operating grant for rare disease associations … · supporting the eurordis therapeutic advisory...

OPERATING GRANT FOR RARE DISEASE ASSOCIATIONS

EURORDIS FY2010

Cont rac t 20093204 F i n a l Re p o r t

A – Techn i ca l Imp lemantat ion Repor t

EURORDIS OPERATING GRANT N°20093204

FINAL REPORT

A. TECHNICAL IMPLEMENTATION REPORT

TABLE OF CONTENTS

A. TECHNICAL IMPLEMENTATION REPORT

1. Executive Summary

2. “Qualitative evaluation of the execution of all tasks mentioned in Annex I of Grant

Agreement 2009 32 04”:

2.1. Introduction to Activity Tables

2.2. Achievement of Strategic Objectives and List of Activities for 2010 (Activity tables)

A. Communication and Information to patients organisations, stakeholders and the public a.1. EURORDIS Website a.2. Electronic Newsletter in 6 languages b. Activity Report, brochures; update of main reference documents on RD; translation c. European RDD 2010 and media monitoring services d. Awareness video & photo contest e. Maintenance of EURORDIS RD community databases f. Strengthening support to more volunteers representing EURORDIS in EU Committees g. Strengthening international dialogue

B. Capacity building for patient representatives 1. Health policy, POs networking and empowerment

a. Outreach to POs and members, dissemination of information, consultation of members, focus on new Member States incl. fellowship b. Support to the EU Network of National Alliances c. Support and strengthening of the Network of EU RD specific Federations d. Fact sheets on RD topics and policy aspects for capacity‐building purposes

2. Support and information services to patients a. Development and access to Respite Care Services and Therapeutic Recreation Programmes b. Support and strengthening of the EU Network of RD Help Lines c. Development of Online Patients Communities services

3. Research and Therapeutics Development

a. Patient involvement in EMEA activities 1. Support to the participation of patient representatives in the EMA Committees (COMP,

PDCO, CAT, PCWP) and in protocol assistance 2. Supporting the EURORDIS Therapeutic Advisory Group (TAG) 3. Monthly reports compiling feedback from each Committee

b. Review and validation of public information on RD therapies c. Supporting the involvement of more RD patient representatives in the OD, PD, ATMP policies and

in drug development 1. Support to EURORDIS Task Forces on Orphan Drugs, Paediatrics, Drug Information,

Transparency & Access (DITA). Survey on compassionate use on OD. 2. Summer School 2010 3. Development of e‐Learning on specific and advanced aspects of drug development,

clinical trials and regulatory affairs d. Support to capacity‐building activities of patient advocates in HTA e. Supporting good practice relations between POs & sponsors on RD clinical trials based on

EURORDIS Charter on Clinical Trials (CCT) f. Promoting RD research policy and patient involvement in research

2.3. Deliverables (outputs)

2.4. Indicators (metrics)

2.5. List of seminars, conferences, etc. indicating the organiser, the number of participants, etc.,

and the travel and subistence costs associated in connection with the implementation of the

programme of activities

1. EXECUTIVE SUMMARY

The Operating Grant “EURORDIS FY2010 – n°2009 32 04” was granted to the European Organisation for

Rare Diseases (EURORDIS) for a second time in 2010. Throughout the year, the Operating Grant

significantly contributed to the implementation of its Strategy 2010‐2015 and the achievement of its

Work Plan 2010 as approved by its members at its annual General Assembly meetings. The Operating

Grant of EURORDIS covers its recurrent core activities with the exceptions of those in the area of

governance and advocacy activities.

The following Report on the technical implementation of the beneficiary’s Work Programme and the

Financial Statements referred to Art. 1.4 and 1.5 of the Grant Agreement1 aim to show how the

activities have been performed and resources allocated. All the activities listed in the Description of

Work (page 27, Annex I of Grant Agreement, “Strategic Objectives and Specific activities for the year in

question”) and all the Deliverables (page 42 of the Grant Agreement) have been achieved in due time

and according to the contract and its amendment.

In 2010, the Operating Grant allowed for the development of areas which, compared to 2009,

significantly expanded. This growth in recurrent core activities stems from EURORDIS’ progressively

increasing outreach to patient associations and communities, successful public awareness raising, as

well as a growing number of technical activities in which to involve patient representatives so to turn

the new EU and national rare disease policy frameworks into reality. This is the case of the

communication activities revolving around the Rare Disease Day 2010; the outreach to patients and

patient organisations and their empowerment; the support to the activities in the therapeutic area;

support to the involvement of volunteers in EU and national policy working groups; support to

international activities.

Notwithstanding, in 2010 EURORDIS was forced to restrain its activities in other sectors to take account

the reduction of the Operating Grant awarded in 2011 (EAHC decision communicated in July 2010). This

reduction affected primarily two areas: the research policy activities (the planned hire of a Research

Policy Manager was put on hold until after 2011) and the support & information services for patients,

including Respite Care Services, Therapeutic Recreational Programmes and the European Network of

Help Lines (following the departure of the Health Policy Project Coordinator, Shane Lynam, in charge of

1 Grant Agreement 20093204 between the European Organisation for Rare Diseases (EURORDIS) and the Executive Agency for Health and Consumers (EAHC).

Operating Grant Agreement n°20093204 Final Report 1. Executive Summary - Page 1

the development of these services, no replacement could be made because of the Operating Grant

reduction in 2011 and potential replacement is on hold until 2012).

Altogether, this showed 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 to reach the Core Values and General Objectives

of EURORDIS, as described in the Grant Agreement (page 25); 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; EU Committee of Experts on Rare Diseases,

Commission Roadmap for the implementation of the Communication and Recommendation on Rare

Diseases).

The qualitative evaluation of the execution of all tasks mentioned in Annex I of the Grant Agreement

is reported in the Section 2 of this Report. In each of the tables describing therein the performed

activities in detail, the analysis of the results achieved is highlighted in a specific box.

For the Strategic Objective “Communication and information to patient organisations, stakeholders

and the public”, the new EURORDIS website, Version 3, has been developed further in 2010 and

achieved a greater turnover of information, new sections were created and tailored to the needs of Rare

Diseases Policy in Europe (National Plans) and aimed to people living with rare diseases (“Share your

story”). The organisation of the Rare Disease Day 2010 “Patients and Researchers: Partners for Life” was

another EURORDIS key activity, which brought rare disease stakeholders together to discuss the future

of rare disease research as well as bringing closer researchers, patient groups and policy makers. It

involved 46 participating countries (50% more than in 2009); the campaign website had 39 000 hits from

56 countries and the media outreach was strong, with an impressive minimum 1800 press clippings;

remarkably, over 1000 events were organised all over the world. The survey “Patients and Research”

was presented at the Brussels event. A video contest has been added this year to the successful photo

contest organised around the Rare Disease Day, as two additional tools to raise public awareness.

10 issues of the electronic newsletter were published in 6 languages (English, French, German, Spanish,

Italian and Portuguese) throughout the year, with a revamped design and layout, tuned to the new

EURORDIS website. The subscription rate went up 10%, with an increased number of subscribers up to

7862, still gathering our main audience of patient organisations. An improved version of the Annual

Activity Report was published and a new presentation brochure was also produced. The entire structure

of information has been revamped and aligned through this brochure, annual report and website, so to

increase understanding, readability and accountability.

The database of EURORDIS RD community has reached 4870 contacts in 3920 organisations, including

1544 patient organisations, 447 of which are members of EURORDIS, the remaining being mostly

members of either National Alliances or European Federations, and some still being isolated groups; this

database is updated every week and the list of members is interfaced with EURORDIS website.


Finally, in 2010, an organisation has been put in place to support EURORDIS’ volunteers representing

EURORDIS in key European Medicines Agency (EMA) and European Commission (EC) Committees,

internal EURORDIS committees and task forces, to support their involvement in the implementation of

the EU pharmaceutical legislations as well as the EC Communication and Council Recommendation on

Rare Diseases and its translation into national policies. A similar organisation is also in place to support

staff and volunteers in fulfilling obligations arising from partnerships with 5 European and international

not‐for‐profit organisations working on transversal issues relevant for rare disease patients (NORD,

EPPOSI, ICORD, DIA, EPF). Of particular note, EURORDIS has signed a long term strategic partnership

with the National Organisation for Rare Disorders (NORD), the US umbrella organisation which is also a

member of our Council of National Alliances; this partnership is based on the respective strategic

reviews of NORD and EURORDIS and on their respective Strategy 2010‐2015; in 2010, several areas of

collaboration are already very active including in public policy, orphan drugs, Rare Disease Day, Patients

On Line Communities, and other areas. In order to reach the Strategic Objective “Capacity‐building for

patient representatives”, three main Objectives have been identified:

“Health policy, POs networking and empowerment”. In 2010, EURORDIS strengthened its outreach to

patient organisations and members, and put in place a range of activities which link up European and

national policies and provide support to members in implementing national policies on rare diseases.

Last year, a growing number of patient representatives (approximately 1200 person/days) from

EURORDIS membership were mobilised to participate to events, conferences, trainings and workshops

organised, building their capacity and acquiring skills. The European Network of 23 Rare Diseases

National Alliances has been consolidated further, with 3 new members joining (after supporting their

creation at national level, during several years) and the organisation of two European Workshops of its

Council of National Alliances, mostly focused on the organisation of Rare Disease Days 2010 and 2011,

on the development of National Plans for Rare Diseases, the support to the organisation of the 15

National Conferences on Rare Diseases in the framework of the EUROPLAN project, and the 5th survey

on orphan drugs availability to patients in Europe organised jointly with 10 national alliances. The

European Network of 30 Rare Disease European Federations or Informal Networks held one European

Workshop of its Council of European Federations, focused on capacity‐building in EMA activities,

cooperation with the European Reference Networks of Centres of Expertise (in preparation of the future

Directive on Cross Border Health Care & Patient Mobility), the Charter of Clinical Trials and organisation

of the Rare Disease Day 2011. The meeting was followed by a one‐day training on how to apply for EU

funds. A pilot “EURORDIS Grant Programme for European Federations” funded 6 meetings of European

Rare Disease Federations; this new and pilot programme is greatly appreciated by European Federations

as an effective and flexible instrument to support and leverage their activities. Five Policy Fact Sheets on

rare disease related topics were developed to provide member organisations and their representatives

with advocacy tools to encourage them to implement the key recommendations of EU rare disease

policy and facilitate their transposition into national plans on rare diseases.

As far as the Strategic Objective “Support and information services to patients” is concerned, the

European Network of Rare Disease Help Lines received 11 applications for membership, organised a

training session for help line respondents and one Workshop, analysed and disseminated the results of


the 2nd Europe‐wide Caller Profile Analysis finalised at the end of 2009. Guidelines for Best Practices for

Respite Care Services (RCS) and Therapeutic Recreation Programmes (TRP) were finalised in early 2010,

which should allow for better quality specialised services and access for patients and their families; a

timely activity as new services are being foreseen within national strategies and plans under

development. Two Policy Fact Sheets respectively on RCS and TRP as well as two other Policy fact Sheets

on National Help Lines and on the European Network of Help Lines were produced. Online information

was updated but unfortunately, these activities were put on hold due to the departure in July of the

responsible Health Policy Project coordinator who could not replaced due to insufficient funds allocated

under the Operating Grant 2011.

