Clinicians and patients needs and expectations from registries

Transcription

1 Clinicians and patients needs and expectations from registries Luciano Vittozzi National Centre Rare Diseases National Institute for Health Rome Italy EPIRARE is a project co-funded by the European Commission (Grant n ) Web address:

2 EPIRARE: focus on content and use «Building Consensus and Synergies for the European Registration of Rare Disease Patients» Needs of the stakeholders (registry holders, clinicians, patients, industry, health authorities) Agreement on the Platform Scope, Governance and long-term sustainability Agreement on a Common data set, disease-specific data collection and data quality assurance Tools and other facilities supporting the operation of the platform users Key issues for a legal basis for data sharing

3 EPIRARE: focus on content and use «Building Consensus and Synergies for the European Registration of Rare Disease Patients» Needs of the stakeholders (registry holders, clinicians, patients, industry, health authorities) Agreement on the Platform Scope, Governance and long-term sustainability Agreement on a Common data set, disease-specific data collection and data quality assurance Tools and other facilities supporting the operation of the platform users Key issues for a legal basis for data sharing

4 The evidence base A few registry holders as expert advisors Survey of registries conditions and needs Survey of collected variables, definitions and formats Survey of patients expectations Consultation/survey of industry expectations Consultation of EMA experts Consultation/Information of national policy-makers Consultation of payer associations experts Review of policy actions and funding initiatives Liaison with US ORDR and RD-Connect (IRDiRC) Participation in EU initiatives and RD Committees

5 The survey of registries The survey scope Aims and scope of existing RD registries Sources, data collected, methodology and quality issues Data protection and ethical issues Governance, communication and data sharing Financial sustainability Needs, expectations, opinions on the platform Valid Respondents 220 registries

6 Overall registry needs Answer options Responses (%) Gather financial support 119 (59.8) Motivate data providers 104 (52.3) Assess the quality of your data 92 (46.2) Communicate and disseminate your results 92 (46.2) Recruit new data providers 74 (37.2) Link your register with other registers 72 (36.2) Widen the geographical coverage of the register 51 (25.6) Link your register with biobanks and bioinformatics 49 (24.6) Strengthen the relation with patient associations 40 (20.1) Gather technical help to refurbish your IT system 33 (16.6) Improve training of the register s staff 26 (13.1) Improve the data protection/security system 25 (12.6) Establish a more robust system of governance 23 (11.6)

7 Registry needs for tools Answer options Responses(%) IT tools (e.g. database software and secure 135 (68.9) data exchange) Tools for networking among partners and 112 (57.1) registries Quality control systems, quality experts 111 (56.6) advice, etc. Legal advice 94 (48.0) Model documents (e.g. informed consent 92 (46.9) form) Facilitated access to useful data sources 87 (44.4) Expert technical advice 76 (38.8) Others 6 (3.1)

8 Divergent interests (answers in order of frequency) EPIRARE WP8 survey EPIRARE WP5 survey Current registries aims Patient expectations 1. Epidemiological research 1. Healthcare and social service planning 2. Natural history 2. Treatment evaluation 3. Patient recruitment 3. Patient recruitment 4. Disease surveillance 4. Natural history 5. Genotype-phenotype correlations 5. Epidemiological research 6. Treatment evaluation 6. Genetics 7. Healthcare service and social planning 7. Disease surveillance

9 Agreed tools and services Essential to promote voluntary participation of registries in the platform Start up support: advice on data protection, ethics and ethical approval, quality control, standards and nomenclature common resources (Patient information form, informed consent procedures; data access policies) software applications for secure data collection data quality management criteria and procedures Operational support: access to useful external resources networking among experts, centres and patients reports; case studies; Best practice and Guidelines for clinical management

10 European platform outputs the added value Stable, basic, regular, multi-purpose and multi-disease reporting activity Long-term sustainability of data collection Higher representativeness of estimates (wider population coverage) New information, not in the reach of individual registries/centres Possibly, Multiple data sources to increase accuracy, to reduce sample biases and to estimate underreporting Assessments and comparisons across diseases Comparisons of performances (benchmarks) of care and services Quality control and validation

11 EPIRARE Deliverables and publications EPIRARE Reports and publications visit: Publications Taruscio et al: The current situation and needs of rare disease registries in Europe. Pub. Health Genomics 2013;16: Vittozzi et al: A Model for the European Platform for Rare Disease Registries. Pub. Health Genomics 2013;16: Taruscio et al: National registries of rare diseases in europe: an overview of the current situation and experiences. Public Health Genomics. 2015;18(1):20-5. Taruscio et al: The EPIRARE proposal of a set of indicators and common data elements for the European platform for rare disease registration. Arch Public Health. 2014;72(1):35. Santoro et al: Rare Disease Registries Classification and Characterization: A Data Mining Approach. Public Health Genomics Feb 7. [Epub ahead of print]

12 Thank you for your attention! Luciano Vittozzi

13 Registry needs (answers in order of frequency) Overall Needs 1. Financial support 2. Motivate data providers 3. Assess data quality 4. Dissemination of results 5. Update data collection form 6. Recruit further data providers 7. Link with other registries 8. Widen the geographical coverage of the registry 9. Link with biobanks and bioinformatics 10. Strengthen the relations with patients associations

14 Registry needs (answers in order of frequency) Needs for tools 1. Database application and secure data exchange 2. Tools for networking among partners and among registries 3. Quality control systems, advice on data quality 4. Legal advice 5. Model documents (e.g. informed consent forms) 6. Facilitated access to useful data sources (e.g. death registries)

15 The Identified Platform Outputs Indicators ( policy-makers, industry, patients) Indicators of relevance for Rare Diseases selected by the RDTF Indicators of RD policy implementation (EUROPLAN) Information for RD policies and services planning ( policymakers) Monitoring RD-dedicated health services and integration in the NHS Monitoring use of OD and other treatments/procedures/devices Information for HTA ( payers, patients) Effectiveness of OD and other treatments/ procedures/devices Support to research and care ( patients, industry) Patient recruitment Patient care benchmarking

16 The organization of the data set 3) Integrated outcome measures Quality of Life index score Health services, procedures and devices Metadata of data sources Case notification completeness, patient recruitment Risk factors, health service monitoring Burden of disease, Advocacy, HTA, Benchmarks

17 The organization of the data set 3) Integrated outcome measures Quality of Life index score Health services, procedures and devices Metadata of data sources Case notification completeness, patient recruitment Risk factors, health service monitoring Burden of disease, Advocacy, HTA, Benchmarks

18 The initial steps of the platform Promotion of harmonization: adaptation and evolution of the data collected and the procedures used use of common tools, standards, procedures and strategies to improve quality and comparability of registered data Support to data collection with a longitudinal design for diseases for which registries are not yet established and where disease-specific registries are not sustainable Based on a core set of common key data elements and extensions to more detailed and disease-specific data