The European Organisation for Rare Diseases (EURORDIS) is the voice of 30 million people affected by rare diseases throughout Europe.
EURORDIS is a non-governmental patient-driven alliance of organisations and individuals active in the field of rare diseases in Europe.
EURORDIS represents 716 rare disease patient organisations in 63 countries out of which 27 EU member states, covering more than 4000 rare diseases and rare cancers.
EURORDIS advocates for people living with rare diseases, fosters patient engagement in Commission Expert Groups, at the European Medicines Agency and EUnetHTA, provides services to patients such as information, networking and capacity building on all aspects of their condition such as therapeutic developments, access to medicines, social services and healthcare. This is done through tools such as the eurordis.org website, Summer School, Rare Disease Day, RareConnect.org , the survey programme Rare Barometer Voices, Rare Diseases International and the European Conference on Rare Diseases & Orphan Products.
The EURORDIS Specific Grant Agreement 2017 continues the work of SGA 2016 within the overarching Framework Partnership Agreement 2015-2017. This grant's main strategic objectives are to consolidate the rare disease patient community and strengthen the voice of rare disease patients; to actively engage rare disease patient representatives to contribute to the adoption, effective implementation and monitoring of relevant EU legislation/strategies and support their integration at national level; to build capacities and empower rare disease patients advocates, members and volunteers; to sustain human, financial and organisational resources.
The Specific Grant Agreement FY2017 n°748373 was granted to the European Organisation for Rare Diseases (EURORDIS) as the ninth operating grant, and third specific grant agreement, in 2017, in the Framework Partnership Agreement 2015-2017. Throughout the year, the Specific Grant Agreement significantly contributed to the implementation of its Strategy 2015-2020 and the achievement of its Work Plan 2017 as approved by its members at its annual General Assembly in May 2017.
As in previous years, the Operating Grant of EURORDIS covers its recurrent activities with the exceptions of those in the areas of governance and advocacy activities.
The following Periodic Technical Report and the Financial Report aim to show how the activities have been performed and resources allocated. The activities listed in the Workplan & Deliverable Tables in the Evaluation section of this report have been achieved in due time and according to the contract.
The Operating Grant has allowed for the consolidation of the quality of EURORDIS’ recurrent activities that have been within its scope since 2009.
However, EURORDIS is increasingly limited by the current level of support from the Operating Grant to cope with the growth in recurrent activities that stems from EURORDIS’ progressively increasing outreach to patient organisations and communities, successful public awareness raising, as well as a growing number of technical activities in which patient representatives are involved so to turn the new EU and national rare disease policy frameworks into reality. This is the case of the activities related to increased patient engagement in European Reference Networks and the development of the ePAGs; EMA Committees and HTA; as well as the increased communication activities revolving around Rare Disease Day 2017; the outreach to patients and patient organisations internationally and their empowerment; the steady growth of EURORDIS membership; the support to the involvement of volunteers in EU and national policy working groups; the growing support to patient training and involvement.
Altogether, this confirmed the great importance of the Operating Grant in support of the recurring activities of EURORDIS and therefore its direct impact on the operations of the organisation. Overall, the Operating Grant proved to be once again instrumental in fulfilling the Key Operational Objectives of EURORDIS for 2017, as described in the Grant Agreement (page 55); and it allowed the organisation to continue to play its pivotal role in the definition and implementation of the EU strategy on rare diseases (European Commission Communication on Rare Diseases, December 2008; Council Recommendations for actions in the area of rare diseases, June 2009; the recommendations of the EU Committee of Experts on Rare Diseases; the Commission Roadmap for the implementation of the Communication and Recommendation on Rare Diseases; the EU Directive on Patients’ Right to Cross-border Healthcare).
The structure of EURORDIS’ Work Plan 2017, found in detail in Annex I, of the Operating Grant Agreement is organised along the following main strategic objectives
WP.1. To consolidate the rare disease patients’ community; WP.2. To actively engage rare disease patient representatives in the effective implementation and monitoring of legislation and strategies; WP.3. To build capacities and empower members and volunteers; WP.4. To sustain human, financial and organisational resources.
