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EURORDIS_FY2014 [EURORDIS_FY2014] [20133304] - Operating Grant
Mission and vision of the operating grant holder

EURORDIS is a patient-driven alliance of Patient Organisations(POs) and individuals active in the field of rare diseases (RDs). It represents the voice of approximately 30 million citizens in the EU.

EURORDIS` mission is to build a strong pan-European community of POs and People Living With RDs (PLWRD), to be their voice at the European level and to fight against the impact of RDs on their lives.

To this end, EURORDIS undertakes activities on behalf of its members:

• Empowering RD patient groups
• Advocating RDs as a public health priority
• Raising public awareness on RDs
• Improving access to information, treatment, care and support for PLWRD
• Improving quality of life
• Encouraging good practices in relation to these issues
• Promoting scientific and clinical research on RDs
• Promoting the development of treatments and medicines for people with RDs

EURORDIS plays a pivotal role in:
- the implementation of the EU strategy on RDs(Commission Communication and Council Recommendations), through its participation in the EU Committee of Experts on RD (EUCERD), as well as in the elaboration and monitoring of National Plans/Strategies on RDs
- the shaping and implementation of:
the specific EU health policies and legislations impacting on the RD field (EU Directive Cross Border Healthcare) through development of European Reference Networks for RD, as well as participation in the EUnetHTA Stakeholders Forum;
the EU Regulations on Medicines (Orphan Medicinal Products, Paediatrics, Advanced Therapies) through participation in the EMA Scientific Committees which also serves at improving the EU framework for drug development, approval and access to address RD patients` needs
- the revision of EU legislation on Clinical Trials, Data Protection and Transparency.

Strategic objectives and specific activities

1.Consolidate the EU RD patients community & voice
Be the voice of all RD patients, genetic or not, including rare cancers as well as the most isolated PLWRDs
Promote RDs as a public health priority at national, European & global levels
Raise public awareness on RDs, including international Rare Disease Day (RDD)
Organise the biannual European Conference on Rare Diseases and Orphan Products (ECRD)
Focus on key EU policy issues relevant to PLWRD

2.Facilitate the shaping & implementation of EU legislation/strategies
-Support effective implementation of EC Communication & Council Recommendations on RDs
Promote RD national plans;
Analyse legislation & strategies and evaluate potential impact on RD patients;
Participate in EUCERD & related Working Groups.
-Support the development of EU/national health care policies
Promote EU Reference Networks of Centres of Expertise and patient registries;
Participate in activities on screening & gene testing.
-Support therapeutic development & access to OMPs, pediatrics & ATMPs
Participate in EMA committees (COMP,PDCO,CAT, as well as PRAC,CHMP) & WPs (SAWP,PCWP)in
order to bring in patient perspective;
Inform & involve more members/volunteers in therapeutic activities;
Facilitate dialogue between patient groups, experts, companies, regulators, NCA & HTA.
-Support RD research policy & priorities
Promote the development of EU research networks and infrastructures;
Participate in activities of the International Rare Diseases Research Consortium
Support the participation of patients in research & ethics committee.
-Support development & access to information & specialised social services
Consolidate national Help Lines & EU Networking;
Produce validated information on respite centres, therapeutic recreation programmes,
resource centres and adapted housing;
Generate/share patient-based knowledge.

3.Build capacities & empower members and volunteers
Maintain high level of legitimacy and professionalism within the membership;
Organise the annual EURORDIS Membership Meeting (EMM);
Broaden the patient group membership base with special focus on EU and Central & Eastern Europe;
Identify, involve and support volunteers in more activities;
Intensify networking & collaborative process with/between National Alliances and RD Specific Federations;
Share good practices & organise training for patient reps.

