RDI Action Plan 2019 and Strategic Priorities

FINAL DRAFT ACTION PLAN 2019 Prepared by YLC and PT for discussion with and adoption by RDI Council (Nov 2018) Based on Draft prepared to base the budget and the funding strategy (June 2018) and Officers meeting (July 2018)

RDI MILESTONES IN 2019 RDI established as a legal independent NGO from January 2019 Major combined events in New York City around Rare Disease Day 2019: RDI AGM + RDI 5th Annual Open Meeting + Global Commission to end Diagnositc Odyssey of Children with a RD Report Launch at Microsoft HQ Times Square on Feb 20 + Second High Level Policy Event of the NGO Committee for Rare Diseases at UN HQ New York on Feb 21 Establishes regular relationship with UN Member states Permanent Missions in Geneva and New York including an informal network Friends of Rare Diseases and Briefing breakfasts Sign MoU WHO-RDI Collaborative Framework 2019- 2021 (1S2019)

RDI MILESTONES IN 2019 Focus its advocacy to secure RD inclusion in UN Political Declaration on UHC High Level Meeting & UN Plenary Assembly - Sept 2019 Grow its advocacy toward a UN Resolution on Rare Diseases horizon 2020 RDI establishes a Secretariat of 3 FTE staff/4 persons, formal offices and develops its budget (from 160 K to 560 K ) RDI Conference Programme: promoted & scaled up: CORD/RDI/ ReACT- Toronto in May; ALIBER, Mexico in Sept ; APARDO, Singapour in Oct; WODC Washington, April & WODC Barcelona, Nov; other Conferences in LMIC (2ndS 2019) RDI Fellowship Programme launched & promoted RDI Faculty Experts launched & partly established

RDI STRATEGIC PRIORITIES 1. Strengthen RDI as a well-governed and sustainable organisation 2. Build a legitimate Global RD Patient Alliance 3. Advocate for RDs to be an International Public Health Priority and Raise Awareness of RDs worldwide 4. Advocate in favour of PLWRD worldwide in key areas 5. Build Capacities of Members and Patient Advocates

1. STRENGTHEN RDI AS A WELL-GOVERNED AND SUSTAINABLE ORGANISATION 1.1 Governance 1.2 Secretariat 1.3 Communications 1.4 Financial Sustainability: EURORDIS 1.5 Financial Sustainability: Resource Development & RDI Alliance of Companies for Patient Centred Action

1. 1 GOVERNANCE Legal incorporation in France, as Association Loi 1901, as an NGO, official publication based on Statutes Council to adjust and adapt By Laws before AGM Feb 2019 Council TC every other month; Officers TC every other month Two F2F Meetings of Council Council Elections (on-line Nov-Dec 2018) 7 members renewed by third every year, exceptionally 4 in 2019 Annual General Assembly 20 Feb 2019: Adoption Annual Activity & Financial Report 2018 Adoption Work Programme: Action Plan & Budget 2019 RDI Open Meeting (open to non members and other stakeholders to attract new members and develop synergies with other actors in the RD ecosystem) Policy of Relations with Commercial Companies

1. 2 SECRETARIAT 1 FTE Director Paloma Tejada, based in Geneva 1 FTE Manager (to be recruited 1S2019) 1 Part-time Public Affairs Manager, Clara Hervas, based in Brussels office 1 Part-time Consultant Corporate Relations Advisor , Jill Bonjean, based in Paris RDI offices established in Brussels on 11 rue D Egmont Brussels 1050 (in-kind EURORDIS, addendum MoU) and in New York on 521 5th Avenue New York NY 10175 (in-kind corporate VOZ)

1. 3 COMMUNICATIONS Website updates Communication material Pending recruitment of Programme Manager New visual identity New Brochure New PPT Presentation Template New Kakemonos Active Social Media on Twitter and Instagram

1. 4 FINANCIAL SUSTAINABILITY : EURORDIS EURORDIS Support: MoU signed for 5 years 2019-2023 EURORDIS will continue to provide professional services: a) employ 1 FTE staff (RDI Director) b) provide 0.5 FTE public affairs staff for advocacy dedicated to RDI and related international activities c) provide financial & administrative services for management of accounts d) provide hosting, maintenance and update of website e) Provide official Office in Brussels

