Workshop 3 - "Feedback from the 15 National Conferences on Registries"

Main results of the EUROPLAN National Conferences on

“Registries”

Gábor Pogány Ph.D.

Rare Diseases Hungary

13-14 May 2011

EURORDIS Annual Membership MeetingAmsterdam 13-14 May 2011

The EUROPLAN project

Developing recommendations on how to define a strategic plan for rare diseases, with priority areas and actions of intervention

Elaborated indicators for monitoring national plans or strategies

Discussed the recommendations with stakeholders in the specific context of each Member State and European synthesis

EURORDIS supported the organisation of 15 National Conferences on Promoting National Plans or Strategies for Rare Diseases based on the Commission Communication , the Council Recommendations and EUROPLAN Recommendations.

EUROPLAN is a three-year project co-funded by the EU Commission for the development of National Plans within the Program of Community

action in the field of Public Health (2003 - 2008)


15 National Conferences


7 major Areas

AREA 1. PLANS AND STRATEGIES IN THE FIELD OF RARE DISEASES

AREA 2. ADEQUATE DEFINITION, CODIFICATION AND INVENTORYING

AREA 3. RESEARCH ON RARE DISEASESAREA 4. CENTRES OF EXPERTISE AND EUROPEAN REFERENCE

NETWORKS FOR RARE DISEASESAREA 5. GATHERING THE EXPERTISE ON RARE DISEASES AT

EUROPEAN LEVELAREA 6. EMPOWERMENT OF PATIENT ORGANISATIONSAREA 7. SUSTAINABILITY


Outputs

• Stimulated discussion and reached a consensus on the importance of national plans / strategies

• Promoted the development of national plans / strategies for rare diseases within EU MS

• Provided instruments and tools (recommendations and indicators) to support Countries in elaboration of national plans / strategies for rare diseases


Reports of the EUROPLAN National Conferences

15 Final Conference Reports + 1 Synthesis Report.

Each Final Report includes: An overall assessment of the usefulness of the EUROPLAN

Recommendations for the advancement of a national strategy in the country and the transferability of the EUROPLAN Recommendations in your country.

Report of the discussion and outcomes of the thematic Working Groups - important conclusions on each theme have been delivered by the 15 Conferences including on Registries

The National and the Synthesis Reports are available on: http://www.eurordis.org/content/europlan-guidance-national-plans-and-conferences#EUROPLAN National Conference Final Reports

http://www.eurordis.org/content/europlan-guidance-national-plans-and-conferences#EUROPLAN%20%20National%20Conference%20Final%20Reports

http://www.eurordis.org/content/europlan-guidance-national-plans-and-conferences#EUROPLAN%20%20National%20Conference%20Final%20Reports


What is a Patient Registry?

• It is an organized system that uses observational study methods to collect uniform data (clinical and other)

• It evaluates specified outcomes for a population defined by a particular disease, condition, or exposure, and that

• It serves a predetermined scientific, clinical, or policy purpose


Key Characteristics of Registries

• Data are collected in a naturalistic manner • Registry is designed to fulfill specific purposes, and these purposes

are defined in advance of collecting and analyzing the data • Registry captures data elements with specific and consistent data

definitions • Data are collected in a uniform manner for every patient. • Data collected derive from and are reflective of the clinical status of

the patient (by history, examination, laboratory test, or patient reported)

• At least one element of registry data collection is active, meaning that some data are collected specifically for the purpose of the registry


Typical Goals and types of Patient Registries

• Track natural history of a disease process • Measure or monitor safety and harm• Evaluate clinical, comparative or cost

effectiveness• Measure and/or improve quality of care

Possible types:

Device; Procedure; Clinical event; Disease; Group of diseases; etc.


Features of a „good” Registry

• Research quality (scientific process)• Planning; design; data elements & data

sources; ethics, privacy and governance• Evidence quality (data/findings)

• Patients; data elements & data sources; Quality assurance ; analysis; reporting

Evaluation criterias - Quality component analysis


Research Quality• PLANNING: A written study plan documents: goals, design, study population,

recruitment, data collection, human subject protection, data element, sources, & review/Quality Assurance. Feasibility is considered at the outset.

• Plans address how data will be evaluated, incl. what comparative information, if any, will be used to support study hypotheses or objectives.

• DESIGN: The size required to detect an effect, should it exist, or achieve a desired level of precision is acknowledged, whether or not met.

