The EURORDIS Specific Grant Agreement 2016 continues the work of SGA 2015 within the overarching Framework Partnership Agreement 2015-2017. EURORDIS main strategic objectives are to consolidate the Rare Disease patient’s community and strengthen the rare disease patients’ voice; 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. All activities within the SGA 2016 work to this effect.
The Specific Grant Agreement FY2016 n°709986 was granted to the European Organisation for Rare Diseases (EURORDIS) as the eighth operating grant, and second specific grant agreement, in 2016, 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 2016 as approved by its members at its annual General Assembly.
As in previous years, the Operating Grant of EURORDIS covers its recurrent core 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. All 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.
In 2016, the Operating Grant allowed for the quality development of areas which consolidated since 2009, when the first Operating Grant was awarded.
However, EURORDIS is increasingly limited by the current level of support from the Operating Grant to cope with the growth in recurrent core 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 communication activities revolving around Rare Disease Day 2016; the outreach to patients and patient organisations internationally and their empowerment; the steady growth of EURORDIS membership; the support to the activities in the therapeutic area; the support to the involvement of volunteers in EU and national policy working groups; the support to the ever increasing international activities.
Altogether, this confirmed the great importance of the Operating Grant in support of the recurring core 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 2016, 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 2016, 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 to contribute to the adoption, 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 2016 reaching 733 members by the end of the year, including 36 new members in EU Member States. EURORDIS has members in 64 countries and 27 countries in the EU.
In 2016, 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 international public health priority; the RDI governance structure was consolidat
The structure of EURORDIS’ Work Plan 2016 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 2016 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 7% in EURORDIS Membership between January 2016 and December 2016 with 51 new members bringing the total membership to 733 members
A.1.2. Outreach & support to rare disease patient groups at international level, through:
- Promotion of rare diseases as an international public health priority in partnership with international patient organisations. 2016 saw the consolidation of Rare Diseases International with the election of its first governance board, the RDI Council. In addition, in October 2016 the NGO Committee for Rare Diseases was officially inaugurated at the UN Headquarters in New York.
- Bilateral partnership agreements with international patient organisations. EURORDIS has signed Memoranda of Understanding with 5 international patient groups: 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 Russian Patient Union (RPU). In 2016, EURORDIS continued to monitor its existing partnership agreements and provide support and mutual exchanges with its international partners.
- 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 & other Scientific Committees; involvement of preparation of IRDiRC conference in February 2017.
A.1.3. Raise public awareness through:
- Rare Disease Day (RDD) 2016 and planning of RDD 2017. 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 2016, RDD was held on 29 February. The overarching theme was Patient Voice, the theme: Join us in making the voice of rare diseases heard. As with previous years, EURORDIS hosted a symposium in Brussels on 23 February 2016 on the occasion of Rare Disease Day. The event was entitled: “Multi-stakeholder Symposium on Improving Patient Access to Rare Disease Therapies” with attendance from multiple representatives in Brussels. Since the first Rare Disease Day was launched in 2008, participating countries have steadily increased from 18 in 2008 to 84 in 2016.
A.1.4. Produce, disseminate and exchange information and experiences:
- 21 bi-weekly eNews reports were be produced and disseminated to over 10,500 subscribers (no eNews in August 2016);
- 10 monthly Member News will be produced and disseminated to the over 10,000 EURORDIS members (no Member News in August 2016). 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 available in 7 languages (EN, FR, DE, IT, ES, PT, RU). Website sections were maintained and updated regularly and new website sections were added (Get Involved!, European Reference Networks). In addition, the website hosted the annual EURORDIS Photo Contest. In 2016, the Photo Contest was also present on Instagram for the first time.
- Management of EURORDIS online communications tools including social media (Facebook, Twitter, Flickr, Youtube), streamlined with the EURORDIS website;
- Production and dissemination of reference documents, which included the Activity Report 2016;
- Translation activities into the 7 official EURORDIS languages (EN, FR, DE, IT, ES, PT, RU)
A.1.5. Provide a platform for multi-stakeholder dialogue on rare diseases through the European Conference on Rare Diseases and Orphan Products (ECRD) 2016 Edinburgh held on 26-28 May with over 750 attendees. The ECRD is a biennial event
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: 51 new members that joined in 2016 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: 2016 saw the consolidation of the governance structure of Rare Diseases International; further recruitment of RDI members and the promotion of the RDI brand, including logo and dedicated website
o International partnerships with 5 MoUs signed with international patient organisations so far
o Consolidation of EURORDIS’ role in the governing bodies of the International Rare Disease Research Consortium, with Yann Le Cam being the Chair of the TSC until October 2016 and major contributions to the redaction of IRDiRC TSC recommendations
o Promotion of RD Information and sensitisation with a growth of Rare Disease Day awareness campaign in 84 countries, with over 1000 events
o Enhanced community building through social networking and online tools for RDD (150 000 unique visits); social media activity increased significantly (58 000 likes
on Facebook and 13 000 followers on Twitter)
o EURORDIS targeted newsletters in the form of eNews and Member News with: 21 eNews issues distributed to over 10 000 subscribers in 7 languages. Articles from EURORDIS eNews are often reproduced and reused by National Alliances and patient organisations in their own printed or electronic newsletters. 10 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 with more visits on eurordis.org compared to 2015 reaching 460 000 annual visits (420 128 visits in 2015) and 30 000 visitors / month; and an improvement of website navigation using User Centered Design (UCD) approach.
