Rare Diseases Europe STRATEGIC APPROACH 2007-2009 & WORKPLAN 2007 Paris, March 2007 www.eurordis.org
Contents Our strategic approach 2007 2009 o Eurordis in 2010 o Strategic orientations o Priorities 2007-2009 Our action plan 2007 o Key priorities o Key topics Budget 2007 Significant European activities Eurordis Workplan 2007 2
Our Strategic Approach 2007-2009 Eurordis in 2010 Eurordis is the European movement of rare disease patient groups and individuals and their European voice. Eurordis bring added value through advocacy, information, capacity building and services Eurordis added value is perceived by our members, patients & families and stakeholders Eurordis has a renewed long term vision and strategy. Strategic orientations Fewer priorities to achieve measurable results More innovative therapies, patient-centred healthcare and patient driven research bridging science & society More integrated approach between EU and national levels, and between Eurordis and national alliances Based more deeply on individual patient and families Develop private funding for resource diversification and for long term financial self-sustainability Promote Eurordis as a brand name in everything we do Web Communications at the centre of what we do: members, volunteers, staff, patient and families as producers of information More international openness and perspective Build a long term and prospective vision Eurordis Workplan 2007 3
Priorities 2007-2009 Rare Diseases: a priority visible on the European agenda Promote rare diseases public health policies at EU and national levels Focus on key advocacy issues Web communications for everything we do Annual European rare disease awareness campaign 2008 & 2009 A broader grassroots patient-centred community Centre our action on our members Reinforce our patient group membership base Special focus on new and future Member States Develop individual membership Involve more volunteers in our activities Organise capacity building and training sessions for patient representatives Better structure our network (new national alliances, European network of national alliances, European specific disease networks, country liaison officers) Budget growth and funding diversification Increase EU support to Eurordis projects and other rare disease activities Reinforce current funding base: AFM, members, health industry Develop private funding and partnerships with corporations and foundations outside the health sector Develop individual support and fund raising events Consolidation of two major events Membership meeting 200X City (annual) European Rare Disease Conference 200X City (every other year) Shaping the Rare Disease European Environment Shifting our priorities toward more public health activities and services to patients and families: o Access to quality care and European networks: Centres of Expertise and European Reference network Patient mobility Good practices diagnosis, gene testing, counseling, screening Good practices based on consensus medical care o Access to quality services and European networks: Help Lines Isolated patients Web patient communities Therapeutic Recreation Programmes Integration at School Respite Care Services Activities on therapy development focused on: o Orphan drugs: speed up development and availability o Advanced therapies: gene therapy, cell therapy o Inform more and involve more members and volunteers Eurordis Workplan 2007 4
Patient driven European research infrastructures addressing bottlenecks toward therapies : o Priority on biobanks o Priority on patient registries o Priorities on clinical trials Bridge the gap between patient needs and research in biomedicine, public health, social, economy, policy and ethic Cross-cutting Priority Develop office support services for better quality work, increased efficiency and better quality at work for all: o Contact Management Database o Advanced Financial Information System o Advanced Human Resource Policy o Advanced Information Technology as a support for projects management, work with volunteers and virtual office to ease mobility o Optimise synergies between websites, project databases, contact management database o Synergy between Eurordis and national alliances o New office support and environment Eurordis Workplan 2007 5
Our action plan 2007 Key priorities Commission s Communication & Council Recommendations Centres of Expertise & European reference networks Future Regulation on Advanced Therapies Implementation of the new Regulation on Paediatric Drugs European Conference on Rare Diseases 2007 Lisbon Networks of services for patients and families of patients Membership Meeting 2007 Paris and capacity building in research for rare disease patient groups Consolidation of activities on orphan drugs 1 or 2 new EC projects: patient group capacity building in science; cooperation and exchange of best practices between patient groups; EuroBioBank II; e-health Strategy and plan for private funding (1st step) Strategy and plan for volunteers (1st step) Eurordis Workplan 2007 6
