The European Organisation for Rare Diseases – EURORDIS – represents the voice of an estimated 30 million people living with a rare disease in the EU, and their families. The proposal submitted for Operating Grant FPA 2015-2017 is structured around four main objectives of utmost importance for the European rare disease patients’ community:1. To consolidate the Rare Disease patients’ community and strengthen the RDs patients’ voice;2. To actively engage rare disease patients’ representatives into the effective implementation and monitoring of relevant EU legislation / strategies and support their integration at national level;3. To build capacities and empower rare disease patients advocates, members and volunteers;4. To sustain human, financial and organisational resources. EURORDIS achieves these objectives through the following activities: collecting data as well as gathering, disseminating and sharing information; support capacity-building to empower patients’ advocates; providing patients, families, carers, decision-makers and other stakeholders with relevant expertise to inform policy-making; generating EU added-value and support Member States efforts; promoting translational research towards therapeutic intervention for patients; serving as a relay of two-ways information flow between policy makers and rare disease patient groups; promoting access to high quality and safe healthcare services for rare disease patients throughout the EU. Through these activities, EURORDIS will contribute to the implementation of the priorities of the Third Public Health Programme which are to complement, add value and support national policies and strategies implemented by Member States to improve health, reduce health inequalities, support health capacity-building, generate and disseminate health information, contribute to innovative, efficient and sustainable health systems, support cross border healthcare in order to facilitate access to better and safer healthcare.

The Specific Grant Agreement FY2015 n°671358 was granted to the European Organisation for Rare Diseases (EURORDIS) as the seventh operating grant in 2015. Throughout the year, the Operating Grant significantly contributed to the implementation of its Strategy 2010-2015 and the achievement of its Work Plan 2015 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 2015, the Operating Grant allowed for the development of areas which consolidated and expanded since 2009, when the first Operating Grant was awarded. This growth in recurrent core activities 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 2015; 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 2015, as described in the Grant Agreement (page 52); 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).

Activities supported by the OG also covered a number of areas of the Health Programme 2014-2020. In particular, they addressed the following thematic priorities:
• Contribute to innovative, efficient and sustainable health systems:
− Health Technology Assessment – participation in HTA network activities, in the EUnetHTA stakeholder forum (co-chaired by EURORDIS) and preparation with other patients and consumers’ groups the third Joint Action “EUnetHTA“ (for details see question 6).
− Implementation of Union legislation in the field of medical devices, medicinal products and cross-border healthcare.
− European Reference Networks. EURORDIS provide regular information to members and capacity building with dedicated webinars, training and meetings, as well as relevant updates via the eNews and member news. A specific session was organised at the EMM 2015 (May) and a training day was organised in October for the Council of European Federations (CEF) and the Council of National Alliances (CNA) on ERNs addressing the following topics: Overall process for the call and assessment of ERNs; Assessment manual & tool box; Operational criteria; rare disease grouping (as in EUCERD Recommendation addendum); groups of RD where there is already an application under development; how EU

The structure of EURORDIS’ Work Plan 2015 was organised along the following main strategic objectives
A.1. To consolidate the Rare Disease patient’s community and strengthen the rare disease patient voice; A.2. 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; A.3. To build capacities and empower rare disease patients advocates, members and volunteers; A.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 2015 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 membership increased by 7.5% in 2015 with 65 new members bringing the total to 695. 5 country visits to National Alliances took place throughout the year.

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.
In 2015, EURORDIS organised the launch of Rare Diseases International with over 80 participants at the inaugural meeting in Madrid.
- Bilateral partnership agreement with international patient organisations. EURORDIS has signed Memoranda of Understanding with 3 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).
In 2015, EURORDIS signed two further MoUs with: Rare Voices Australia (RVA) & the Russian Patient Union (RPU).
- 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; attendance of the annual IRDiRC conference
A.1.3. Raise public awareness through:
- Rare Disease Day (RDD) 2015 and planning of RDD 2016. 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 2015, the 8th edition of RDD was held on 28 February with over 84 countries participating. The annual RDD Policy Event was held in Brussels with attendance by multiple stakeholders including Commissioner of Health and Food Safety, Vytenis Andriukaitis. The RDD video was produced by EURORDIS and garnered over 1.5 million views on social media.
- European Year for Rare Diseases 2019.
In 2015, EURORDIS continued efforts to build a campaign in favour of the nomination of European Year for Rare Diseases in 2019. A dedicated website section was set up.
A.1.4. Produce, disseminate and exchange information and experiences:
- 29 eNews reports were produced and disseminated to almost 10 thousand subscribers;
- 6 new monthly Member news were produced and disseminated to over 700 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). New website sections added in 2015 included European Reference Networks and International Activities while a dedicated website on Rare Diseases International was also set up. In addition, the website hosted the annual EURORDIS Photo Contest.
- Management of EURORDIS online communications tools including social media (Facebook, Twitter, Flickr, Youtube), streamlined with the EURORDIS website; EURORDIS TV
- Production and dissemination of reference documents, which include the Activity Report 2014 and Financial Report 2014; ECRD brochures for researchers and a fact sheet on declaring financial statements with in-kind support
- Translation activities into the 7 official EURORDIS languages (EN, FR, DE, IT, ES, PT, RU) as well as a further 20 languages for the Rare Disease Day video

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 to be held on 26-28 May with approx. 800 attendees expected.
During 2015, activities related to the organisation of the conference were carried out, including selection of venue, appointment of

The structure of EURORDIS’ Work Plan 2015, found in Annex I, page 21 of the Operating Grant Agreement is organised along the following main strategic objectives
A.1. To consolidate the Rare Disease patient’s community and strengthen the rare disease patient voice; A.2. 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; A.3. To build capacities and empower rare disease patients advocates, members and volunteers; A.4. To sustain human, financial and organisational resources.

