The database network

taking it to the next level .....

Bodo Grimbacher, MD

University of Freiburg, Germany

Head of ESID patient registries

RegistryThe existing ESID Registry

Up-to-date patient- and research-databases

The ESID Main Patient Registry

The CVID Subregistry

Requirements

Time schedule

Support for the documenting centers

Summary

Prospectives

a non-profit organization established in 1983, became a society in 1994•to facilitate the exchange of ideas and information among doctors, nurses, biomedical studies,

patients and their families, collaboration with IPOPI and INGID•to establish common diagnostic criteria (together with PAGID)

(Clin. Immunol. 1999; 93:190).•to promote research on causes, mechanisms and treatment

- summer schools (education, pathogenesis, treatment of primary immunodeficiencies)- biennual scientific meetings (next: 21-24 October, 2004 in Versailles, France)

All ESID CountriesAustriaBelgiumCroatiaCzech-RepublicDenmarkEstoniaFinlandFranceGermanyGreeceHungaryIcelandIrelandIsraelItalyNetherlandsNorwayPolandPortugalRussiaSerbia-Montenegro SpainSwedenSwitzerlandTurkeyUnited-Kingdom

 

                          

                        

President: Prof. L. D. Notarangelo, Brescia, Italy; Secretary: Prof. H. Wolf, Vienna, Austria

67%

18%

7%6% 2%

Antibody deficiencies T-cell / combined IDPhagocytic disorders Complement deficienciesOther PID

Main patient registry10,717 recorded cases of Primary Immunodeficiencies in Europe

RegistryThe existing ESID Registry

Up-to-date patient- and research-databases

The ESID Main Patient Registry

The CVID Subregistry

Requirements

Time schedule

Support for the documenting centers

Summary

Prospectives

What features should a patient- and research database have?

Longitudinal Analysis of a single patient and a cohort of patients

Cross-sectional Analysis of patient cohorts

Multivariate Analysis and complex database queries

Variable programming of queries

Easy implementation of new databases via XML-programming

Data-entry via electronic Import, Scanning-Function or manually

Connecting medical centers world-wide

Accessable any time from any place

Patient-Report-Generation

Recall-Function

Security: Implementation of rules for patient‘s data protection

Access: controlled by complex User-Role-Management

USER ROLE RIGHTS

•User A

•User B

•User C

•User ...

•Administrator

•Documentator

•Monitor (QC)

•Sponsor

•Analyst

•...

•Write 1

•Write 2

•Write ...

•View 1

•View 2

•View ...

ESID database System

Concept and architecture of an internet

based ESID database networkUser-Role management

•User-Password: Handles access to the ESID-database system

XML

View Model

Controller

DB

Java2EnterpriseEdition Server

XMLInitialisierung

Client access via Standard InternetBrowser

Server

ClientClient

Thin Client-Server Architecture

OperatingSystem: Unix

3.Additional

Subregistrie

s on demand

ESID Main

Patient Registry

CVID SubregistryDGS SubregistryNBS SubregistryXLA SubregistryHIGM SubregistrySCID SubregistryNeutropenia Subregistry...Additional Subregistries

ESID Main Registry & Subregistries

2.CVID

Subregistry

1.ESID Main Registry

RegistryThe existing ESID Registry

Up-to-date patient- and research-databases

The ESID Main Patient Registry

The CVID Subregistry

Requirements

Time schedule

Support for the documenting centers

Summary

Prospectives

Core data-subset

PATIENT: Sex, date of birth, region of residence (clustered in regions with > 30 millions inhabitants)

DIAGNOSIS: Date of diagnosis, onset of symptoms, disease/diagnosis

THERAPY: Date, drug, dose per weight, dose interval, route (oral / SC / IV), from, until, side-effects, reason stopped, compliance

PHARMACO-ECON: Days in hospital, days missed at school, days missed at work

IMMUNIZATIONS: Date, vaccine, postvaccinal titres

LABORATORY: Date, time, label, value (IgG, IgA, IgM, CD3, CD4, CD8, CD19 or CD20, CD56, Leukocytes, Thrombocytes, Erythrocytes, Lymphocytes, Granulocytes, Hb, Eosinophils, Basophils, Macrophages) in percent or/and in absolute values

