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

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Workshop 3 - Registries
"Feedback from the 15 National Conferences
on Registries"
Gabor Pogany, HUFERDIS, Hungary

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  • The EU definition on RDs is supported. It is generally agreed that one European definition is useful for facilitating cooperation and community actions. Conferences’ participants broadly expressed the necessity and expectation to (continue to) use the European definition, especially in view of laying down legally binding legislation, as required by European policy documents. Nevertheless, voices of disagreement have been recorded, in Denmark and Sweden, in particular, and in the Netherlands to a certain extent.  The swift adoption of ICD-11 classification system is unanimously supported in all countries when ready (2014), because of the specific arrangements for rare diseases envisaged therein and unfortunately lacking in ICD-10. While waiting for the release of the ICD11, it is suggested to introduce existing coding systems such as the ICD10 or the Orphanet code, possibly with cross-referring across systemsto account for and overcome the respective shortcomings of the two different systems.An early introduction of a RD-oriented coding system is necessary not only for inventorying purposes, but also to raise greater awareness and knowledge on RDs with healthcare professionals. 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, possibly by laying down the legal framework necessary, defining competences and establishing the coordination mechanisms among public institutions, administrations and/or private centres. Coordination purposes may be better achieved by creating a (nation-wide) epidemiological portal defining a minimum data set for the existing databases and registries and supporting the interoperability of their content (see, as an example, the 2nd French NP). Registries sustainability must be achieved by optimising resources and reducing fragmentation and overlapping. In each country, this should be the preliminary step, before developing or advancing any policy actions on registries. Focus should be on a better use and better information on existing knowledge and existing information sources. Government resources are generally considered indispensable by all Conferences’ participants to sustain the functioning of RD registries. National quality standards, integrity issues, privacy requirements and networking efforts with other (EU) countries were demanded by many Conferences.Specific proposals for the sustainability of registries included the preferential creation of registries which fulfil multiple purposes (from epidemiological studies to the feeding into clinical trials) and are supported by a variety of stakeholders.
  • RDs must be a priority in medical research in the country and ad hoc national research measures should be dedicated to RDs. This call creation of a specific programme for RD research emerged from all Conferences but the German one, and it was supported in countries with either a tradition of non-thematic approach to research or countries where the absence of dedicated RD funds results rather from limited resources, lack of funds or lack of political willingness.A body should be created at national level which steers and advises on RD research; develops public private partnerships with industry and associations; create close links with centres of expertise; and acts as a one-stop shop for all information on RD research and potential incubator for enterprises. RD research programmes must be supported by adequate public funds, even though public-private partnerships were not excluded. Dedicated research programmes for rare diseases would help to streamline scarce and scattered resources. National programmes should especially encourage an approach to RD research which is multidisciplinary (with more professionals involved, from different scientific backgrounds) and covers all research areas, but chiefly focus on translation research. Socio-economic research (i.e. research on quality of life, living conditions, etc.) is extremely importantand should become a national priority, not only for public health planning, but also for provision of services which help to provide an answer to the needs of patients in their daily life and empower them. This message resonated strongly in very many EUROPLAN Conferences (Italy, Denmark, Sweden, Romania, France, Spain, Germany...).
  • Qualified patients must become fully-fledged research partners.Patients association should take and should be recognised a more proactive role as research partners. For an optimal support by patient organisations, qualifying training of patient representatives and financial support to patient representatives should be ensured. Centres of Expertise must play a pivotal role in closing the gap between research andcare. Centres of Expertise are crucial for researchers and patients: good healthcare infrastructures, where patients meet, provide significant possibilities to research. Appropriate legal and financial frameworks should support this role of Centres of Expertise. For the Centres of Expertise to play this role, it is important to establish mechanisms allowing, on the one side, researchers to fully integrate with clinical services and, on the other one, clinicians to devote time to research without compromising care. At the Centre level, clinical and basic science could be connected with social and political sciences in order to optimise the provision of patient care and of those services which go beyond healthcare. The creation of quality patient registries should be a primary objective and a basic requirement to develop RD research. Quality registers for rare diagnoses are needed: structure needs to be clarified, as well as long-term funding. Clearer definitions of rules on the storage of data, the definition of quality standards to ensure trustworthiness, the development of uniform data structure and software platforms are required.  Multi-centre national and international studies are an absolute necessity for the organisation of clinical trialsto address the limited number of patients and the scarcity of expertise. This Recommendation of EUROPLAN is fully supported, with the addition that it is essential to promote clinical and preclinical testing in cooperation with the pharmaceutical industry.