The web tool “Rare Disease Communities” was developed jointly with the US organisation NORD, as a

multilingual enabled online social network for patients and families. The platform is organised to host

disease‐specific communities. At present, 2 pilot online patient communities are live and 10 in

development. This new tool and related methodologies to create communities, train and support

moderators as well as to promote good practices for social networking are based on the experience

gained by EURORDIS with 25 patient mailing lists, in partnership with Médicalist. Three Workshops were

held to train volunteer moderators. The functioning of the portal is being boosted up since the hire in

August of a dedicated Online Patient Communities Coordinator. The “Rare Disease Community” tool was

awarded the audience prize “Best Start‐Up Idea of the conference” at the prestigious global Health 2.0

Conference, held in Paris in October 2010.

For the Strategic Objective “Research and Therapeutic Development”, EURORDIS continued its support

to the participation of patient representatives in the Committees (COMP, PDCO, CAT, PCWP) of the

European Medicines Agency’s, cumulating up to 104 days of meeting and 519 dossier examined; the

Therapeutic Action Group (TAG), composed of all EURORDIS representatives in these Committees and

support staff, held monthly conference calls and organised a face‐to‐face meeting in July prior to the

Board of Directors meeting, discussing, amongst other, how to improve collaboration between

committees and communication with EURORDIS Board; 11 monthly Therapeutic Activity Reports were

produced on the activities of the EMA (European Medicines Agency) Committees and EURORDIS’ patient

representatives in these Committees; they were disseminated to all TAG members, Task Forces,

EURORDIS Board and participants to EURORDIS Summer School.

Another major activity was the further consolidation of the three Task Forces involving 34 patient

representatives and volunteers, from different rare diseases and different EU Member states, trained

and active in issues concerning Orphan Drugs, Paediatric Drugs and Drug Information & Transparency &

Access (DITA). The Task Forces held at least one Workshop each, with the DITA holding two. The DITA, in

particular, compiled a survey mapping compassionate use programmes for orphan drugs in Europe,

including existing programmes, national contacts and published literature. The analysis of the results has

been performed and the survey will be completed in early 2011.

EURORDIS is also 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 123 Public

Summaries of Opinion of Orphan Designations (PSOs), 5 European Public Assessment Reports (EPARs)


and 7 Package Leaflets (PLs) were reviewed by EURORDIS staff members, in conjunction with relevant

patient groups when appropriate, in order to ensure the quality of the information disseminated by the

Agency to the general public.

The EURORDIS Summer School, a four‐day session for 34 selected volunteers on drug development,

clinical trials and EU regulatory process, is now a consolidated and consistently successful activity. The

2010 edition focused on extending the competence of advanced patients’ advocates using peer‐based

real case studies from various committees at the EMA, coupled with a first introduction to Health

Technology Assessment (HTA); an e‐learning tool, in English only, is being established based on the

topics discussed at the Summer School using different learning methods. Moreover, five more patient’s

advocates benefited from the training opportunity on HTA last year in February, when they attended the

four‐day HTA Programme organised by the UMIT University in Hall im Tyrol (Austria).

During 2010, EURORDIS continued its engagement to implement its “Charter for Clinical Trials in Rare

Diseases”, which has been signed now by 6 pharmaceutical companies. In the framework of the Charter,

which aims to regulate the relationships between clinical trial promoters and the patient organisation

concerned by their studies, the first two Agreements of Understanding have been signed up.

Altogether, 2010 has been an important year for the recognition of the role of patient organisations in

rare disease research, with the completion and presentation at the Rare Diseases Day of the survey

“Rare Disease Patient Organisations in Research”, performed by EURORDIS and its partner the CNRS‐the

French “École des Mines”.

Based on the initial assessment of potential risks, none has emerged over the year.

A comprehensive set of activity indicators was developed and data were collected throughout the year.

These indicators appear in the description of each activity and in table 2.5 of this Report.

The list of seminars and conferences, the list of travel and subsistence costs and the published

material and website addresses with publicity of Community funded are annexed to this Report.

Altogether the implementation of the work programme played an essential role in the accomplishment

of EURORDIS strategic objectives and paved the way to significant developments in the organisation and

to the reflection which led to the definition of the Work Plan 2011 along the lines of the new Strategy

2010‐2015.

The final financial statements and comments, based on the Grant Agreement and amendment, are

reported separately. The financial statements are accompanied by the audit certificate of Deloitte &

Associates.


2.1. INTRODUCTION TO THE ACTIVITY TABLES

The following Activity Tables represent the core part of the technical implementation report. They are

meant to provide a list of the activities performed by EURORDIS in 2010 in compliance with the

Operating Grant, as well as an overview of the execution of the tasks in the Work Plan 2010 is detailed

activity by activity.

The Activity Tables are indeed based on the structure of EURORDIS Work Plan 2010, which can be found

in Annex I, page 24 of the Operating Grant Agreement. They are therefore organised along the two

Strategic Objectives and, in the second one, three main Objectives laid out therein:

A. Communication and Information to patients organisations, stakeholders and the public

B. Capacity building for patient representatives

1. Health policy, POs networking and empowerment

2. Support and Information service to patients

3. Research and Therapeutics Development

Each Table corresponds to one specific Activity and includes:

Title

Responsible and Supervising Staff

Deliverable(s), i.e. the outputs associated to each activity. Of course, a number of activities have

been performed that are not associated to a specific OPERA Deliverable. In any case, the full list

of Deliverables is provided in this report (section 2.4).

Indicators, which correspond to the metrics associated to each activity. Again, a table with the

overview of all Indicators is provided in section 2.5 of this report.

A Calendar of activities, summarising the main dates of the achievements relevant to the

concerned activity.

A qualitative Description of the Activity, which explains how the activity has been performed.

The Results achieved with each activity, described separately in order to highlight what has

been accomplished thanks to the Operating Grant by the end of the contract period.

Annexes, i.e. the published material related to the described activity, which is listed at the end

of the Activity Table in order to create a link between each activity and the material itself. The

latter is provided separately in the part C of this report, named “Contractual Annexes”.

Operating Grant Agreement n°20093204 Final Report 2.1. Introduction to Activity Tables - Page 1

Activity A.b. Activity Report, brochures; update of main reference documents on RD; translation

Responsible and supervising staff

Responsible: Paloma Tejada

Supervising: Yann Le Cam

Deliverable No specific deliverable is associated to this activity

Indicators • External communication materials: 2

• Press kit: 2

Calendar of activities

• 2009 Activity and Financial Report produced in March 09

• New presentation brochure disseminated in October 2010

• Press Kit for ECRD 2010 Krakow

• Press kit for Inauguration French Rare Disease Platform

Description of the Activity

Produced an improved version of its Activity Report reporting on all activities undertaken by EURORDIS’ staff and volunteers, highlighting main events. The Activity Report included a list of members and funders. The Financial Report included in‐depth analysis of the revenue and expenses of the organisation.

The Activity Report was broken down by area of activity, and the same organisation was used in the ‘What We Do’ section of the website and in the new presentation brochure. The brochure has been printed in English and French and has already been translated into the other languages of the website (Spanish, Italian, German and Portuguese).

The Activity Report was sent out to 800 member and non‐member patient organisations, funders and other public and private stakeholders. The Financial Report was distributed to all members who attended the General Assembly (approx. 100) and to all financial donors.

Sent out a press release for Rare Disease Day to more than 5000 media contacts using PR Newswire and produced two press kits: ECRD 2010 Krakow (in May) and for the Inauguration of the Rare Disease Platform (in October).

Results achieved

Improved communication with members and other key stakeholders of the rare disease community.

Increased awareness about rare disease policy by the general public.

Relevant Annexes

• Activity Report

http://download.eurordis.org/documents/pdf/Activity_Report_EURORDIS_2010.pdf

• Financial Report

http://download.eurordis.org/documents/pdf/3.%20Rapport%20financier%20EURORDIS%282010BD%29.pdf

• Presentation Brochure

http://download.eurordis.org/documents/pdf/EURORDIS_EN.pdf

http://download.eurordis.org/documents/pdf/Activity_Report_EURORDIS_2010.pdf

http://download.eurordis.org/documents/pdf/3.%20Rapport%20financier%20EURORDIS%282010BD%29.pdf

http://download.eurordis.org/documents/pdf/EURORDIS_EN.pdf

Activity A.c. European Rare Disease Day 2010 and media monitoring service




Deliverables • D10: Rare Disease Day 2010 and media monitoring service

Indicators • Number of participating countries: 46

• Number of events: around 1000

• Number of press clippings (on‐line): 1 967

• Number of visits on RDD website: 67 638 from 170 countries

Calendar of activities • On and around 28 February 2010 – Hundreds of awareness‐raising events all across Europe and in many countries outside Europe (USA, Canada, China, Japan, Brazil, Argentina, Australia and others)

• 1 March 2010 ‐ European Workshop “Bridging Patients and Researchers to Build the Future Agenda for Rare Disease Research in Europe”, Brussels

Description of the Activity Rare Disease Day 2010 took place on 28 February 2010. The campaign has been implemented in 46 countries, of which 27 European countries (newcomers Latvia, Lithuania and Slovenia.) EURORDIS and 22 Rare Disease National Alliances, together with 24 patient groups acting as country organisers, mobilised hundreds of patient organisations throughout 5 continents.

In line with this year’s slogan: “Patients and Researchers: Partners for Life!”, EURORDIS organised a workshop bringing rare disease stakeholders together to discuss the future rare disease research agenda in Europe. The event Workshop entitled «Bridging Patients and Researchers to Build the Future Agenda for Rare Disease Research in Europe» took place on March 1st, 2010 at the Centre de Presse International in Brussels. It was co‐organised with E‐RARE (ERA‐Net on rare diseases) in partnership with the European Commission, Orphanet and the EC‐funded project EUROPLAN. 100 participants from 15 countries attended, including European and national research authorities, researchers, patient organisations and industry.

At the workshop, EURORDIS presented the key findings of a survey conducted amongst more than 300 rare disease patient organisations representing 110 rare diseases, which aimed at exploring the role of patients in research and their priorities for the future. The results of this study were widely publicised during Rare Disease Day as part of EURORDIS advocacy efforts to increase investment in rare disease research.

In addition, patient organisations were invited to nominate a scientist, who has helped advance research into their disease, to the Research Hall of Fame. EURORDIS received 46 nominations, which were visible on the RDD website for 10 months.

The RDD 2010 website was created, which contained 46 country pages (including one for Europe), with a calendar of events organised in each country. The website contained a downloadable tool kit with an Information Pack, logo, poster and other materials accessible to organisers; a section for patients to upload photos and videos; press releases for journalists and links to 4 social media platforms (Facebook, YouTube, Flickr, and Tweeter) especially created for the Day.

More than 10 000 fans joined RDD Facebook and 700 people followed the campaign on Tweeter.

Other interesting features this year included: the Photo and Video Contest with 276 and 36 entries respectively (see Activity Table A.d.); a news media feed which displayed media clippings on a loop and ‘Friends of Rare Disease Day’ and a new dedicated section where all Rare Disease Day supporters could register and describe their contribution to the campaign. In total 189 individuals, patient organisations, industry, health professionals, research and public authorities signed up.

800 posters were printed and sent out to National Alliances, European Federations and member patient organisations.

More than 1700 news articles were collected through a media monitoring service.