o The EURORDIS Photo Contest 2016 with 400 photos submitted from 54 countries; 207 000 votes on Facebook; 29 200 fans on facebook.com/eurordis; 128 followers on Instagram
WP.2. Actively engage rare disease patient representatives into the effective implementation and monitoring of relevant legislation/strategies
o Active involvement in the European Commission Expert Group on Rare Diseases (CEGRD) with the adoption of the “Recommendation to support the incorporation of rare diseases into social services and policies”; and accompaniment of the development of European Reference Networks for Rare Diseases (ERNs) with the support of the EU Joint Actions for Rare Diseases (RD-Action).
o Active involvement in the European Commission Expert Group on Cancer Control. EURORDIS is a partner of the EU Joint Action on Rare Cancers, which started on 1st October 2016.
o Review of public information on medicines with a total of 235 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 resulting in the contribution to the scientific evaluation of 686 dossiers at the EMA COMP (296), CAT (60), PDCO (330) and participation of 46 patients’ representatives in Protocol Assistance procedures
o Analysis of a survey on off-label use, called “Tell us how you take your treatment”
o On shortages, coordination with EAHP, the European Association of Hospital Pharmacists to increase awareness of shortages at the EU level; presentation at a meeting organised by the Slovak Presidency of the EU Council; presentation at a conference organised by the Belgium Regulatory Affairs Society, 24 November in Brussels
o EURORDIS participated in discussions in progress with E
Deviations from expected outcomes were few:
Council of National Alliances (CNA)
Following the results of a survey sent out to the members of the Council of National Alliances (CNA) in 2015, in which respondents clearly stated a preference for two annual meetings of the CNA, an additional face-to-face meeting was added to the yearly planning (originally only one meeting had been planned). This additional meeting was held back to back with the EURORDIS Membership Meeting 2016 Edinburgh in May and was deemed a success with 43 participants attending and focus on the following topics: Rare Disease Day; Reimbursement issues of new orphan medicines; ERN, Member endorsement of CoE, ePAGS; RD-Action National EUROPLAN workshop; Rare Barometer.
European Patient Advocacy Groups (ePAGs)
Actions performed related to the European Reference Networks accelerated in 2016. To ensure that the patient voice is heard throughout the ERN development process, EURORDIS took the initiative in developing the European Patient Advocacy Groups (ePAGs) with 24 ePAGs having been created and involving over 80 elected patient representatives and 1000 patient organisations. This action had not initially been included in the work plan of the 2016 Operating Grant.
European Year on Rare Diseases 2019 (EYRDs 2019)
The Campaign for a European Year on Rare Diseases 2019 was further investigated in 2016. It was however made clear that there is no possibility of obtaining support from the European Commission for an official European Year. Having consulted the Council of National Alliances regarding the option of continuing to pursue a European Year on Rare Diseases for which there was not political or financial support from the EC, in addition to performing an analysis of the financial and human resources that would be necessary for such an action, which were deemed to be too high, a final decision was taken by the EURORDIS Board of Directors to cancel the objective for a European Year of Rare Diseases. EURORDIS will examine the possibility of pursuing the objectives of the initiative through alternative advocacy and communication tools and activities.
EURORDIS has the potential to reach out to more than 2200 POs across Europe (EURORDIS RD community Database), in particular through its Membership, the EU Networks of National Alliances and European rare disease specific Federations. EURORDIS main target audiences for dissemination purposes are rare disease patients and their families, rare disease patient organisations and advocates, public at large, decision-makers at EU and national levels, pharmaceutical and biotech companies, academic and health professionals, and social workers.
Media tools & campaigns:
The external dissemination strategy includes the following main elements:
• Dissemination of EURORDIS bi-weekly eNews in 7 languages to 10.500 subscribers;
• Dissemination of EURORDIS monthly Member News to 1.500 contacts
• EURORDIS website available in 7 languages (over 360.000 website visitors/year) and other web services and communication tools (Facebook groups, RDD website, Youtube, Twitter, Instagram, online communities, etc. = approximatively 800.000 visits/year);
• Large-scale awareness-raising campaign through the annual international Rare Disease Day, organised in 84 countries (in 2016), including the establishment of a dedicated RDD website and broad media exposure; Subscribers for the specific RDD Facebook group: more than 75.000; Followers for the specific RDD Twitter account: more than 19.000;
• Online patients and advocates communities, and discussion groups (RareConnect) operated by EURORDIS in order to provide a forum for information exchanges, feedback and knowledge sharing for and from patients, advocates and professionals (number of registered users in RareConnect - mailing lists: 19.893; number of online patient communities: 81 at start of 2016 compared to 2 in 2010);
• 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;
• Webinars on a variety of topics including access to medicines, social policy, ERNs, etc. addressed to different audiences;
• Publications: Activity Report, fact sheets, brochures, etc.
• EURORDIS Membership Meeting gathering more than 200 patients and patient representatives;
EURORDIS representatives participating in different fora are also valuable means of external dissemination of key messages (over 80 speeches, presentations & meetings attended).
EURORDIS’ annual Activity and Financial Reports are disseminated to all members prior to the General Assembly (AGA), and are put to a vote by the members present at the AGA. The rate of approval is always high: in 2016 97% voted yes with 3% abstaining.
EURORDIS’ has a wide dissemination network and numerous channels through which to reach its relevant stakeholders. This provides a strength in being able to reach out and provide information to a large number of people in various ways. What is becoming a challenge however is tailoring the outgoing information to the specific needs of each stakeholder group individually, an activity that provides not only extra resources but also additional training in the needs and expectations of each group. Recent steps have been taken in addressing various forms of information individually to groups (i.e. the EURORDIS Member News) but further steps need to be taken and are indeed planned for in the upcoming years.