Towards a United Nations Resolution on Rare Diseases: Objectives, Progress, Working together CH Yann Le Cam Chief Executive Officer, EURORDIS-Rare Diseases Europe Council member , Chair Advocacy Committee, Rare Diseases International

I. Reminder of the overall Strategy

The United Nations: 2030 agenda The 2030 Agenda: The Sustainable Development Goals (SDGs) Universal & inclusive: « Leave no one behind » Return to social development Inter-linkages Post-2015: agreed in a multi-stakeholder process (very diverse) Dramatically expanding the agenda Greater diversity of topics Beauty of the 2030 agenda – all dimensions (social, economic and environmental dimension of development) Universal and inclusive : for both developing and developed countries (focus on the inequalities that still exist within these countries) “if the goals are not met for everyone equally then they are not met” Return to the social development agenda of the Copenhagen Declaration of 1995 and to the fundamentals of the UN (at 1945 creation) SDGs are supposed to talk to each other: foster and develop inter-linkages CONCLUSION OF SECTION: So United Nations is the right platform for advocating for rare diseases at the global level. For decades, the UN has been a driver for: strong public health agenda, social development and inclusion developments in science, technology and innovation human rights for all And now is the right time: As we will explain later when presenting the rationale behind the NGO Committee for RDs, there are a number of synergies between the goals of the rare diseases community and the SDGs.

United Nations: The right place For decades, the UN has been a driver of a strong public health agenda, social development, inclusion, and progress,developments in science, technology and innovation, and human rights for all. So United Nations is the right platform for advocating for rare diseases at the global level. For decades, the UN has been a driver for: strong public health agenda, social development and inclusion developments in science, technology and innovation human rights for all And now is the right time: As we will explain later when presenting the rationale behind the NGO Committee for RDs, there are a number of synergies between the goals of the rare diseases community and the SDGs.

THE UNITED NATIONS SYSTEM New York GENERAL ASSEMBLY 193 members & Committees Security Council Economic and Social Council (ECOSOC) International Criminal Court International Court of Justice UN PROGRAMMES & FUNDS UNDP UNHCR UNICEF UNEP Secretary General DESA CoNGO SPECIALISED AGENCIES FAO UNESCO WHO WTO Geneva CH Very simplified diagram: Peace and security: UN security council and the International Criminal Court Human rights: International Court of Justice, programmes like the UNHCR (refugees) and the Human Rights Council (Geneva) Development: ECOSOC and a number of programmes (UNDP, UNICEF) and specialised agencies (FAO – food & agri; WTO; UNESCO) Specialised agencies are autonomous agencies working with the UN throught the coordinating mechanisms of the Economic and Social Council (ECOSOC) CoNGO is the Conference of NGOs in Consultative Status with the UN – they facilitate the interaction between civil society and the UN There are two so-called « bubbles » in the UN system which are particularly relevant for health issues and for the rare diseases community: The Geneva Bubble with the WHO The New York Bubble with the GA, ECOSOC and the Secretariat (which includes DESA, the Department of Economic and Social Affairs) and CoNGO (mostly based in NY)

THE UNITED NATIONS SYSTEM CH The UN system: Very complex Infinity of accronyms and interlinkages Generally can think of it as divided along the 3 broad themes of the UN: peace, development and human rights

1st UN « bubble»: Geneva Focus on Human Rights: Office of the United Nations High Commissioner for Human Rights United Nations Human Rights Council Focus on Health: World Health Organisation (Specialised Agency) Communicable diseases Increasingly non-communicable diseases Access to medicines debate focused on IP and generics Rare Diseases not at all on the agenda CH Focus on Human rights: bodies like the office of the UN High Commissioner for Human Rights or the UN Human Rights Council Focus on the right to health: WHO Current priorities of WHO include: - communicable diseases (HIV/AIDS, Ebola, malaria and tuberculosis) + UNAIDS Increasingly, non-communicable diseases (sexual and reproductive health; nutrition, food security and healthy eating; substance abuse; driving the development of reporting, publications, and networking). Debate on access to medicines is currently prompted by India & Brazil because they have a growing generic industry

2nd UN « bubble »: New York Rebirth of universal mission of UN with SDGs SDG is a Development agenda: Economic and Social Council (ECOSOC) Department for Economic and Social Affairs (DESA) United Nations Development Program (UNDP) Reviving definition of health: more than the mere absence of disease Health & well-being « Leave no one behind » Universal Health Coverage CH The focus in New York is rather on development. With ECOSOC, DESA and UNDP During many decades the aim was economic development, particularly of developing countries (for the most part through agencies like the World Bank or the IMF funding schemes). Now the agenda has shifted back to the promises of the Copenhagen declaration of 1995: social development and universality – through the work of bodies of the GA and ECOSOC With the 2030 Agenda and the SDGs, the two « bubbles » are converging and the WHO is more and more applying the definition of health in its Constitution: « Health is a state of complete physical, mental and social well-being and not merely the absence of disease”

II. Progress made since 2016

ProGress made since 2016 1st High Level Event of the NGO Committee for Rare Diseases 11 November 2016, United Nations HQs NYC