  • Qualified patients must become fully-fledged research partners.Patients association should take and should be recognised a more proactive role as research partners. For an optimal support by patient organisations, qualifying training of patient representatives and financial support to patient representatives should be ensured. Centres of Expertise must play a pivotal role in closing the gap between research andcare. Centres of Expertise are crucial for researchers and patients: good healthcare infrastructures, where patients meet, provide significant possibilities to research. Appropriate legal and financial frameworks should support this role of Centres of Expertise. For the Centres of Expertise to play this role, it is important to establish mechanisms allowing, on the one side, researchers to fully integrate with clinical services and, on the other one, clinicians to devote time to research without compromising care. At the Centre level, clinical and basic science could be connected with social and political sciences in order to optimise the provision of patient care and of those services which go beyond healthcare. The creation of quality patient registries should be a primary objective and a basic requirement to develop RD research. Quality registers for rare diagnoses are needed: structure needs to be clarified, as well as long-term funding. Clearer definitions of rules on the storage of data, the definition of quality standards to ensure trustworthiness, the development of uniform data structure and software platforms are required.  Multi-centre national and international studies are an absolute necessity for the organisation of clinical trialsto address the limited number of patients and the scarcity of expertise. This Recommendation of EUROPLAN is fully supported, with the addition that it is essential to promote clinical and preclinical testing in cooperation with the pharmaceutical industry.
  • Workshop 3 - "Feedback from the 15 National Conferences on Registries"

    1. 1. Main results of the EUROPLAN NationalConferences on “Registries”<br />Gábor Pogány Ph.D.<br />RareDiseases Hungary<br />13-14 May 2011<br />
    2. 2. The EUROPLAN project<br />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)<br /><ul><li>Developing recommendations on how to define a strategic plan for rare diseases, with priority areas and actions of intervention
    3. 3. Elaborated indicators for monitoring national plans or strategies
    4. 4. Discussed the recommendations with stakeholders in the specific context of each Member State and European synthesis </li></ul> 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.<br />
    5. 5. 15 National Conferences<br />
    6. 6. 7major Areas<br />AREA 1. PLANS AND STRATEGIES IN THE FIELD<br />OF RARE DISEASES<br />AREA 2. ADEQUATE DEFINITION, CODIFICATION AND INVENTORYING<br />AREA 3. RESEARCH ON RARE DISEASES<br />AREA 4. CENTRES OF EXPERTISE AND EUROPEANREFERENCENETWORKS FOR RARE DISEASES<br />AREA 5. GATHERING THE EXPERTISE ON RARE DISEASES AT EUROPEAN LEVEL<br />AREA 6. EMPOWERMENT OF PATIENT ORGANISATIONS<br />AREA 7. SUSTAINABILITY<br />
    7. 7. Outputs<br /><ul><li>Stimulateddiscussion and reacheda consensus on theimportance of national plans / strategies
    8. 8. Promotedthe development of national plans / strategiesfor rare diseases within EU MS
    9. 9. Providedinstruments and tools (recommendations and indicators) to support Countriesinelaboration of nationalplans / strategies for rare diseases</li></li></ul><li>Reports of the EUROPLAN National Conferences<br />15 Final Conference Reports + 1 Synthesis Report. <br />Each Final Report includes:<br /><ul><li>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.
    10. 10. 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
    11. 11. 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</li></li></ul><li>What is a Patient Registry?<br /><ul><li>It is an organized system that uses observational study methods to collect uniform data (clinical and other)
    12. 12. It evaluates specified outcomes for a population defined by a particular disease, condition, or exposure, and that
    13. 13. It servesa predetermined scientific, clinical, or policy purpose</li></li></ul><li>Key Characteristics of Registries<br /><ul><li>Data are collected in a naturalistic manner
    14. 14. Registry is designed to fulfill specific purposes, and these purposes are defined in advance of collecting and analyzing the data
    15. 15. Registry captures data elements with specific and consistent data definitions
    16. 16. Data are collected in a uniform manner for every patient.
