The European registry of rare anemias RADeep currently includes over 5,000 patients registered across nine countries

On Thursday, September 18th, the 3rd annual in-person meeting of the RADeep Data Access Committee (DAC) took place at the Vall d’Hebron Research Institute (VHIR), with nearly full participation from members representing Belgium, Cyprus, Denmark, France, Greece, Italy, Norway, Spain, and the Netherlands.

RADeep (Rare Anemia Disorders European Epidemiological Platform) is a GDPR-compliant patient registry launched in 2017. It centralizes data from referral centres across multiple countries, including national and local registries, to support research, surveillance, and treatment access for rare anaemias in Europe and to enable standardized, cross-border clinical data sharing. It currently includes data on over 5,000 registered patients from 9 countries, covering 16 documented rare diseases as well as undiagnosed cases. Anemia is the most common blood disorder worldwide, affecting nearly 1 in 4 people globally, and among rare anemias, sickle cell disease (SCD) and thalassemia (THAL) are the most prevalent groups.

The RADeep DAC was established to review requests for accessing RADeep data from third parties, ensuring data use aligns with ethical, legal, and scientific standards. Its members include representatives of the Steering Committee, patients, data providers, IT and statistical experts, and legal and ethical advisors. The DAC meetings aim to review the registry status, share regulatory updates, define objectives, and foster collaborative work across the network.

The 2025 meeting, led by Dr María del Mar Mañú Pereira, Head of the Rare Anaemia Disorders Laboratory at the Childhood Cancer and Blood Disorders group at VHIR, RADeep Coordinator and Scientific Coordinator of ERN EuroBloodNet—the European Reference Network for rare hematological diseases—covered RADeep's objectives for 2025–2026, strengthening collaborations and disease-specific working groups, implementing the RADeep Data Quality Framework aligned with European Medicines Agency (EMA) recommendations, and presenting new tools available to members. A key topic was the European need for shared data quality principles essential for the European Health Data Space.

RADeep’s Data Quality Framework transforms raw data into high-value information through integrated data architecture, standardization, database structure, and dashboards. As of September 2025, RADeep has 15 collaborations and 202 healthcare providers in 14 EU countries, with 9 actively contributing data from over 5,000 patients, summarized regularly on the RADeep website epidemiological dashboards.

Participants agreed to launch disease-specific working parties focusing on Case Report Form (CRF) revision and research methodology to improve clinical trial design and evaluation in rare anemias. As an example, members discussed how many sickle cell disease trials have failed due to suboptimal endpoints and definitions and emphasized building future studies on existing pharma data while defining new clinically meaningful endpoints co-created by clinicians and patients. High-quality real-world data and patient-reported outcomes from RADeep are vital to ensure European trials evolve beyond US-driven definitions, leveraging ERN–EMA collaboration to reinforce Europe’s regulatory science leadership.

Dashboards and tools for monitoring data completeness and active patients were displayed, reinforcing RADeep's commitment to quality data for research, AI, and regulatory goals.

Teams refined the RADeep Annual Baseline and the harmonized Annual Survey to launch in early 2026, targeting rare anemia patients across the EU to demonstrate registry representativeness for EMA.

RADeep at ASH 2025

The outcomes of this meeting were showcased at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2025, Orlando, Florida. RADeep presented five abstracts highlighting its impact on rare anemia research in Europe:

• Oral presentation: “Sickle cell disease in Europe: A cross-border real-world data analysis from the RADeep registry”
• Poster: “Implementing patient-centered PROMs in sickle cell disease care: A European consensus approach”
• Poster: “Data validation and quality framework for building a European multimodal real-world dataset for clinical outcome research in sickle cell disease”
• Poster: “Patient-driven insights in sickle cell disease: Results from a global survey informing public health strategies”
• Poster: “Thalassemia in Europe: A cross-border real-world data analysis from the RADeep registry”

These presentations reflect RADeep’s ongoing commitment to advancing evidence-based care for rare anemias across Europe through high-quality real-world data.

This comprehensive update highlights RADeep’s progress, collaborations, and strategic direction towards improving research and care for rare anemia patients in Europe through robust data governance and international partnerships.