Opening of Conference
Ichiro Kanazawa
(Professor, Dean, International University of Health and Welfare, former President of the Science Council of Japan, Local Chair of ICORD 2012, Japan)
Welcome Ichiro Kanazawa
Successful examples of clinical research in Rare Diseases
Jan-Inge Henter (ICORD Board member, Karolinska Institute, Karolinska University Hospital, Stockholm, Sweden)
Successful Academic Clinical Trials in Rare Diseases can be Valuable also for Common Diseases: Examples and Implementation
Access to and Reimbursement for Diagnosis and Therapy
Stefaan van Gool (University Hospital Gasthuisberg, Belgium)
Improved survival in the brain tumor malignant gliomas by successful academic clinical trials using tumor vaccination
Regulatory Aspects of Orphan Drugs
Agnes Saint-Raymond (EMA, UK)
Regulatory Aspects
Debra Lewis (FDA, USA)
Regulatory Aspects of Orphan Drugs – FDA Perspective
Marc Dunoyer (GSK Rare Disease, UK)
Vision for Creating New Therapies for Patients with Rare Diseases: View from Industry
Regional Pan-Pacific session – providing access to knowledge and collaboration
Naoko Yamamoto (Disease Control Division, Ministry of Health, Labour and Welfare, Japan)
Rare Disease Policies in Japan
Patient groups – Their connection and needs
John Forman (ICORD President-Elect and President NZORD, New Zealand Organization for Rare Disorders)
Presentation slides by John Forman
Tateo Itoh (Japan Patients Association, Japan)
Activities of patient groups in Japan
Supporting Product Development and Venture Capital
Ségolène Aymé (Orphanet, France)
Pre-competitive tools to speed up R&D for rare diseases: the Orphanet perspective
Jin Shiomura (Nobelpharma Company Ltd., Japan)
Development of rare disease treatment drugs is a collaborative project among patients, industry, academia and government
Ron Bartek, President (Friedreich’s Ataxia Research Foundation, USA)
Joint-Venture Philanthropy and Public-Private Partnership
International health policies for rare diseases and orphan drugs
Virginia Llera (ICORD President, elected 2012, and President GEISER Foundation, Argentina)
Reflections about rare diseases organizations and their impact on the policies. The ICORD experience in LA&C
Steve Groft (ICORD Board member, Director, Office of Rare Diseases, NIH, USA)
Presentation slides by Stephen Groft
John Forman (ICORD President-Elect and President NZORD, New Zealand Organization for Rare Disorders)
ICORD – Yukiwariso Declaration
The value of and promotion of basic research in RD – The future for international research collaboration
Kenji Hayashi (President, National Institute of Public Health, Japan)
Characteristics of a medical care program for specific diseases in Japan
Steve Groft (ICORD Board member, Director, Office of Rare Diseases, NIH, USA)
Translational Research Initiatives at the NIH, USA
It’s All about the Patients
Mark Krueger (Mark Krueger and Associates, USA)
Developing Advocacy
Virginia Beakes-Read (Global Regulatory Policy and Intelligence, Eisai, Inc, USA/Japan)
Global Environment for Development of Drugs for Rare Diseases
Selected Posters (Abstracts)
S Kobayashi et al (Ehime University, Japan)
Clinical Data Modeling for National Surveillance of Rare Diseases in Japan
K Ikeda et al (PRIP Tokyo, Japan)
Data Integration on Rare Disease and Orphan Drug with Open Source Drug Discovery Database
H Guillette et al (University of South Florida College of Medicine, USA)
PRISM: A Shared Resource for Global Data Standards in Patient Registries
Connolly et al (Children’s Hospital Boston, USA)
The Manton Center for Orphan Disease Research: Gene Discovery Core
D Gavhed et al (ICORD Secretariat, Karolinska Institute, Sweden)
A survey about rare diseases among Swedish General Practictioners (Abstract)
Closing Session
Yukiko Nishimura (ICORD Board member, Chief secretariat of ICORD2012, Tokyo University, Japan)
Why we wanted to host ICORD 2011/2012