Session I: Opening and Plenary Lectures. Policies: The main steps for recognizing the societal value of diagnosis, prevention and treatment of rare diseases
Opening
ICORD’s capabilities. Domenica Taruscio (ICORD Past-President, Italy)
ICORD’s immediate challenges. John Forman (ICORD President-Elect, New Zeeland),
ICORD – a platform for bridging and cooperation. Virginia Llera (ICORD-President, Argentina
Plenary Lectures
Health policy for rare diseases in The Netherlands. Fred Krapels (Dutch Ministry of Health, The Netherlands)
EU major policies on the patients’ perspective and international engagement. Yann Le Cam (EURORDIS, France)
The current state of rare diseases in Mexico. Mario Alanis Garza (Federal Commission for Protection against Health Risks, COFEPRIS, Mexico)
The challenging process from a global vision to a global social value impact. Virginia Llera (ICORD, Argentina)
Session II: Key-note plenary lectures (presentations not available)
TI PHARMA Lecture: Addressing the needs of the neglected: control of schistosomiasis in young African children and infants. Russell Stothard (Liverpool School of Tropical Medicine, United Kingdom)
ICORD Lecture: Mitochondrial drug development: from bench to bedside. Jan Smeitink (Radboud University Medical Centre, The Netherlands
Session III: Ways forward: Evaluation, development and societal impact of orphan drugs. Joint session with ZonMw
Evaluation of Orphan Drugs: Ways forward. Carla Hollak (Academic Medical Center, The Netherlands)
Novel developments in HTA methodology (Health Technology Assessment). Leona Hakkaart (Institute of Health Policy and Management, The Netherlands)
The politics of rare diseases and orphan drugs. Rob Hagendijk (University of Amsterdam, The Netherlands)
Novel business models for Orphan Drug development. Hans Büller (ErasmusMC, The Netherlands)
Session IV: Poster session (no presentations available)
Session V: Special Lecture; International collaboration
The Biopontis Alliance for Rare Diseases (BARD) – a new collaborative model for bridging from discovery to cures candidates. Erik Tambuyzer (BARD, Belgium)
International school of public health for rare diseases. Domenica Taruscio (Istituto Superiore di Sanità, Italy)
New strategic direction at GSK. Martin Andrews (GSK, United Kingdom)
Session VI: Primary prevention of neural tube defects. Making a common disease rare
Congenital Anomalies as Preventable Rare Diseases. Amanda Julie Neville (Universitaria di Ferrara, Italy)
Folate and prevention of neural tube defects: Tracking red blood cell concentrations will help guide policy decisions about fortification. Derrick Bennett (University of Oxford, United Kingdom)
Could folic acid supplementation bridge the gap of the results of fortification? State of the art in Europe and future perspectives. Amanda Julie Neville (Universitaria di Ferrara, Italy) and Stefania Ruggeri (Agricultural Research Council, Italy)
Patient and family perspectives. Lieven Bauwens (International Federation for Spina Bifida and Hydrocephalus, Belgium)
Public health decision making frameworks. John Forman (New Zealand Organisation for Rare Disorders, New Zeeland)
Session VII: Orphan drugs and personalized medicine (part II). Joint session with Dutch Clinical Trial Foundation (DCTF)
Marjon Pasmooij (Medicines Evaluation Board, The Netherlands): Regulatory Perspective
Angela Brand (Maastricht University, The Netherlands): Academic Perspective
Barbara Wuebbels (Audentes Therapeutics, USA): Patient Perspective
Session VIII: ICORD Working Groups
An integrative model of rare diseases management in a tertiary hospital: from basic research to clinical practice and beyond. Eduardo Tizzano and Joan Comella (Vall d’Hebron University Hospital, Spain)
Beyond orphan drugs: cooperation of a biopharmaceutical company with other rare diseases’ stakeholders. Fernando Royo (Fundación Genzyme, Spain)
Session IX. Registries and Biobanks
National registries of rare diseases in Europe: an overview by the EPIRARE project. Domenica Taruscio (Istituto Superiore di Sanità, Italy)
Spanish rare diseases registries research network – SpainRDR. Manuel Posada (Institute of Rare Diseases Research, IIER, Spain)
Spanish national rare disease biobank. Veronica Alonso (Instituto de Salud Carlos III, Spain)
Session X: The International Rare Diseases Research Consortium (IRDiRC)
Introduction to IRDiRC. Ségolène Aymé (IRDiRC Scientific Secretariat, France)
Recommendations to speed up the R&D process in the field of orphan drugs. Gert-Jan van Ommen (Leiden University, The Netherlands)
Initiatives to speed up data mining in the field of rare diseases. Ségolène Aymé (Orphanet, INSERM, France)
Initiatives to speed up data sharing. Anthony J Brookes (University of Leicester, United Kingdom)
Session XI: Patient perspectives on the societal value of rare diseases and orphan drugs
Perspectives from the USA. Sharon Terry (Genetic Alliance, USA)
Perspectives from The Netherlands: International lessons from a Dutch reimbursement procedure on Pompe and Fabry disease. Cees Smit (Dutch Genetic Alliance, European Genetic Alliances Network, The Netherlands)
Perspectives from Mexico. Carlos David Peña (FEMEXER, México)
Perspectives from Japan: The activity of Werner syndrome patient/family group in Japan. Satoshi Tanaka, Werner syndrome patient/family group in Japan (Japan)