FESCA Strategic Plan 1 Strategic Plan 2 Vision

FESCA Strategic Plan 1

Strategic Plan 2

Vision and Mission 3

FESCA Vision Our vision can be defined as: “A world in which everyone with Scleroderma receives appropriate access to the same level of successful care from fully informed healthcare professionals. ” Our vision is one of a world in which: Scleroderma is recognised as an important disease Society knows about Scleroderma, and those with the disease do not feel so isolated People with Scleroderma have equal access to successful treatments Medical practitioners have better knowledge and access to treatments Consistent care for SSc is provided across the globe Scleroderma becomes a curable disease 4

FESCA Mission The FESCA mission statement: “To make Scleroderma a well-known disease and, in collaboration with doctors, to secure for those with Scleroderma the best possible treatments, care, and ultimately a cure. ” Our mission will be achieved by: Becoming the main representative of the EU national organizations Working with and encouraging the active involvement of politicians and pharmaceutical companies Working with doctors to secure top treatments, care, and ultimately a cure Facilitating collaboration among national associations to share ideas and projects Ultimately we will: “Educate the world, patients, and doctors about Scleroderma” 5

Objectives and Strategy 6

FESCA Objectives The objectives of FESCA, as the representative of all its member national organizations, are: • To work with politicians to raise awareness and improve access to appropriate treatment and care • To become a recognized non-governmental organization (NGO) in the EU parliament in Brussels, and to subsequently acquire grants • To raise awareness of Scleroderma throughout the EU • To increase understanding of Scleroderma • To empower people with Scleroderma • To improve access to appropriate treatment and care • To help develop a cure • To encourage the development of, and assist in setting up, national Scleroderma organizations where none exist 7

FESCA Strategy FESCA can: • Connect with the medical community and put companies in touch with key opinion leaders (KOLs) • Facilitate access of people with SSc to others through its network of members • Provide intelligence/market research – with proviso that it must always fully explore ethical considerations • Lobby and build relationships with politicians to enable their understanding and seek their support • Promote the goal of rapid access to medical treatment in all member countries, noting different rates of access in member states • Disseminate news about treatments and care 8

Environmental opportunities & threats 9

Opportunities World Congress of Scleroderma organized by EUSTAR and North America Emerging data e. g. that Scleroderma affects approximately 1 in 10, 000 people in the UK – amounting to around 500, 000 Involvement of EU members --now 11 countries, 14 members Increased involvement of EULAR with connective tissue diseases (CTDs) such as Scleroderma Several new treatments in development (e. g. for PAH) Quality of life studies being conducted; opportunity to highlight the impact of SSc on quality of life EULAR-PARE as potential partner Potential funding from governments Commitment and dedication of its members 10

Threats Incidence and prevalence of systemic Scleroderma not known (establishment is one of the aims of EUSTAR) Incidence and prevalence of localized Scleroderma not known Very few (perhaps 1%) of the talks at EULAR are on Scleroderma Little evidence base of effective treatments for Scleroderma Low number of people with Scleroderma Little interest from pharmaceutical companies due to small size. Market size estimate – 500, 000 patients; £ 20, 000 a year per patient drug course £ 10 billion a year. Low prevalence (or numbers) of people with Scleroderma within musculoskeletal conditions bracket Reimbursement issues – protocols that exclude Scleroderma despite the fact that it requires lifelong care. 11

Target audiences 12

FESCA target audiences Governments/politicians Media National organizations Healthcare professionals Pharmaceutical companies 13

Government/Politicians Members of the European Parliament Need to: • Be educated about Scleroderma as they currently know little about the condition • Know that it afflicts children as well as adults (usually localized) • Know that earlier diagnosis and treatment would reduce the health economic burden • Know that Scleroderma can be life-threatening Want to: • Understand the financial impact of the disease on their populations • Know the number of people who have the disease • Be visible for having achieved something – FESCA needs to propose projects e. g. building specialized centers 14

Media Specific outlets will include: Newspapers Women’s magazines Health magazines Local daily newspapers CNN Newswires News feeds The International Herald Tribune The internet, etc. For effective communication with the media: A media campaign will be planned by FESCA in collaboration with partners (EULAR, EUSTAR, etc. ) to address medical treatments and information about the disease News should be fresh and new (scoops) despite having been planned out in advance Scientific authority should be given News will be regular European Scleroderma Day 15

National organisations FESCA will provide essential support for national associations by: • Providing information, best practice guidance and other support to help them improve their services • Developing a resource for new/fledgling patient associations to ease start- up, including core materials, leaflets about the disease, patient literature, etc. • Establishing authority/credibility on issues relevant to people with Scleroderma to enable FESCA to issue statements on their behalf across Europe • Serving as an authoritative information resource with top medical backup on medications, treatment options, and current research • Acting as a central communications point within the EU 16