4.Sustain human, financial and organisational resources
Good governance & financial transparency;
Web communications embedded in work process;
Integrative IT infrastructures;
Development and diversification of resources;
Provide volunteers with appropriate support (technical tools,fee waivers,fellowships, etc) to enhance integration of their activities

Expected outcomes

A. Information & communication to POs, patients, stakeholders and the public:
a) EURORDIS RD community databases and dissemination of information
b) EURORDIS Website & weekly eNews in 7 languages (English, French, German, Italian, Spanish, Portuguese & Russian)
c) Raise awareness through EURORDIS TV, Social Media, patient stories, videos & photos
d) Support the development of RD Help Lines and promote shared tools and training
e) Promote and expand the network of 39 online patient communities (RareConnect)
f) RDD 2014 on 28/02; preparation of RDD 2015
g) Reference documents on RD: brochures, fact sheets; translations
h) Recurrent activities linked to preparation, follow-up & reporting of ECRDs:
- ECRD 2014 Berlin: support the planning & follow-up; programme development; promotion of registrations; call for posters; ECRD report & dissemination, fellowship programme - 800 participants
- ECRD 2016 Edinburgh: programme committee; announcement; selection of venue, hotels and providers
i) Promoting RD as an international priority
j) International RD partnership with NORD, CORD, JPA, RPU, ICORD, Orphanet
k) Involvement of Health & Research Stakeholders beyond RD such as EPF, IAPO, DIA, EFPIA/EBE, EuropaBio

B. Capacity building for patient representatives:
1. Health Policy, POs networking and empowerment:
a) Outreach to 1700 POs, 590 members, 132 volunteers; consultation; support to local networking & fellowships, focusing on new MS, Eastern & Central Europe; country visits
b) Support the development of CoE, ERN, Registries, good clinical practices, the involvement of patients in these activities & collect patient feedback on functioning of CoE
c) Support the EU Network of National Alliances: involvement in RDD; capacity building on RD National strategies; integration of EU/national policies; 1 Workshop of the CNA
d) Support the Network of EU Federations: involvement in RDD; empowerment on EU policies; 1 Workshop for the CEF; support to networking meetings of RD specific networks; dissemination and sharing of good practices
e) Support volunteers & staff representing EURORDIS in EU committees & conferences
f) EURORDIS Membership Meeting 2014 Berlin and preparation of EMM 2015 Madrid (250 participants)

2. Research & Therapeutics Development and Information & Access to Treatments:
a) Patient involvement in EMA activities:
- Support the participation of patient representatives in the EMA Committees (COMP, PDCO, CAT), Working Parties (Scientific Advice, Patients & Consumers) and as external experts (Protocol Assistance, SAG/CHMP, PRAC)
- Support the EURORDIS Therapeutic Action Group (TAG) composed of all Rare Disease patient representatives at EMA to exchange information and coordinate actions
- Monthly report compiling feedback from each committee and WP
b) Review and validate public information on RD therapies disseminated by EMA at the time of designation (PSOs) and benefit/risk evaluation (EPARs, Package Leaflet) & further disseminate the information to patients via compiled documents
c) Support involvement of more RD patient representatives in OMP, PD, ATMP & PhV policies and in drug development via information dissemination and capacity building activities on clinical trials (CT) and EU regulatory affairs, through:
- EURORDIS Task Force on Drug Information, Transparency & Access (DITA) - trained volunteers
- Summer School 2014 on drug development, CT & regulatory affairs based on experience exchange & case studies
- Online learning on clinical trials, statistics & ethics
d) Promote RD research policy and patient involvement in research
- Create & support EURORDIS Scientific Affairs Committee
- Develop partnership with learned societies such as European Federation of Internal Medicines, ISPRO, HOPE, ESHG, International Federation of Social Workers, International Women’s Health Institute
- Support participation of staff & volunteers in scientific conferences and involvement as advisors in research projects

Start date: 01/01/2014
End date: 01/01/2015
Duration: 12 month(s)
Current status: Finalised
Programme title: Second Programme of Community action in the Field of Health 2008-2013
EC Contribution: € 770,00
Portfolio: Rare diseases