    17. 17. Data collected derive from and are reflective of the clinical status of the patient (by history, examination, laboratory test, or patient reported)
    18. 18. At least one element of registry data collection is active, meaning that some data are collected specifically for the purpose of the registry </li></li></ul><li>Typical Goals and typesof Patient Registries<br /><ul><li>Track natural history of a disease process
    19. 19. Measure or monitor safety and harm
    20. 20. Evaluate clinical, comparative or cost effectiveness
    21. 21. Measure and/or improve quality of care</li></ul>Possibletypes:<br />Device; Procedure; Clinicalevent; Disease; Group of diseases; etc. <br />
    22. 22. Features of a „good”Registry<br />Evaluationcriterias- Qualitycomponentanalysis<br /><ul><li>Research quality (scientific process)
    23. 23. Planning; design; data elements & data sources; ethics, privacy and governance
    24. 24. Evidence quality (data/findings)
    25. 25. Patients; data elements & data sources; Qualityassurance; analysis; reporting</li></li></ul><li>Research Quality<br /><ul><li>PLANNING: A written study plan documents: goals, design, study population, recruitment, data collection, human subject protection, data element, sources, & review/QualityAssurance. Feasibility is considered at the outset.
    26. 26. Plans address how data will be evaluated, incl. what comparative information, if any, will be used to support study hypotheses or objectives.
    27. 27. DESIGN: The size required to detect an effect, should it exist, or achieve a desired level of precision is acknowledged, whether or not met.
    28. 28. 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.
    29. 29. DATA ELEMENTS: Outcomes are clinically meaningful and relevant, i.e., useful to the medical community for decision-making.
    30. 30. ETHICS, PRIVACY, GOVERNANCE: Registry has received review by required oversight committees
    31. 31. COMMUNICATION PLAN for results is addressed.</li></li></ul><li>EvidenceQuality<br /><ul><li>PATIENTS: Participants are similar to the target population; attention is paid to minimize selection bias to the extent feasible.
    32. 32. For safety studies, registry personnel are trained to ask about AdverseEvents in a consistent, clear & specific manner, and know how to report.
    33. 33. DATA ELEMENTS & SOURCES: Data are reasonably complete.
    34. 34. QualityAssurance: 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.
    35. 35. ANALYSIS: Accepted analytic techniques are used; they may be augmented by new or novel approaches.
    36. 36. 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.</li></li></ul><li>Results of countriesbyindicators<br />
    37. 37. KEY MESSAGES by AREA<br />Area 2 – Definition, coding and inventorying<br />The EU definition of RDs is supported although with a few exceptions.<br />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.<br />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.<br />Patient registries sustainability must be achieved by optimising resources and reducing fragmentation and overlapping. Sustainable funding at EU level was also considered essential.<br />
    38. 38. KEY MESSAGES by AREA<br />Area 3 – Research on RDs<br />RDs must be a national priority in medical research and ad hoc national research measures should be dedicated to RDs. <br />RD research programmes must be supported primarily by adequate public funds, even though public-private partnerships were not excluded. <br />RD research should be multidisciplinary and cover all research areas, but chiefly focus on translational research.<br />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.<br />
    39. 39. KEY MESSAGES by AREA<br />Area 3 – Research on RDs<br />Qualified patients advocates must become fully-fledged research partners. <br />Centres of Expertise must play a pivotal role in closing the gap between research and care. <br />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- usingnewtechnologiesadvised.<br />Multi-centre national and international studies are an absolute necessity for the organisation of clinical trials.<br />
    40. 40. MESSAGES fromthee-HEALTHWEEK<br /><ul><li>John Dalli Commissioner for Health and Consumer Policy: Commission wants to give initiatives on patient registries
    41. 41. Commission Vice-President for digital agenda NeelieKroes: New action plan of e-Health will be worked out before the end of 2011.
    42. 42. E-Health Governance High-level Group was created.
    43. 43. Common European e-Health Interoperability Road Map (Calliope) together with recommendations will be published by the end of 2011.
    44. 44. Common European electronic ID management will be established.</li></li></ul><li>EUROPLAN<br />THANK YOU! <br />RareDiseases Hungary<br />Website: http://www.rirosz.hu<br />e-mail: pogany@rirosz.hu<br />Address: H-1082, Budapest, Üllőiút 82.<br />

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