ProGress made since 2016 Rare Diseases and the Rights of persons with disabilities 1) Conference of States parties to Convention on the Rights of Persons with disability in New York – 12 to 14 June 2018 Oral Statement delivered by Clara Hervas (EURORDIS) on behalf of NGO Committee for Rare Diseases 2) Report of the United Nations Special Rapporteur on the Rights of persons with disabilities: Catalina Devandas Mention to rare diseases in report to the UN General Assembly in September 2018 “Health systems must respond to the needs of the diversity of persons with disabilities. […]. States should consider developing and implementing policies and practices targeting the most marginalized groups of persons with disabilities (e.g., persons with multiple or severe impairments, rare diseases or deaf- blindness) in order to accelerate or achieve de facto equality in access to health care.”

ProGress made since 2016 Rare Diseases and Human Right to Health Contribution to report of the Office of the High Commissioner for Human Rights (OHCHR) on Health and SDGs Official Written Statement at the Human Rights Council June-July 2018

ProGress made since 2016 Rare Diseases and the World Health Organisation Nata Menabde, Executive Director of WHO Office to UN in New York invited to speak 1st High Level Event of the NGO Committee for Rare Diseases, 11 November 2016, United Nations HQs NYC Letters to 4 candidates to position of Director-General of WHO – Mar/Apr 2017 Dr Tedro’s statement of support when he was candidate to the position of WHO Director-General for First Rare Disease Policy Event in Geneva, February 10, 2017 Peter Beyer, of WHO Essential Medicines invited to speak at First Rare Disease Policy Event in Geneva RDI contributions to Public Consultation on WHO 13th General Programme of Work 2019-2023 in October and November 2017

ProGress made since 2016 Rare Diseases and the World Health Organisation Meeting with high level officials at WHO Geneva Secretariat on November 22nd, 2017 -Mariângela Simão, Assistant DG Access to Drugs, Vaccines and Pharmaceuticals -Dr Naoko Yamamoto, Assistant DG Universal Health Coverage and Health Systems Cluster -Rudiger Krech, Director Health Systems and Innovation -Sarah Garner, Coordinator - Innovation, Access and Use, Essential Medicines an Products Invited to propose a set of ambitious goals and areas of concrete collaboration to address the challenges in the field of rare diseases within the 2030 Sustainable Development Agenda and WHO 13th General Programme of Work. Internal consultation with members throughout 2018

ProGress made since 2016 Rare Diseases and the World Health Organisation Formal submission of proposed WHO-RDI Collaborative Framework, November 2018 5 Ambitions with specific Goals 2030 & Actions (2019-2023) Awareness & Visibility UHC & Healthcare Diagnosis Medicines & Assistive Technologies National strategies

ProGress made since 2016 Rare Diseases and the World Health Organisation Invited to request and submit MoU with WHO (based on Collaboration Framework) Ongoing process of finalisation/ submission of MoU Meeting with WHO Director-General Dr Tedros on Rare Disease Day – February 28th! WHO Delegation: Dr Tedros, General-Director Dr Bernhard Schwartlander, Chef de Cabinet Dr Soumya Swaminathan, Deputy Director- General Programmes Dr Naoko Yamamoto, Assistant DG Universal Health Coverage and Health Systems Cluster Rudiger Krech, Director Health Systems and Innovation

ProGress made since 2016 Universal Health Coverage Preparing towards High Level Meeting on UHC during UNGA 74th Session (Sept 2019) Outreach and briefings to co-facilitator countries (Hungary and Thailand) and to drafting countries (France, Japan) and champion countries (EU Delegation, Philippines, Kuwait, …) Participation to Universal Health Coverage Day, 12 December 2018: High Level Panel organised by WHO & the UN Group of Friends of UHC Participation of representative: Ms. Durhane Wong-Rieger, RDI Chair and member of the Inception Executive Board of the NGO Committee for Rare Diseases

III. A Resolution of the General Assembly of the United Nations to address the challenges of people living with a rare disease

What is a UN GA resolution? The term « UN resolution » can mean many things: GA, Security council, WHA, ECOSOC, HRC UN GA Resolution = 193 Member States views GA is organised in groups: EU, G-77, FP & GH Not binding on MS but acts as soft law, set a course of action Not binding on Specialised Agencies (WHO, World Bank, IMF) – but influence as de facto coordinator of UN system Binding on the Secretariat (DESA, OHCHR) and UN Funds & Programmes (UNDP,UNICEF) There are many organs of the UN that adopt resolutions: The Security Council on peace and security matters The WHA at the level of the WHO ECOSOC also adopts resolutions But the highest-level comes from the General Assembly which assembles 193 nation-states as members GA Resolutions reflect the degree of intergovernmental agreement, the evolution of political ideas and the state of global cooperation on a given topic. Not binding for MS but it can act as soft law – Include policy recommendations and identify actions to be taken by MS and UN System GA resolution assign mandates to the UN secretariat and bodies responding to the GA. They are binding to the Funds and Programmes: UNDP and UNICEF for example. GA Resolutions are not binding on Specialised Agencies like the WHO because they have their own intergovernmental structure (World Health Assembly) – but even if the head of the WHO is not accountable to the Secretary-General, they still recognise their coordinating role within the UN system.

