SESSION II. Facilitating Cooperative Efforts of the Regulatory Processes: Progress on Collaborative Regulatory Requirements for the Orphan Product Designation Process between Office of Orphan Products Development (OOPD), Food and Drug Administration (FDA), USA and Committee for Orphan Medicinal Products (COMP)/European Medicines Agency (EMEA), Europe
- Trans-Atlantic Opportunities for Orphans Timothy R. Coté
- Common EMEA/FDA Application form for Orphan Medicinal Product Designation Kerstin M. Westermark
SESSION III. Linking Academic Discoveries and Industry Product Development Strategies
- Evaluation of Dietary Supplements for the Treatment of Inborn Errors of Metabolism (presentation) Paul M. Coates
- Rapid Access to Interventional Development (The RAID Pilot: A ROADMAP Program) at NIH David G. Badman (Download abstract)
- The Use of Antidotes in Hospitals and Communities, Supply Issues, and Emerging Research Needs Gregory M. Bogdan
- E-Rare Project: Networking research programmes on Rare Diseases in Europe Igor Beitia Ortiz de Zarate
- Rare and Neglected Diseases: Pieces of the Puzzle Bonny Harbinger
- Identifying Present Activities and Future Opportunities James C. Cloyd
- The Center for Rare Disease Therapies; Keck Graduate Institute Matt T. Reed
Session IV. Linking Patients to Research Programs and Treatment Centers
- Histiocytosis in Argentina since 1991 Jorge L. Braier
- Challenges in Developing Innovative Drugs Specifically for Rare Disorders (presentation) Stuart W. Peltz
- Linking Patients to Protocols: An Automated Registry Communication System Rachel L. Richesson
SESSION V. Research Methodology and Statistical Analyses for Trials of Rare Diseases and Orphan Products — Strength of Evidence: How Much Evidence is Necessary
- Getting Reliable Evidence Simon Day
- Research Methodology and Statistical Analyses for Trials of Rare Diseases and Orphan Products Annalisa Trama
SESSION VI. Stimulating Awareness and Research on Rare Diseases and Orphan Products through the Media
- Stimulating Awareness and Research on Rare Diseases and Orphan Products Through the Media Abbey S. Meyers
- Alliance for Chronic Rare Diseases, Germany (presentation) Arnd Brauer
- Rare Diseases and Media Bo Piela
- Stimulating Awareness and Research on Rare Diseases and Orphan Products Through the Media Virginia A. Llera
SESSION VII. Rare Diseases Research Activities at the NIH
- After the Human Genome Project: Applying Genomics to Health Alan E. Guttmacher
- The NIH Office of Rare Diseases: Current and Future Activities, States as Part of a Global Approach Stephen C. Groft
SESSION VIII. WHO International Classification of Diseases and Rare Diseases Emphasis
- WHO ICD-X and ICD X-CM Update and Revision Process David Berglund
- International Classification of Diseases in the field of rare diseases; ICD XI Revision Process Ségolène M. Aymé
SESSION IX.
- The Value and Need for International Collaboration Josep Torrent-Farnell
- Directions in Global Health Michael P. Johnson
- Report from Latin American Congress (ER2008LA) Emilio J. Roldán
- Neglected Diseases Luis Alejandro Barrera
- Orphan Drugs Policies: Dynamic Global Landscape Alice L. Pomponio
SESSION X. A Global Look at Policy Initiatives for Rare Diseases Research and Orphan Products – Current Activities and Future Needs
PANEL A. Global policy needs and what is being done?
- Policies on rare diseases research and orphan drugs in the Netherlands Sonja van Weely
- General Office of Drugs/ ANVISA Brazil Antonio Bezerra
PANEL B. Europlan and National Plans for Rare Diseases Research and Orphan Products Development
- Steps to a national policy on rare diseases: the case of Bulgaria Rumen Stefanov
- National Plans for Rare Diseases; The French plan 2005-2008 Ségolène M. Aymé (Download abstract)
- Recommendations for Rare Diseases national plans development Domenica Taruscio
- Draft of the Portuguese Programme on Rare Diseases José Marques Robalo
SESSION XI. Genetic Testing and Screening Approaches
- GeneTests: State of the Art and Current Projects Roberta A. Pagon
- EuroGenTest/Orphanet Database: New Services Ségolène M. Aymé (Download abstract)
- Genetic Reference Materials Lisa Kalman
- Patients’ Interest in Genetic Testing Sharon F. Terry
- Expanding the CETT Genetic Test Development Program Andy Faucett
SESSION XII. Meeting Patient and Family Needs Across the Lifespan — Access to Health Care, Psychological, and Social Support Programs
- Global Approaches for Rare Diseases and Orphan Products Anders Olauson
- Meeting Patient and Family Needs across the Lifespan: Psychological and Social Support Programs Vicky Whittemore
- The NZ Organisation for Rare Disorders John Forman
- Consulta Nazionale Malattie Rare Annalisa Trama
SESSION XIII. Gaining Access to Information on Rare Diseases and to Orphan Products: Policy Issues and Needs
- Pharmaceutical Industry Perspective Erik Tambuyzer
- The Role and Value of Help Lines Yann Le Cam
- Experiences of the Genetic Alliance Natasha Bonhomme
- Experiences at NORD Mary H. Dunkle
- Genetic and Rare Diseases Information Center (GARD) sponsored by ORD and NHGRI Janine Lewis
- Future Directions; Telefono Verde Malattie Rare – Toll Free Number for Rare Diseases Annalisa Trama
SESSION XV. Parallel Working Group Sessions, Workshops on Planning Future Activities and to Determine Future Needs, Goals, Venues and Implementation Mechanisms
- Small Clinical Trials for Orphan Drugs: How are these received at FDA? Timothy R. Coté
- Personalized Medicine: Viewing Product Approval through Mechanism of Action versus Disease State Marlene E. Haffner
- Gaining Access to Orphan Products: Sustainability Erik Tambuyzer
- Presentation of WG: Genetic Testing for Rare Diseases in International Settings — Genetic Reference Materials, Clinical Validity and Utility of Genetic Tests and Genetic Test Standards Joe Boone and Lisa Kalman