Results achieved Improved knowledge via public awareness on rare diseases, improved access to information for patient representatives, patients and their families, as well as enhanced patient to patient exchange of information on their disease through social networking and new online tools.

Rare Disease Day 2010 was also the opportunity to establish a dialogue with new stakeholders, like scientists and researchers. The Brussels Workshop, in particular, constituted an important platform for patient organisations, industry and academia to meet up, exchange views and present their latest findings on that status of rare disease research.

Relevant Annexes • Logo

• Poster

• Information Pack

• RDD website

• Agenda RDD 2010, Workshop Brussels

Activity A.a.1. EURORDIS website


Responsible: Denis Costello


Deliverable • D4: Electronic Newsletter and V.3 of EURORDIS website/ V.3 of EURORDIS website

Indicators • Number of website visitors/month: approx. 17 420

• Number of websites linking to EURORDIS website: 14 900

Calendar of activities • Weekly Web Editorial Meeting

• Monthly insertion of new articles in 6 languages

• Regular updates of Events calendar for both EURORDIS Membership and the rare disease community

• March 2010 ‐ Meeting with EUROPLAN Coordinator to determine concept for new website section dedicated to European National Plans

• June 2010 ‐ Call for Tender to source developer to design a “Share your story” function aimed at patients and families

• All year long ‐ Invitation to Key Opinion Leaders within the rare disease community to share opinion and participate in online debate by blogging on rarediseaseblogs.net

• November/December 2010 ‐ Advertising campaign on Google to promote eurordis.org

Description of the Activity The EURORDIS website provides information relating to the role of patient organisations in the development of rare disease and orphan drug policy in patient‐friendly language translated into 6 languages, while also outlining the activities provided by EURORDIS.

During 2010 the EURORDIS web‐team sought and achieved a greater turnover of information on the website. The team also developed new website sections tailored to meet the needs of the evolving situation of rare disease policy in Europe. In particular, a section on the activities carried out to promote National Plans in the context of the EC‐funded project EUROPLAN, was added.

Work was also completed in optimising existing features within the site, namely the signup process to the EURORDIS Newsletter, which had been judged to be slightly cumbersome. A daily automatic synchronisation with the in‐house contact database (previously a manual task) provided a time and cost saving exercise.

An openness to content written from outside the website was also incorporated by developing a real time linkup with the EURORDIS sponsored blogging platform rarediseaseblogs.net.

Results achieved • Weekly updates of the homepage and events calendar to reflect activities of EURORDIS and the rare disease community.

• New website section providing access to key documents and details relating to National Plans for Rare Diseases in European Member States.

• Daily update of Membership database from in‐house CRM Contact Database.

• Development of a “Share your story” feature aimed at patients and families living with a rare disease.

• Optimisation of Newsletter signup process.

• Integration of opinion from Key Opinion Leaders via RSS feed from rarediseaseblogs.net

• Improved presentation of financial reporting to reflect greater transparency of funding sources

Relevant Annexes • EURORDIS website: www.eurordis.org

http://www.eurordis.org/

Activity A.a.2. Electronic Newsletter




Deliverable • D4: Electronic Newsletter and V.3 of EURORDIS website

Indicators • Number of subscribers: 7862

• Number of issues: 10

Calendar of activities • Issues sent out in Jan, Feb, Mar, Apr, May, Jun, July, Oct, Nov, Dec

Description of the Activity The activity entails a monthly publication of an electronic newsletter in 6 languages (English, French, Spanish, Italian, German and Portuguese). The purpose of the e‐Newsletter is to generate information for the rare disease community and extend EURORDIS' outreach to rare disease patients and/or their representatives. More specifically, the Newsletter aims to:

• Encourage patient groups to learn from the experience of one another;

• Build groups' capacity to support research projects or to advocate for better healthcare at European and national levels;

• Promote networking and joint projects across Europe and across diseases.

The e‐Newsletter content links to the EURORDIS website and other external resources. It includes in average 1 editorial, 4 main articles (1 1 article on the news, 1 insight, 1 special report and 1 on “leaving with RD”), 1 news in brief, 1‐2 announcements, events, calendar, orphan drug designations and market authorisations.

The e‐Newsletter design and layout changed in January 2010 in accordance with the new design and layout of EURORDIS’s website. The revamping of the e‐Newsletter also includes new features: RSS feed, PDF download, multimedia (photos on the newsletter itself, embedded videos and links to the EURORDIS YouTube Channel). Also, more links to other EURORDIS web services, such as the On‐Line Patient Communities and the blog EURORDIS‐NORD (US National Organization for Rare Diseases), were made. Finally, the signup process to the e‐Newsletter, judged slightly cumbersome, was optimised.

In 2010, 10 issues of the electronic newsletter were produced (no publication in August and September).

Results achieved • Growing number of subscriptions from around the globe, especially from Eastern Europe and Latin America

• Increased traffic to EURORDIS’ website and to other EURORDIS‐managed websites such as: ECRD 2010 (European Rare Disease Conference) in Krakow, Rare Disease Day, On‐Line Patient Communities, Rare Disease Blog, EuroBioBank.

• The e‐Newsletter subscription rate went up 10 % in 2010 compared to 2009 (1480 French, 1022 Spanish, 479 German, 844 Italian, 447 Portuguese), leading to a total of 7862 subscriptions.

• The EURORDIS e‐Newsletter and the related Website have become one of the two major sources of information about rare diseases, rare disease policy and best practices, along with OrphaNews Europe published by Orphanet. It is also one of the main, if not the most comprehensive source of information in Europe about patient groups, patient advocacy, capacity building and patient testimonies.

• Articles from EURORDIS e‐Newsletter are often reproduced and reused by patient organisations in their own printed or electronic newsletters.

Relevant Annexes • 1 example of E‐Newsletter (email)

Activity A.d. Photo and Video Contest




Deliverable • D10: Rare Disease Day 2010

Indicators • Number of photo entries: 272

• Number of countries represented: 34

• Number of diseases: 80

• Number of video entries: 36

• Number of countries represented: 25

• Number of diseases: 21

• Number of viewings of videos: 35 906

• Number of visits to winning video: 10 670

Calendar of activities • Contest launched in November 2009

• Contest closed on 31 March 2010

Description of the Activity EURORDIS Photo & Video Contest was held during the Rare Disease Day campaign from November 2009 to March 2010. The contest was promoted through the EURORDIS website (www.eurordis.org) and the Rare Disease Day website (www.rarediseaseday.org). People from all over the world, representing a variety of rare diseases, ‘told their story’ with the help of a photo or video camera.

The contest attracted 272 photo entries and 36 video entries. The entries were submitted via a Flickr photo wall and YouTube. People from all over the world, representing a variety of rare diseases, ‘told their story’ with a little help from a camera.

Photo contest. Photo submissions were received from 34 different countries, and showcased over 80 different rare diseases, as well as the activities of rare disease patient groups and alliances across Europe and the Americas.

The photos have been viewed more than 36000 times. Viewers connected from around the globe: India and China, the Americas, and especially from every country in Europe.

All of the photos submitted to the contest can be found in the Rare Disease Day photo gallery: http://www.rarediseaseday.org/yourstory/gallery.

The photo of two women living with SMA (Spinal Muscular Atrophy, Type IIa) was chosen as the winner by a selected jury of 10 persons. Ilona Brandt shares a moment of high spirits between herself and her twin sister, Claudia, as they cool their toes in a fountain. The winning photo can be found on the EURORDIS website: http://www.eurordis.org/content/winners‐2010‐photo‐video‐contests

Video contest. The winning video, entitled “Garret’s story”, features

http://www.eurordis.org/

http://www.rarediseaseday.org/

http://www.rarediseaseday.org/yourstory/gallery

http://www.eurordis.org/content/winners-2010-photo-video-contests

the daily life of an Epidermolysis Bullosa sufferer. Garrett’s story was the most visited video receiving more than 10,000 visits in five months. The winning video can be found on the EURORDIS website: http://www.eurordis.org/content/video‐contest‐winner‐rare‐disease‐day‐2009

Video submissions were received from more than 25 different countries, and showcased more than 21 different rare diseases, as well as the activities of rare disease patient groups and alliances across Europe and the Americas.

The videos have been viewed more than 35 000 times. Viewers connect from around the globe, from India and China, the Americas and especially from every country in Europe.

All of the videos submitted to the contest can found on EURORDIS’ Rare Disease Day channel of YouTube: http://www.youtube.com/rarediseaseday.

Results achieved • Improved public awareness on rare diseases via photo and videos.

• Enhanced patient‐to‐patient exchange of information on their disease through social networking and new online tools.

Relevant Annexes • Winning photo, and information about the winner: http://www.eurordis.org/content/eurordis‐2011‐photo‐and‐video‐contest

• All photos: http://www.rarediseaseday.org/yourstory/gallery • Winning video, and information about the winner:

http://www.youtube.com/watch?v=0TISlcYhsU8

• All videos: http://www.youtube.com/rarediseaseday

http://www.eurordis.org/content/video-contest-winner-rare-disease-day-2009

http://www.eurordis.org/content/video-contest-winner-rare-disease-day-2009

http://www.youtube.com/rarediseaseday

http://www.eurordis.org/content/eurordis-2011-photo-and-video-contest

http://www.eurordis.org/content/eurordis-2011-photo-and-video-contest

http://www.rarediseaseday.org/yourstory/gallery

http://www.youtube.com/watch?v=0TISlcYhsU8

http://www.youtube.com/rarediseaseday

Activity A.e. Maintainance of the EURORDIS RD community database


Responsible: Patrice Regnier

Deliverable D3: Update of EURORDIS RD community database

Indicators • Number of entries in the patient organisation section of the contact database: 1544

• Number of entries in the member organisation section: 447

• Contact database entries: 4870 contacts, 3920 organisations

Calendar of activities All year ‐ ongoing

Description of the Activity The community database gathers data of 4870 contacts in 3920 organisations, 8000 diseases, 1544 patient organisations, including 447 EURORDIS member organisations, 20 EURORDIS projects and 52 events.

The database allows:

‐ E‐mailing or paper mailing to all contacts or sub‐groups of contacts

‐ Search of patient groups or experts by disease

‐ Direct link to Orphanet database by disease

‐ Follow‐up of member fees payments & statistics

‐ Recording of participation of contacts in events & projects, including role (i.e. speaker, chair, participant, etc.)

‐ Recording of activities performed (i.e. mailing, meeting, trainings, etc.)

The database is used and updated daily by all EURORDIS staff members.

Results achieved • Update of the emailing system and adaptation to the website section

• Enhancement of the ergonomics of the application.

Relevant Annexes • Membership section of database: http://www.eurordis.org/membership

http://www.eurordis.org/membership

Activity A.f. Strengthening support to volunteers representing EURORDIS in EU Committees


Responsible: Ariane Weinman


Deliverable No specific deliverable is associated with this activity

Indicators • Volunteers total number in 2010: 57 (except volunteer translators)

• 5 volunteer translators (who work on regular basis)

• Volunteers in projects: 11

• Volunteers involved in EURORDIS Task Forces: 34

• Number of presentations at European, international conferences and workshops: 46

Calendar of activities • 16 December 2009 ‐ EURORDIS’ application to the EU Committee of Experts on Rare Diseases (EUCERD)

• 27 July 2010: Commission’s decision on the appointment of 8 patient representatives (4 full members and 4 alternates) on the EUCERD

• 9 – 10 December 2010: First meeting of the EUCERD. All the 8 patient representatives attended.

