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Literature DB >> 22037026

Clinical pharmacology as a cornerstone of orphan drug development.

Edward D Bashaw¹, Shiew-Mei Huang, Timothy R Coté, Anne R Pariser, Christine E Garnett, Gilbert Burckart, Lei Zhang, Angela Y Men, Christine D Le, Rosane Charlab, Jogaro V Gobburu, Lawrence J Lesko.

Abstract

A recent US Food and Drug Administration (FDA) advisory committee meeting highlighted the potential of clinical pharmacology to overcome challenges in orphan drug development.

Mesh：

Year: 2011 PMID： 22037026 DOI： 10.1038/nrd3595

Source DB: PubMed Journal: Nat Rev Drug Discov ISSN： 1474-1776 Impact factor: 84.694

5 in total

Review 1. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years.

Authors: M Miles Braun; Sheiren Farag-El-Massah; Kui Xu; Timothy R Coté
Journal: Nat Rev Drug Discov Date: 2010-06-07 Impact factor: 84.694

Review 2. Rare diseases, orphan drugs and their regulation: questions and misconceptions.

Authors: Erik Tambuyzer
Journal: Nat Rev Drug Discov Date: 2010-11-09 Impact factor: 84.694

3. Applications of physiologically based pharmacokinetic (PBPK) modeling and simulation during regulatory review.

Authors: P Zhao; L Zhang; J A Grillo; Q Liu; J M Bullock; Y J Moon; P Song; S S Brar; R Madabushi; T C Wu; B P Booth; N A Rahman; K S Reynolds; E Gil Berglund; L J Lesko; S-M Huang
Journal: Clin Pharmacol Ther Date: 2010-12-29 Impact factor: 6.875

4. Impact of pharmacometric reviews on new drug approval and labeling decisions--a survey of 31 new drug applications submitted between 2005 and 2006.

Authors: V A Bhattaram; C Bonapace; D M Chilukuri; J Z Duan; C Garnett; J V S Gobburu; S H Jang; L Kenna; L J Lesko; R Madabushi; Y Men; J R Powell; W Qiu; R P Ramchandani; C W Tornoe; Y Wang; J J Zheng
Journal: Clin Pharmacol Ther Date: 2007-02 Impact factor: 6.875

5. A Pharmacometric Approach to Substitute for a Conventional Dose-Finding Study in Rare Diseases: Example of Phase III Dose Selection for Emicizumab in Hemophilia A.

Authors: Koichiro Yoneyama; Christophe Schmitt; Naoki Kotani; Gallia G Levy; Ryu Kasai; Satofumi Iida; Midori Shima; Takehiko Kawanishi
Journal: Clin Pharmacokinet Date: 2018-09 Impact factor: 6.447

5 in total

Clinical pharmacology as a cornerstone of orphan drug development.

Review 1. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years.

Review 2. Rare diseases, orphan drugs and their regulation: questions and misconceptions.

3. Applications of physiologically based pharmacokinetic (PBPK) modeling and simulation during regulatory review.

4. Impact of pharmacometric reviews on new drug approval and labeling decisions--a survey of 31 new drug applications submitted between 2005 and 2006.

5. Leveraging prior quantitative knowledge in guiding pediatric drug development: a case study.

1. The discovery of medicines for rare diseases.

Review 2. Molecular pharming's foot in the FDA's door: Protalix's trailblazing story.

Review 3. Hypoxia-induced alternative splicing in human diseases: the pledge, the turn, and the prestige.

4. Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease.

5. A Pharmacometric Approach to Substitute for a Conventional Dose-Finding Study in Rare Diseases: Example of Phase III Dose Selection for Emicizumab in Hemophilia A.