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Literature DB >> 20624867

Policy alternatives for treatments for rare diseases.

Abbas H Panju¹, Chaim M Bell.

Abstract

Mesh：

Year: 2010 PMID： 20624867 PMCID： PMC2988562 DOI： 10.1503/cmaj.081429

Source DB: PubMed Journal: CMAJ ISSN： 0820-3946 Impact factor: 8.262

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25 in total

1. Managing public payment for high-cost, high-benefit treatment: enzyme replacement therapy for Gaucher's disease in Ontario.

Authors: J T Clarke; D Amato; R B Deber
Journal: CMAJ Date: 2001-09-04 Impact factor: 8.262

2. The price of innovation: new estimates of drug development costs.

Authors: Joseph A DiMasi; Ronald W Hansen; Henry G Grabowski
Journal: J Health Econ Date: 2003-03 Impact factor: 3.883

3. Clinical trials and rare diseases.

Authors: Stephen W Lagakos
Journal: N Engl J Med Date: 2003-06-12 Impact factor: 91.245

4. Rare disease research gets boost.

Authors: Tracy Hampton
Journal: JAMA Date: 2006-06-28 Impact factor: 56.272

5. Accountability for reasonableness: opening the black box of process.

Authors: Andreas Hasman; Søren Holm
Journal: Health Care Anal Date: 2005-12

6. Orphan drug development is progressing too slowly.

Authors: Roberta Joppi; Vittorio Bertele; Silvio Garattini
Journal: Br J Clin Pharmacol Date: 2006-03 Impact factor: 4.335

7. Cardiovascular events associated with rofecoxib in a colorectal adenoma chemoprevention trial.

Authors: Robert S Bresalier; Robert S Sandler; Hui Quan; James A Bolognese; Bettina Oxenius; Kevin Horgan; Christopher Lines; Robert Riddell; Dion Morton; Angel Lanas; Marvin A Konstam; John A Baron
Journal: N Engl J Med Date: 2005-02-15 Impact factor: 91.245

8. Orphan drug legislation: lessons for neglected tropical diseases.

Authors: Stefano Villa; Amelia Compagni; Michael R Reich
Journal: Int J Health Plann Manage Date: 2009 Jan-Mar

9. The safety and efficacy of oral methylnaltrexone in preventing morphine-induced delay in oral-cecal transit time.

Authors: C S Yuan; J F Foss; J Osinski; A Toledano; M F Roizen; J Moss
Journal: Clin Pharmacol Ther Date: 1997-04 Impact factor: 6.875

10. Outpatient gatifloxacin therapy and dysglycemia in older adults.

Authors: Laura Y Park-Wyllie; David N Juurlink; Alexander Kopp; Baiju R Shah; Therese A Stukel; Carmine Stumpo; Linda Dresser; Donald E Low; Muhammad M Mamdani
Journal: N Engl J Med Date: 2006-03-01 Impact factor: 91.245

11 in total

Review 1. Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now?

Authors: Devidas Menon; Derek Clark; Tania Stafinski
Journal: Healthc Policy Date: 2015-08

2. Decision Models for Assessing the Cost Effectiveness of Treatments for Pediatric Drug-Resistant Epilepsy: A Systematic Review of Economic Evaluations.

Authors: Jesse Elliott; Sasha van Katwyk; Bláthnaid McCoy; Tammy Clifford; Beth K Potter; Becky Skidmore; George A Wells; Doug Coyle
Journal: Pharmacoeconomics Date: 2019-10 Impact factor: 4.981

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

Policy alternatives for treatments for rare diseases.

1. Managing public payment for high-cost, high-benefit treatment: enzyme replacement therapy for Gaucher's disease in Ontario.

2. The price of innovation: new estimates of drug development costs.

3. Clinical trials and rare diseases.

4. Rare disease research gets boost.

5. Accountability for reasonableness: opening the black box of process.

6. Orphan drug development is progressing too slowly.

7. Cardiovascular events associated with rofecoxib in a colorectal adenoma chemoprevention trial.

8. Orphan drug legislation: lessons for neglected tropical diseases.

9. The safety and efficacy of oral methylnaltrexone in preventing morphine-induced delay in oral-cecal transit time.

10. Outpatient gatifloxacin therapy and dysglycemia in older adults.

Review 1. Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now?

2. Decision Models for Assessing the Cost Effectiveness of Treatments for Pediatric Drug-Resistant Epilepsy: A Systematic Review of Economic Evaluations.

3. Economic Evaluation of Stiripentol for Dravet Syndrome: A Cost-Utility Analysis.

Review 4. A systematic review of moral reasons on orphan drug reimbursement.

5. Some Numbers behind Canada's Decision to Adopt an Orphan Drug Policy: US Orphan Drug Approvals in Canada, 1997-2012.

6. Determinants of orphan drugs prices in France: a regression analysis.

7. Rare diseases: Canada's "research orphans".

8. Funding innovation for treatment for rare diseases: adopting a cost-based yardstick approach.

Review 9. Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries.

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.