• Regular exchanges and consultations throughout the year amongst the volunteers members of the EURORDIS European Public Affairs Committee (EPAC)

• 15 EUROPLAN national conferences on national plans and strategies for rare diseases

• Participation to 63 conferences throughout the year

Description of the Activity As stated in its mission, EURORDIS represents the rare disease patients’ voice at the EU level, and consequently in EU Institutions, European and international conferences. One essential way EURORDIS does it as European umbrella is to have spokesperson from different countries and diseases.

One specific challenge is to identify highly qualified volunteers who can become strong European patient advocates to further develop initiatives and activities aimed at improving the life of rare disease patients and their families at the EU and national levels.

These volunteers must: a) be committed to the cause of rare diseases, b) master English, c) be able to get involved in high‐level EU committees and/ or conferences and speak on the same level with other healthcare stakeholders (ex: policy makers, academia, industry).

EURORDIS insists on the quality and the commitment of its volunteers rather than on the number of volunteers. A policy to select volunteers has been established and an organisation put in place to support them.

EURORDIS volunteers are considered as experts. Since the creation of the organisation, they have greatly contributed to its recognition in the EU rare diseases policy‐making scene.

Profile of EURORDIS’ volunteers:

Due to EURORDIS’ advocacy activity at the EU level, our volunteers naturally share the same profile:

‐ the rarity of the disease leads affected people to become experts of their disease. Therefore, most of EURORDIS’ volunteers are either patient or parent of patient;

‐ by being an expert of their own disease, our volunteers are in the best position to advocate for better healthcare services and access to drugs for patients;

‐ the volunteers have a good (and sometimes excellent) level of spoken and written English;

‐ the volunteers have followed the same evolution: they first got involved in the activities of their own disease’s association, then they moved to national activities (National Alliance) and eventually joined EURORDIS. They go beyond their own suffering to help other rare diseases’ patients all over Europe and even worldwide ;

‐ all the volunteers at EURORDIS have a university degree. They have a solid educational background (BA or MA, MS, MD, PhD) allowing them to learn about European policies and EU institutions;

‐ Lastly, about more than a half of EURORDIS’ volunteers has slowed down his/her professional activity (due to the impact of the disease on their life).

Representation of EURORDIS in key EMA and EC Committees (see also Activity Table B.3.a.1. )B.3.a.1. B.3.a.1.

Over the past ten years, EURORDIS has always been able to identify high‐level volunteers able to represent the rare disease patients’ community on EMA and EC committees. EURORDIS selects potential representatives based on a call for expression of interests. Our strict rules have enabled us to propose good candidates to EMA and EC Committees. As a result, these candidates have been nominated.

Volunteer representatives in 2010:

‐ EMA Committee for Orphan Medicinal Products: Two EURORDIS representatives out of three patients’ representatives: Ms Lesley Grenne and Ms Birthe Byskov Holm, who is the Vice‐Chair of the COMP. Dr Maria Mavris (staff) is an Observer.

‐ EMA Committee for Paediatric Drugs: One EURORDIS’ representative out of three patients’ representatives: Dr. Tsveta Schyns.

‐ EMA Committee on Advanced Therapies: One EURORDIS’ representative, Dr Fabrizia Bignami (staff), and her alternate Dr. Michele Lipucci di Paola (volunteer) out of three patients’ representatives.

‐ EMA Patients’ and Consumers Working Party: The Co‐Chair, Ms Lise Murphy, is a volunteer EURORDIS’ representative. Mr François Houÿez (staff) also seats on the EMA PCWP.

‐ EU Committee of Experts on Rare Diseases: The 8 seats (four full members and four alternates) for patients are hold by EURORDIS’ representatives (selected based on their skills, advocacy track

records, representativeness of large disease groups as well as geographic outreach). Mr Le Cam is the only staff member, other representatives are volunteers: Ms Dorica Dan, Mr Torben Gronnebaek, Ms Christel Nourissier, Mr Jan Geissler, Mr Gabor Pogany, Ms Bianca Pizzera and Mr Alastair Kent.

It is important to underline that alternates’ work is as important as full members’.

EURORDIS organisation to support volunteers involved in committees and in other advocacy activities

EURORDIS has established different volunteer groups, coordinated by EURORDIS staff. These groups are aimed at supporting the work performed by EURORDIS’ volunteers in EU committees as well as developing initiatives in favour of rare disease patients in different fields (therapeutics, public health, social services…)

‐ The Therapeutic Action Group (TAG): This group is composed of the 8 representatives in EMA scientific committees. They have monthly conference calls to discuss on the work performed in their respective committees and to share relevant information. The TAG is coordinated by Dr. Maria Mavris, Drug Development Manager.

‐ The Policy Action Group (PAG): Composed by all the members of EURORDIS representatives on the EUCERD (seven volunteers and our CEO, Mr Yann Le Cam), Ms Flaminia Macchia, European Public Affairs Director and Ms Ariane Weinman, Volunteers and NGO Partners Coordinator. The group organises regular conference calls to better follow up the work of the EUCERD.

‐ The European Public Affairs Committee (EPAC): This internal committee plays an active and key role in EURORDIS’ governance and advocacy activities. It is composed of 30 members: 18 volunteers (Board members, TAG members and full members representing patients on the EUCERD), as well as 12 staff members (our CEO, Directors and managers). Each EPAC member has a specific mandate to represent EURORDIS in institutions and in conferences.

The EPAC members communicate regularly by emails and discuss important issues related to care for rare disease patients such as trans‐border healthcare directive, information to patients, etc… The exchanges of opinions are coordinated by Flaminia Macchia.

Each EPAC member is entitled to vote for the adoption of a position of EURORDIS on specific issues.

‐ The EURORDIS Task Forces:

EURORDIS has established three internal Task Forces:

a) Orphan Drugs Task Force (coordinated by Dr. Maria Mavris)

b) Paediatric Drugs Task Force (coordinated by Dr. Maria Mavris)

c) Drug, Information, Transparency and Access (DITA) Task Force

(coordinated by Mr François Houÿez, Health Policy Officer)

These EURORDIS Task Forces comprise 34 members who all are volunteers and mainly patients or parents of patients. They have been recruited based on a call for expression of interest. The calls to participate in the Orphan Drugs and Paediatric Drugs Task Forces were launched after the EURORDIS Summer School (cf. B.3.C.2) in 2009.

Each Task Force has its own agenda. The volunteers involved also support the work performed by other EURORDIS volunteers in EMA scientific committees. As an example, the members of the DITA Task Force provided their inputs for EURORDIS’ comments on the “EMA Reflection Paper on ethics of clinical trials in third countries”.

European Council Recommendation and National Plans and Strategies on Rare Diseases

In addition, the work performed by EURORDIS’ volunteers on rare diseases at the European level is crucial to translate the EC Commission Communication and the Council Recommendation on Rare disease into national policies and plans for people living with a rare disease. We have witnessed that volunteer patient advocates have been able to catalyse the participation of all stakeholders in strategies for rare diseases at the national level. This is one of the key success factors to develop a sustainable national policy.

In 2010, EURORDIS coordinated the organisation of 15 national conferences on national plans for rare disease within the framework of EUROPLAN, a DG Sanco co‐funded project. Mr Yann Le Cam and Ms Valentina Bottarelli, European Public Affairs Advisors, teamed up with six Advisors, patient advocates, who were in charge of coordinating two to three national conferences. A portion of their time was financially compensated to their respective National Alliance, within EUROPLAN’s budget. However, these 6 Advisors spent much more time in liaising with different National Alliances, between them and with the staff, with EUROPLAN project leaders etc…

Results achieved • EPAC Communication:

Over 80 exchanges of emails (apart from consultations)

6 Consultations: ‐ Why Research on Rare Diseases (3 March)

‐ EMA new Road Map (16 march)

‐ EUROPLAN ‐New draft of the EUROPLAN

Recommendations (22 March)

‐ Information to Patients ( 23 march)

‐ Letters of support to the adoption of National

Plans on Rare Diseases (7 October )

‐ Cross‐Border Healthcare (October)

• EUCERD: The EC appointed the 8 EURORDIS candidates to represent patients with rare diseases on the EUCERD (July 27 2010)

• EURORDIS’ volunteer representatives in European / International conferences:

‐ In 2010, EURORDIS sent representatives in 49 conferences

‐ 12 volunteers represented EURORDIS, made presentations, co‐

chaired sessions, participated in panels (some of them participated in

several conferences)

Relevant Annexes • List of EPAC members

• List of participation of EURORDIS’ representatives in conferences in 2010

• EURORDIS’ application letter to the EU Committee of Experts on Rare Diseases (EU CERD)

• EURORDIS' application to EUCERD_ Memorandum Eurordis

Activity A.g. Strengthening international dialogue


Responsible: Ariane Weinman



Indicators • Number of presentations at European, international conferences and workshops: 46

• EURORDIS sent representatives in 49 international conferences

Calendar of activities • 22nd Annual DIA EuroMeeting, March 8th‐10th, Monaco

• 6th International Conference on Rare Diseases and Orphan Drugs (ICORD), March 18th‐20th, Buenos Aires

• NORD Corporate Council, May 18th, Washington D.C.

• DIA Management Board meeting, June 12th, Washington D.C.

• EPPOSI HTA closed workshop: June 29th, Brussels

• DIA Orphan Drugs Conference, November 3rd, Paris

• DIA 2nd Health Technology Assessment (HTA) conference, November 4th and 5th, Paris

• EPPOSI HTA open workshop: November 17th, Brussels

• 11th EPPOSI workshop on partnering for rare disease therapy development, November 29th and 30th, Prague

• EPPOSI 2nd workshop on Chronic Conditions, December 9th, Brussels

• “Fifth Eastern European Conference for Rare Diseases and Orphan Drugs “Rare Diseases in the Focus of Personalized Medicine” &

The First All‐Russian Conference for Rare Diseases and Rarely Used Medical Technologies “Lifeline”, Saint‐Petersburg, Russia, July 2‐4

• First International South Caucasian Conference on Rare Diseases and Orphan Drugs Yerevan, Armenia, Осtober 7‐8

Description of the Activity EURORDIS has developed partnerships with several European and international not‐for‐profit organisations to work on transversal issues relevant for patients affected with rare diseases.

The partners are:

‐ NORD – The US organisation for rare disorders

‐ EPF – The European Patients’ Forum

‐ EPPOSI – The European Platform for Patients’ Organisation, Science and Industry

‐ DIA – Drug Information Association

‐ ICORD – The International Conference on Rare Diseases and Orphan Drugs

The staff and EURORDIS volunteers involved in NGO partners’ activities are supported by Ariane Weinman.

NORD:

Since its creation in 1997, EURORDIS has established a longstanding relationship with NORD to join efforts in improving the life of rare disease patients on both sides of the Atlantic.

NORD is a member of the EURODIS Council of National Alliances of rare disease patient organisations.

In 2009, Yann Le Cam and Peter Saltonstall, President and CEO of NORD, decided in liaison with each organisation’s Board, to further strengthen our partnership.

