthroughs that last far beyond the moment.
The MassBio Patient Advocacy Summit celebrates that commitment to purpose. It is a moment to reconnect with why we do this work and to lead with compassion, conviction, and courage, even when the pressure is greatest. It’s just one morning, but for many past attendees, what they heard from speakers and the conversations they had one-on-one stayed with them long after the last session ended.
MassBio Patient Advocacy Summit
📅 November 6, 2025 | 9:00 AM – 1:00 PM
📍 MassBioHub, Cambridge, MA
Walk away with:
- Actionable insights from patient advocacy leaders and industry innovators
- Real-world strategies for integrating patient voices into research and policy
- Meaningful connections with peers across biotech, healthcare, advocacy, and patient communities
- Renewed clarity on your purpose in this mission-driven work
Seats are limited. Register today to be part of this inspiring and empowering event.