For the Strategic Objective “WP.1. Consolidate the Rare Disease patients’ community”, EURORDIS’ membership base continued to grow in 2017 reaching 779 members by the end of the year, including 34 new members in EU Member States. EURORDIS has members in 69 countries and 28 countries in the EU.
In 2017, EURORDIS played a central role in the efforts for the development of an international rare disease movement. Rare Diseases International continued to grow and promote rare diseases as an intern
The structure of EURORDIS’ Work Plan 2017 was organised along the following main strategic objectives
WP.1. To consolidate the Rare Disease patient’s community and strengthen the rare disease patient voice; WP.2. To actively engage rare disease patient representatives to contribute to the adoption, effective implementation and monitoring of relevant legislation/strategies; WP.3. To build capacities and empower rare disease patients advocates, members and volunteers; WP.4. To sustain human, financial and organisational resources.
In order to achieve the above objectives EURORDIS organised meetings & training sessions; produced and published documents, reports & information material; reached out to the community and stakeholders through webinars, conf calls and face to face meetings.
Further details as to the methods, means and work carried out during 2017 can be found in the section below.
The work performed during the reporting period is detailed in the work programme below:
WP.1. Consolidate the rare disease patients’ community:
A.1.1. Outreach to rare disease patient organisations (POs).
EURORDIS had a membership increase of 8% between January 2017 and December 2017 with 57 new members bringing the total membership to 779 members. EURORDIS has 2712 patient organisations in its contact database.
A.1.2. Outreach & support to rare disease patient groups at international level, through:
- Participation in the International Rare Disease Research Consortium (IRDiRC) through face to face Executive Committee & Therapies Scientific Committee (TSC) meetings and related conference calls; Chairmanship of the TSC; interaction with IRDiRC Scientific Secretariat & the Interdisciplinary Scientific Committee. In 2017, EURORDIS was involved in the preparation of the new IRDIRC roadmap to achieve the new goals elaborated in February 2017 during the 3rd IRDiRC conference held in Paris, which was also attended by EURORDIs representatives.
- Promotion of rare diseases as an international public health priority in partnership with international patient organisations. In 2017 EURORDIS monitored and maintained its partnership with 7 international organisations: the National Organisation of Rare Disorders – NORD (USA), the Canadian Organisation for Rare Disorders - CORD (Canada), the Japanese Patient Association - JPA (Japan), Rare Voices Australia (RVA), the Chinese Organisation of Rare Diseases (CORD), the Russian Patient Union (RPU) and the Rare Diseases Foundation of Iran (RADOIR).
A.1.3. Raise public awareness through:
- Rare Disease Day (RDD) 2017 and planning of RDD 2018. In order to raise public awareness on rare diseases, EURORDIS launched Rare Disease Day in 2008, which is traditionally held on the last day of February. In 2017, RDD was held on 28 February and had the theme “Research” within the overarching theme of “Patient Involvement”. For the 6th year in a row EURORDIS produced a well-received video was translated into 33 languages and viewed over 200.000 times on EURORDIS social media. Events took place in 94 countries (up from 18 in 2008 and 84 in 2016).
A.1.4. Produce, disseminate and exchange information and experiences:
- 21 bi-weekly eNews reports will be produced and disseminated to over 10.800 subscribers (no eNews in August 2017);
- 11 monthly Member News translated in 6 languages were produced and disseminated to the over 1500 EURORDIS members (no Member News in August 2017). EURORDIS Member News was introduced in 2015, and consists of a news report tailored specifically to the needs and interests of EURORDIS’ membership.
- Content management of the EURORDIS website. Website sections are maintained and updated regularly. The website had 580.000 visitors in 2017 and there was an improvement of the website navigation with the use of the User Centered Design (UCD) approach, which ensures that structure, navigation architecture, content and features adapts to the expectations of the users. The annual EURORDIS Photo Contest was moved from eurordis.org to the Black Pearl Awards website.