Key Topics Advocacy Level 1 priorities Commission s Communication and Recommendations to the Council and European Parliament on Rare Diseases (2007-2008) Rare Diseases as a Public Health Priority in Commission programmes and in Member states: o EU Research Framework: promote rare diseases in FP 7 (2008-2013)+ input in call for proposal + consensus panels o EU Public Health: promote rare diseases in Work Programme (2008-2013) + input in call for proposals + Rare Disease Task Force o ECRD 2007 Lisbon under Portugal s EU Presidency o Sustainability: propose stronger cooperation between Member States with creation of EU High Level Group on Rare Diseases Centres of Reference and European Reference Networks Patient mobility (part of Services of general interest) Patient access to diagnosis: delay and conditions, proposals to improve earlier diagnosis Orphan Drugs: o Common needs, global development: harmonisation EU/US for designation and parallel procedure toward converging EU/US requirements (joint position paper with NORD) (+ COMP WGIP) o Patient access to authorised orphan drugs in the EU: common policy and criteria for health technology assessment and for pricing and reimbursement (position paper) (+ EMEA COMP) Paediatric Drugs: implementation of EU Regulation + appointment to Paediatric Committee at the EMEA Advanced Therapies: support the future EU Regulation Transparency of NGOs Internationalisation in Rare Diseases Advocacy Level 2 priorities Patient databases, registries and cohorts: promote policy recommendations Rare Disease Biological Resource Banks and European Network: promote policy recommendations and the Charter Clinical Trial Directive: assessment of the impact of the implementation of the Directive on rare disease clinical research in Europe (toward legislative process for revision of the Directive in 2008/10) Orphan Drugs: clinical research grant programmes, unmet medical needs (toward 2008/9 level 1 priority through COMP WGIP) Diseases are rare, patients are many : a quantitative analysis of rare disease numbers Comprehensive approach to rare diseases (beyond medical care) Organ Donation and Transplant for rare disease patients Members and patient groups Membership Meeting 2007 Paris May 3-5 Increase interaction and recognition: favour direct contacts; send more information; ask for opinion; call for volunteers; involve people in projects, workshops, conferences; training sessions Eurordis Workplan 2007 7
Priority recruitment of European Federations and EU Enlargement to new and future Member States 2 European Workshops of National Alliances (Paris May 3 & Lisbon November 26) 20 European Workshops of Rare Disease specific networks (Paris May 3 & Lisbon November 26) Country visits and support: Czech Republic, Greece, Norway, Poland, Portugal, Romania, Health policy: Rare Disease Patient Solidarity Project Centres of Reference and European Network: solid recommendations New Services for patients: towards tangible & measurable outcomes o European network of helplines o Isolated patients o Therapeutic recreational programmes o Respite care services o Internet Patient Communities European Conference on Rare Diseases 2007 Lisbon Therapeutic development Orphan drugs: improve follow up of COMP, Protocol Assistance, marketing authorisations, product information, availability to patients Advocate on orphan drugs in EU/US and on better patient access to drugs Drug Information and Transparency: EMEA PCWP Paediatric Committee ready for implementation Implementation of the Charter on Clinical Trials ERTC Workshops o Availability of Orphan Medicines in Europe o Level of evidence for Proof of concept studies Establish Eurordis Task Forces on: o Drug Information, Transparency and Access Re-establish Eurordis Task Forces on: o Orphan drugs o Paediatric Drugs DIA EuroMeeting Vienna March 2007: o Track for patients o Tutorial o Fellowship programme Capacity building sessions in clinical trials (CAPOIRA project): o Common pedagogic tool and evaluation o Sessions in Denmark, Spain and Italy Research European Workshop «Gaining Access to Rare Disease Research Resources» 3 and 4 May 2007, Paris Establish a Eurordis Research Task Force, composed of volunteers Develop priorities for FP 7 and develop concept of European Agency for Rare Diseases Develop two patient driven pilot health research projects for FP 7 (2007-08) o Capacity building in research II o EuroBioBank II or e-health Eurordis Workplan 2007 8
Communication and development New EC DG Sanco Project for 2008 2011 Website V3 finalised Print-base communication material Celebrating 10 years of Achievements (Eurordis 10 year anniversary) Prepare 2008: o Rare Disease Awareness Campaign 2008 o Develop short & long term fund raising strategy for unrestricted funds Finance and office support Budget reporting and forecast cash flow Support budgeting project development Quality processes: written procedures Volunteer policy Contact management database New offices Eurordis Workplan 2007 9
Budget 2007 * Individual and corporate donations Revenue 2007 = 2 369 000 euros Expenses 2007 = 2 369 000 euros
Eurordis 2007 budget compared to 2006 accounts REVENUE 2 006 2 007 Difference Patient Organisations 695 877 763 282 67 405 AFM 661 387 743 282 81 895 Membership fees and Grants 34 490 20 000-14 490 Volunteers 168 693 168 693 0 European Commission 123 972 544 810 420 838 National authorities 79 919 150 399 70 481 Industry and Foundations 450 061 667 670 217 609 Industry 346 525 443 890 97 365 Foundations and NPOs 103 536 223 780 120 244 MIscellaneous 62 357 74 000 11 643 EXTraordinary incomes 94 622 0-94 622 PARtners 86 225 0-86 225 OTHers 8 397 0-8 397 Sub-total (I) 1 675 500 2 368 854 693 354 Report of non-used income 24 102 0-24 102 TOTAL REVENUE 1 699 602 2 368 854 669 252 EXPENSES 2 006 2 007 Difference Staff 882 365 1 010 657 128 291 Volunteers 168 693 168 693 0 Travel and subsistence 146 373 435 021 288 648 Services 343 952 686 116 342 164 Honorary 173 642 312 626 138 984 EC project's partners 29 728 102 578 72 850 Telecom and post 22 221 29 416 7 195 Rent 96 771 96 660-112 Others 21 590 144 836 123 246 Purchase 55 270 68 305 13 035 Miscellaneous 5 393 0-5 393 Extraordinary expenses 75 004 0-75 004 Partners 75 004 0-75 004 Others 0 0 0 Sub-total (I) 1 677 051 2 368 792 691 741 Commitment on assigned income 0 0 0 TOTAL EXPENSES 1 677 051 2 368 792 691 741 PROFIT OR LOSS 22 551 62
Significant European activities Eurordis Workplan 2007 11 EURORDIS Plateforme Maladies Rares 102 rue Didot Paris 75014 France Tel + 33 1 56 53 52 10 Fax + 33 1 56 53 52 15 Brussels office: Tel/fax + 32 2 733 81 10 eurordis@eurordis.org www.eurordis.org