Literature DB >> 24671833

An Orphan Drug Framework (ODF) for Canada.

David K Lee, Barbara Wong.   

Abstract

An orphan drug is a pharmaceutical or biological drug that is used to treat a rare disease. The development of a Canadian orphan drug framework is intended to help translate scientific discoveries into meaningful results for Canadian rare disease patients.

Entities:  

Mesh：

Year:  2014        PMID： 24671833

Source DB:  PubMed          Journal:  J Popul Ther Clin Pharmacol        ISSN： 2561-8741


  12 in total

1.  We need a "made in Canada" orphan drug framework.

Authors:  Hugh J McMillan; Craig Campbell
Journal:  CMAJ       Date:  2017-10-16       Impact factor: 8.262

2.  Engagement of Canadian Patients with Rare Diseases and Their Families in the Lifecycle of Therapy: A Qualitative Study.

Authors:  Andrea Young; Devidas Menon; Jackie Street; Walla Al-Hertani; Tania Stafinski
Journal:  Patient       Date:  2018-06       Impact factor: 3.883

3.  Metabolic Clinic Atlas: Organization of Care for Children with Inherited Metabolic Disease in Canada.

Authors:  Monica F Lamoureux; Kylie Tingley; Jonathan B Kronick; Beth K Potter; Alicia K J Chan; Doug Coyle; Linda Dodds; Sarah Dyack; Annette Feigenbaum; Michael Geraghty; Jane Gillis; Cheryl Rockman-Greenberg; Aneal Khan; Julian Little; Jennifer MacKenzie; Bruno Maranda; Aizeddin Mhanni; John J Mitchell; Grant Mitchell; Anne-Marie Laberge; Murray Potter; Chitra Prasad; Komudi Siriwardena; Kathy N Speechley; Sylvia Stockler; Yannis Trakadis; Lesley Turner; Clara Van Karnebeek; Kumanan Wilson; Pranesh Chakraborty
Journal:  JIMD Rep       Date:  2015-02-26

4.  Pharmaceutical expenditure on drugs for rare diseases in Canada: a historical (2007-13) and prospective (2014-18) MIDAS sales data analysis.

Authors:  Victoria Divino; Mitch DeKoven; Michael Kleinrock; Rolin L Wade; Tony Kim; Satyin Kaura
Journal:  Orphanet J Rare Dis       Date:  2016-05-21       Impact factor: 4.123

Review 5.  Exploring patient and family involvement in the lifecycle of an orphan drug: a scoping review.

Authors:  Andrea Young; Devidas Menon; Jackie Street; Walla Al-Hertani; Tania Stafinski
Journal:  Orphanet J Rare Dis       Date:  2017-12-22       Impact factor: 4.123

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

Authors:  Nigel S B Rawson
Journal:  Orphanet J Rare Dis       Date:  2017-03-23       Impact factor: 4.123

7.  EUPATI and Patients in Medicines Research and Development: Guidance for Patient Involvement in Regulatory Processes.

Authors:  David Haerry; Cordula Landgraf; Kay Warner; Amy Hunter; Ingrid Klingmann; Matthew May; Wolf See
Journal:  Front Med (Lausanne)       Date:  2018-08-17

8.  Factors associated with positive and negative recommendations for cancer and non-cancer drugs for rare diseases in Canada.

Authors:  Fernanda Naomi Inagaki Nagase; Tania Stafinski; Jian Sun; Gian Jhangri; Devidas Menon
Journal:  Orphanet J Rare Dis       Date:  2019-06-07       Impact factor: 4.123

9.  Biologic medicine inclusion in 138 national essential medicines lists.

Authors:  Raphaël Kraus; Rae S M Yeung; Nav Persaud
Journal:  Pediatr Rheumatol Online J       Date:  2021-09-06       Impact factor: 3.054

10.  Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand.

Authors:  John I McCormick; L Diana Berescu; Nabil Tadros
Journal:  Orphanet J Rare Dis       Date:  2018-01-30       Impact factor: 4.123
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