I have no personal or financial interests to declare

I have no financial support from an industry source at the current presentation.

대한혈액학회 Korean Society of Hematology

COI disclosureName of author ： María del Mar Mañú Pereira

The Rare Anaemia Disorders European Epidemiological Platform

www.eurobloodnet.eu/radeep

Dr. María del Mar Mañú PereiraERN-EuroBloodNet Scientific DirectorUniversity Hospital Vall d’HebrónBarcelona - Spain

What is ERN-EuroBloodNet?

Member Sate nº HCPBelgium 5

Bulgaria 2

Cyprus 1

Czech Republic 1

Germany 4

Spain 1

France 12

Ireland 1

Italy 21

Lithuania 1

The Netherlands 6

Poland 1

Portugal 3

Sweden 1

United Kingdom 6

Members 66

Co-funded by the Health Programme of the European Union

ERN-EuroBloodNetresults from a joint effort of many pieces

ERN-EuroBloodNet is a collaborative network of 66 healthcare providers (HCPs) in 15 MS that brings together individuals and institutions committed to improving healthcare services in Rare Hematological Diseases

Oncological diseases HubCoordinated by Prof P. FenauxAP-HP, Hôpital St Louis, Paris

Non -oncological diseases HubCoordinated by Prof B. GulbisCUB-Hôpital Erasme, Brussels

•Myeloid malignancies•Lymphoid malignancies

• Rare red blood cell defects • Bone marrow failures and rare haematopoietic disorders• Rare bleeding-coagulation disorders and related diseases• Haemochromatosis and rare hereditary iron metabolism disorders

Oncological diseases coordinators:

• Myeloid malignancies

23 HCPs from 9 MS

P. Fenaux (France)

U. Platzbecker (Germany)

S. Wintrich (UK) - ePAG

• Lymphoid malignancies

22 HCPs from 10 MS

A. Engert (Germany)

C. Thieblemont (France)

P. Aumont (France) -ePAG

Non-Oncological diseases:

• Rare Red blood cell defects

36 HCPs from 12 MS

B. Gulbis (Belgium)

N. Cappellini (Italy)

L. Brunetta (Italy) - ePAG

• Bone marrow failures

20 HCPs from 8 MS

A. Iolascon (Italy)

R. Peffault (France)

M. Piggin (UK)

• Rare bleeding-coagulation disorders

35 HCPs from 10 MS

M. Makris (UK)

F. Peyvandi (Italy)

A. Bok (UK) - ePAG

• HH and and hereditary iron metabolism disorders

15 HCPs from 6 MS

G. Porto (Portugal)

D. Swinkels (The Netherlands)

ERN-EuroBloodNet – Disease coverage and coordinators

Objectives and Transversal Fields of Action (TFAs)

Improvement in clinical patient management

Improve equal access to highly specialized healthcare delivery for RHD

Promote best practices in prevention, diagnosis and safe clinical care

Disseminate cutting-edge knowledge and facilitate continuing medical education in the field of RHD

Provide inter-professional consultation by sharing of expertise and safe exchange of clinical information

Foster European cooperation in highly specialized procedures for diagnosis, innovative treatments and research

Objectives and Transversal Fields of Action (TFAs)

Improvement in clinical patient management

Cross-border health

Best practices

Clinical trials and research

Continuing medicaleducation

Telemedicine

Objectives and Transversal Fields of Action (TFAs)

Improvement in clinical patient management

Cross-border health

Best practices

Clinical trials and research

Continuing medicaleducation

Mapping of services for best clinical care

Availability of Highly specialized procedures

Model for cross border referral system

Assessment of guidelines

Task force on TaxonomyEQAS

Public database of reliable guidelines

Identification of educational gaps, with EHA and ESH

Short fellowships of health professionals

Telemedicine

Clinical Patient Management System

Mapping of CTs

Sponsoring of CTs

Systematic retrieval for international ca

European epidemiological surveillance

RADeep, the Rare Anaemia Disorders European Epidemiological Platform, is a joint venture conceived in the core of ERN-EuroBloodNet, as an umbrella for both new and already existing European patients’ registries in rare anaemias

Why RADeep?

