EFHSP

1. EFHSP First things first

2. Rare!TogetherPRO’s • Contact between people from different countries and various cultural backgrounds sharing the same special disability • - learning from each other - for daily living and other issues • - giving advice and support • - making friends • Collection and publication of information about the disability on an international level • Collection of data (addresses, information on different structures or procedures of national organizations, etc) • Common website + networking – “together we’re strong!” • Cooperation with, networking and training of medical professionals • Patient networks can enhance cooperation of professionals who did not work on common projects earlier

3. Rare!TogetherPRO’s • Common projects (youth exchange, summer camps, workshops and conferences) • Creation of common standards of care, rehabilitation, treatment • Centers of expertise - on a national or European level • Political representation of common interests on European (or international) level • Promotion of research on all aspects of the disorder • Transfer of knowledge and equipment, access to treatment and support for people in less-developed countries • Support for small national groups in the process of setting up • Exchange of best practises and guidelines (public relations, fundraising, organize a conference…) • Others

4. Rare!TogetherCON’s • For contact between individuals, it might be easier / advisable to join and use other existing networks like “Facebook”, discussion groups or listservs, etc • For work on a European political level, perhaps this could be reached by joining and being active in an umbrella organization like EURORDIS (European Organisation for Rare Diseases) and others who represent different national groups. Reaching political goals is maybe better as a part of a larger organization* - any new European network could and should join that umbrella organization • *altho this could be a PRO for federation

5. Rare!TogetherCON’s • Economic problems – financial resources & access to external subsidies will be difficult, especially during the starting period. National members will need to give towards: • The Federation’s operational costs, eg, an annual membership fee which may be a burden for small national organizations, costs to attend meetings, workshops, conferences may be another burden • Language barriers: problems to use a common language and to find representatives with acceptable language skills • Lack of human resources: generally, volunteers are rare, the foundation of a European federation might be difficult due to the lack of competent and capable representatives

6. Rare!Together • Often, a uniting theme can bring groups together quickly (ie, multiple myeloma and thalidomide) • Will new federations do conferences, symposia, internet forums, training visits by professionals, establishing treatment centres? Translations where possible, organisational development of new groups (fundraising workshops, etc).

7. Rare!Together • OI – bridge between researchers and community, even just between researchers • Paid staff, budget – problem • Dedicated person(s) needed

8. Rare!Together • CF - QOL, patient representation, raising awareness of the disease, promoting optimized treatments, offering a forum for exchange • Network building, up-to-date info to patients, they partner with clinicians, patient empowerment on a local level • Professionalisation – very important

9. Rare!Together • MM – “no policy without patients” • R&D, Drug Development, Regulatory concerns, Access and Standard of Care. • Relations with sponsors are important, a regulatory dialog, access and pricing • They have a multi-year (7-yr) working / strategic plan. They are very professionalised, although not well-funded • ENGLISH is a concern

10. Rare!Together • New federations - more research, drugs, and investigation. Tension between basic science (genetics) and applied research • more weight to the patients’ voice, which wants more translational (applied) work done. • Long-term health

11. Rare!together • The future: Both groups will work separately in the morning and come together in the afternoon, to work on agenda / work plans / priorities (details of statutes etc) then a session on the wiki followed by a teach-in on • fundraising, • advocacy at an EU level, • CABs, • working with EU institutions like DG Research, • getting more in the public eye – awareness • The existing federations also underlined how helpful Rare!Together is for them in their future work, whether it be setting up a broader network (beyond Europe) or clarifications about best country as HQ, etc

12. Statutes • Where? • We can always establish ‘working offices’ where needed.

13. Statutes • Objectives of EFHSP • Community involvement in research & treatments?? • Promote collaboration w/ researchers (between each other and with us) • social, political and cultural issues re: patients affected by any hereditary neuropathological degenerative condition & improving the exchange ofs/p/c information • International community collaboration (incl. joining other groups) • Collect $ for research and community issues • PHARMA????

14. Statutes • Scientific board??? • Fees for each type of member????? • Termination – 4/5 of GA, 2/3 of BOD??? • And reinstitution???

15. Statutes • BOD – how many? • Must a scientist be a member? Must at least one person with HSP be a member? • More than one position on BOD??? • Who elects the Chair? • 2nd in command – Secretary or Vice-Chair? • Please see by-laws in certain circumstance???

16. Statutes • Quorum? • Except for reasonable limitations as to time and space, the accounts will be available for inspection by the members??? • Internal auditors??? • Big issues – quorum is??? 2/3???

17. Statutes • Paying members??? • Minutes in French and English • Co-optation