The objectives of the European Conference on Rare Diseases & Orphan Products are:
- To disseminate the most up-to-date health information related to the rare disease environment to all relevant stakeholders (patients and patients’ representatives, academics, health care professionals, industry and policy makers);
- To demonstrate the importance of EU actions in the field of rare diseases and review progress made to date;
- To elaborate strategies and mechanisms for developing further exchange of information between stakeholders: people living with rare diseases, volunteers, health professionals, policy makers, researchers and industry at national and EU levels;
- To exchange knowledge and best practices on all relevant health issues related to the rare disease environment;
- To sustain efforts for rare disease policies at both the European and the national level;
- To stimulate dialogue on policies for rare diseases in some of the Member States having recently joined the EU;
- To present specific, achievable objectives at both European and national levels in order to reduce health inequalities for rare disease patients.
The target number of participants for the Conference is 700. This number represents all categories of participants: patients and patients representatives, health professionals, academia, industry and policy makers. This number also includes Scientific and Steering Committee Members, speakers, patient fellowship recipients, poster presenters and supportive staff.
The anticipated percentage of each category of participant at the conference, based on figures from previous conferences, in particular the ECRD 2012 Brussels, is as follows:
43 % Patient Organisations (POs) representing over 45 countries of which 27 Member States and including patient fellowship recipients from Central and Eastern European countries
32 % Health professionals & academia & scientists
19 % Pharmaceutical and biotech industry representatives
6 % Policy-makers / EU officials / National officials
As an illustration, 649 participants attended ECRD 2012 Brussels from the following countries:
Finland, Romania (17 each)
Hungary, Poland (11 each)
and others (85)
The target participants fall into 5 distinct categories which are briefly described below:
1) All RD patients and patients representatives throughout Europe and beyond: they will benefit from the knowledge and information disseminated about RD policies and actions at a European and national level and will also directly benefit from the capacity-building workshops on the first day of the Event, the networking opportunities and information exchange with other patients and stakeholders.
2) Academia & scientists active in the field of rare diseases and in particular leading scientists involved in EU and E-Rare Funded Projects and all researchers listed on ORPHANET: they are interested in learning the most up-to-date information about policy orientations, concepts and priorities concerning rare diseases at a European level and who will benefit from the networking events.
3) Health professionals, in particular the health professionals of Centres of Expertise identified through national plans of RD and listed on ORPHANET and health professionals reached through Learned Societies, such as the European Society of Human Genetics, European Federation of Internal Medicines & the European Academy of Paediatrics: they are eager to learn more about rare diseases and benefit from the experiences, best practices and forum for exchange of ideas and networking.
4) Pharmaceutical and biotech industry representatives involved in orphan drug development: they are interested in the knowledge and information disseminated about policy orientations, concepts and priorities concerning RD at European and national levels who will directly benefit from the sessions on orphan products and the networking opportunities with other patients and policy makers.
5) Policy makers from around Europe working in the health domain and representatives of Member States involved in different EMA Committees and European Commission relevant committees such as the EUCERD, the Committee on Cross Border Health Care and Corporate Responsibility in the field of Pharmaceuticals.
The Conference annoucements and the Call for Posters will be disseminated to EURORDIS full database containing over 7000 qualified contacts including over 1900 patient organisations and to the 4000 attendees of the 25 National Conferences taking place in the context of the EUROPLAN II/EUCERD Joint Action in 2012-2013, representing all stakeholders in the RD community who will be targeted by our communications. This figure is based on our experience organising 15 National Conferences in the context of EUROPLAN I which gathered 2400 participants.
Access for persons with disabilities will be facilitated.
Simultaneous translation of the plenary will be provided in up to 5 languages. The conference is by no means restricted to Europeans. Participants from other parts of the world are encouraged to attend.
A patient fellowship programme (funded outside of the scope of this Conference Grant application) is developed for patient advocates, giving them the opportunity to attend the conference without incurring high travel and accommodation expenses.
The entire Event will take place in Berlin over 3 days.
- The 1st day will be dedicated to capacity-building and networking satellite meetings: (a) The EURORDIS Membership Meeting with parallel workshops on national strategies and EU policies targeted at patients representatives
(b) Annual meetings of RD Patient Groups, National Alliances, ORPHANET and the ExCom meeting of the IRDiRC
(c) DIA Tutorials targeted at industry and academic representatives
The costs of the 1st day are not included in this grant: they are covered either by EURORDIS core funding, or by projects (satellite workshops,capacity-building workshops for patients and their representatives).
- The Conference itself (for which the grant is requested) will take place over the 2 last days of the Event.
The Conference starts the 2nd day of the event with a half-day plenary officially launching the Conference with patients representatives, health professionals, policy decision-makers and officials from the EC. This part of the ECRD will focus on the overall state of the art of RD in Europe, EU policy priorities, status on national plans and a broad overview of the main themes addressed. It will involve the EU presidency next trialogue Italy/Latvia/Luxembourg.
The remaining 1.5 days of the Conference will be dedicated to parallel "tracks" developing specific themes: 6 simultaneous tracks of 6 sessions each. A total of 30 sessions will be proposed. Each session lasts 1.5 hours.
The sessions will include: EU Policy; Research Policy; Infrastructures & Projects, including registries, biobanks, clinical research networks, international research consortium; Quality of Life & Specialised Social Services & Social Guidelines; Integration of RD into Social Policy; Public Health: screening, gene testing & counselling, Information (helplines, webserver, social media) & public awareness; Offer for Care: centres of expertise & European reference networks, health care pathways, good clinical practices on diagnostic & care, cross border healthcare & mobility, Orphan products & rare disease therapies: OD development, RD advanced therapies; OD development: clinical trials; compassionate use & early access, off-label use, shortages in authorised medicines for rare diseases; Improving access to orphan products: HTA and CAVOMP, relative effectiveness and post-marketing research plan, European mechanism for coordinated access to OD and EU Transparent Value Framework.
A list of potential speakers will be drafted at the 1st Scientific Committee Meeting scheduled for April 2013. Speakers will be invited subsequent to this meeting.
Based on a call for posters, selected posters will be displayed on day 2 and 3 of the Conference. Two Conference sessions will be dedicated to pre-selected posters for oral communication.
Detailed budget is attached to this document.
*** Budget ***
Given the uncertainties related to the scheduling of the Work Plan calls and the fact that such conferences have to be prepared 2 years in advance, preparation costs (selection of venue, constitution and meetings of Programme and Steering Committees, announcement, update of website) are budgeted under the annual Operating Grant.
All staff costs are therefore budgeted under the Operating Grant and this grant will cover all invoices directly linked to the two days of the event (venue, audio-visual, missions, catering, etc).
Estimation of costs is based on previous experience and call for bids for this event:
- Room rental, equipment, catering costs are based on the venue call for bid best quote
- Travel management, interpreters and miscellaneous are based on previous experience
- Registration will be managed by our partner DIA
- Travel and Subsistence allowances costs are based on our experience in Brussels.