- Management of EURORDIS online communications tools including social media (Facebook, Twitter, Flickr, YouTube), streamlined with the EURORDIS website; and EURORDIS TV
- Production and dissemination of reference documents, which included the Activity Report 2016; a new ‘About EURORDIS’ leaflet; two position papers on Early access to medicines in Europe: Compassionate use to become a reality and Breaking the Access Deadlock to Leave No One Behind; in addition to a Rare Barometer survey report and infographic on Juggling care and daily life: The balancing act of the rare disease community
- Translation activities into the 7 official EURORDIS languages (EN, FR, DE, IT, ES, PT, RU) for Website, eNews and Members’ News, and into 33 languages on an ad hoc basis (RDD video translation)
A.1.5. Provide a platform fo
Overall the key outcomes achieved during the Operating Grant for each strategic
objective were as follows:
WP.1. Consolidate the Rare Disease patient’s community and strengthen the rare disease patients’ voice
o EURORDIS membership: 57 new members that joined in 2017 and at least 1000 person/days from EURORDIS membership were mobilised to participate to events, conferences, trainings and workshops organised throughout the year.
o Promotion of rare diseases as an international public health priority through continued participation and support of Rare Diseases International and the UN Committee on Rare Diseases.
o International partnerships with 7 MoUs signed with international patient organisations so far
o Participation in the preparation of the new International Rare Disease Research Consortium (IRDiRC) roadmap for the achievement of the new goals following the 2017 3rd IRDIC conference
o Raising public awareness with a growth of Rare Disease Day awareness campaign in 94 countries, with over 1000 events
o EURORDIS targeted newsletters in the form of eNews and Member News with: 21 bi-weekly eNews issues distributed to over 10.000 subscriber. Articles from EURORDIS eNews are often reproduced and reused by National Alliances and patient organisations in their own printed or electronic newsletters. 11 monthly Member News were developed and distributed to over 1500 contacts at EURORDIS member organisations with a 60% open rate (average for this type of mailing is 20%)
o Improvement of EURORDIS website navigation using User Centered Design (UCD) approach
WP.2. Actively engage rare disease patient representatives into the effective implementation and monitoring of relevant legislation/strategies
o Review of public information on medicines with a total of 158 EMA documents for public information reviewed by EURORDIS staff members or volunteers in order to ensure the quality of the information disseminated by the EMA to the general public
o Providing support to patient representatives in pre-marketing activities with a total number of over 172 days spent by EURORDIS representatives at EMA Committees
o Survey report on access, called “Access to treatment: unequal care for European rare disease patients”
o EURORDIS contributed to two documents for the European Commission’s call for contribution to the HTA legislation Principles of Patients and Consumers engagement in HTA and Criteria for prioritisation of technologies for join assessment
o Two face to face meetings of the DITA Task Force in Paris.
o Over 500 exchanges by email discussing issues and reports relevant to people living with rare diseases, e.g. access to care, orphan medicinal products, paediatric drugs, HTA, access to social services
WP.3. Build capacities and empower members and volunteers
o The EURORDIS Membership Meeting 2017 Madrid saw 250 participants from 38
countries (75% patient representatives) and 40 fellowships in support to patient representatives from 19 countries
o The Council of National Alliances had 2 meetings in 2017
o The Council of European Federations grew to 62 members whereas 13 beneficiaries were provided with the necessary funding to organise their meetings with very satisfactory results
o 13 help lines from 11 countries participated in the activities of the European Rare Disease Help Line Network with 1 face to face training training on pharmacovigilance and how to take complex calls held in March 2017. The Help Lines were involved in the 10th Annual Caller Profile Analysis.
o EURORDIS Summer School 2017 had 62 participants attending from 27 countries representing 43 diseases. A Spanish version of the Summer School was also organised, with 85 participants in total. An online pre-training programme was implemented – the online training is free and available to all.
o Conduct surveys on patient perspectives regarding their experience, needs & expectations. The panel of rare disease patients who answer on a regular basis to EURORDIS’
Deviations from the Work Plan 2017 have been few and are summarised below:
Support patient involvement in Commission Experts Groups (Rare Diseases & Cancer Control): At the Commission’s decision, the mandates of the Expert Groups on Cancer Control and Rare Diseases were not renewed in 2017. The work of the new horizontal working group that is meant to replace them, has yet to commence. Due to this, there was no activity in the area of patient engagement in Commission Expert groups.