This partnership is aimed at converging strategies between the two organisations for the period 2009 – 2015. Each organisation brings mutual support to develop the following activities:

‐ Exchange of information and advice on EU and US national policies on orphan drugs and rare diseases;

‐ Raise international awareness of rare diseases as a public health priority on the occasion of the Rare Disease Day;

‐ Develop online rare disease patients’ communities with a common strategy as well as co‐branded blogs;

‐ Exchange on orphan drugs development in the EU and the US, work on the harmonisation of marketing authorisation process for ODs; work on the report of the US Institute of Medicine on ODs development together with the NIH and FDA;

‐ bring together more closely the NORD Corporate Council and

the EURORDIS Round Table of Companies (ERTC); coordinate the topics addressed, cooperate to promote companies

membership in each other's forum.

EURORDIS advised NORD to create a US conference on rare diseases and orphan drugs based on the model of the European Conference on Rare Diseases (ECRD). NORD eventually established a partnership with the DIA to organise a global conference on rare diseases. The conference will be taking place on October 11 through 13 2011 in Washington D.C.

EPF: Mr. Anders Olauson, former EURORDIS Board member, has been officially appointed by our Board to represent EURORDIS in EPF.

Mr. Olauson is the current President of EPF. He was the co‐founder of Agrenska in Sweden, a centre for disabled people, and was EURORDIS’ president from 1999 to 2001.

EURORDIS works on transversal issues with EPF: EU Directive on cross‐border healthcare; information to patients; animal welfare; EMA Reflection Paper on Ethical and GCP Aspects of Clinical Trials Conducted in Third Countries. The members of the EPAC (see Activity Table A.f) exchange their views and opinions on these issues.

EPPOSI: Mr Yann Le Cam is a Board member of EPPOSI and the Treasurer. EURORDIS participates in the governance of EPPOSI and provides inputs on its strategy.

In 2010, EURORDIS was involved in the Organising Committees of all the EPPOSI workshops. Ariane Weinman provides support to EURORDIS’ representatives in Organising Committees and identifies EURORDIS members who can attend EPPOSI workshops.

Health Technology Assessment ‐ Brussels:

Mr Yann Le Cam participated in the closed workshop (June). In the open workshop (November), he chaired the session on: “How should we engage with patients to ensure that HTA is truly patient‐centred?

28 patient representatives attended the HTA open workshop, included 4 staff members and 15 members of EURORDIS.

11th workshop on Rare Diseases ‐ Prague:

Ms Lesley Greene (volunteer, parent of patient) represented EURORDIS in the Organising Committee and was Co‐Chair of the workshop. Ms Greene also represents EURORDIS on the EMA Committee for Orphan Medicinal Products and served as EURORDIS President from 2001 to 2003.

Ms Greene chaired the session on: “Regulation: The EU and Beyond – Developing Future Regulations and Policies for Better Rare Diseases Therapies”. Mr Le Cam made a presentation on “Developing new tools for coordination between Member States: Clinical Added Value of Orphan Drugs”. 43 rare disease patients’ representatives attended the workshop out of 106 participants. 2nd workshop on Chronic Conditions, Brussels: Dr Bernd Quadder, volunteer and patient, represented EURORDIS in the Organising Committee. Dr Frank Brunsmann, volunteer and patient, made a presentation on behalf of EURORDIS on: “The EURORDIS View on Electronic Health Records (EHR): Draft EHR checklist”. 15 patients’ representatives were invited by EPPOSI to attend this meeting.

DIA: EURORDIS (Yann Le Cam) and the DIA (Dr Yves Juillet) established “The DIA patient fellowship programme”. This programme provides 47 fellowships to European patient advocates across diseases to speak in sessions or attend the DIA Annual EuroMeeting. The successful fellows are selected based on their involvement in clinical trials and advocacy track records. At the EuroMeeting 2010 in Monaco, 21 members of EURORDIS attended this Forum as fellows. The special DIA booth for patients was manned by Ariane Weinman. The participation of patients was a great success. They are now recognised as equal stakeholders by the representatives of industry and health national agencies.

ICORD:

The ICORD mission is to improve the welfare of patients with rare diseases and their families world‐wide through better knowledge, research, care, information, education and awareness. Its brings together renowned international rare diseases’ organisations.

EURORDIS brings its longstanding experience as a patient organisation involved in the field of orphan drugs and rare diseases policy making at the European level.

EURORDIS and NORD have both agreed to play pivotal and coordinated role in ICORD to help expand the movement of rare diseases / disorders at the international level. In particular, they will collaborate to develop the ICORD Work Programme, the ICORD Position Paper of "Rare Diseases: an International Public Health Challenge", and the international network of patient organisations.

Beyond EU:

EURORDIS supported new patients groups in Russia, Armenia and Georgia to organise international rare diseases awareness‐raising conferences in their respective countries. EURORDIS’ representatives attended conferences showing our strong support on a long term basis.

Results achieved • Successful workshops and great participation of patients’ representatives

• Greater visibility of patients’ representatives in international conferences and workshops

• Common work with NGO Partners to produce recommendations on healthcare policies relevant for people affected with rare diseases

Relevant Annexes • Memorandum of Understanding: Strategic Partnership between NORD and EURORDIS – July 4th 2009

• DIA EUROMEETING 2010 – March 8th ‐10th , Monaco, summaries of sessions from the Patient Fellowship “Report Team”

Activity B.1.a. Outreach to POs and members, dissemination of information, consultation of members, focus on new Member States

Responsible staff, supervising staff

Responsible: Anja Helm

Supervising: François Houyez


Indicators • Total number of members: 447

• New members: 44

• Number of members in EU MS and candidates EU MS: 29

• Internal consultations with members: 14

• Lost members: 3

Calendar of activities All year – ongoing

Description of the Activity EURORDIS is a grassroots movement of patient organisations active in the field of rare diseases. The membership of EURORDIS (447 patient organisations) is involved in the organisation’s activities and in its decision‐making process through regular mailings sent out to all members, surveys (on‐line surveys or simple e‐mail consultations) and face‐to‐face meetings. EURORDIS staff and volunteers participate in a great number of conferences and meetings across Europe and notably in the new Member States (on volunteers, see also Activity Table A.f).

Patient representatives attend EURORDIS events such as: the General Assembly, the European Conference on Rare Diseases, the Summer School Training and different workshops, such as the Council of National Alliances, the Council of European Rare Disease Federations, workshops of the European Round Table of Companies or workshops for Online Communities.

For the majority of workshops and trainings, patient representatives travel and hotel expenses are covered.

The European Conference on Rare Diseases 2010 (ECRD 2010) that took place in Krakow in May 2010, offered 40 fellowships to patient representatives from new Member States, allowing them to participate, which otherwise would have been quite impossible.

The 3 withdrawn members are

‐ German OI Society (withdrawn because their European federation is a member already and the same person heads the two organisation)

‐ GOLD (closed completely end of 2009 for lack of funding)

‐ Asociacion Hemofilia, de Madrid (withdrawn for financial reasons)

Results achieved • A growing number of patient representatives attend conferences, workshops and trainings, building their capacities and acquiring skills.

• EURORDIS membership grows by an average of 13% per year.

• Not less than 1200 person/days from EURORDIS membership were mobilised to participate to events, conferences, trainings and workshops organised throughout the year.

Relevant Annexes

Activity B.1.b. Two European Workshops of National Alliances of Rare Diseases



Supervising: François Houyez, Yann le Cam

Deliverable D2: Workshops of the EU Networks/ 2 Workshops of the CNA

Calendar of activities • 13 May 2010 ‐ One‐day CNA Workshop, Krakow

• 12 December 2010 ‐ One‐day CNA Workshop, Paris

Description of the Activity National RD Alliances serve to bring together all the many RD organisations in a particular country. The CNA (Council of National RD Alliances), established by EURORDIS, allows national representatives of rare disease patients to work together on common European actions. Currently, 17 EU and 3 non EU countries participate in the CNA.

In 2010, the CNA worked to support its members 1) in the elaboration of national plans on rare diseases; 2) the organisation of the national conferences (under the EUROPLAN project); and 3) the organisation of the Rare Disease Day.

In 2010, two CNA Workshops have been held.

The Workshop of 13 May 2010, held in Krakow at the occasion of the European Conference on Rare Diseases (ECRD 2010), gathered 27 representatives of National Alliances. The following topics were discussed:

‐ Feedback from Rare Disease Day 2010 and preparation of Rare Disease Day 2011

‐ National plans: EUROPLAN update and preparation of national conferences

The Workshop of 12 December 2010 gathered 18 representatives and focused on the following topics:

‐ Cross‐Border Healthcare Directive

‐ National plans & the EUROPLAN project: outcomes of the national conferences

‐ Rare Disease Day 2011 – preparation

‐ Survey on orphan drug availability: collaborative work of EURORDIS and 10 National Alliances

Results achieved • Involvement of patient organisations in the development of national plans and strategies for rare diseases, through participation of 15 National Alliances in EUROPLAN and the development and organisation of national conferences.

• Networking and information sharing at European level.

• Increasing awareness at the national level on RD via the Rare Disease Day.

• Development of real collaborative work between National

Alliances, as in the Survey on orphan drug availability, Rare Disease Day and in the support to organise national conferences on national plans on rare diseases.

Relevant Annexes • Report of the CNA Workshops of 13 May 2010

• Report of the CNA Workshops of 12 December 2010

Activity B.1.c. Support and strengthening of the network of EU RD specific Federations



Supervising: François Houyez, Yann le Cam

Deliverables • D2: Workshops of the EU Networks/ Workshop of the Council of European RD Federations

• D5: Wiki guide for EU RD Federations

Indicators • Participants to a one‐day Workshop and a one‐day training: 95

Calendar of activities • 1 July 2010 ‐ Workshop of the Council of European RD Federations (CEF)

• 2 July 2010 ‐ Training session “How to apply to EU Funds in the field of Rare Diseases?”


Representatives of European Rare Disease Federations gathered in Paris in July 2010 to discuss issues that are important across Europe and across diseases:

- Why & how involve your patient organisation in EMA activities

- Rare Disease Day 2011

- EURORDIS support to European Federations

- Collaboration with European Reference Networks

- Clinical Trials Charter

The next day, the representatives participated in a one‐day capacity building session entitled “How to apply to EU Funds in the field of Rare Diseases?”

In 2010, EURORDIS launched as well a pilot project in support of European Rare Disease Federations, called “EURORDIS Grant Programme for European Federations”. The smallest and/or youngest of such organisations often have great difficulties in financing their network meetings (Board meetings, network meetings, conferences, etc.).

Through this pilot programme, EURORDIS granted 6 European RD Federations financial support to help them organise their different meetings. A total of 15.000 € has been granted for 6 network meetings:

- South Eastern European CF conference ((3000 €)

- Board meeting of the European Network for Ichthyosis (472 €)

- European Aniridia Network meeting (1200 €)

- Osteogenesis Imperfecta project committee meeting (1400 €)

- Euro‐HSP 1st General Assembly preparatory meeting (3000 €)

- European congenital Heart Diseases 1st General Assembly (3000 €)

Finally, the wiki‐like guide http://raretogether.eurordis.org has been redesigned and updated. This website was created in the framework of

http://raretogether.eurordis.org/

the Rare!Together mentoring project, which aims to help in the creation, operation and management of European Rare Disease Federations. This knowledge base shall become a good practice reference handbook and a toolkit reaching out to patient organisations planning to set up their European Federation, as well as to existing European Federations.

