Real-World Evidence For Rare Diseases
Gain faster, more meaningful insights into how rare diseases and treatments affect real patients in everyday care.
Our Mission
To unlock the full value of clinician-reported and patient-reported real-world data in rare diseases—empowering research, improving care, and accelerating access to life-changing therapies.
We are here to make meaningful data accessible, structured, and impactful—where it matters most.
Our Vision
A future where every rare disease patient contributes to science without extra burden—through seamless data collaboration between clinics and industry.
We envision a world where real-world evidence is not an afterthought, but a foundational pillar in drug development and health system planning—especially for diseases that have been historically underserved.
Why Real-World Evidence in Rare Disease Needs a Rethink
As regulators, payers, and clinicians increasingly demand data beyond the clinical trial setting, real-world evidence (RWE) has become a cornerstone of drug development and access strategy. But in rare diseases, the data foundation is often missing or deeply flawed.
Self-reported data is unreliable
Patients with chronic or rare conditions are often unable or unmotivated to consistently report symptoms and outcomes, especially without support or incentives..
Clinician insights are underutilized
The most valuable data often lives in clinicians' hands, yet they lack the technical and financial means to structure it into registries or research-grade formats.
Fragmented and outdated registries
When rare disease registries exist, they are often siloed, incomplete, or paper-based making them unusable for modern research or regulatory needs.
A Trusted Data Hub for Rare Disease Research
Carepile bridges the gap—delivering the depth of clinic insights with the structure and security pharma requires.
Clinician-led Data Capture
We partner directly with specialist clinics to gather structured, anonymized data on rare disease patients.
- Ready-to-use templates
- Customizable data fields by indication
- Minimal burden for clinic staff
Secure, Compliant Infrastructure
GDPR-ready, research-grade data platform built for pharma use with enterprise-level security.
- Patient location insights
- Cohort identification
- CTIS integration
Where We Focus
We specialize in rare respiratory and autoimmune diseases where RWE needs are high and data is scarce..
Idiopathic Pulmonary Fibrosis (IPF)
Pulmonary Arterial Hypertension (PAH)
Systemic Sclerosis
Idiopathic Pulmonary Fibrosis (IPF)
These areas are characterized by:
- Small, dispersed patient populations
- High unmet medical need
- Active and growing drug pipelines
- Low existing data infrastructure
Help More Patients. Do Less Admin.
You’re already generating valuable insights every day—but you may not have the time, tools, or infrastructure to turn them into research-ready data. Carepile is here to change that.
A Ready-Made Registry Platform
No need to build a registry from scratch or hire IT consultants. Carepile gives your clinic:
- A pre-built, disease-specific registry tailored to your specialty
- Secure, GDPR-compliant infrastructure to protect patient privacy
- An intuitive interface that integrates into existing workflows
Revenue Sharing from Pharma Access
Unlike traditional registries, Carepile believes in fair value for contribution. When pharmaceutical companies license your data:
- Receive a share of the revenue
- Get transparent reporting on data usage
- Reinvest funds into clinic, staff, or research programs
Less Time on Research Requests
Pharma companies and academic collaborators often request data, but formatting and sending it takes time. With Carepile:
- Data is already structured, anonymized, and ready for use
- No need to respond to one-off data requests manually
- We handle compliance, formatting, and licensing
Make a Meaningful Contribution to Science
Clinicians often want to contribute to research but lack the capacity. With Carepile:
- Become part of a growing research network across Europe
- Your patients contribute to new treatment development
- Generate evidence for conditions that desperately need it
Accelerate Your Rare Disease Pipeline
Rare diseases demand smarter evidence strategies. Clinical trials are harder to run. Regulators want more context. And payers need to understand value, beyond the trial setting.
We deliver curated, clinician-reported, real-world data from underrepresented patient populations
Gain access to structured, high-integrity data:
- Plan and optimize clinical trials
- Strengthen regulatory filings (EMA, FDA)
- Support pricing and reimbursement strategies
- Customize data packages with biomarkers and longitudinal insights
Our Business Model
B2B Data Licensing
Curated datasets for regulatory and commercial use
Custom RWE Projects
Tailored data panels with deep analytics
Feasibility Services
Trial site and patient cohort identification
Security & Privacy
Built for Compliance. Designed for Trust. Patient data security and privacy are the foundation of everything we do.
What We Guarantee
Full Anonymization
No directly identifiable patient information collected
GDPR Compliance
Fully compliant with EU data protection regulations
Ethical Data Use
Strictly for research and evidence generation purposes
Clinic Control
Partner clinics retain control over data sharing
Technical Safeguards
- End-to-end Encryption (data in transit and at rest)
- Secure Cloud Hosting in EU-based, ISO 27001-certified data centers
- Role-based Access Controls (RBAC) with strict authentication
- Data Minimization: Only necessary fields collected
- Regular Security Audits & Penetration Testing
Built by experts in RWE, HEOR and Market Access
Carepile was founded by professionals with deep expertise across clinical development, real-world evidence, and rare disease strategy.
Why Now?
Accelerating Innovation
Advances in genomics, expedited regulatory pathways, and increased investment have catalyzed significant expansion in rare disease drug development.
Data Infrastructure Gap
While scientific innovation accelerates, supporting data infrastructure remains underdeveloped and fragmented, posing critical challenges.
Perfect Timing
Carepile was created to close this gap by anticipating where real-world data demand is heading and building the infrastructure to meet it.
Case Studies
Real examples of how Carepile is transforming rare disease research and patient care through structured real-world evidence.
Accelerating IPF Drug Development
A leading pharmaceutical company needed real-world evidence to support their regulatory submission for a novel IPF treatment. Traditional patient registries were incomplete and outdated.
- 6-month data collection across 12 European clinics
- 450+ patient records with longitudinal outcomes
- Regulatory submission accepted by EMA
“Carepile delivered the structured, clinician-reported data we needed in half the time of traditional registry approaches.”
— Clinical Development Director, Major Pharma
Optimizing Myasthenia Gravis Care Pathways
A network of neurology clinics wanted to understand treatment patterns and outcomes for MG patients to improve care coordination and identify best practices.
- 8 specialist clinics across 4 countries
- Real-time insights into treatment effectiveness
- 25% improvement in care pathway efficiency
“The platform transformed how we share insights across our clinic network. Patient outcomes have measurably improved.”
— Head of Neurology, European Medical Cente
Ready to Talk? Interested in Collaborating?
Whether you represent a clinic seeking to digitize and structure patient data, or a pharmaceutical company in need of high-quality real-world evidence, Carepile is ready to support you.
Email Us
Get in touch to learn more about how our tailored solutions can address your specific needs and drive meaningful results.
Carepile Key Features
Clinician-Led Data Collection
Structured, anonymized real-world data captured directly from specialist clinics
Compliant, Secure Infrastructure
GDPR-compliant platform with ISO 27001-certified hosting and encryption
Rare Disease Registry Templates
Pre-built, customizable data templates for faster onboarding
Pharma-Ready Data Exports
Clean, structured datasets for clinical trials and regulatory submissions
Trial Feasibility & Site Intelligence
Integrated tools to identify high-potential trial sites and patient clusters
Revenue Sharing Model
Fair compensation for clinics contributing valuable data