RADeep’s Principle is to maximize public benefit from data on RAs opened-up through the platform with the only restriction needed to guarantee patient’s rights and confidentiality in agreement with EU regulations for cross-border sharing of personal data

RADeep general objective is to engage clinical research to improve the delivery of best healthcare

For patients with RAs, to map at the European levelo Demographyo Survival rateo Diagnosis methodso Main clinical manifestationso Treatments

Benefits

possibility to share and pool data, essential for RAsreach critical numbers

perform studies, researchknowledge generation (evidence)

better healthcare for RAs patients

Why RADeep?

Coordination and Steering Committee: Policy and Data Access committee Data quality and interoperability Harmonization including Common Data Set Technical platform and safeguards for secure Exchange of data International collaborations and long-term sustainability

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Disease specific Scientific Committee:- Deployment of disease specific RADeep arm- Address disease specific needs- Expansion of RA - Common Data Set- Gathering of Data- Clinical and Policy reports

Implementation of RADeep

Registries

Medical centres

Patients

Providers of data

Users of data

Researchers

Medical centres

Patients

Industry

Policy makersRADeep is built in line with the EC-Joint Research Centre European Platform on RD registration

https://eu-rd-platform.jrc.ec.europa.eu/

Implementation of RADeep

Data Access Committee

Pooling of expertsInvolvement of stakeholders

Outreach of the Registry: target groupsInternational collaborations & sustainability

RADeep in the context of the ERNs and the EC Program for RDs

Point of information

RADeep in the context of the ERNs and the EC Program for RDs

RADeep in the context of the ERNs and the EC Program for RDs

New General Data Protection RegulationPersonal Data:

• Data containing any identifiers that make possible to find out who the subjects are, including codes orpseudonyms.

• Data and/or factors that in tandem allow the re-identification of data subject, although by indirect means.

Sharing of Personal Data (for both clinical practice and research):• Informed consent is required

• Have to be done with the appropriate safeguards ----- pseudonymisation tool

RADeep in the context of the ERNs and the EC Program for RDs

RD-Registries using EUPID are supported in being GDPR-compliant

Why EUPID?

EUPID has been designed to facilitate secondary use of datasets in Biomedical Research and Healthcare by addressing the following major requirements:o prevent duplicate registration of patients

o avoid creating a transparent universal patient ID but

o provide distinct pseudonyms for patients in different contexts

o keep a protected link between the different pseudonyms in the background

o which supports creating merged, datasets for secondary use

o preserve the possibility for re-identification by a trusted third party

RADeep in the context of the ERNs and the EC Program for RDs

Data provider: Medical centre or national registryGenerates the pseudonymKeep locally the link betweenpseudonym and personal data

Trusty Third Party

RADeep in the context of the ERNs and the EC Program for RDs

Pseudonym

RA - Diseasespecific Data Set

RA-Common Data Set92 items

38 Mandatory51 Optional

RD-CommonData Set16 items

RADeep specific objectives and Common Data Set

RA – Rare Anaemias

RD – Rare Diseases

Primary objective

Assessment of prevalence and incidence of RAs in Europe:

o What is the prevalence/incidence of the disease?

o Is it changing over time?

o Where are the patients?

Secondary objectives

Assessment of survival

Main clinical manifestations

Treatments

o How severe is the disease?

RADeep specific objectives and Common Data Set

• Information about the status of the patient and geographical location for

prevalence studies and survival (Date of birth, gender, patient status, country,

medical centre)

RA-CDS based on:o EU-RD Platform / French RD Registry

o Belgium SCD Registry / French THAL & PKD Registry / Spanish SCD &THAL Registry / Italian initiatives for THAL and CDA / German

registry for SCD / American registry for SCD/ UK Registry for SCD and THAL

o 92 ítems - 38 Mandatory – 23 to be updated

- 51 Optional – 38 to be updated

- 3 automatically generated

RADeep specific objectives and Common Data Set

• Information to appreciate methods used for diagnosis and to calculate

diagnosis delay and diagnosis wavering (Clinical and genetic diagnosis, onset, first symptoms)

• Hematological parameters

• Serological data: Rate of infections

• Clinical manifestations (Neonatal manifestations, Iron overload, cholelithiasis, splenomegaly, heart disease, endocrine complications)

• Treatments (Blood transfusion requirement and annual transfusion information; chelation; splenectomy; HSTC; use of specific

treatment)

• Assessment of inclusion in CT protocols

Data provider: Medical centre or national registryGenerates the pseudonymKeep locally the link betweenpseudonym and personal data

TTP

Pseudonym

RADeep in the context of the ERNs and the EC Program for RDs

Data provider: Medical centre or national registryGenerates the pseudonymKeep locally the link betweenpseudonym and personal data

TTP

Pseudonym

Aggregated DataNo personal Data

RADeep in the context of the ERNs and the EC Program for RDs

Web-based RADeep platform – mySQLServer in Cyprus – Data storage in Cyprus

Entering data in RADeep

- Manually entering of data in RADeep

o Data Managers in Medical centres with high number of patients

- Users / Accounts password protected

o Define levels of access (read, edit, specific items..)