The ESIDMain

Registry

The ESIDMain

Registry

The ESIDMain

Registry

The ESIDMain

Registry

RegistryThe existing ESID Registry

Up-to-date patient- and research-databases

The ESID Main Patient Registry

The CVID Subregistry

Requirements

Time schedule

Support for the documenting centers

Summary

Prospectives

Datamodel: CVID database-system

The CVIDSubregistr

y

TheCVID

Subregistry

TheCVID

Subregistry

TheCVID

Subregistry

TheCVID

Subregistry

TheCVID

Subregistry

TheCVID

Subregistry

TheCVID

Subregistry

TheCVID

Subregistry

TheCVID

Subregistry

TheCVID

Subregistry

RegistryThe existing ESID Registry

Up-to-date patient- and research-databases

The ESID Main Patient Registry

The CVID Subregistry

Requirements

Time schedule

Support for the documenting centers

Summary

Prospectives

Positive statement of data protection officer and/or ethics board

Signed consent form from patients

Database models

Requirements

Data protection approval: source document

Ethics vote: source document

Positive statement of data protection officer and/or ethics board

Signed consent form from patients

Database models

Requirements

Statement of consent for the maintenance of patient data

on an internet database for research (Informed Consent) The Department of Rheumatology and Clinical Immunology of the University Hospital Freiburg participates in the research project “ESID OnlinePatient- and Research-Registry”. This is a pseudo-anonymous internet database with password-protected access. Pseudo-anonymity means thatthe data obtained from the database are anonymous for the receiver i.e. the confidentiality of records identifying the subject will be maintained. In amedical emergency the treating doctors could retrieve a specific patient’s data using a highly protected encryption code. Patient data relating to amedical condition are saved and stored automatically without the patients personal data.

The aim of this project is to compile clinical and laboratory data in order to provide improved diagnosis, classification, prognosis and therapy for thepatient.

The aim of the database is a continuous long-term compilation of data on PID (Primary Immunodeficiency disease) patients.

Therefore, the patient data obtained in the course of their treatment is not deleted but instead stored in a pseudo-anonymous and coded form in apassword-protected internet database. These pseudo-anonymous data can be made available to centers that specialize in treating PID (PrimaryImmunodeficiency disease) patients, to laboratories that are researching the cause of PID (Primary Immunodeficiency diseases), to pharmaceuticalcompanies developing or improving medication, or to epidemiologists for evaluation.

Participation in this research project is voluntary. Refusal to participate will involve no penalty or loss of benefits to the subject, and the subject maydiscontinue participation at any time without penalty or loss of benefits.

I give my consent that confidential data relating to my PID (Primary Immunodeficiency disease), obtained in the course of the research project“ESID Online Patient- and Research-Registry”, can be compiled and evaluated as described above.

Date: Patient’s name and DOB:

Name of doctor involved:

Doctor’s Signature Patient’s Signature

Consent form for patients

Positive statement of data protection officer and/or ethics board

Signed consent form from patients

Database models

Requirements

PID Classification & Steering committees

RegistryThe existing ESID Registry

Up-to-date patient- and research-databases

The ESID Main Patient Registry

The CVID Subregistry

Requirements

Time schedule

Support for the documenting centers

Summary

Prospectives

Time schedule

Sept. 1st 2003 Start of projectJan. 27th, 2004 CVID-datamodel submittedJan. 29th, 2004 NBS-datamodel submittedFeb. 2nd, 2004 DGS-datamodel submittedFeb. 19th, 2004 osteopetrosis-datamodel submittedMarch 8th, 2004 The beta-version goes onlineMay 15th, 2004 End of test drive

June 15th, 2004 The ESID-registry will go onlinewith the ESID-main-registry and the CVID subregistry

October 1st, 2004 additional 3 subregistries implementedfrom there on: additional 4 subregistries per year

RegistryThe existing ESID Registry

Up-to-date patient- and research-databases

The ESID Main Patient Registry

The CVID Subregistry

Requirements

Time schedule

Support for the documenting centers

Summary

Prospectives

The ESID registry working party has made major steps towards an ”ESID Online Patient- and Research-Registry”.

We are about to develop a multi-centred data collection system in European and Non-European countries for patients with Primary Immunodeficiencies. The data collected in the participating centres shall be pseudonymized and thereafter be entered and made accessible in a central online registry.The registry will consist of multiple 'disease-specific subregistries'.

Data from e.g. national registries will be automatically transferrable to eliminate the need of a second registration. The system will enable the user to enter any data at any time for any visit. We are asking for yearly updates of the records. With PPTA (Plasma Protein Therapeutics Association), the major financial sponsor of this project, we are about to arrange a financial compensation for the use of secretarial help for the documentation.

For this project, ESID is asking for support from all physicians and researchers involved in the diagnosis, care, treatment, and support of patients with PID.

