MEETING ABSTRACT Open Access State of the art of rare disease activities around the world: overview of the non-European landscape Durhane Wong-Rieger From 6th European Conference on Rare Diseases and Orphan Products Brussels, Belgium. 23-25 May 2012 With support from patient associations, political frame- works for rare diseases have been established through- out the world albeit with varying definitions for rare diseases. In the USA, the National Organization for Rare Disor- ders was instrumental in passing the 1983 Orphan Drug Act and the 2002 Rare Disease Act, which includes medical devices and dietary products as orphan products. In 2011, the House passed bills supporting research for undiagnosed diseases and preserving regulatory fee excep- tions for orphan drugs. In 2012, the Canadian government awarded five-year rare disease research grants. With advocacy from the Canadian Organization for Rare Disorders, Health Canada concluded consultations on an orphan drug regulatory fra- mework. Several provinces have implemented orphan drug access programs and expanded newborn screening. In Argentina, the Geiser Foundation led advocacy result- ing in the 2011 Rare Disease Law, obliging health and social systems to provide assistance. A central committee, including patients, will coordinate activities like neonatal screening and patient registries. In 2010, Colombia passed the Orphan Disease Law and hosted the 2 nd National Forum of Orphan Diseases. In 2011, Peru passed legislation promoting treatment and a national strategy including diagnosis, surveillance, prevention, care, and rehabilitation. Through the 1972 Medical Care Program for Specific Diseases, Japan provides medical cost subsidy to patients affected by “56 rare and intractable diseases.” The 1993 Orphan Drug Law supports research and development. In 2008, Supporting Organizations for Patients with Rare Diseases was formed. Since 1991, Singapore’ s Orphan Drugs Policy allows patients with life-threatening and severely debilitating diseases with no other treatment options to access approved drugs prescribed by their practitioner. The Taiwan Foundation for Rare Disorders helped secure the Rare Disease and Orphan Drugs Act in 2000. Diseases affecting fewer than 1 in 10,000 that are offi- cially recognized are eligible for medical coverage. In Korea, the Orphan Drug Centre supplies medicines for diseases affecting fewer than 1 in 20,000. The Genetics and Rare Disease Centre supports national reference centres and research. In China, in 2011, medical professionals called for legislation to support healthcare, research, orphan drug development, and epidemiological studies for diseases affecting fewer than 1 in 10,000. Australia’s 1987 Orphan Drugs Policy makes available rare disease drugs, based on US regulatory information. In 2010, consultation for a national strategy was posted online. In 2012, Rare Voices Australia was formed. Acknowledgment EUCERD 2012 Report on State of the Art of Rare Diseases Activities in Europe Published: 22 November 2012 doi:10.1186/1750-1172-7-S2-A2 Cite this article as: Wong-Rieger: State of the art of rare disease activities around the world: overview of the non-European landscape. Orphanet Journal of Rare Diseases 2012 7(Suppl 2):A2. Correspondence: [email protected] Canadian Organization for Rare Disorders, Toronto, Ontario, Canada M5S 1S4 Wong-Rieger Orphanet Journal of Rare Diseases 2012, 7(Suppl 2):A2 http://www.ojrd.com/content/7/S2/A2 © 2012 Wong-Rieger; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Page 1: State of the art of rare disease activities around the world: overview of the non-European landscape

MEETING ABSTRACT Open Access

State of the art of rare disease activities aroundthe world: overview of the non-EuropeanlandscapeDurhane Wong-Rieger

From 6th European Conference on Rare Diseases and Orphan ProductsBrussels, Belgium. 23-25 May 2012

With support from patient associations, political frame-works for rare diseases have been established through-out the world albeit with varying definitions for rarediseases.In the USA, the National Organization for Rare Disor-

ders was instrumental in passing the 1983 Orphan DrugAct and the 2002 Rare Disease Act, which includes medi-cal devices and dietary products as orphan products. In2011, the House passed bills supporting research forundiagnosed diseases and preserving regulatory fee excep-tions for orphan drugs.In 2012, the Canadian government awarded five-year

rare disease research grants. With advocacy from theCanadian Organization for Rare Disorders, Health Canadaconcluded consultations on an orphan drug regulatory fra-mework. Several provinces have implemented orphan drugaccess programs and expanded newborn screening.In Argentina, the Geiser Foundation led advocacy result-

ing in the 2011 Rare Disease Law, obliging health andsocial systems to provide assistance. A central committee,including patients, will coordinate activities like neonatalscreening and patient registries. In 2010, Colombia passedthe Orphan Disease Law and hosted the 2nd NationalForum of Orphan Diseases. In 2011, Peru passed legislationpromoting treatment and a national strategy includingdiagnosis, surveillance, prevention, care, and rehabilitation.Through the 1972 Medical Care Program for Specific

Diseases, Japan provides medical cost subsidy to patientsaffected by “56 rare and intractable diseases.” The 1993Orphan Drug Law supports research and development.In 2008, Supporting Organizations for Patients with RareDiseases was formed.

Since 1991, Singapore’s Orphan Drugs Policy allowspatients with life-threatening and severely debilitatingdiseases with no other treatment options to accessapproved drugs prescribed by their practitioner.The Taiwan Foundation for Rare Disorders helped

secure the Rare Disease and Orphan Drugs Act in 2000.Diseases affecting fewer than 1 in 10,000 that are offi-cially recognized are eligible for medical coverage. InKorea, the Orphan Drug Centre supplies medicines fordiseases affecting fewer than 1 in 20,000. The Geneticsand Rare Disease Centre supports national referencecentres and research.In China, in 2011, medical professionals called for leg-

islation to support healthcare, research, orphan drugdevelopment, and epidemiological studies for diseasesaffecting fewer than 1 in 10,000.Australia’s 1987 Orphan Drugs Policy makes available

rare disease drugs, based on US regulatory information.In 2010, consultation for a national strategy was postedonline. In 2012, Rare Voices Australia was formed.

AcknowledgmentEUCERD 2012 Report on State of the Art of Rare Diseases Activities inEurope

Published: 22 November 2012

doi:10.1186/1750-1172-7-S2-A2Cite this article as: Wong-Rieger: State of the art of rare diseaseactivities around the world: overview of the non-European landscape.Orphanet Journal of Rare Diseases 2012 7(Suppl 2):A2.

Correspondence: [email protected] Organization for Rare Disorders, Toronto, Ontario, Canada M5S 1S4

Wong-Rieger Orphanet Journal of Rare Diseases 2012, 7(Suppl 2):A2http://www.ojrd.com/content/7/S2/A2

© 2012 Wong-Rieger; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative CommonsAttribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction inany medium, provided the original work is properly cited.

mailto:[email protected]

http://creativecommons.org/licenses/by/2.0