Extracting data from RADeep

- A set of predefined “Queries” implemented by RADeep (no limit)

- Export data in:

- Excel

- Multiple CSV files

- Import multiple CSV files in a local Data Base (DB)

- RADeep guidance for constructing the local DB “in line” ----- Server at the national level

Data provider: Medical centre

RADeep - IT Platform solution

Web-based RADeep platform – mySQLServer in Cyprus – Data storage in Cyprus

Entering data in RADeep

- Manually entering of data in RADeep

o Data Managers in Medical centres with high number of patients

- Users / Accounts password protected

o Define levels of access (read, edit, specific items..)

Extracting data from RADeep

- A set of predefined “Queries” implemented by RADeep (no limit)

- Export data in:

- Excel

- Multiple CSV files

- Import multiple CSV files in a local Data Base (DB)

- RADeep guidance for constructing the local DB “in line” ----- Server at the national level

Data provider: Medical centre

Supporting a) the existing registries becoming interoperable and b) the setting up of new registries

RADeep - IT Platform solution

Web-based RADeep platform – mySQLServer in Cyprus – Data storage in Cyprus

Entering data in RADeep

- Manually entering of data in RADeep

o Data Managers in Medical centres with high number of patients

- Users / Accounts password protected

o Define levels of access (read, edit, specific items..)

- Pseudonym

o Personal Data only at the local level

Extracting data from RADeep

- A set of predefined “Queries” implemented by RADeep (no limit)

- Permission to access National individual Data – Agreement required

- Export data in:

- Excel

- Multiple CSV files

- Import multiple CSV files in a local Data Base (DB)

- RADeep guidance for constructing the local DB “in line” ----- Server at the national level

National Registry (New registries or without specific software in place)

Supporting a) the existing registries becoming interoperable and b) the setting up of new registries

RADeep - IT Platform solution

Web-based RADeep platform – mySQLServer in Cyprus – Data storage in Cyprus

Entering data in RADeep

- Individual Data

- Manually entered by Data managers or Semi-automatically transfer of data (need to analyse case

per case)

Extracting data from RADeep

- A set of predefined “Queries” implemented by RADeep (no limit)

- Export data in:

- Excel

- Multiple CSV files

- Import multiple CSV files in a local Data Base (DB)

- RADeep guidance for constructing the local DB “in line” ----- Server at the national level

National Registry (With specific software in place)

Supporting a) the existing registries becoming interoperable

RADeep - IT Platform solution

RADeep - Policy

Challenge:

RADeep dissemination and long-term sustainability

Action plan:

Involvement of many parties• Support the development of National / Regional / Local registries• Proposal to help gathering the data (data managers)• Proposal to link existing registries to RADeep

RADeep is partially co-financed by pharmaceutical companies HOWEVERindustry will not have access to personal data allowing patients’identification, but only to pseudoanonymous and/or aggregated(statistical) data, following the same established policy for the transfer ofdata to other third parties.

Property remains ALWAYS in RADeep members (coordinators and dataproviders) regulated by specific legal contracts

RADeep - Policy

1. RADeep answers to the EC request to ERNs for working on EU Platforms for registry of patients affected

by RDs

2. RADeep Policy is established in line with EU-RD Platform and is GDPR-compliant

3. Providers of information and users of information are identified and transparent rules for data sharing

have been developed

4. Pseudonymisation constitutes an appropiate measure for achieving data protection through Technology

5. Comprehensive common data set has been established based on real examples of existint registries and

in agreement with RADeep’s primary and secondary objectives

6. RADeep offers a technical solution with appropiate safeguards GDPR-compliant for:

o Development of European registries i.e. PKD, CDA, HSt

o Support the integration of existing National registries i.e. SCD, THAL and the creation of new ones

by giving guidance

Messages

Work together is better than alone