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Articles (55)

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1-10 of 55 results for Orphan drug designation

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Select article: Orphan Drug Development — International Program and Study Design Issues

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First published 1998-1

Orphan Drug Development — International Program and Study Design Issues

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Select article: Development of Orphan Drugs in Japan: Characteristics of Orphan Drugs Developed in Japan *

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First published 2000-7

Development of Orphan Drugs in Japan: Characteristics of Orphan Drugs Developed in Japan *

Makoto Shiragami, Kiyohito Nakai

Therapeutic Innovation & Regulatory Science

Select article: Designing Clinical Trials to Study Rare Disease Treatment

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First published 1998-1

Designing Clinical Trials to Study Rare Disease Treatment

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Select article: Efforts to develop orphan drugs: The FDA experience

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Research article

First published 2010-2

Efforts to develop orphan drugs: The FDA experience

Linda C. Ulrich

Pharmaceuticals Policy and Law

Select article: Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Open access

Research article

First published 2023-03

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Richa Madan Lomash, Oleg Shchelochkov, Randy J. Chandler [...]

Human Gene Therapy

Select article: Orphan Drug Designation and Exclusivity for “Same Drugs”

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Research article

First published 2019-6

Orphan Drug Designation and Exclusivity for “Same Drugs”

Phebe Hong, Ameet Sarpatwari, Aaron S. Kesselheim

The Journal of Law,Medicine & Ethics

Select article: Assessing the Performance of the EMEA's Centralized Procedure: A Comparative Analysis with the US FDA

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First published 2008-1

Assessing the Performance of the EMEA's Centralized Procedure: A Comparative Analysis with the US FDA

Laura B. Faden, Kenneth I. Kaitin

Therapeutic Innovation & Regulatory Science

Select article: Orphan Drug Development: Opportunities and Challenges from a Midsized to Large Company Perspective

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First published 2010-3

Orphan Drug Development: Opportunities and Challenges from a Midsized to Large Company Perspective

Rosann Reinhart, Krysten Madrzjewski

Therapeutic Innovation & Regulatory Science

Select article: The Current Environment in Orphan Drug Development

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First published 2003-10

The Current Environment in Orphan Drug Development

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Select article: Orphan Drugs: The United States Experience

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First published 1999-4

Orphan Drugs: The United States Experience

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

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