The Vall d’Hebron Research Institute (VHIR) hosted the fourth annual meeting of the RADeep (Rare Anemia Disorders European Epidemiological Platform) Data Access Committee on 17 September. RADeep is a European platform that brings together clinical information on nearly 7,000 patients with rare anaemias from ten countries. The meeting brought together 21 representatives from nine countries and provided an opportunity to review the progress made by the registry and establish its main priorities for the next two years.

Established in 2017, RADeep is a patient registry that complies with the General Data Protection Regulation (GDPR) and integrates information from reference centres and local and national registries. Its aim is to promote research, improve epidemiological surveillance and facilitate access to treatments for people with rare anaemias in Europe, while promoting the standardised exchange of clinical data between countries.

The registry currently involves 180 hospitals, 115 of which are actively entering patient data. The platform collects information on 16 groups of rare diseases, as well as undiagnosed cases, and allows users to regularly consult epidemiological data through the dashboards available on the RADeep website.

A European network to promote research and improve data quality

The meeting was chaired by Dr María del Mar Mañú Pereira, RADeep Coordinator and Head of the Rare Anaemias Laboratory within VHIR's Childhood Cancer and Haematological Diseases Research Group. Dr Mañú Pereira is also Co-coordinator of EuroBloodNet, the European Reference Network (ERN) specialising in rare haematological diseases, and President of the 24 European Reference Networks (ERNs).

The meeting was attended by members of the Steering Committee, principal investigators and national representatives from Belgium, Spain, France, Greece, Italy, Norway, the Netherlands, Portugal and Switzerland, which joined the network this year.

The Data Access Committee is responsible for assessing third-party requests to access the registry and ensuring that the use of its information complies with ethical, legal and scientific requirements. Its meetings also provide an opportunity to share regulatory updates, review the status of the platform and strengthen collaboration among participating countries.

Progress in data quality and regulatory readiness

One of the main topics discussed was data quality and its potential to support studies for regulatory purposes. RADeep is collaborating with the European Medicines Agency (EMA) on the preparation of a joint ERN-EMA workshop, scheduled for spring 2027, focusing on updates to the guidelines for registry-based studies. Sickle cell disease will be one of the use cases illustrating how a registry can be qualified for regulatory purposes.

Furthermore, RADeep's Data Quality Framework is already approximately 80% aligned with the requirements envisaged for the future European Health Data Space (EHDS). Centres connected to the central platform exceed the minimum threshold of 75% data completeness, while an annual benchmarking survey makes it possible to assess how representative the registry is of the European patient population.

New platforms to generate scientific evidence

The meeting also featured presentations on two platforms currently under development that will expand research opportunities using RADeep data: RADiANT-CT, focused on clinical trials and regulatory studies, and RADiANT-CORE, dedicated to advanced research, including the integration of omics data, artificial intelligence and federated analyses.

One of the challenges identified is the lack of standardised criteria and appropriate outcome measures (endpoints) in some clinical trials on sickle cell disease. In this context, RADeep is exploring how real-world data can contribute to the development and validation of robust, patient-relevant outcome measures.

In terms of scientific output, the network is preparing its first manuscript on sickle cell disease. In addition, it presented three abstracts at the 2026 European Hematology Association (EHA) Congress and has submitted two abstracts to this year's American Society of Hematology (ASH) Annual Meeting.

New initiatives to strengthen the registry

Among the priorities for 2026–2027 is the revision of the Case Report Form (CRF) by five expert working groups, with the aim of completing this task before April 2027. The groups will focus on laboratory parameters, treatments, transplantation, thalassaemia and ultra-rare anaemias.

Training sessions on HGVS genetic nomenclature will also be organised for the teams responsible for data entry. At the same time, a pilot project is underway in Catalonia to collect patient-reported outcome measures (PROMs), and the automated extraction of information from electronic health records will be explored. RADeep data will serve as the reference standard for validating this methodology.

Through these initiatives, RADeep continues to move towards a more comprehensive, interoperable European registry that is better equipped to meet the needs of clinical and regulatory research. Its ultimate goal is to generate high-quality evidence that contributes to improving the diagnosis, monitoring and treatment of people living with rare anaemias.