1. 5 FINANCIAL SUSTAINABILITY RESOURCE DEVELOPMENT RDI secures revenue to: cover its own external expenses hire the Programme Manager (1 FT) hire the Consultant in Resource Development (90 days) Raise unrestricted funds by setting up a Corporate Group: RDI Alliance of Companies for Patient-Centred Action Raise restricted targeted funds for Specific Programmes: Conferences, Fellowships and Faculty Expert In-kind coporate contributions

1. 5 RESOURCE DEVELOPMENT RDI ALLIANCE OF COMPANIES FOR PATIENT CENTRED ACTION Made of commercial companies Different industry sectors from the beginning: pharma, biotech, diagnostic, device, digital, CRO, distribution, PR & Law firms, other Membership Fees: 20 000 EUR /year, looking for a long term commitment Provides unrestricted resources for core activities Counter-part offer: a) invited to the Annual RDI Open Meeting, b) invited to RDI Policy events, c) invited to all Level-3 RDI Conferences; d) invited to attend 2 dedicated Webinars per year Code of Conduct to be signed by all members Policy on Relations with Commercial Companies

2. BUILDING A LEGITIMATE GLOBAL RARE DISEASES PATIENT ALLIANCE 2.1 Next patient organisations to be invited to join 2.2 Strategic partnerships in RDI eco-system

2. 1 NEXT ORGANISATIONS TO BE INVITED TO JOIN 56 Members as end 2018: 33 National Alliance and 14 International Federations and 4 Regional Networks Expand membership base > +10, toward 70 members as end 2019 Actively recruit more International Federations PHA International, Tuberous Sclerosis, CF Worldwide, Duchenne World, Dravet Foundation, Rett Syndrome International, FOP, IPWSO, OIFE Target US based international federations at NY events Recruit National Alliances in following countries: Peru, Brazil, Chile, Philippines, Turkey, Algeria Maintain informal relations with TFRD, KORD Bring in regional networks: APARDO, African-Rare.org Call for Membership Fees

2. 2 STRATEGIC PARTNERSHIPS NGO Committee for Rare Diseases Executive Board International Rare Diseases Research Consortium (IRDiRC) - Patient Advocates Constituency Committee (PACC) Orphanet Global figures on RDs+ ICD-11 and OrphaCode + promote Orphanet in new languages eg Arabic, Persian International Federation of Pharmaceutical Manufacturers & Associations (IFPMA) Working Group on RD Roundtable on Barriers to Expanded Access to OD in LMICs Asia Pacific Economic Cooperation (APEC) LSIF Rare Disease Network patient input in APEC Rare Disease Action Plan 21 countries Global Commission on Ending the Odyssey for the Diagnosis of Children with Rare Diseases patient input in

3. ADVOCATE FOR RD TO BE AN INTL PUBLIC HEALTH PRIORITY & RAISE AWARENESS FOR RD WORLDWIDE 3.1 Building up our capacities in Advocacy Actions 3.2 Promoting RDs as a priority within UN Sustainable Development 2030 Agenda and towards a UN Resolution on RDs 3.3 Placing RDs in the Universal Health Coverage Agenda top priority for WHO 3.4 Establishing WHO-RDI Collaborative Framework 3.5 Towards an evidence-based Advocacy

3.1 BUILDING UP OUR CAPACITIES IN ADVOCACY ACTIONS Reach out to a critical mass of countries Permanent Missions (20) in Geneva and New York, select future country champions in each region of the G77 in close collaboration with RDI Member National Alliances Establish an informal network Friends of Rare Diseases in Geneva and New York, organised briefings, breakfast meetings and small awareness or education conferences RDI Advocacy Committee: add TIF and FADEPOF; web meetings every other month; focus on WHO Collaborative Framework, Position Papers, Side events Ad hoc Drafting Groups on UHC and UN Resolution Staff: Clara Hervas, International Public Affairs

3.2 PROMOTING RD AS PRIORITY IN UN SDG 2030 & TOWARDS UN RESOLUTION ON RD Co organise with NGO Committee for Rare Diseases Second High Level Policy Event in New York on February 21st Develop a first draft of UN Resolution on RDs with Ad Hoc Drafting Group, Advocacy Committee, Permanent Missions from 3 to 5 countries, consult UN experts Possible High Level Conference of Perm Reps from G77+ China on RDs sponsored and organised by one MS (tbc)