Results achieved • European Federations have benefited from sharing experiences at the Workshop and building their capacities in raising funds.

• The 6 beneficiaries of the pilot project were able to set up meetings which otherwise they could not have organised; they are very satisfied with the results.

• The wiki‐like website helps European networks across Europe develop their Federation.

Relevant Annexes • Report of the Council of European Federations Workshop

• Programme of Support to European Rare Disease Federations ‐ Terms & Conditions

Activity B.1.d. Fact Sheets on RD topics and policy aspects for capacity‐building purposes


Responsible: Anna Kole



Indicators • Number of Fact Sheets produced: 5

• Number of Fact Sheets distributed: approximately 5000

• Number of visitors to EURORDIS website page dedicated to Factsheets: approximately 400

Calendar of activities • Publication of Fact Sheets:

- Respite Care Services (May 2010)

- RD Research (June 2010)

- Therapeutic Recreation Programmes (November 2010)

- Orphanet (November 2010)

- Registries (December 2010)

• Distribution to participants of the European Conference on Rare Diseases, ECRD (May 2010), the Council of European RD Federations, CEF (July 2010), the Council of National RD Alliances, CNA (Dec 2010) and EUROPLAN conferences (May – Dec 2010).

Description of the Activity To provide the Council of National Alliances, the Council of European Federations, and individual patient groups and representatives with advocacy tools encouraging the implementation of key recommendations in the European Commission’s Communication on Rare Diseases & Council Recommendation on Rare Diseases, and facilitating their transposition into national plans and strategies on rare diseases.

Specific objectives include:

• Create Fact Sheets with input from patient advocates and experts for patient advocates;

• Create Policy Fact Sheets corresponding to the main pillars of the Commission Communication & Council Recommendation on RD to support their implementation at the national level and their transposition into national plans;

• Provide these Policy Fact Sheets as downloadable documents on the EURORDIS website;

• Provide these Policy Fact Sheets in a format that may be reproduced in hard copy in an easy and cost‐effective manner.

Results achieved • 5 Fact Sheets produced on the following topics: Needs and Priorities in RD Research, RD Registries, Orphanet, Therapeutic Recreation Programmes, Respite Services

• Dissemination of hard copies to 550 participants of the European Conference for Rare Diseases 2010

• Dissemination of hard copies to CNA and CEF

• Dissemination of hard copies to EUROPLAN conference organisers

• Publication of all factsheets on EURORDIS website

• Development of Advocacy Pack for patient advocates including Fact Sheets, the Commission Communication and the Council Recommendation on Rare Diseases.

• Continued positive feedback from CNA and CEF on the utility of these supportive documents

Relevant Annexes • 5 Fact Sheets

Activity B.2.a. Development and access to Respite Care Services and Therapeutic Recreation Programmes


Responsible: Shane Lynam (until July 2010), Robert Pleticha (as from 1 September 2010)

Supervising: François Houÿez


Indicators • Number of TRPs (Therapeutic Recreation Programmes) listed: 40

• Number of RCSs (Respite Care Services Programmes) listed: 15

Calendar of activities • 1st quarter 2010 – Finalisation of Guidelines of best practices for RCSs and TRPs

• 2nd quarter 2010 – Production of one Fact Sheet on RCSs

• 2nd quarter 2010 ‐ Update of information on RCSs and TRPs on website

• July 2010 – End of contract of Shane Lynam, project coordinator (not replaced in the absence of sufficient resources in the Operating Grant 2011)

• November 2010 ‐ Robert Pleticha, Online Patient Community Coordinator, briefed to maintain the information service for patients on RCSs and TRPs

• 4th quarter 2010 – Production of one Fact Sheet on TRPs

Description of the Activity These activities are a continuation of the work that was done during the RAPSODY project to increase the quantity and quality of Therapeutic Recreation Programmes and Respite Care Services for Rare Diseases that exist around Europe.

Results achieved • Updated online information

• Production and dissemination of good practices guidelines

• Two policy Fact Sheets produced on, respectively, Respite Care Services and Therapeutic Recreation Programmes

Relevant Annexes

Activity B.2.b Support and strengthening of the EU Network of RD Help Lines


Responsible: Shane Lynam (until July 2010)

Supervising: Yann Le Cam, François Houÿez

Deliverable • D2: Workshops of EU RD Networks / 1 Workshop of the European Network of RD Help Lines

Indicators • Number of Members applying to become member of the ENRDHLs (European Network of Rare Disease Help Lines) ‐ Charters signed up: 11

• Participants to the Workshop on RD Help Lines in Krakow: 20

Calendar of activities • 25‐26 February 2010 – Training session for help line respondents, Bucharest

• 13 May 2010 ‐ ENRDHLs Network Meeting, Krakow

Description of the Activity • Final network application form and handbook were produced

• Applications received from 11 help lines were analysed and compared to criteria to become a network member, 1 of which passed the criteria, 5 almost passed them, and 5 did not at all

• Letters were prepared to explain to help lines which progresses are needed to become full members, as no help lines satisfy the criteria (in particular, no help line has adopted the Orpha code for disease classification)

• Results of the Caller Profile Analysis conducted in 2009 were analysed, discussed with the members and disseminated.

• All activities have been on hold since the departure from EURORDIS of Shane Lynam, project coordinator in charge of the ENRDHLs. He was not replaced due to insufficient resources in the Operating Grant 2011.

Results achieved • One network meeting was necessary to analyse the help lines difficulties in satisfying the requested criteria to become a network member and longer delays were asked to implement the measures.

• Information of interest to all help lines was post on the web site.

• A training session for help line respondents on “How to find validated biomedical information on the internet” was organised in Bucharest on 25‐26 February at the National Alliance for Rare Diseases in Romania, RONARD.

Relevant Annexes • ENRDHLS Network Application handbook

• Caller Profile Analysis 2009 final results

• Analysis of the ENRDHLS network

Activity B.2.c. Development of Online Patient Communities services


Responsible: Denis Costello, Robert Pleticha as from 1 September 2010


Deliverable • D7: Online Patient Community Portal

Indicators • Number of online communities live: 2


• April 2010 ‐ Call for tender for IT development of additional functionality for the service

• 3 Workshops to train volunteer moderators:

- 13 May 2010, Krakow

- 4 September 2010 , Amsterdam

- 19 December 2010, Paris


The web tool “Rare Disease Communities” is a multilingual enabled online social network for patients & families allowing them to connect with one another to support and share vital experiences on aspects of living with a rare disease. Organised into disease specific communities, this platform will also provide links to quality information and involve patient associations in the governance and growth of each community. The project is a joint collaboration between EURORDIS (European Rare Disease Organisation) and NORD (US National Organization for Rare Disorders).

Project management by an external programmer and a graphic designer continued, with the objective of improving existing usage and functionality and adding new features continued.

Three training workshops took place in order to further involve patient organisations in the governance and management of future online communities. EURORDIS was also present at a medical congress (World AutoInflammation Congress) in order to raise awareness of its efforts in this domain with medical professionals.

EURORDIS presented the tool at prestigious global conferences, such as Health 2.0 (Paris, April 2010), where it was awarded the audience prize of “Best Start‐Up Idea of the conference” and which merited an invitation to the Health 2.0 Conference in San Francisco in October 2010. Given that the project is a partnership with the US National Organization Rare Disorders (NORD), EURORDIS also spent some time with them, as a team building exercise in order to build parallel capacity. This included weekly conference calls since October 2010.

In August 2010, EURORDIS recruited an Online Patient Communities Coordinator, Robert Pleticha, who works with patient groups in Europe and the US to help them start and manage their own community. He also started a series of weekly webinars to inform new groups about the service.

Médicalistes.

Maintenance of 30 Mailing lists (listservs) for patient advocacy and patient support through a partnership with Médicalistes.

- 7 advocacy and governance mailing lists

- 23 disease specific support mailing lists for the following disorders:

Genetic disorders at the long arm (q) of chromosome 11

Achondroplasia ‐ achondroplasie

AHC ‐ Eurordis Mailing List

Aicardi syndrome ‐ Syndrome d'Aicardi

Alström syndrome

Aniridia

Aniridia mailing list for teenagers ‐ Aniridie: liste de discussion pour les moins de 25 ans

Anorectal malformations

Behçet's disease

Ehlers‐Danlos Mailing list

EURORDIS HSP Mailing List

Ichthyosis ‐ ichtyoses

Locked‐in syndrome mailing list ‐ La liste de discussion/diffusion sur le Locked‐in syndrome

Narcolepsy in childhood mailing list ‐ Liste de discussion sur la Narcolepsie de l'enfant

Relapsing polychondritis ‐ Polychondrite chronique atrophiante

Prader‐Willi

Propionic Acidemia / Acidémie propionique

Patients' Registries Discussion List

Sanfilippo syndrome or Mucopolysaccharidosis type III (MPS III)

sarcoidosis ‐ sarcoïdose

TSC

Willebrand disease / Maladie de Willebrand

Fragile X syndrome mailing list

In 2010, a total of 1221 users of the service were recorded, i.e. a 20% increase on 2009.

Results achieved • Addition of 3 new languages to the service (ES, DE, IT) in addition to the default EN & FR.

• New IT features include on‐demand translation service in 20 language combination of all messages posted by patients

• 2 Online Patient Communities live: Cryopyrin Associated Periodic Syndromes, Von Hippel Lindau disease

• 10 Online Patient communities in active development

• 3 Workshops (Krakow, Amsterdam, Paris) to provide training to volunteer moderators on best practises relating to patient exchanges on the internet with over 50 attendees in all.

• Hiring of Online Patient Communities Coordinator

• Weekly demonstration webinars with interested patient groups from Europe and US

• Team building visit to National Organisation for Rare Disorders

• Presentation of tool at Health2.0 conferences in Paris and San Francisco

• Total visits in 2010 = 9294

• 180 members

Relevant Annexes • URL of web tool: http://www.rarediseasecommunities.org

http://www.rarediseasecommunities.org/

Activity B.3.a.1. Support to the participation of patient representatives in the EMA committees (COMP, PDCO, CAT, PCWP) and in Protocol Assistance


Responsible: Maria Mavris

Supervising: Fabrizia Bignami, Yann Le Cam

Deliverable • D9: Participation in EMA Committees (COMP, PDCO, CAT, PCWP) and update through the TAG

Indicators EURORDIS nominated patients’ representatives in EMA Committees and working parties:

• Number of patients representatives in COMP: 2

• Number of patients representatives in PDCO: 1

• Number of patients’ representatives in CAT: 1 member + 1 alternate

• Number of patients’ representatives in PCWP: 2

• Number of Protocol Assistance (PA) dossiers received: 66

• Number of patients’ representatives included in PA: 16

• Number of Committee meetings per year:

COMP: 11 for 2‐3 days each

CAT: 11 for 2‐3 days each

PDCO: 12 for 3 days each

PCWP: 4‐5 for 1 day each

• Number of dossiers examined: 519, distributed as follows:

COMP: 157 dossiers

CAT : 46 dossiers

PDCO: 326 dossiers

Calendar of activities • Monthly meetings for COMP (2 days/month), PDCO (3 days/month) and CAT

• Four meetings annually for PCWP (2 days/month)

• Participation in Protocol Assistance dependent upon dossiers

Description of the Activity Patients’ representatives involved in the scientific committees and working parties at the EMA dedicate their time, experience and expertise to the task of evaluating dossiers of medicinal products requesting orphan drug designation, evaluation for use in paediatric patients, evaluation of advanced therapies as well as ensuring accurate, transparent and available information to patients on authorised medicinal products.

