Own Your Data, Accelerate Your Cure
A guide for Foundations moving from “funding research” to “owning the cure.” Discover the blueprint for Patient-Led Drug Development using Castor Catalyst.
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A new era of agency
“For too long, rare disease families have been told to wait. Wait for academic interest. Wait for pharmaceutical investment. Wait for a clinical trial to open. With 95% of rare diseases lacking an FDA-approved treatment, the ‘wait and see’ model has failed. We are entering the era of Patient-Led Drug Development.”
– Julia Vitarello, Founder, Mila’s Miracle Foundation | Global Genes Rare Patient Advocacy Summit
Today, successful patient communities are no longer just raising awareness. They are building their own infrastructure. They are acting as “virtual biotechs,” funding the science, de-risking their own therapeutic assets, and creating the epicenter of their disease ecosystem.
The power of ownership
Historically, foundations raised money to fund academic grants. While vital, relying solely on a single academic center to hold your registry often leads to data silos. Similarly, if you allow a vendor to aggregate your data for free, they often own and monetize it. Building and owning your registry infrastructure gives you four capabilities that passive donors never have:
1. Break academic silos
When you own the data, you can give it back to multiple academic researchers globally at the same time, forcing open collaboration, expediting peer review, and speeding up the science.
2. Fuel AI discovery
Organizations like Every Cure use AI to find repurposed drugs. They cannot do this without structured data. By owning a regulatory-grade registry, you provide the exact fuel needed to find a cure sitting on a pharmacy shelf today.
3. Sustainable revenue
High-quality longitudinal data is incredibly valuable. You can license de-identified data to pharmaceutical companies for clinical trial recruitment or synthetic control arms, generating revenue to fund even more research.
4. Follow the disease better
By capturing continuous data directly from patients via Patient-Reported Outcomes (ePROs), you build a real-time understanding of disease progression, validating the exact endpoints that matter to families.
Pioneers who proved the model
Patient ownership is proven. These communities didn’t just advocate. They built the infrastructure that directly enabled drug approvals and survival gains.
The gold standard
CFF accredited care centers to ensure high-quality data entry. As the trusted steward, they drove standard-of-care guidelines and made their registry the backbone of a global drug development program.
Result: 4 FDA-approved disease-modifying therapies. Median survival age more than doubled, from 29 years in 1990 to 68 years in 2023.
Trial enablement
PPMD acts as the “sole guardian” of the Duchenne Registry. They proved a patient-reported registry could be structured enough for industry feasibility planning.
Result: Recruited for 82 clinical trials.
Endpoint development
Used their registry to validate new endpoints (caregiver burden), ensuring future trials measured what actually mattered to families.
Result: >2,000 participants across 28 countries.
Global scale
Faced with a highly dispersed population, they prioritized a low-friction digital registry, removing geographical barriers for families worldwide.
Result: 70% of participants reside outside the US.
Fast learning
Their registry data revealed that trial endpoints were overly focused on seizures, while families cared deeply about communication. This fast learning reshaped future trial designs based on the patient and caregiver voice.
Result: Trial endpoints redesigned to reflect what matters most to patients and caregivers, directly influencing future study design.
How it works: the data retrieval engine
You do not need to be a software engineer to build a regulatory-grade registry. Castor Catalyst automates the hardest part: getting the data out of the hospital and into a structured database. By combining direct-to-patient consent with modern APIs, we build a complete medical history without burdening the patients, caregivers, or physicians.
The "self-driving" record retrieval workflow
1. Recruitment and eConsent
Patients join online, sign HIPAA release forms, and authorize data sharing. The entire consent process is digital, reducing friction and speeding enrollment.
2. Data retrieval (FHIRFast Healthcare Interoperability Resources — a modern API standard that allows electronic health records to be securely shared and retrieved across hospital systems in real time. + HIPAAHealth Insurance Portability and Accountability Act — the US law governing the privacy and security of personal health information. A HIPAA release form authorises a provider to share a patient’s medical records with a third party.)
FHIR APIs (Fast)
Instant pull of structured Labs, Vitals, and Medications via modern healthcare APIs.
HIPAA release (Deep)
Full unstructured PDFs and Physician Notes retrieved via traditional record requests.
3. AI and clinical review
Castor Catalyst extracts data from unstructured clinical notes, verified by human experts via Visual Audit Trail. Every data point is traceable back to its source document.
4. Foundation-owned registry and dataset
Regulatory-grade data populates your registry, ready for FDA submission. The foundation owns the data, the governance, and the intellectual property.
The catalyst for action: the TFE3 story
This model is not theoretical. It is happening right now. The story of the TFE3 Foundation provides a blueprint for what is possible when parents refuse to wait.
Halcyon's journey
In October 2022, Castor’s founder and CEO, Dr. Derk Arts, met the parents of a child attending the same school as his son. They shared the story of their four-year-old daughter, Halcyon. Born seemingly healthy, she began missing critical developmental milestones and experiencing hearing loss. At age two, she was diagnosed with an extremely rare genetic mutation in the TFE3 gene.
