Speakers’ Presentations
Presentations at the 5th ICORD meeting, Rome, Italy, 23-25 Feb
Monday, February 23, 2009
Spreading the Word of Rare Diseases Internationally – Rare Disease Day 2008 & 2009: Experiences and Plans
Facilitating Cooperative Efforts of the Regulatory Processes:
Progress on Collaborative Regulatory Activities OOPD/FDA, USA and COMP/EMEA, Europe
- European Union – Kerstin Westermark
- United States – Timothy Coté
- Japan – Yukiko Nishimura
- Canada – Maurica Maher
WHO International Classification of Diseases and Rare Diseases Emphasis
- Orphanet Classification of Rare Diseases – Ana Rath
- ICD XI Revision Process and Rare Diseases Topic Advisory Group and WHO ICD-X and ICD X-CM Update and Revision Process – Segolene Ayme
- Office of Rare Diseases ResearchTerms in the MeSH System of the National Library of Medicine USA – Stephen Groft
Global Look at Policy Initiatives for Rare Diseases Research and Orphan Products – Current Activities and Future Needs
- The National Program on Rare and Intractable Diseases – Yukiko Nishimura
- Current Activities in South Korea – Soo Kyung Koo
- Review of Rare Diseases Research and Orphan Products Development Activities by the USA National Academy of Sciences and Institute of Medicine
- Review of Rare Diseases Research and Orphan Products Development Activities by the European Commission – Kerstin Westermark, Josep Torrent, Antoni Montserrat Presentations 2009Westermark.ppt
Europlan and National Plans for Rare Diseases Research and Orphan Products Development
- Italy – Domenica Taruscio
- Portugal – Jose Robalo
- Bulgaria – Rumen Stefanov
- Germany – Mirjam Mann
Tuesday, February 24, 2009
Linking Academic Discoveries and Industry Product Development Strategies
- E-Rare Project – Sophie Koutouzov
- TEDDY –Task Force in Europe for Drug Development in the Young – Adriana Ceci
Activities at the Academic Research Centers: Identifying Present Activities and Future Opportunities
Linking Patients to Research Programs and Treatment Centers – The Value of Patient Registries and Experiences in Recruiting Patients for Clinical Trials
- Overview: Ronald A. Christensen
- Utilization and Expansion of a Patient Contact Registry to Recruit Patients to the NIH Rare Diseases Clinical Research Network – Rachel Richesson
- ECRIN – Arrigo Schieppati
- EUROCAT – Epidemiological Studies – Fabrizio Bianchi
- Piedmont and Valle d’Aosta Registry of rare diseases – Dario Roccatello
The Value and Need for International Collaboration
Wednesday, February 25, 2009
Research Methodology and Statistical Analyses for Trials of Rare Diseases and Orphan Products
- Bayesian Methods to ‘Strengthen’ Limited Trial or Study Data – Simon Day
- Methodology Issues for Trials in Rare Diseases – Paolo Bruzzi
Future Emphasis