Support Patient Engagement in ERNs: The deployment of the European Reference Networks necessitated a lot more action in the area of patient engagement in ERNs. EURORDIS developed the ePAGs (European Patient Advocacy Groups), one for each ERN disease grouping, for the purpose of ensuring that the patient voice is heard throughout the ERN development process. There are 24 ePAGs, involving over 250 ePAG representatives and and 1000 patient organisations including non-members of EURORDIS.
In 2017, two ePAG face-to-face meetings were organised by EURORDIS with 37 and 40 participants respectively. Throughout the year, 77 conference calls were held with the 24 ePAGs and ERNs to discuss topics of relevance with the ERN leads and ePAG representatives. EURORDIS also developed online ePAG communities in order to enhance ePAG communication. 190 ePAG representatives have been invited to join Facebook Workplace. The EURORDIS membership meeting held in Budapest in May included a capacity building workshop on ERNs & ePAGs and the joint CNA/CEF meeting in Paris in October included an item on the Development of ERN & ePAGs. In addition to the meetings organised by EURORDIS, EURORDIS facilitated ePAG representation and ePAG satellite meetings at 23 ERN board and kick off meetings throughout 2017.
EURORDIS has the potential to reach out to more than 2500 POs across Europe (EURORDIS RD community Database), in particular through its membership; the EU Networks of National Alliances, RD specific Federations and Rare Disease Help Lines; the membership of Rare Diseases International; the EURORDIS Rare Barometer survey programme with currently 6000 registered participants; and the RareConnect platform of online communities (26000 registered users & 90 communities). EURORDIS’ main target audiences for dissemination purposes are a) RD patients & families; b) RD patient organisations & advocates; c) decision makers; d) industry; e) care providers (health and social care) and f) the general public.
External dissemination included:
• Dissemination of 11 EURORDIS eNews in 7 languages to over 10500 subscribers; Frequency: monthly; Method: Active; Main target audience: all EURORDIS’ stakeholders
• Dissemination of 22 EURORDIS member news to over 1500 contacts at member patient organisations; Frequency: bi-monthly; Method: Active; Main target audience: EURORDIS’ members
• EURORDIS websites available in 7 languages (approx. 460.000 website visitors/year); Frequency: permanent; Method: passive, Main target audience: Patient organisations & advocates followed by all stakeholders
• Web services and communication tools (Facebook groups, Twitter, RDD website, RDI website, YouTube, Flickr, Instagram, online communities); Frequency: daily; Method: active; Main target audience: RD patients & families and RD patient organisations & advocates, followed by all stakeholders
• Large-scale awareness-raising campaign through the annual international Rare Disease Day, organised in 94 countries (in 2017), including a dedicated RDD website and broad media exposure; Subscribers for the specific RDD Facebook group: more than 87 000; Followers for the specific RDD Twitter account: more than 19 000; Visits of EURORDIS RDD 2017 website on February 28, 2017: over 42 000; Frequency: annual over the month of February; Method: active, Main target audience: General public, Care providers, Decision makers, followed by all stakeholders
• Public contributions, statements, comments, press releases, position papers, etc. widely circulated to relevant stakeholders, such as national and European decision-makers, industry representatives, regulators, patient communities, health professionals; Frequency: Throughout the year as needed; Method: active, Main target audience: Adapted to stakeholders as needed
o Activity Report, Frequency: Annual – Adopted at May General Assembly; Method: Active; Main target audience: EURORDIS members, followed by all other stakeholders
o Fact sheets, brochures, etc., Frequency: Regular updates & additions throughout the year; Main target audience: Adapted to stakeholders as needed
• EURORDIS Membership Meeting 2017 Budapest gathering more than 250 patients and patient representatives; Frequency: Annual; Method: Active; Main stakeholder: EURORDIS members
• ECRD 2018 Vienna: Announcement and programme dissemination, dedicated web site, flyers distributed at key conferences and regular emailing for the organisation of the conference, executive summary publication and dissemination; Frequency: Annual; Method: Active; Main stakeholder: All stakeholders
• EURORDIS representatives participating in different fora are also valuable means of external dissemination of key messages (over 80 speeches, presentations & meetings attended per year, on average); Frequency: Throughout the year; Method: Active; Main stakeholder: All stakeholders