A Decade of Discoveries and Current Challenges in the World of Rare Diseases and Orphan Drugs
15-16 October 2015, Mexico City
On behalf of ICORD we would like to thank the local organisers (FEMEXER, GEISER and Proyecto Pide Un Deseo México), the excellent speakers and everyone who attended the 10th ICORD Annual Meeting in Mexico City, Mexico. About 200 delegates, from all continents and representing many different stakeholders such as patients, patient support groups, health care professionals, researchers, industry, politicians, regulator and health authorities, came to take part of an inspiring programme, to network and to share their experiences in the field of rare diseases and orphan drugs. The ICORD meeting was part of the “Global Rare Diseases Week, Mexico 2015″ and back to back with the 4th Latin American meeting of Rare Diseases on 12 October and the Discoveries and Innovations in Orphan Drugs Congress, 13-14 October.
We would like to extend special thanks to the local organisers, David and Celita Peña, for all of the hard work, dedication and for creating such a warm and friendly atmosphere.
The ICORD Secretariat
Read more about the conference in a publication in Rare Diseases and Orphan Drugs.
Session I: Global Strategies for Rare Diseases
– John Forman (ICORD President, New Zealand) – ICORD’s vision and the Yukiwariso Declaration
– Ségolène Aymé (IRDiRC Scientific Secretariat, France) – IRDiRC update
– Paloma Tejada (EURORDIS, Switzerland) – Rare Diseases International
Session II: Update on The Latin American Rare Diseases Status
– Mikel Arriola (Federal Commission for Protection against Health Risks, COFEPRIS, Mexico), Regulatory facts and needs
– Ignacio Burgos (Ex-senator and Adviser of the health minister, Spain), Progress on regional laws
– Alison Joslyn (Shire, Brazil), Challenges and opportunities for rare diseases and orphan drugs in Latin America – an industry perspective
– Virginia Llera (GEISER, Argentina), About the rare diseases Latin American day, building a regional identity
Session III: The ethics of resource allocation for Rare Diseases
Mariana Kruger (Stellenbosch University, South Africa) – Keynote lecture
Session IV: Inborn errors of metabolism as an example of RD
– Stephanie Sacharow (Harvard University, USA), Medical perspective. A short introduction to metabolic disorders
– Kelly du Plessis (Rare Disease Society of South Africa, South Africa), Patient perspective
– Luis Barrera (Javeriana University, Colombia), Research perspective
– Fernando Royo (Fundación Genzyme, Spain), Industry perspective
– Ezperanza Quiroz (Seguro Popular, Mexico), Authority perspective
– Panel discussion with all presenters. Current challenges, differences around the globe.
Session V: Prevention
– José F Cordero (University of Georgia, USA) – Prevention and birth defects
– Domenica Taruscio (Istituto Superiore di Sanità, Italy) – European Recommendations on policies for the primary prevention of congenital anomalies
Session VII: Patients’ voices: International cooperation within patient groups. Why, Who and How?
– Gema Chicano (EURORDIS, Spain), Perspective of EURORDIS
– Natasha Bonhomme (Genetic Alliance, USA), Perspective of Genetic Alliance
– David Peña (FEMEXER, Mexico), Perspective of FEMEXER
Session VIII: Building the Undiagnosed Diseases Program to a global network
Cynthia Tifft (Undiagnosed Diseases Program, NIH, USA), Keynote lecture
Session X: Spreading the word – Incentives for OD development are needed around the world
– Catarina Edfjäll (CSL Behring, Switzerland), A short update of existing OD incentives in different countries and regions
– James Reese (Office of Orphan Products Development, FDA, USA), Updates on recent changes in incentives for rare disease drug development in the United States
– Fernando Fon Méndez (Mexican Association of Pharma Investigation Industries, AMIIF), A Mexican overview
Session XI: Development and accessibility to international registries and biobanks
– Yaffa Rubinstein (NIH, USA), The NIH/NCATS GRDR® Program-Global Rare Diseases Patient Registry Data Repository: Linking patient registries data to bio-specimens data
– Manuel Posada (Instituto de Salud Carlos III, Spain), Medical ontologies and registering activities. What are their added values?
– Michelle Durborow (The Life Raft Group, USA), Poster presentation: The Global Gist Patient Registry and Tissue Bank
Session XII: Examples of successful rare disease collaborations. Progresses in the RD field the last 10 years and futuristic outlooks.
– Rashmi Gopal-Srivastava (ORDR, NIH, USA) – The Rare Diseases Clinical Research Network, RDCRN
– Antoni Matilla Dueñas (Health Sciences Institute Germans Trias i Pujol, Spain) – The RIBERMOV Latin-American collaboration
– Janine Lewis (GARD, US), The Genetic and Rare Diseases Information Center (GARD): Twelve years of improving access to hard-to-find genetic and/or rare diseases information and resources
– Daria Julkowska (French National Research Agency, France), Poster presentation: E-RARE efforts towards patients’ involvement in rare diseases research and funding activities