Different elements of a GA Resolution – the example given is fictional. Always have preambular paragraphs that link to existing resolutions or conventions, or to the current context. Operative paragraphs: take note, calls upon, encourages, decides, requests

a) Simple / Awareness-raising Resolutions Examples: 61/225. World Diabetes Day (2006) 62/139. World Autism Awareness Day (2007) International Universal Health Coverage Day (2017) If internet - Open links to show length Diabetes day: Resolution recognised that diabetes was a chronic, debilitating and costly disease Welcomed the observation of 14 November as World Diabetes Day since 1991 with WHO co-sponsorship and made it official UN Day Encouraged Member States to develop national policies for the prevention, treatment and care of diabetes World Autism Awareness day: Resolution designated 2nd April as official day Encouraged MS to raise awareness Sickle-cell anaemia: Resolution asked to raise awareness on 19th June Urged MS to establish national programmes for it In 2017 there was a UN Resolution on Bycicle Day…

b) Content-rich & requiring Substantial action Examples: Recognition of sickle-cell anaemia as a public health problem (2008) - lighter 58/173. The right of everyone to the enjoyment of the highest attainable standard of physical and mental health (2003) 65/277. Political Declaration on HIV and AIDS: Intensifying Our Efforts to Eliminate HIV and AIDS (2011) 72/140 Persons with albinism (2017) 64/265. Prevention and control of non-communicable diseases (2010) 65/186. Realizing the Millennium Development Goals for persons with disabilities towards 2015 and beyond (2010) Much more details as to which UN agencies should act, what programmes should be put in place or conventions should be ratified Non-communicable diseases: Resolution decided to convene a high-level meeting of the General Assembly in September 2011, with the participation of Heads of State and Government (and had a 3rd high level meeting on this in September 2018) Millenium Dev Goals for persons with disabilities: asked for report by Sec-Gen two years after resolution on progress made The one on HIV and AIDS is special: it is a Resolution adopting a Political Declaration by Member States (recalling past declarations 2001 & 2006) – 105 points (note, recall, recognise, commits, pledges)

EXAMPLE: Persons with Albinism

Example: Disability and MDgs

Tabling a Resolution at the UN GA: Process UN GA Resolutions require support of at least one UN Member State as sponsor Can be co-sponsors Process: Sponsor drafts resolution Sponsor tables resolution = technical term for formally submitting a draft resolution text to the GA The draft resolution must be tabled under a specific agenda item A draft resolution/decision can be adopted by consensus or by a vote (simple majority) Concretely, how do you do it? The GA is ‘in session’ for the entire year, but mid-September to Christmas is the main part of the GA when most work is carried out by the Committees. Sometimes resolutions can be tabled through Committees of the GA: 2nd Committee: Economic and Financial Committee – macroeconomic policy, operational activities for development financing for development, groups of countries in special situations, Globalization and interdependence, Eradication of poverty, information and communication technologies for development, Agriculture and food security, sustainable development Support: Usually DESA 3rd Committee: Social, Humanitarian and Cultural Committee Promotion and protection of human rights Support: Office of the High Commissioner for Human Rights (OHCHR) is the main substantive secretariat to the Committee

IV. WORKING TOGETHER is the key for a MEANINGFUL RESOLUTION and for ADOPTION

A mode of action FOR our advocacy on UHC and UN Resolution Engaging in advocacy activities to create the right environment and a network – great potential! Integrating a mention to rare diseases in UN texts, Several attempts this year and success with 2018 report of Special Rapporteur on Rights of Persons with Disabilities to the General Assembly + ongoing UHC Political Declaration (Sept 2019) Meetings with Permanent Missions in NYC (May, June and October 2018, February 2019) Argentina Belgium Brazil Colombia Cyprus EU Delegation Integrate mention to rare diseases into the Foreign Policy and Health Initiative resolution? Next chair 2019 is France Emirates Estonia France Germany Greece Hungary India Indonesia Iran Japan Kuwait Luxembourg Malaysia Mexico The Netherlands Romania Serbia Spain Turkey Uruguay

WORKING TOGETHER For a meaningful and impactful resolution RDI Advocacy Committee All RDI members participate in internal consultations in 2019-2020 on draft UN Resolution on Rare Diseases Background: RDI Joint Declaration, already debated at large and adopted Send information or reflexion about the situation in your country or for your disease which you think would make the resolution more meaningful or impactful – state of the art Identify people you know who could help us as experts in these matters or as influencers

ProGress made since 2016 For the adoption of the resolution Your Ministry of Foreign Affairs Your Ministry of Health Your Permanent Mission to UN in New York Your Permanent Mission to UN in Geneva Mind your supportive ministers and parlementarians Possible creation of a « Friends of Rare Diseases » bringing together all UN Member states supporting We will have to work on certain countries were there are no national alliances for rare diseases _ Int Federations