3.3 PLACING RARE DISEASES IN THE UHC AGENDA RDI Contribution to UN Political Declaration on UHC (Feb) WHO 72nd World Health Assembly Informal Side Event on RD & UHC (May) Outreach and briefings to co-facilitator countries (Hungary and Thailand) and to drafting countries (France, Japan) and champion countries (EU Delegation, Philippines, Kuweit, ) High Level Meeting on UN Political Declaration on UHC in New York & UN Plenary General Assembly (Sept) Possible side meetings with Perm Reps, small briefing meetings before the HLM RDI Members to advocate to their MoH and MoFA and Perm Reps to attend the WHO WHA Side event in May and support inclusion of RDs in the Declaration

3.4 ESTABLISH WHO-RDI COLLABORATIVE FRAMEWORK Draft and sign Memorandum of Understanding WHO-RDI Collaborative Framework 2019- 2021 on 5 ambitions WHO 72ndWHA informal side event and secure MS and NSA support Mobilise Perm Reps in Geneva with Friends of RDs , small meetings, breakfast briefings Explore the feasibility of a light Resolution on RDs at WHO Executive Board of January 2020 Apply to enter into NSA in Official Working Relations with WHO in July 2019 for decision in January 2020 (earliest, might have to delayed one year)

3.5 TOWARDS AN EVIDENCE-BASED ADVOCACY Advocacy Fact Sheets on key figures in RDs (prevalence, global impact, children/ adult, genetic / non-genetic) Advocacy Fact Sheets on surveys on social impact of RDs Explore partnership to perform the International State of the Art of RDs in a standard, progressive and sustainable way

4. ADVOCATE FOR PLWRD AT A GLOBAL LEVEL IN KEY AREAS 4.1 Engagement in Research: IRDiRC and ReACT 4.2 Access to Diagnosis: Global Commission to end the diagnosis Odyssey of Children with a Rare Disease 4.3 Access to Medicines: WHO and IFPMA

4.1 Engagement in Research: IRDiRC and ReACT RDI is a member of IRDiRC, represented by Ritu Jain Take part in IRDIRC s Consortium Assembly Contribute to IRDIRC s goals and mission Promote membership amongst new countries RDI supports all patient reps members of RDI in IRDIRC PACC (9 out of 12) Contribute to PACC s policy work and contribute to design and dissemination to RDI members of survey on patient engagement in research Partner with ReACT Conference, Toronto, Canada (May 5 - 8) with BLACKSWAN Foundation

4.2 Access to Diagnosis: Global Commission to End Diagnostic Odyssey of Children with RD The Global Commission initiated and co-chaired by EURORDIS, Microsoft and Shire, overall members of the Global Commission include patient reps from RDI members: CORD, NORD, CORD China, EURORDIS Support and involve all RDI members in Launch events on 20 February at Microsoft Global Headquarters, simultaneous 2ndlaunch in Brussels at Friends of Europe and 3rdlaunch event in Beijing on Feb 28 by CORD China RDI will take part in 2 out of 4 pilots Use the Recommendations as basis to develop RDI position on diagnosis Bring the recommendations from Global Commission experts into Collaborative Framework with WHO

4.3 Access to Medicines Propose a Collaborative Framework with WHO that includes actions to improve access to medicines in LMIC Mobilise political support of Member States through Perm Reps in Geneva to include access to medicines for rare diseases in WHO Fair Pricing agenda Organise a first Roundtable on barriers to Expanded Access Programme in LMIC with IFMPA RD Working Group in Geneva (1st Q 2019) Organise a second Roundtable on Innovative Access Programmes with IFPMA, WHO, HTAi and other global actors in Geneva (4th Q 2019)

5. BUILD CAPACITIES OF MEMBERS & PATIENT ADVOCATES 5.1 RDI 5thAnnual Meeting, Feb 20, New York 5.2 RDI Conference Programme 5.3 RDI Fellowship Programme 5.4 RDI Faculty Experts 5.5 RDI Peer to Peer Exchange & Networking Virtual Platform & Educational training tool kits (Staff: programme manager when recruited)

