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Rare diseases: Canada's "research orphans".

作者信息

Gupta Samir

机构信息

Department of Medicine, Division of Respirology, University of Toronto, Toronto, Ontario.

出版信息

Open Med. 2012;6(1):e23-7. Epub 2012 Feb 28.

PMID:22567079

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3329116/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b2cf/3329116/560f5902095a/OpenMed-06-e24-g001.jpg

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本文引用的文献

Abstracts of the 5th European Conference on Rare Diseases (ECRD 2010). Krakow, Poland. May 13-15, 2010.

Orphanet J Rare Dis. 2010;5 Suppl 1(Suppl 1):O1-32, P1-28. doi: 10.1186/1750-1172-5-s1-o1.

National plans and strategies on rare diseases in Europe.

Adv Exp Med Biol. 2010;686:475-91. doi: 10.1007/978-90-481-9485-8_26.

Policy alternatives for treatments for rare diseases.

CMAJ. 2010 Nov 23;182(17):E787-92. doi: 10.1503/cmaj.081429. Epub 2010 Jul 12.

Rare disease research gets boost.

JAMA. 2006 Jun 28;295(24):2836-8. doi: 10.1001/jama.295.24.2836.

Adopting orphan drugs--two dozen years of treating rare diseases.

N Engl J Med. 2006 Feb 2;354(5):445-7. doi: 10.1056/NEJMp058317.

Ethical issues in funding orphan drug research and development.

J Med Ethics. 2005 Mar;31(3):164-8. doi: 10.1136/jme.2003.007138.

Orphan products: hope for people with rare diseases.

FDA Consum. 2003 Nov-Dec;37(6):10-5.

Orphan drugs: legal aspects, current situation.

Haemophilia. 2002 May;8(3):194-8. doi: 10.1046/j.1365-2516.2002.00643.x.

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相似文献

Rare diseases: Canada's "research orphans".

Open Med. 2012;6(1):e23-7. Epub 2012 Feb 28.

The study of drugs for rare disorders: harnessing research contributions by Canadian academic institutions.

J Popul Ther Clin Pharmacol. 2014;21(1):e47-55. Epub 2014 Feb 23.

Canada's progress toward an orphan drug framework: 2014.

J Popul Ther Clin Pharmacol. 2014;21(1):e41. Epub 2014 Feb 23.

Fair pricing of "old" orphan drugs: considerations for Canada's orphan drug policy.

CMAJ. 2015 Apr 7;187(6):422-425. doi: 10.1503/cmaj.140308. Epub 2015 Feb 23.

Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products.

Br J Clin Pharmacol. 2006 Sep;62(3):264-71. doi: 10.1111/j.1365-2125.2006.02654.x.

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J Popul Ther Clin Pharmacol. 2020 Jun 1;27(2):e58-e67. doi: 10.15586/jptcp.v27i2.673.

The Pediatric Research Equity Act Moves Into Adolescence.

JAMA. 2017 Jan 17;317(3):259-260. doi: 10.1001/jama.2016.18131.

Rare diseases, orphan drugs, and orphaned patients.

Mol Interv. 2006 Aug;6(4):186-91. doi: 10.1124/mi.6.4.2.

An Orphan Drug Framework (ODF) for Canada.

J Popul Ther Clin Pharmacol. 2014;21(1):e42-6. Epub 2014 Feb 23.

Don't Forget the Orphans.

Eur J Health Law. 2015 Apr;22(2):107-11. doi: 10.1163/15718093-12341351.

引用本文的文献

Application of observational research methods to real-world studies for rare disease drugs: A scoping review protocol.

PLoS One. 2025 Mar 28;20(3):e0304540. doi: 10.1371/journal.pone.0304540. eCollection 2025.

Health technology assessment of new drugs for rare disorders in Canada: impact of disease prevalence and cost.

Orphanet J Rare Dis. 2017 Mar 23;12(1):59. doi: 10.1186/s13023-017-0611-7.

Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework.

Pharmacoeconomics. 2015 Mar;33(3):255-69. doi: 10.1007/s40273-014-0235-x.

本文引用的文献

Abstracts of the 5th European Conference on Rare Diseases (ECRD 2010). Krakow, Poland. May 13-15, 2010.

Orphanet J Rare Dis. 2010;5 Suppl 1(Suppl 1):O1-32, P1-28. doi: 10.1186/1750-1172-5-s1-o1.

National plans and strategies on rare diseases in Europe.

Adv Exp Med Biol. 2010;686:475-91. doi: 10.1007/978-90-481-9485-8_26.

Policy alternatives for treatments for rare diseases.

CMAJ. 2010 Nov 23;182(17):E787-92. doi: 10.1503/cmaj.081429. Epub 2010 Jul 12.

Rare disease research gets boost.

JAMA. 2006 Jun 28;295(24):2836-8. doi: 10.1001/jama.295.24.2836.

Adopting orphan drugs--two dozen years of treating rare diseases.

N Engl J Med. 2006 Feb 2;354(5):445-7. doi: 10.1056/NEJMp058317.

Ethical issues in funding orphan drug research and development.

J Med Ethics. 2005 Mar;31(3):164-8. doi: 10.1136/jme.2003.007138.

Orphan products: hope for people with rare diseases.

FDA Consum. 2003 Nov-Dec;37(6):10-5.

Orphan drugs: legal aspects, current situation.

Haemophilia. 2002 May;8(3):194-8. doi: 10.1046/j.1365-2516.2002.00643.x.