The permanent members of the scientific committees and PCWP make up the Therapeutic Action Group (B.3.a.2.).

Participants in Protocol Assistance are selected from our membership database and as often as possible have received training via the EMA in Quality Review of Documents or via the EURORDIS Summer School

(B.3.c.2).

Results achieved • Contribution to the evaluation of 519 dossiers at the EMA.

• Lise Murphy, patient representative at the PCWP, was elected as co‐chair of that working party.

• Birthe Byskov Holm, patient representative at the COMP, remains as Vice‐Chair.

• The participation of patients’ representatives in 6 Protocol Assistance procedures altered the outcome of the procedure.

Relevant Annexes • Protocol Assistance participation is referenced in the summary section of the monthly Therapeutic Reports (see Annexes of B.3.a. 3.)

Protocol Assistance information sent to patients’ representatives:

• Protocol Assistance letter of invitation and explanation

• EURORDIS Protocol Assistance flyer

• Regulatory diagram to explain Protocol Assistance

Activity B.3.a.2. Supporting the EURORDIS Therapeutic Advisory Group (TAG)




Deliverable • D9: Participation in EMA Committees (COMP, PDCO, CAT, PCWP) and update through the TAG

Indicators • Number of days at Committees: 123, distributed as follows:

COMP: 42 days

CAT: 35 days

PDCO: 24 days

PCWP: 22 days

• Number of consultations/meetings of TAG: 10

Calendar of activities • Monthly conference call with all members

• 1 July – TAG Meeting in Paris

• 2 July – TAG Meeting in Paris with EURORDIS Board of Directors

Description of the Activity The Therapeutic Action Group (TAG) is composed of EURORDIS representatives in the scientific committees and working party at the EMA (PDCO, PCWP, CAT, COMP): Fabrizia Bignami (CAT), Lesley Greene (COMP), Birthe Byskov Holm (COMP), François Houÿez (PCWP), Lise Murphy (PCWP), Michele Lipucci di Paola (CAT) and Tsveta Schyns (PDCO). These patients’ representatives dedicate their time, experience and expertise to the task of evaluating dossiers of medicinal products requesting orphan drug designation, evaluation for use in paediatric patients, evaluation of advanced therapies as well as ensuring accurate, transparent and available information to patients on authorised medicinal products.

A monthly report of activities of EMA Committees and EURORDIS’ patients’ representatives in these Committees is produced and disseminated to all members of TAG, Task Forces, Eurordis Summer School participants and EURORDIS’ Board (see Activity Table B.3.a.3).

Results achieved • The TAG was created in January, 2009 and has successfully established a monthly conference call and a yearly face to face meeting to discuss matters related to regulatory affairs and therapeutic development.

• A face to face meeting on July 1, 2010 in Paris took place prior to the Eurordis Board of Directors meeting. The members of the TAG present included Fabrizia Bignami, Lesley Greene, Birthe Byskov Holm, Francois Houyez, Michele Lipucci di Paola, Lise Murphy and Tsveta Schyns. Major issues discussed included collaboration between different committees and improvement on communication between EURORDIS Board and TAG members.

Relevant Annexes • Agenda from TAG face to face meeting, 1 July 2010

• Agenda from TAG conference call, 17 December 2010

Activity B.3.a.3. Monthly reports compiling feedback from each Committee





Indicators • Number of monthly reports: 11

Calendar of activities • Monthly conference call with all members to exchange information and receive updates to include in monthly report

• Publishing and dissemination of monthly Therapeutic Report on activities of patients’ representatives in regulatory activities in the EU.

Description of the Activity A monthly report of activities of EMA Committees and EURORDIS’ patients’ representatives in these Committees is produced and disseminated to all members of the TAG, EURORDIS Task Forces, EURORDIS Summer School participants and EURORDIS’ Board.

The report is compiled based on feedback received from members of the TAG, monthly reports published by the European Medicines Agency, searching of the EMA website for relevant news and information, meetings attended relating to regulatory affairs by TAG members and presence of Maria Mavris at COMP monthly COMP meetings.

Results achieved • Eleven monthly “Therapeutic Reports” were published in 2010

• Targeted dissemination of the Reports to:

- Previous EURORDIS Summer School attendees

- EMA staff

- EURORDIS Board of Directors

- Staff of French muscular dystrophy association (AFM)

- EURORDIS staff

Relevant Annexes • Three examples of Therapeutic Reports are provided:

- May 2010

- September, 2010

- December, 2010

Activity B.3.b. Review and validation of public information on RD therapies disseminated by EMA (PSOs, EPARs, PLs)


Responsible: Maria Mavris, François Houÿez

Supervising: François Houÿez, Fabrizia Bignami


Indicators Public Summaries of Opinion of orphan designation (PSO), European Public Assessment Report (EPAR) summaries and Package Leaflets (PL) reviewed EURORDIS staff members:

• Number of PSOs: 123

• Number of EPARs: 5

• Number of PLs: 7

Calendar of activities • PSOs validated monthly upon positive opinion given by COMP and designation awarded by EC

• EPARs and PLs – at time of Marketing Authorisation

Description of the Activity The European Medicines Agency (EMA) is responsible for providing information about medicines authorised via the centralised procedure that includes information directed to the patient and the public. During the preparation of this information, the Agency interacts with patients and consumers’ organisations to ensure that it is adequately formulated and comprehensible to the target audience.

EURORDIS is extensively involved in these activities and has reviewed a total of 135 EMA dossiers for public information this year.

Documents that are destined for the general public include:

• Public Summary of Opinion (PSO) of orphan drug designations explain in lay terms the disease, the number of individuals potentially affected in the EU, the medicinal product and the stage of development. A link to the Sponsor and to EURORDIS and Orphanet are provided in the PSOs to help patients obtain more information.

• EPAR summaries (EPAR) are shorter documents based on the European Public Assessment Reports that are published at the time of Marketing Authorisation. The EPARs contain information about the development of the product and how the committee reached it recommendations.

• The Package Leaflet (PL) contains information on what the drug is, what it is used for, how to take the drug, possible side effects and how to store the drug. It is important that this information is easily understandable for the general public.

Results achieved A total of 123 PSOs, 5 EPARs and 7 PLs were reviewed by EURORDIS staff members in order to ensure the quality of the information disseminated by the EMA to the general public.

Relevant Annexes • Link to PSOs on EMA website: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/ orphan_search.jsp&murl=menus/medicines/medicines.jsp&mid=WC0b01ac058001d12b

http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/%0Borphan_search.jsp&murl=menus/medicines/medicines.jsp&mid=WC0b01ac058001d12b



• Link to EPAR summaries and PL on EMA website: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/epar_search.jsp&murl=menus/ medicines/medicines.jsp&mid=WC0b01ac058001d125

http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/epar_search.jsp&murl=menus/%0Bmedicines/medicines.jsp&mid=WC0b01ac058001d125



Activity B.3.c.1. Support to EURORDIS Task Forces on Orphan Drugs, Paediatrics, Drug Information, Transparency & Access (DITA)



Supervising: Fabrizia Bignami, François Houÿez

Deliverable • D8: Meetings of the Task forces: Orphan Drugs, DITA and Paediatrics

Indicators • Number of consultations with Task Forces: DITA‐TF: 9

OD‐TF: 5

P‐TF: 6

• Number of volunteers involved in Task Forces: 34

Calendar of activities • 4 June – DITA Task Force meeting, Paris

• 30 July – Orphan Drugs Task Force meeting, Paris

• 28 November – DITA Task Force meeting, London

• 3 December – Paediatric Task Force meeting, Paris

Description of the Activity The Task Forces represent a group of volunteers who are trained (via the EURORDIS Summer School) and active in issues concerning therapeutic development of orphan drugs.

The Task Forces support and/or advise the EURORDIS representatives who participate in EMA scientific committees and working parties.

They are also consulted on papers prepared by EURORDIS.

Existing Task Force (TFs) include:

- DITA‐TF, Drug Information, Transparency and Access

- OD‐TF, Orphan Drugs

- P‐TF, Paediatrics

The General Terms of Reference, which apply to all TFs, were approved by the Board of Officers on April 20, 2009.

The Task Forces participate in conference calls when necessary (if certain issues need to be discussed) and at least one face‐to‐face meeting per year.

Finally, in the last quarter of 2010, the DITA performed a “Survey on Compassionate Use of Orphan Drug”, a full account of which is given in the following Table.

Results achieved • DITA Task Force meetings held in Paris on 4 June, and in London on 28 November, 2010.

• Paediatric Task Force meeting held in Paris on 3 December, 2010.

• Orphan Drug Task Force meeting held in Paris on 30 July, 2010.

• Nomination of future ‘alternate’ member of Paediatric Committee (PDCO) at EMA and nomination of observers at PDCO to ensure continuation of patient representative presence and support for PDCO member.

Relevant Annexes • Agenda of DITA meeting 4 June, 2010

• Agenda of DITA meeting 28 November, 2010

• Agenda of Orphan Drugs Task Force, 30 June, 2010

• Agenda of Paediatric Task Force, 3 December, 2010

Activity B.3.c.1 (additional table). DITA’s Survey on Compassionate Use of Orphan Drugs in Europe


Responsible: Blanca Vera‐Gargallo (from 20/09/2010 to 31/12/2010)

Supervising: François Houÿez, Fabrizia Bignami


Indicators • Number of pharmaceutical companies responding to the survey: 26 of which 16 exploitable / 55

• Number of patient organisations responding to the survey: 28 / 330

Calendar of activities • 20 September to 20 October: development of the questionnaire to industry and to patient organisations

• 20 October to 15 December: survey period

• 15 December to 31 December: analysis of results

• 20 September to 31 December: bibliographic search, collection of resources and resource persons


The activity consisted in compiling information on compassionate use programmes and administrative schemes for orphan medicinal products in Europe. It included the collection of information from published literature, national competent authorities and pharmaceutical companies with a compassionate use programme in progress, number of patients who benefit, understanding why the programme can be run in a country and not in another one, identification of administrative schemes used, estimates of the costs, and estimates of the health benefits.

Objectives:

• Expand our knowledge about Compassionate Use Programmes for Orphan Drugs in Member States and stakeholders’ point of views on them;

• Inform patient organisations, industries, Member States and National Agencies about Compassionate Use Programmes;

• Make recommendations to companies and agencies about how and when to give an opinion and to Member States about how to implement Compassionate Use Programmes;

• Create a bank of data containing contacts and useful bibliography and legal texts.

Results achieved • A definition of Compassionate Use Programmes was developed.

• Two questionnaires were developed, one for the industry (disseminated via EBE/EuropaBio), one for patient organisations (disseminated by EURORDIS).

• These questionnaires were online (Survey Monkey) and were available in French, English, Spanish, German, Portuguese and

Italian.

• Preliminary analysis of results is done (more responses are due in 2011).

• Identification of person in charge of compassionate use programmes in some National Competent Authorities. And collection of national rules in some EU Member States.

• A compilation of published articles and thesis was achieved (28 references, and 9 presentations from workshops on compassionate use).

• A contact list of experts in compassionate use was updated (15 contacts in the industry, 17 in patient organisations, 130 authors).