• Follow-up time needed to detect events of interest is acknowledged, whether or not feasible to achieve. To the extent feasible, follow-up time is adequate to address the main objective.

• DATA ELEMENTS: Outcomes are clinically meaningful and relevant, i.e., useful to the medical community for decision-making.

• ETHICS, PRIVACY, GOVERNANCE: Registry has received review by required oversight committees

• COMMUNICATION PLAN for results is addressed.


Evidence Quality

• PATIENTS: Participants are similar to the target population; attention is paid to minimize selection bias to the extent feasible.

• For safety studies, registry personnel are trained to ask about Adverse Events in a consistent, clear & specific manner, and know how to report.

• DATA ELEMENTS & SOURCES: Data are reasonably complete.• Quality Assurance: Reasonable efforts have been expended to assure that

appropriate patients have been systematically enrolled and followed in as unbiased a manner as possible; reasonable efforts have been devoted to minimize losses to follow-up. Data are checked using range and consistency checks.

• ANALYSIS: Accepted analytic techniques are used; they may be augmented by new or novel approaches.

• REPORTING: Results are reported for all main objectives; follow-up time is described so readers can assess its impact on conclusions drawn; report clearly states any conclusions drawn and implications of results, as appropriate.


Results of countries by indicators

Country Is there official RD registry?

Registering activity Number of diseasesincluded

BG No Multiple RD registries, not Standardised More than 20

HR No Multiple RD registries, not Standardised Some

DK No Multiple RD registries, Standardised Several

F Yes Centralized RD registries Around 130

D No Multiple RD registries, Standardised Around 80

GR No No registry at all 0

H No Multiple RD registries, not Standardised 26

IRL No Multiple RD registries, not Standardised Some

I Yes Centralized RD registries Around 130

NL No Multiple RD registries, not Standardised (?) Some

PL No Multiple RD registries, not Standardised Some

RO No Multiple RD registries, not Standardised 6

E No Multiple RD registries, Standardised Several

S Yes Multiple RD registries, Standardised Several

GB No Multiple RD registries, Standardised Several


KEY MESSAGES by AREA

1. The EU definition of RDs is supported although with a few exceptions.

2. While awaiting ICD-11, it is suggested to introduce existing coding systems such as the ICD10 or the Orphanet code, possibly with cross-referring systems.

3. Day-to-day registration of rare diseases should be organised in such a way that the workflow, as far as possible, is integrated into existing administrative systems.

4. Patient registries sustainability must be achieved by optimising resources and reducing fragmentation and overlapping. Sustainable funding at EU level was also considered essential.

Area 2 – Definition, coding and inventorying



1. RDs must be a national priority in medical research and ad hoc national research measures should be dedicated to RDs.

2. RD research programmes must be supported primarily by adequate public funds, even though public-private partnerships were not excluded.

3. RD research should be multidisciplinary and cover all research areas, but chiefly focus on translational research.

4. Socio-economic research (i.e. research on quality of life, living conditions, etc.) was highlighted in most National Conferences as extremely important and should become a national priority.

Area 3 – Research on RDs



5. Qualified patients advocates must become fully-fledged research partners.

6. Centres of Expertise must play a pivotal role in closing the gap between research and care.

7. All National Conferences called for the creation of quality patient registries as a primary objective and a basic requirement in the development of RD research - using new technologies advised.

8. Multi-centre national and international studies are an absolute necessity for the organisation of clinical trials.

Area 3 – Research on RDs


MESSAGES from the e-HEALTH WEEK

John Dalli Commissioner for Health and Consumer Policy: Commission wants to give initiatives on patient registries

Commission Vice-President for digital agenda Neelie Kroes: New action plan of e-Health will be worked out before the end of 2011.

E-Health Governance High-level Group was created. Common European e-Health Interoperability Road Map (Calliope)

together with recommendations will be published by the end of 2011.

Common European electronic ID management will be established.


EUROPLAN

THANK YOU!

Rare Diseases HungaryWebsite: http://www.rirosz.hue-mail: [email protected]: H-1082, Budapest, Üllői út 82.

mailto:[email protected]





Workshop 3 - "Feedback from the 15 National Conferences on Registries"

Documents

europlan recommendations

national strategy

rare diseasesreports

rare diseasesbased

patient registry

uniform data clinical

fieldof rare diseasesarea

council recommendations