Clinical Research

Clinical Research is a cornerstone of ERDERA’s ambition to improve rare disease diagnosis and treatment across Europe.

Clinical Research

By harnessing data, technology, and a strong network of clinical partners, ERDERA enables cutting-edge research that brings science back to the bedside—ensuring that patients and their families benefit directly from innovation.

ERDERA Clinical Research Services

ERDERA supports a pan-European Clinical Research Network (CRN) to streamline and accelerate rare disease research across borders. This ecosystem connects European Reference Networks (ERNs), national diagnostic centres, and undiagnosed disease programmes to provide shared access to harmonised data and innovative trial platforms.

Through its Clinical Research Services, ERDERA is building the infrastructure to:

Integrate and re-analyse thousands of existing genomic datasets
Enable data access and interoperability across institutions and countries
Launch coordinated studies, including N-of-1 trials and ATMP readiness
Develop patient-centric outcome measures to guide future interventions

Rare Disease Diagnostic Data Sharing

ERDERA is driving a Europe-wide effort to collate and standardise 10,000 diagnostic datasets within the first year, enabling powerful analyses and insights that support faster, more accurate diagnosis.

Data Federation & Re-analysis

By implementing distributed and federated data models, ERDERA enables secure and privacy-preserving access to clinical and genomic datasets for large-scale re-analysis and new discoveries.

Variant Interpretation & Best Practices

ERDERA is coordinating efforts across disease groups and ERNs to improve the interpretation of genetic variants. This includes developing consensus-driven methodologies and shared tools to ensure consistency and accuracy.

Equitable Access to Genomic Medicine

A key goal is to expand access to genome sequencing for rare disease patients in underrepresented countries, ensuring that all European patients can benefit from the latest diagnostic advances.

Real World Data Integration

Multiple real-world datasets will be integrated and quality-optimised to support research and care. ERDERA is also laying the legal groundwork to access relevant primary healthcare data.

Patient-Centred Outcomes

ERDERA is developing regulatory frameworks and study protocols for Patient-Centred Outcome Measures (PCOMs and PROMs), tailored to the needs of specific disease groups and therapies, including Advanced Therapy Medicinal Products (ATMPs).

N-of-few and N-of-1 Trial Platforms

Work is underway to design and launch precision trial platforms that accommodate highly personalised therapies. This includes defining criteria for patient identification, trial design, outcome measures, and treatment site capabilities.

Clinical Research