For the Strategic Objective “A.1. Consolidate the Rare Disease patient’s community and strengthen the rare disease patient voice”, EURORDIS’ membership base continued to grow in 2015 reaching 695 members by the end of the year, including 36 new members in EU Member States. EURORDIS has members in 65 countries and 26 countries in the EU.
2015 also saw the official launch of Rare Diseases International with over 80 participants at the inaugural meeting adopting the “Joint Declaration: Rare Diseases as an International Public Health Priority” and promoting the longstanding goal of EURORDIS to provide support to rare disease patient groups at international level.

The EURORDIS website, was developed further in 2015. The EURORDIS website has over 300.000 visits per year and is continually updated. New website sections were added in 2015 including European Reference Networks and International Activities while the dedicated Rare Diseases International website was also launched later on this year. A new donation platform was implemented to receive donations over the Christmas period and the Photo Contest section was redesigned for the 2015 edition. The EURORDIS website hosts the annual Photo contest which has seen an increase in its success rate with a great success with 376 photos received from 42 countries

The organisation of Rare Disease Day remains another key EURORDIS activity which in 2015 involved over 80 participating countries; the campaign website had over 150.000 visits in January and February. For the 4th time in a row EURORDIS proceeded with the production of a RDD video which was the most successful one to date. The video was a resounding success, with 1.5 million views on EURORDIS social media alone. The video was translated into 27 languages (up from 21 languages in 2014). As with previous years, EURORDIS hosted a policy event in Brussels on the occasion of Rare Disease Day. The event was entitled: “Rare but Real: Talking Rare Diseases” with attendance from multiple representatives such as the Commissioner of Health and Food Safety, Vytenis Andiukaitis. There were 150 attendants on site and participants from 30 different countries tuned in to the live event in parallel online.

For the Strategic Objective A.2. 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, EURORDIS continued to be extensively involved in the EMA’s activities related to the provision of information to patients and the public about medicines authorised via the centralised procedure. A total of 215 EMA dossiers for public information, including 187 Public Summaries of Opinion of Orphan Designations (PSOs), 9 European Public Assessment Reports (EPARs) and 19 Package Leaflets (PLs), were reviewed by EURORDIS staff members in order to ensure the quality of the information disseminated by the Agency to the general public.
In 2015, EURORDIS also proceeded with the qualitative analysis of the Access Campaign which includes a survey for the purpose of gathering patient experiences regarding access difficulties. The number of responses received exceeded the expectations: 1.961, of which 1.844 from Europe

For the Strategic Objective A.3. To build

There were only minor deviations from expected activities such as the following:
EURORDIS commitment to the International Rare Disease Research Consortium (IRDiRC) was more time-consuming than initially envisaged. This was also due to EURORDIS chairing the Therapies Scientific Committee of IRDiRC.
The newly appointed Commission Expert Group on Cancer Control also went beyond initially predicted scope of work by producing an analysis of the commonalities and differences between rare cancers and rare diseases to provided added insight to the development of policy recommendations.
EURORDIS HTA related activities were also more time for 2 main reasons: increased involvement of patient representatives in SEED due to more pilots performed than those anticipated; and an increase in the intensity of work within the HTA Stakeholder Forum especially due to the fact that EURORDIS was elected as co-chair.
With respect to actions performed related to the European Reference Networks, the technical guidance and capacity building of patient advocates and the interactions with lead clinicians (health professionals) and policy makers at Member State level was slower than anticipated due to delayed implementation.
The new EURORDIS survey programme was also slower in being implemented as more time was spent in the necessary strategic planning for its implementation.

EURORDIS has the potential to reach out to more than 1800 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.

The external dissemination strategy includes the following main elements:
• Dissemination of EURORDIS weekly eNews in 7 languages to almost 10 000 subscribers;
• EURORDIS website available in 7 languages (over 328.000 website visitors/year from over 190 countries) and other web services and communication tools (Facebook groups, RDD website, Youtube, Twitter, Flickr, online communities and newly established discussion groups, etc. = approximatively 800.000 visits/year);
• Large-scale awareness-raising campaign through the annual international Rare Disease Day, organised in 84 countries (in 2015), including the establishment of a dedicated RDD website and broad media exposure; Subscribers for the specific RDD Facebook group: more than 62.000; Followers for the specific RDD Twitter account: more than 18.000;
• 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;
• Publications: Activity Report, fact sheets, brochures, etc.
• EURORDIS Membership Meeting gathering more than 250 patients and patients 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 2015 97% voted yes with 3% abstaining.