Healthcare professionals Key health professional targets for FESCA include: • International key opinion leader (KOLs) physicians • Lead specialists in teams coordinating SSc healthcare e. g. rheumatologists or immunologists • Collaboration with all KOLs to provide special access to people with Scleroderma and support in research grant applications Partnership with key opinion leaders is to involve: • Helping execution of their research plans • Providing Epidemiology data – FESCA can facilitate contact to people with Scleroderma through the FESCA database of National Organizations • Facilitating access to people with SSc 17

Pharmaceuticals: industry leaders and products Company Product(s) Encysive Thelin® (sitaxsentan) GSK/Myogen Ambrisentan® , Flolan® for PAH Wyeth Enbrel® etanercept for ankylosing spondylitis United Therapeutics Remodulin® (treprostinil sodium) Injection for PAH Bayer Adalat® (nifedipine) - hypertension, etc. Ventavis® – PAH Digna Biotech/ISDIN P 144 - topical cream; NAFB 001* Actelion Tracleer® (bosentan) – PAH Pfizer Revatio® - (sildenafil citrate) – PAH; Norvasc®/Istin® (anlodepine) – hypertension, etc. Bosch Atlin® (atenolol) – hypertension Caloc® – (amlodipine besylate) – hypertension Roche Torem® (Torasemide) – hypertension, renal failure, CHF Neotigason® (Acitetrin) – prescribed to treat severe psoriasis and other skin diseases characterized by thickening and shedding of the skin Abbott Humira® (adalimumab ) – inflammation and systemic Teveten® (eprosartan mesylate) – Angiotension II Novartis Visudyne® (verteporfin) – eye drop for dry eyes Schering Ventavis® (iloprost) – PAH 18

Pharmaceuticals The needs of pharmaceutical companies include: • Need to conduct trials • Need for information (market research) • Need to gain acceptable access to people with Scleroderma through the correct channels • Need to get people with Scleroderma educated about the disease and treatment options (members of FESCA and member associations) • Need for advisers in strategy intelligence • Need for testimonials and case studies 19

FESCA Comparators 20

Comparator strengths and weaknesses Association Strengths Médecins Sans Frontières (MSF) 30 years Strong Media (on TV, bringing aid and help to stricken areas) Transparent image Not politicized Funding (independent individuals, Bill Gates; Governments) European Lupus Erythematosus Federation (ELEF) PAH alliance Weaknesses One patient on the board Perceived as a nice gathering once a year with no results Lost NGO status Not active Umbrella group for pulmonary hypertension Wanted to go too far outside of Europe – but there was a split in the board Lack of cohesion Lack of focus Lack of control of their strategic planning 21

FESCA Strategy 22

FESCA strengths 1. Expertise in Scleroderma – from the patient’s perspective 2. Capability to meet a healthcare need 3. Links with professional groups EUSTAR and EULAR-PARE 4. Status as young, vibrant, umbrella EU organization with links to local associations, professional groups, etc. 5. Balanced, multidisciplinary leadership from various backgrounds e. g. pharmaceuticals, journalist, etc. 6. Non-profit organization 7. Cohesive/coordinated focus 23

FESCA weaknesses 1. Unknown territory for the group 2. Need for a secretariat 3. No core funding yet 4. Limited numbers of known patients 5. Members are patients with health issues 6. Members are not all English-speaking 7. Members cannot meet frequently enough 8. Not enough Northern and Eastern European representation yet 9. No website yet 24

The FESCA Position 25

FESCA position “For governments, politicians, doctors, the pharmaceutical industry, and the media, FESCA will be the main focal point for all enquiry. As the EU’s only independent, not-for-profit, focused, umbrella patient organization with deep expertise in Scleroderma, FESCA is in a position to achieve this. Run with the motivation and energy of those whose lives are affected by the disease, FESCA has the support of EUSTAR and EULAR-PARE and disseminates expert knowledge while facilitating communications for all European Connective Tissue Disease stakeholders. FESCA is a clearing house for patients and the medical community, serving as a conduit for the dissemination of funds in support of research and outreach from a multiplicity of sources. With complete transparency in support of research and outreach, FESCA operates in pursuit of a world in which everyone with Scleroderma receives appropriate access to a comparable standard of successful care. ” 26

FESCA Programs, Activities, and Initiatives 27

FESCA themes for the next 3 years 2007 – Foundation 2008 -2010 – Education of doctors and sufferers, and raising awareness 2009 – EU launch of EU Scleroderma Day* *EU Scleroderma Day to evolve into World Scleroderma Day in time 28

