Speaker Presentations

Opening Session: Leave no person living with a rare disease behind

The NGO Committee for Rare Diseases: working on a common cause at the global level (Anders Olauson, Chair of the NGO Committee for Rare Diseases; Chairman, of Ågrenska Foundation)

Remarks by the Host (H.E. Mr. Sven Jürgenson, Ambassador and Permanent Representative, Permanent Mission of Estonia to the United Nations)

Rare Diseases and the Sustainable Development Goals (Leslie Wade, Chief ECOSOC and Interorganizational Cooperation Branch)

Human Rights of people living with a rare disease (Andrew Gilmour, Assistant Secretary-General for Human Rights and Head of the Office of the High Commissioner for Human Rights in New York)

Universal Health Coverage for people living with a rare disease (H.E. Vitavas Srivihok, Ambassador and Permanent Representative, Permanent Mission of Thailand to the United Nations)

Remarks by Chair of the UN Group of Friends of Universal Health Coverage (H.E. Toshiya Hoshino, Ambassador and Deputy Permanent Representative, Permanent Mission of Japan to the United Nations)

Session 2: Recognising rare diseases as a policy priority: Empowering people living with a rare disease

Introduction to the session and the Rare Disease Campaign (Terkel Andersen, President of the Board of Directors, EURORDIS-Rare Diseases Europe)

A need for visibility within support systems (Lieven Bauwens, Secretary General, International Federation for Spina Bifida and Hydrocephalus; Member of Inception Executive Board NGO Committee for Rare Diseases)

Inclusion of rare diseases in scientific research (Irene Norstedt, Acting Director responsible for the Health Directorate within the Directorate-General for Research and Innovation, European Commission and Head of Unit, Innovative and Personalised Medicine Unit; Member of the International Rare Diseases Research Consortium (IRDiRC))

Session 3: Universal Health Coverage and reduction of inequalities for inclusive development

The extreme of ‘Leave no one behind’ – undiagnosed patients (William Gahl, Chair, Undiagnosed Diseases Network International (UDNI); Clinical Director, National Institutes of Health, National Human Genome Research Institute)

New opportunities to improve the diagnosis of children living with a rare disease (Simon Kos, Chief Medical Officer and Senior Director, Worldwide Health, Microsoft & member of the Global Commission on ending the Diagnostic Odyssey of Children with Rare Diseases)

The need to address the ‘Big 5’ from a lifelong perspective: healthcare, social care, school, insurance and labour (Robert Hejdenberg, Chief Executive Officer, Ågrenska Foundation)

Session 4: Ensuring inclusion and participation of people living with a rare disease through national policies and strategies

Introduction to the session (Daniela Bas, Director of Division for Inclusive Social Development, UN Department of Economic and Social Affairs (DESA), United Nations Secretariat)

Nan-Byo Strategy – Japan (Toshi Ezoe, Counsellor, Permanent Mission of Japan to the United Nations)

Implementing a national rare disease plan starting with national registry – Colombia (Germán Escobar Morales Director of Health, ProPacífico)

Establishing an APEC Rare Disease Registry Network: QUT and a framework for collaboration (Matthew Bellgard, Director of eResearch, Division of Research and Innovation, Queensland University of Technology and Chair of APEC LSIF Rare Disease Network)

Networks for applying research to diagnosis and care – Canada (Kym Boycott, Professor of Pediatrics, University of Ottawa; Care4Rare)

Rare Diseases Clinical Research Networks – United States of America (Marshall Summar, Director, Rare Disease Institute at Children’s National, Washington, D.C; Chairman of Board of Directors of the National Organization for Rare Disorders (NORD), USA)

Resource centres for rare diseases – Romania (Dorica Dan, President, RONARD (Romanian National Alliance for Rare Diseases); Member of Board, EURORDIS-Rare Diseases Europe)

Building a grassroots approach for persons living with a rare disease – Iran (Hamid. R. Edraki, Managing Director, Rare Diseases Foundation of Iran)

The case of rare diseases in China (Shuyang Zhang, Vice President of Peking Union Medical College Hospital (PUMCH), Director of Clinical Pharmacology Research Center, PUMCH)

Integration of rare diseases in the national health system – Philippines (Cynthia Magdaraog, President of the Philippine Society for Orphan Disorders, Inc. (PSOD))

Closing session

Official video statement from Mrs. Michelle Muscat, Spouse of the Prime Minister of the Republic of Malta

Official video statement from Mrs. Tamara Vucic, Spouse of the President of the Republic of Serbia