The bonus system is set up as follows:

ESID suggests to compensate for secretarial assistence with 5 per documented patient for whom a core dataset �(red fields) has been documented and 10 for each �documented patient for whom an extended dataset (blue fields) has been documented.

In addition, there shall be an annual award for the five best publications of the five disciplines (Antibody deficiencies, T-cell or combined deficiencies, Phagozytic disorders, Complement disorders, Other PIDs) within PID using data of the ESID registry of about EUR 2.000,00 per publication and an extra award of EUR 5.000,00 for the best publication over all.

Furthermore, a center which has reached a specific number of documented patients shall be rewarded by travel grants to either the ESID summer school or the biannual ESID scientific meetings. Free registration to the meetings is also beeing discussed.

Each center may install the database locally to be able to work with personalized data, since the application will be able to generate patient reports to be sent e.g. to the home-physician. In addition, the database will support features like 'recall functions' which will remember the physician if a patient has not shown up for his/her regular follow-up visit.

There will be a patient consent to be signed to ensure that the patient supports the initiative. It will be the centers' responsibility to obtain the patient consent. However, all PID patient organizations so far supported this project since it is designed to increase awareness and diagnosis of PID and to facilitate research in the field.

We are prepared to provide you with all documents you will need for patients consents and ethics approvals.

Thank you for your collaboration!

By now about 150

confirmations of intended

colla- boration by

single documentation

centers

Letter to the ESID members

Bonus system:

Compensation for secretarial assistance (EUR 10 per patient per year)

Annual award for the best publications within PID using data of the ESID Registry

Travel grants to ESID meetings and summer school

Possibility to work with personalized data plus recall-functions

RegistryThe existing ESID Registry

Up-to-date patient- and research-databases

The ESID Main Patient Registry

The CVID Subregistry

Requirements

Time schedule

Support for the documenting centers

Summary

Prospectives

Industry

• access to a core

dataset

• additional data for

post licensing

surveillance

• data for marketing

• advertisement,

publicity

• close contact to a

world-wide network of

high-profile research

institutions

Patients

• optimize research

• implement standards

for diagnosis and

therapy

• improve quality of life

• meet the patient‘s

needs

• support the spirit of

ESID

Physicians

• research across

centers using a huge

database

• enable genetic, and

therapeutic trials

within ESID

• secretarial assisitance

• automated patient

reports

Why support the ESID-online registry?

RegistryThe existing ESID Registry

Up-to-date patient- and research-databases

The ESID Main Patient Registry

The CVID Subregistry

Requirements

Time schedule

Support for the documenting centers

Summary

Prospectives for participating countries

Decision 1:

Do I need a signed consent form for the registry in my country? Do I

need a vote from my ethical review board?

Decision 2:

Do I wish to have a personalized version of the ESID registry as a

national registry, running behind the the firewall of my university?

Question 1:

What do I need from ESID to facilitate the registration of my patients?

Does my center have the technical requirements for runnig the

ESID database?

Question 2:Can I improve the ESID registry?

Prospectives

The online

patient and research

database network

Bodo Grimbacher MD; Dominic Veit MA; Barbara Frisch MA

University of Freiburg, Germany

ESID patient registriesgrimbacher@medizin.ukl.uni-freiburg.de frisch@medizin.ukl.uni-freiburg.deveit@medizin.ukl.uni-freiburg.de

Concept and architecture of an internet

based ESID database networkSecurity and Dataprotection

Input requires a positive statement from the local data protection officer and a signed consent from the patient(s) or legal tutor (approved form provided in 6 languages)

• SSL encrypted data transmission

• Secure Server Net (audited Firewall of the Hospital of Freiburg)

• User-Role Authentication

• Passwords are stored in encrypted form

• Multiple-User-Roles restrict views and presented data

• Patient randomization (patient ID’s)

• Only coded data allowed (ONLY the patient’s physician can match a specific data-set with his patients)

• No demographical data or ‘free text’ that potentially directly identifies the patient is stored in the system

• Meet the standards set by FDA CFR 21 Part 11 for Good Clinical Practice and ISO 9001 for Quality Control

Concept and architecture of an internet

based ESID database networkThe network structure

ESID registry:Disease-specific

subregistries

Ab

Local patient registry

a

B ...

National patient registries

Centers

AIEOPCINECA

Italy

National DB

Local physiciansHospitals/INGID

ESID mutation databases

IPOPI

Existing registries/databases in:

Italy 04/03 Spain 09/03 Poland 10/03 Czech 11/03 Paris 12/03 Hannover 02/04 Munich 03/04 Ulm Belgium Hungary The Netherlands Portugal Sweden ...