Programs, activities and initiatives 29

FESCA Projects 30

Project responsibility Activity FESCA Leader Actions Strategic plan and slides Beata Garay Toth Ann Tyrrell Kennedy Create, circulate, collate feedback, and prepare for Florence meeting Website development Ann Tyrrell Kennedy Nele Caeyers Beata Garay Toth Design a brief, liaise with web company, decide site map, URL, copy development, circulation of copy Brochure/Press pack Ilaria Galetti Carmela Scarano Brief to be prepared Ann Tyrrell Kennedy to edit final copy Press pack - ATK to be point of contact E-bulletin/ newsletter Vanda Serras Beata Garay Toth Ann Tyrrell Kennedy Content to come out of website Survey/research Beata Garay Toth Options to be considered Project 7 modules Kim Fligelstone Confirmation from EUSTAR September 07 EUSTAR/World Congress of Scleroderma Kim Fligelstone Confirmation from EUSTAR September 07 Funding Ann Tyrrell Kennedy Kim Fligelstone Beata Garay Toth Identify and contact the pharmaceutical company marketing managers; use strategic plan and slides to show what is scoped out to secure support and funding/allocation Funding Vanda Serras Despo Charalambous Project team to be put together EU introduction Peter Bakker Prepared list of 30 MEPs with interest in healthcare 31

Funding and Sponsorship 32

Attracting pharmaceuticals Quality of Life/Impact of Scleroderma Survey: Offering market research data from the collation/meta-analysis of completed surveys Offering data from quality of life/Impact of Scleroderma Surveys in countries that have not had a recent survey Quality of life survey results would highlight the life-threatening nature of the disease, loss of interpersonal relationships, loss of autonomy, impact on employment, etc. Regular market research: Regular market research surveys conducted once a year using an independent company to help prepare the study E-bulletin/newsletter: An e-bulletin or an A 3 folded newsletter sent out at 3, 6, or 12 month intervals A feature schedule to include disease; politics; drugs; FESCA news; events etc. 33

Immediate funding requirements Project area EUSTAR; flights; accommodation; registration fee; room hire for meeting; etc. Budget needs € 12, 000 Brochure; 6 -8 pages; content development; editorial; copy approval; printing and production € 4, 500 Website; site map development; content development; design and coding € 7, 500 Press pack; content development; design; pack production; distribution € 4, 500 TOTAL € 28, 500 34

Projected 2008 funding requirements Project area Budget needs EUSTAR; flights; accommodation; registration fee; room hire for meeting; etc. € 12, 000 Start-up pack for new societies; content development; design; pack production; distribution € 18 -23, 000 EULAR stand; stand fee; registration fee; leaflet/flyer production € 2, 500 Project 7 - Education Modules; FESCA curriculum development; onsite meeting attendance; distribution; dissemination €TBC EU Scleroderma Day* set-up (Jan); meeting to set-up team; travel; accommodation € 37, 000 EU Scleroderma Day* team (Oct) € 44, 500 Total * EU Scleroderma Day to evolve into World Scleroderma Day in time €TBC 35

Projected 2009 funding requirements Project area Budget needs EUSTAR (£ 25 k); flights; accommodation; registration fee; room hire for meeting; etc. € 37, 000 Core materials (£ 20 K); content development; copy approval; reiterative review; scientific committee approval; etc. € 30, 000 March PA plans (£ 75 K); meeting for PAs to present plans; flights; accommodation; venue hire; meeting materials and support; moderator; meeting report; € 111, 500 EU Scleroderma Day (£ 125 K); core materials distribution; communications with members; secretariat support; themeing; € 185, 500 TOTAL (£ 245 K) € 364, 000 36

Projected 2010 funding requirements Project area Budget needs EUSTAR; flights; accommodation; registration fee; room hire for meeting; etc. € 12, 000 EU Scleroderma Day (£ 125 K); core materials distribution; communications with members; secretariat support; themeing; € 185, 500 Additional support activities; brochures, website, meetings, etc. TBC €TBC TOTAL (£TBC) €TBC 37

Glossary of Abbreviations CHF Congestive Heart Failure CNN Cable News Network CTD Connective Tissue Diseases ELEF European Lupus Erythematosus EU European Union EULAR European League Against Rheumatism EULAR-PARE EULAR-People with Arthritis/Rheumatism in Europe EUSTAR European Scleroderma Trials and Research Group FESCA Federation of European Scleroderma Associations GCM General Council Meeting GPs General Practitioners (family doctors) KOL Key Opinion Leader MEP Member of EU Parliament MSF Médecins Sans Frontières NGO Non-governmental organization PAH Pulmonary hypertension Qo. L Quality of Life RA Rheumatoid Arthritis WCS World Congress of Scleroderma 38