Domenica Taruscio. Director National Centre for Rare Diseases Istituto Superiore di Sanità Rome, Italy Brussels, 23 May 2012

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1 Domenica Taruscio Director National Centre for Rare Diseases Istituto Superiore di Sanità Rome, Italy

2 Background More than 500 RD registries in the EU No uniform standards for the storing and management of data Registries set up for 20% of rare diseases, more registries for the same RD Need to increase data sharing and exchange Need to harmonise existing regulations (EU, national and regional) The EU Council Recommendation on Rare Diseases (adopted in June 2009) recommends, among other topics, the implementation of registers and databases for epidemiological purposes. Therefore, many initiatives may appear in the coming years.

3 Building Consensus and Synergies European Platform for Rare Disease Registries EPIRARE Co-funded by the EU Commission (DG SANCO) Starting date: April 2011 Duration: 30 months (April 2011 October 2013) Coordinator of the project: National Centre for Rare Diesases Italian National Institute of Health (Istituto Superiore di Sanità) Rome, Italy

4 8 Workpackages: WP1 WP4 The project is organised in 8 Workpackages: WP1 - Coordination of the project WP Leader: Domenica Taruscio National Centre for Rare Diseases, Istituto Superiore di Sanità, Rome (Italy) WP2 - Dissemination of the project WP Leaders: Domenica Taruscio, Fabiola Gnessi National Centre for Rare Diseases, Istituto Superiore di Sanità (Italy) WP3 - Evaluation of the project WP Leaders: Rumen Stefanov Information Centre for Rare Diseases and Orphan Drugs, BAPES (Bulgaria); Franco Cavallo - University of Turin (Italy) WP4 - Legal basis WP Leaders: Angela Brand, David Townend- University of Maastricht (Netherlands)

5 8 Workpackages: WP5 WP8 WP5 - Policy scenarios on scope, aims, governance and long-term sustainability WP Leader: Yann Le Cam, Monica Ensini - EURORDIS WP6 - Common data set and disease-specific data collection WP Leader: Fabrizio Bianchi - National Council of Research Pisa (Italy) WP7 - Data quality, validation and data sources integration WP Leader: Manuel Posada de la Paz - Istituto de Salud Carlos III (Spain) WP8 - Current needs of existing registries on rare diseases WP Leader: Sabina Gainotti - National Centre for Rare Diseases, Istituto Superiore di Sanità (Italy) Steering Committee Advisory Board

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7 EPIRARE Objectives General objective To build consensus and synergies to address regulatory, ethical and technical issues associated with the registration of RD patients and to elaborate possible policy scenarios. Specific Objectives To identify the needs of the EU registries and databases on RD To identify key issues to prepare a legal basis To agree on the Register and Platform Scope, Governance and longterm sustainability To agree on a Common data set, disease-specific data collection and data validation To identify tools and other facilities supporting the operation of the platform users

8 The EPIRARE Deliverables The current situation, the challenges and the expectations on Patient Registries and Databases Possible policy scenarios on Scope, Common data set, Governance and Sustainability for RD patients registration: Report on Common data sets and disease-, treatment- and other specific modules Guidelines for data sources and quality of RD Registries in Europe Identification and characterization of services and facilities of a European Platform of EU RD Registries Best practice model of a legal instrument for a European Platform of EU RD Registries Scientific secretariat of an EUCERD WG on registries and databases (cochaired ISS and EURORDIS) European/International Workshop with stakeholders to discuss possible policy scenarios Promotional material, website, and activities intended for dissemination at conferences and distributed to stakeholders

9 Expected results of this meeting Common view of the survey results. Start the discussion of meaning of the survey results in the perspective of defining the added value of the platform, with respect to the registries, in terms of Synergies among the stakeholders interests Transparent and trustworthy governance Sustainability Outputs Offer of technical tools and services. Best practice model of a legal instrument for a EU Platform of Registries Share views on a position paper on the ethical, legal and epidemiological reasons in support to RD patient registration scope, structure, format, target audience lobbying strategy

10 EPIRARE MEETINGS

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12 Thanks for your attention