5. 1 RDI ANNUAL MEETING:AGENDA Toward a UN Resolution on Rare Diseases Horizon 2020 Universal Health Coverage: advocacy priority & achievement 2019 WHO - RDI Collaborative Framework 2019-2021: achievement 2019 Regional RD Patient Networks APARDO APEC ALIBER - LATAM RARE AFRICA EURORDIS EU & Beyond

5.2 RDI CONFERENCE PROGRAMME Level 3 - "Conference co-organised with RDI Level 1 support + RDI in Programme Committee; RDI covers RDI speakers, Fellowships Content aligned with RDI s overall strategy + advocacy topics Ex. CORD/ReACT Level 2 - "Conference in Partnership with RDI Level 1 support + RDI in Programme Committee; RDI sometimes covers some RDI speakers, No Fellowships RDI objective: to build capacities on specific topics of need (national plans, registries..) Ex. ECRD, NORD Summit, WODC, IRDiRC, ISPOR , APARDO, ALIBER Level 1 - "Conference supported by RDI RDI logo, communication and promotion, link in RDI website Ex. Conferences organised by Member NA or IF

5.3 RDI FELLOWSHIP PROGRAMME Objectives: To build capacities of patient advocates and organisations To support networking, exchange of experience, dissemination of patient advocacy knowledge and best practices To identify and train leader patient advocates To enable RDI members and other patient advocates who cannot afford to take part into RDI events Available for: Annual RDI Membership Meeting NY Feb 20 NGO CfRD High Level Policy Event at UN NY Feb 21 all RDI Level 3 Conferences WHO WHA side event Training seminars ie School

5.4 FACULTY EXPERTS Individuals Leaders from different horizons: patient advocates, academics, reps of learned societies, reps of industry, experts by themselves (retired or active) Appointed by the RDI Council; can also apply To provide RDI with a pool of quality speakers for all conferences, workshops, meetings, to serve as speakers customized to local or regional event specific needs Expert resource to RDI members for specific activities and for RDI Advocacy Committee Cultivate committed individuals to link back to their organisations and foster international collaborations/ partnerships and multi-stakeholder engagement To produce scientific / opinion papers for publication

5.5 PEER TO PEER EXCHANGE NETWORKING & KNOWLEDGE BASE Pending recruitment of Programme Manager: Create an RDI community, as a virtual forum for members eg. Facebook Workplace Create a profile of basic information about each member and its activities of potential interest to other members Organise one Webinar each month to present one member, or two members on same topic, and discuss their activities and interests in common , webinars to be recorded and made available to other members Advocacy training tool kits to train patient reps to speak about global initiatives and talk to their governments and policy-makers. Also to educate their membership and support local advocates at local level. Educational training tool kits: pilot on gene therapy. Similar to IAPO s toolkit on biosimilars. Non-static information, web- based. Not branded, generic. Accessible to lay audience. RDI

RDI 2018 Preliminary Budget Expenses Director Manager of Relations with POs Junior Public Affairs (part-time) Corporate Relations Manager Volunteers AMM NY + Council F2F WHO WHA Geneva side meeting 2nd Council F2F -B/B to WODC BCN Communications NGO Committee meeting Travel and Subsistence Overheads (25%) Sub-total Core Expenses EURORDIS RDI TOTAL 86 000 86 000 48 000 24 000 43 000 103 090 18 200 9 550 16 900 5 000 20 000 15 000 97 185 460 153 48 000 24 000 43 000 103 090 18 200 9 550 16 900 5 000 20 000 15 000 71 413 181 413 0 278 740 RDI Conference Programme 3 x Conference level-2 2 x Conference level-3 RDI Fellowship Programme RDI Membership Meeting (AMM) Conferences Level-3 UN related events Capacity building Overheads (10%) Sub-total Programmes 25 000 10 000 15 000 60 000 20 000 20 000 6 000 14 000 25 000 10 000 15 000 60 000 20 000 20 000 6 000 14 000 17 000 102 000 17 000 17 000 0 85 000 Total Expenses 198 413 363 740 562 153 Income Membership fees Volunteers RDI Alliance of companies Programmes EURORDIS Int. Init. Total Income EURORDIS RDI TOTAL 6 000 103 090 180 000 85 000 6 000 103 090 180 000 85 000 198 413 572 503 198 413 198 413 374 090