Citizen Health

Nasha Fitter captures it well. Foundation leaders are often told a natural history study is required before they can move a therapy forward. That's true. What's less understood is that the traditional approach, prospective, multi-year, site-based, rarely works for ultra-rare diseases. It's slow, expensive, and burdens the families you're trying to help. The FOXG1 Research Foundation took a different path. Working with us, they assembled close to 1,000 patient-years of longitudinal data from 100+ patients in under six months. That dataset powered endpoint discovery, external comparator construction, and ultimately secured FDA alignment on a single Phase 1/2 study as potentially registrational under the RDEP pathway without a placebo arm. The case study Nasha references breaks down exactly how that was done. If you lead a foundation or work in rare disease drug development, it's a must read. [Link is in the comments] #rarediseasedrugdevelopment #RWE

How do you generate meaningful evidence in rare disease patient populations?   Elli Brimble, Head of Clinical Strategy at Citizen Health, will share her expertise around complex rare disease data questions in her session, “From Community to Clinic: Patient-Centered Evidence Accelerates Therapeutic Development in Rare Disease,” as part of the Triangle CERSI workshop, “Advancing Novel Surrogate Endpoints for Rare Disease Drug Development,” taking place on Monday, May 18, 2026.   The virtual workshop brings together regulators, researchers, clinicians, and industry leaders to explore how surrogate endpoints are developed and applied in rare disease contexts. If you’re interested in the evolving role of patient-centered data in clinical development, join the webinar next week. You can learn more and register here: https://lnkd.in/esD3Y9RE   #RareDisease #ClinicalStrategy #RealWorldData #DrugDevelopment

Patients with rare diseases aren’t just navigating care; they’re navigating uncertainty and a beast of a medical system.   At Citizen Health, we’re building an AI advocate alongside patients and families to help change that. Hearing from leaders like Sumaira Ahmed (Founder, The Sumaira Foundation) brings that reality into focus for our team and fuels our drive to change the future.   The NMOSD landscape alone has evolved from zero approved therapies to four—driven by advocacy, research, and community leadership from patient leaders like Sumaira.   At Citizen we are constantly in dialogue with inspiring and fierce patient advocates to inform what we build. And we have started a new monthly "Citizen Health Voices" series, bringing our team together in the office to hear directly from the rare disease community, so that we can build better and build faster to solve the care needs of healthcare’s most overlooked patients.   #RareDisease #HealthcareInnovation #PatientAdvocacy #AIinHealthcare

What if every doctor’s visit didn’t start with a clipboard?   Citizen Health was an early adopter of the Centers for Medicare & Medicaid Services (CMS) pledge to give patients easier access to their health data across medical office visits. Now, 60+ organizations have joined the movement to “Kill the Clipboard.”   In TechTarget, Deven McGraw, chief regulatory and privacy officer for Citizen Health, shares what it takes to reduce administrative burden and why collaboration across healthcare matters more than ever.   Learn more here: https://lnkd.in/gZGsBCXX   #HealthTech #PatientExperience #RareDiseases #Interoperability TechTarget

Our co-founder Nasha Fitter joined the Biohub Affiliate Program 2026 Symposium this week. A gathering of leaders from industry, academia, venture, tech, and philanthropy united around one mission: to cure or prevent all disease by combining frontier AI with frontier biology. This is exactly the ecosystem Citizen Health is built to serve. We connect thousands of rare disease patients with 100+ advocacy groups, with a global reach of 400M+. We know firsthand that the breakthroughs discussed at Biohub (multi-dimensional imaging, decoding inflammation, reprogramming immune cells, frontier AI research) only matter when they reach the patients waiting for them. Thank you to Biohub for convening this community, and for betting on the cross-sector collaboration that makes frontier science actually translate to people’s lives. Tania Simoncelli #Biohub #RareDisease #AI #DigitalHealth

To the celebrate the upcoming start of human clinical trials for a first of its kind gene therapy for FOXG1, Citizen Health and FoxG1 Research Foundation Co-Founder Nasha Fitter, sat down with Daniel Levine, host of RAREcast, the Global Genes podcast. Like other ultra-rare diseases, FOXG1 has no approved disease-modifying therapies today. Families are forced to navigate a list of serious symptoms with tools that were designed for other conditions. In this episode, Nasha discusses the data from the natural history study with Citizen Health that helped to shed light on the neurodevelopment disorder, and how she formulated a research agenda as a parent to get to this historic milestone. Listen to learn more 👉 https://lnkd.in/gSRr8VRj #RareDisease #FOXG1 #Research #RARECast

Patients need AI that is powerful and precise but also grounded in real-world data and trust. OpenAI's new life sciences white paper highlights how AI can accelerate discovery by connecting fragmented data, lived experience, and research. We’re proud to be part of that conversation and to support clinical research in rare disease communities. #raredisease #healthdata #AI #AIinHealthtech Nasha Fitter

CNBC spotlighted something we're incredibly proud of and here's why: The FOXG1 Research Foundation built a full natural history study on Citizen's platform using patient medical records. No clinic visits. No long surveys. The FDA aligned with using this data as the control arm in a seamless Phase 1/2 registrational study, which could potentially remove the need for a separate Phase 3 trial and save upward of $80M. This is what it looks like when patients are at the center of research, and not an afterthought. We're also building agentic AI specifically for rare disease families: • Helping navigate insurance appeals, schedule appointments, surface signals in medical records, and connect families to relevant clinical trials • Over 8,000 patients across 350+ diseases are on the platform • 98% choose to share their data with researchers   Follow the CNBC Cures newsletter for more rare disease news and check out our full story: https://lnkd.in/g9aPead7    #RareDisease #AI #PatientAdvocacy #HealthData #CNBCCures

What does it take to build a gene therapy from scratch and a platform to support every rare disease family along the way? Our co-founder Nasha Fitter sat down with PharmaBoardroom to share the story behind both. Following her daughter Amara's diagnosis with FOXG1 syndrome, Nasha co-founded the FOXG1 Research Foundation to develop a gene therapy at a fraction of traditional costs and built Citizen Health to solve the data problem slowing everyone down. In the interview: - How Citizen's patient dataset shaped the FOXG1 clinical program and saved millions - Why natural history data is the key to FDA-defensible, single-arm trials - The new paradigm of parent-entrepreneurs driving rare disease innovation Read the full conversation: https://lnkd.in/gEiYUNQ2 #RareDisease #PatientAdvocacy #GeneTherapy #RealWorldData

For ultra-rare diseases, the traditional clinical trial model wasn't built for this reality. The answer isn't weaker evidence — it's better evidence, collected differently. Longitudinal real-world data. Continuous outcomes tracking. A genuine, ongoing relationship with patients after therapy begins. That's what we're building at Citizen Health. #RareDisease #RealWorldEvidence #AIinHealthtech