Relevant Annexes • Annexe 5 Compassionate use survey questionnaire to industry

• Annexe 6 Compassionate use definitions and terms

Activity B.3.c.2. Summer School 2010



Supervising: Fabrizia Bignami

Deliverable • D6: Summer School 2010 and online training (paedagogic kit)/ Summer School

Indicators • Number of training sessions: 1 sessions of 4 days

• Total number of trainees: 34

• Total number of person‐days in training: 136

• Total number of trainers: 10

Calendar of activities • Summer School Programme held: 26‐30 September 2010, Barcelona

• Report: finished 30 November 2010


The Summer School is a 4‐day capacity building programme for rare disease patient representatives in the areas of drug development, clinical trials and aspects of the EU regulatory process. The 2010 Summer School invited previous participants for an advanced course in Regulatory Affairs and Health Technology Assessment with the following objectives:

• To extend the knowledge of these advanced patient advocates using peer‐based real case studies from the various scientific committees at the EMA;

• To provide patient advocates with an initial introduction to Health Technology Assessment (HTA) and to illustrate the link between the drug regulatory and HTA processes.

Thirty‐four participants attended representing 15 countries and more than 25 different rare diseases. All speakers were recorded and edited footage will be linked to the slide presentations and made available to all participants.

Results achieved The 2010 Summer School provided a practical ‘hands‐on’ experience of the work performed by the representatives at the EMA scientific committees and working parties using:

• Orphan drug designation dossiers from the Committee for Orphan Medicinal Products;

• An example of a waiver request from the Paediatric Committee;

• Protocol Assistance – examples of types of topics for questions, feedback from patient experts who have participated from Scientific Advice Working Party;

• Real examples of crisis management with respect to drug availability, reimbursement and access to drugs by Patients’ and Consumers’ Working Party.

An introduction to Health Technology Assessment (HTA) was included in order to prepare patients’ advocates to become more involved and be armed with adequate knowledge to interact with national agencies in issues concerning effectiveness, pricing and reimbursement of medications.

Relevant Annexes • 2010 Summer School Programme

• Web link on Summer School: http://www.eurordis.org/training‐resources

http://www.eurordis.org/training-resources

Activity B.3.c.3. Development of e‐Learning on specific and advanced aspects of drug development, clinical trials and regulatory affairs

Responsible and upervising staff


Supervising: François Faurisson, Fabrizia Bignami

Deliverable • D6: Summer School and online training (paedagogic kit)/ online training

Indicators

Calendar of activities Tool is being built in PowerPoint using slides which are then uploaded into the web tool ‘Articulate’. The slides have been completed and are currently in validation phase.

Description of the Activity As part of the EURORDIS Summer School, a 4‐day capacity building programme for rare disease patient representatives in the areas of drug development, clinical trials and aspects of the EU regulatory process, an on‐line e‐learning tool for continued education is being established.

The on‐line tool, temporarily named ‘Keys for Clinical Trials’, is made up of four modules and was developed using PowerPoint presentations. The layout of the slides was designed by a graphic designer and then uploaded into Articulate. All slides were elaborated based on presentations from the Summer School and reviewed by Lesley Greene for language and coherence.

The e‐learning tool will cover the topics of described during the Summer School using different learning methods. The topics that are included are presented as modules:

- Statistics

- Methodology

- Ethics

- Clinical research

The user can access the different modules and learn by using the lessons, case studies, vocabulary tests, quizzes or specific documents. Once inside a particular module, the user can move between case studies to lessons to vocabulary or relevant documents.

• Total number of slides: approximately 400

Results achieved The slides are completed and are currently being uploaded and validated in a web tool called ‘Articulate’. Once completed and validated, the tool will go live on the EURORDIS website http://elearning.eurordis.org (targeted date March 2011).

http://elearning.eurordis.org/

Activity B.3.d. Support to capacity‐building activities of patient advocates in HTA



Supervising: Fabrizia Bignami


Indicators


• Four‐day training course in Hall‐im‐Tyrol, Austria by UMIT (The Health & Life Sciences University) in February, 2010

• HTA in 2010 Eurordis Summer School in Regulatory Affairs and Health Technology Assessment in June, 2010


This year, two opportunities for patients to be trained in Health Technology Assessment were provided:

The HTA Programme in Austria is a four‐day course focusing on Health Technology Assessment, Health‐Economic Evaluation and Outcomes Research. This introductory course covers the key elements and methods of HTA and combines lectures, discussions, case study group work, and hands‐on computer lab session.

This course covers key elements and methods of HTA and combines, lectures, discussions, case study group work and hands‐on computer lab sessions. Case examples of the course include technologies from different areas such as pharmaceuticals, devices, public health and prevention strategies, management programmes and health information systems.

In the 2010 Eurordis Summer School, an introduction to Health Technology Assessment was introduced for the first time. One and a half days were devoted to the basic introduction of HTA, introduction to the Quality Adjusted Life Years (QALY) as well as alternatives, ethics in HTA and presentations from all stakeholders including industry, patients and HTA agencies.

Results achieved • Five patients’ advocates were trained for 4 days in the HTA training. These patients’ representatives represented 3 different countries and 4 different rare disease areas.

• The Summer School was the first opportunity for many patients’ advocates to be exposed to Health Technology Assessment for the first time and to participate in some of the HTA evaluation processes. Thirty‐four participants attended. All speakers were recorded and edited footage will be linked to the slide presentations and made available to all participants.

Relevant Annexes • 2010 Summer School Programme

• Web link to Summer School online presentations: http://www.eurordis.org/training‐resources

• Web link on HTADS: http://www.umit.at/page.cfm?vpath=departments/public_health/ htads_continuing_education_program_d/short_courses_d/modeling_for_hta_d

http://www.eurordis.org/training-resources

http://www.umit.at/page.cfm?vpath=departments/public_health/%0Bhtads_continuing_education_program_d/short_courses_d/modeling_for_hta_d

http://www.umit.at/page.cfm?vpath=departments/public_health/%0Bhtads_continuing_education_program_d/short_courses_d/modeling_for_hta_d

Activity B.3.e. Supporting good practice relations between POs & sponsors on RD clinical trials based on EURORDIS Charter on Clinical Trials (CCT)


Responsible: Fabrizia Bignami, François Faurisson


Indicators • Number of companies having signed up to the Charter: 6

Calendar of activities • February 2010 ‐ Second meeting of the European Tuberous Sclerosis (TS) patient organisations (POs)

• May 2010 – Presentation of the Charter at the European Conference on Rare Diseases (ECRD 2010)

• 12‐13 November 2010 ‐ Final meeting for finalisation of the Agreements of Understanding (AoU) between the TS POs and the clinical trials sponsor. Final signature of first two AoUs in the framework of the EURORDIS Charter on Clinical Trials.

Description of the Activity The general aim of the EURORDIS Charter for Clinical Trials in Rare Diseases” (CCTRD) is to regulate the relationship between a clinical trial promoter and the patient organisations representing the disease concerned by the study. Since the second quarter of 2009, EURORDIS is engaged in the implementation phase of the Charter. During 2010 this activities implied the following steps:

Formal adhesion of pharmaceutical companies developing medicinal products for rare diseases to the EURORDIS Charter for Clinical Trials in Rare Diseases.

Implementation of the Charter at the occasion of any clinical trial performed by the signatories in a rare indication. EURORDIS offers its support to help implementing the Charter, and in particular:

• Upon request of a sponsor having signed the EURORDIS Clinical Trials Charter or of a patient group, EURORDIS contacts and gathers together all EU patient groups concerned by a specific clinical trial.

• Then, EURORDIS helps all involved parties to reach an agreement on the level and modalities of the collaboration they all wish to establish for a specific clinical trial.

• Such agreement is officially described in a dedicated AoU (Agreement of Understanding) signed by the parties and published on the EURORDIS website.

A specific section on the EURORDIS website provides information on the EURORDIS Charter for Clinical Trials in Rare Diseases, on the companies who adhered to the Charter and will soon lists the AoU signed.

Results achieved • During 2010, the first two Agreements of Understanding (AoU) have been signed in the framework of the EURORDIS the Charter

have been signed in the framework of the EURORDIS, the Charter for Clinical Trials. These agreements, available on the EURORDIS website, have been drafted with the direct support of EURORDIS and signed by the two concerned parties, the company Novartis and the group of European organisations representing patients affected by Tuberous Sclerosis (TS). This collaboration has also provided the opportunity, for the different TS organisations, to speak a single voice on a very important issue for the TS patients: the clinical development of a product in their disease. This was the first step toward the construction of an official EU network of TS patient organisations.

• The EURORDIS Clinical Trials Charter has been presented at the DIA USA by Maria Mavris.

Relevant Annexes • EURORDIS Clinical Trials Charter

• First two Agreements of Understanding signed by patient groups and clinical trial sponsor.

Activity B.3.f. Promoting RD research policy and patient involvement in research


Responsible: Fabrizia Bignami

Supervising: François Faurisson, Valentina Bottarelli


Indicators

Calendar of activities • January 2010 ‐ Analysis of the results of the survey on “Rare Disease Patient Organisations in Research”

• 1 March 2010, Brussels ‐ Presentation of the results of the survey at the Rare Disease Day European meeting on “Bridging Patients and Researchers to Build the Future Agenda for Rare Disease Research in Europe”.

• March 2010 ‐ Presentation of the draft EURORDIS document on “Why Research on Rare Diseases”

Description of the Activity • One of the major roles of EURORDIS is to advocate for stimulating research on rare diseases so that new diagnostic tools, therapies and improved protocols of care are made available for rare disease patients. In this framework, EURORDIS collaborates with all involved stakeholders (EU Commission and other public funding agencies, researchers, industry and patient groups) to increase as much as possible the budgets allocated to research on rare diseases and to optimise their use. Rare Disease Day 2010 has been the occasion to open the discussion on how to develop a strong policy to advance rare disease research.

• In order to consolidate its advocate role for the rare disease patient community, EURORDIS has collected solid data on what patient groups bring today to research on rare diseases, so that their role is not underestimated, but seriously taken into account in the general picture.

Results achieved • These data have been gathered and analysed in the survey on “Rare Disease Patient Organisations in Research”, completed at the beginning of 2010 by EURORDIS, in collaboration with the group of sociologists from the « Centre de sociologie de l'innovation »(Ecole des Mines, Paris, France).

• The results of this survey have been presented for the first time at the occasion of the European event organised in Brussels for the Rare Disease Day 2010 on “Bridging Patients and Researchers to Build the Future Agenda for Rare Disease Research in Europe”. The meeting was dedicated to present the state of the art of the research on rare diseases in Europe and to reinforce the collaboration of all stakeholders in this area. In particular, this meeting was also the occasion to present and discuss the first draft

of the EURORDIS Discussion Paper on “Why Research on Rare Diseases”

• In the context of its role of advocate for rare disease patients, EURORDIS has also been invited to participate and contribute to the meeting organised in October 2010 in Reykjavik, Iceland, by the EU Commission/DG Research and the NIH (US National Institute for Health), Office of Rare Disease Research, to explore ways to collaborate internationally to stimulate and improve the quality of the research on rare diseases.

Relevant Annexes • EURORDIS document on “Why Research on Rare Diseases” (Ongoing document)

• Fabrizia Bignami presentation on the results of the survey “Rare Disease Patient Organisations in Research”