TFE3 is a “nano-orphan disease” with fewer than 30 known children diagnosed in the US. There was virtually no dedicated research or drug development underway. Halcyon’s parents were desperate and tracking patient stories in a shared spreadsheet. They asked Dr. Arts: “How can we do something for our child?”
Recognizing that the foundational step for any future pharmaceutical interest is highly structured, clinical-grade baseline data, Castor offered its platform to build a detailed, global patient registry.
The parent-owned, expert-backed model
To avoid the friction of requiring local Institutional Review Board (IRB) approvals for individual physicians globally, the registry operates on a patient-led model. Parents legally consent to share their child’s healthcare data. Castor managed the central IRB submission on their behalf. While driven by parents, the registry is formally supported by a Medical Advisory Board of academic researchers who ensure the clinical relevance of the data model.
The direct-to-patient workflow
- eConsent and retrieval Parents digitally sign HIPAA release forms authorizing Castor Catalyst to securely retrieve medical records via FHIR networks and traditional record requests.
- AI processing Catalyst processes years of unstructured clinical notes, extracting the necessary data points directly into the registry’s structured database.
- Quality of life (ePRO) The system integrates Patient-Reported Outcomes to continuously monitor the patient’s real-world developmental updates via surveys sent directly to parents’ and caregivers’ phones.
- Biobanking The digital registry coordinates with an NIH-funded biobank storing skin biopsies from TFE3 patients, giving researchers direct access to study functional genetics alongside the digital phenotype.
90%
Reduction in services cost
Our mission is to enable patient communities to run their own drug trials. Historically, the human services required to run a trial (manual data abstraction, site monitoring, compliance paperwork) made research prohibitively expensive for non-profits. By automating the clinical trial journey with AI, Castor aims to reduce service costs by 90%. That means your foundation’s money goes to the science and not the administrative overhead.
Your step-by-step blueprint
Castor provides the technology; you provide the community.
The "self-driving" record retrieval workflow
1. Gather your community (the foundation)
Establish your patient advocacy group. Create a central website to rally the families, patients, and caregivers. You provide the trust and the mission.
2. Establish academic governance
Partner with academic Key Opinion Leaders (KOLs) to form a Medical Advisory Board. Work with them to define the data model: what specific endpoints and biomarkers do we need to track to satisfy the FDA?
3. Deploy the platform (with Castor)
Castor helps define the Informed Consent Forms (ICFs), provides the registry technology, and manages the IRB and site services (with academic oversight). The parents own the registry and the governance.
4. Hybrid data ingestion (AI + ePRO)
Castor Catalyst uses AI to pull past medical records via FHIR and HIPAA to build the clinical history automatically. We combine this with defining Patient-Reported Outcomes (ePRO), crucial for developmental diseases to actively monitor progression.
5. Governance and data management
You have ultimate governance over who gets access to the data. You can export it at any time. Castor also offers expert clinical data management services on top to ensure everything is FDA submission-ready.
Useful resources to start your journey
A curated list of bootcamps, training programs, and regulatory guidelines specifically designed to empower patient communities and advocacy leaders.
Training and bootcamps
RARE Bootcamp (by Ultragenyx and EveryLife Foundation)
A multi-day forum designed to provide parents seeking to develop therapies with an opportunity to learn from drug development experts. More than 100 organizations have participated to date.
An annual event equipping advocates with the knowledge, skills, and connections to navigate early-stage research with confidence.
Self-paced online courses from NORD covering rare disease development, health data management, and how patient groups can become active research partners.
Step-by-step guidance for starting and growing a rare disease nonprofit, from filing your foundation through preparing your organisation for clinical research.
Registry building and regulatory guidance
NIHNational Institutes of Health — the US government’s primary agency for biomedical and public health research, funding thousands of disease studies each year. / NCATSNational Center for Advancing Translational Sciences — a division of the NIH specifically focused on turning scientific discoveries into real treatments, including support for rare disease patient groups. Patient-Focused Therapy Toolkit
A centralised collection of online resources that help patient groups progress through the research and development process, from the NIH’s National Center for Advancing Translational Sciences.
FDA guidance: natural history studies for drug development
The official FDA document outlining how to design natural history studies to support safe and effective drug development.
Ecosystem partners and inspiration
The Buffalo Initiative
An impact investment fund bridging the “Valley of Death” by empowering foundations to act as venture philanthropists, with capital timelines aligned to drug development, not venture exit pressures.
COMBINEDBrain
A consortium of 25 rare disease patient-advocacy foundations pooling resources to fast-track clinical trial readiness and biomarker identification across neurodevelopmental disorders.
Every Cure
Using AI to scan thousands of approved drugs for new uses, relying on structured registry data to find hidden cures. Backed by $48.3M from ARPA-H.
References
- Cystic Fibrosis Foundation Patient Registry — cff.org
- PPMD The Duchenne Registry — duchenneregistry.org
- Foundation for Prader-Willi Research — fpwr.org
- Cure Mito Foundation — curemito.org
- FamilieSCN2A Foundation — scn2a.org
- TFE3 Foundation — tfe3.org
- Chordoma Foundation — chordomafoundation.org
- Foundation for Sarcoidosis Research — stopsarcoidosis.org
- Children’s PSC Foundation — childrenspsc.org
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