Search Results

There are 39836 results for: content related to: Unintended effects of orphan product designation for rare neurological diseases

  1. You have free access to this content
    An Empirical Review of Major Legislation Affecting Drug Development: Past Experiences, Effects, and Unintended Consequences

    The Milbank Quarterly

    Volume 89, Issue 3, September 2011, Pages: 450–502, AARON S. KESSELHEIM

    Version of Record online : 20 SEP 2011, DOI: 10.1111/j.1468-0009.2011.00636.x

  2. You have free access to this content
    The Evolving Drug Development Landscape: From Blockbusters to Niche Busters in the Orphan Drug Space

    Drug Development Research

    Volume 75, Issue 4, June 2014, Pages: 231–234, Ashish Kumar Kakkar and Neha Dahiya

    Version of Record online : 14 MAY 2014, DOI: 10.1002/ddr.21176

  3. Market uptake of orphan drugs – a European analysis

    Journal of Clinical Pharmacy and Therapeutics

    Volume 37, Issue 6, December 2012, Pages: 664–667, E. Picavet, L. Annemans, I. Cleemput, D. Cassiman and S. Simoens

    Version of Record online : 26 JUN 2012, DOI: 10.1111/j.1365-2710.2012.01364.x

  4. You have free access to this content
    Access to orphan drugs despite poor quality of clinical evidence

    British Journal of Clinical Pharmacology

    Volume 71, Issue 4, April 2011, Pages: 488–496, Alain G. Dupont and Philippe B. Van Wilder

    Version of Record online : 11 MAR 2011, DOI: 10.1111/j.1365-2125.2010.03877.x

  5. Orphan Drugs

    Principles and Practice of Pharmaceutical Medicine, Second Edition

    Lionel D. Edwards, Andrew J. Fletcher, Anthony W. Fox, Peter D. Stonier, Pages: 265–276, 2007

    Published Online : 25 JUN 2007, DOI: 10.1002/9780470093153.ch21

  6. Which Orphans Will Find a Home? The Rule of Rescue in Resource Allocation for Rare Diseases

    Hastings Center Report

    Volume 42, Issue 1, January-February 2012, Pages: 27–34, EMILY A. LARGENT and STEVEN D. PEARSON

    Version of Record online : 13 JAN 2012, DOI: 10.1002/hast.12

  7. The Role of Academic Institutions in the Development of Drugs for Rare and Neglected Diseases

    Clinical Pharmacology & Therapeutics

    Volume 92, Issue 2, August 2012, Pages: 193–202, L D Coles and J C Cloyd

    Version of Record online : 4 JUL 2012, DOI: 10.1038/clpt.2012.83

  8. Orphan Drugs for Rare Diseases: Grounds for Special Status

    Drug Development Research

    Volume 73, Issue 3, May 2012, Pages: 115–119, Eline Picavet, Marc Dooms, David Cassiman and Steven Simoens

    Version of Record online : 23 FEB 2012, DOI: 10.1002/ddr.21005

  9. You have free access to this content
    Orphan drug legislation: lessons for neglected tropical diseases

    The International Journal of Health Planning and Management

    Volume 24, Issue 1, January/March 2009, Pages: 27–42, Stefano Villa, Amelia Compagni and Michael R. Reich

    Version of Record online : 24 APR 2008, DOI: 10.1002/hpm.930

  10. You have free access to this content
    Adopting an orphan

    EMBO reports

    Volume 6, Issue 6, June 2005, Pages: 507–510, Andrea Rinaldi

    Version of Record online : 1 JUN 2005, DOI: 10.1038/sj.embor.7400450

  11. Federal Regulation of Biotechnology Products for Human Use, FDA, Orphan Drug Act

    Standard Article

    Encyclopedia of Ethical, Legal and Policy Issues in Biotechnology

    Robert A. Bohrer

    Published Online : 15 OCT 2002, DOI: 10.1002/0471250597.mur008

  12. Quantitative Analysis to Guide Orphan Drug Development

    Clinical Pharmacology & Therapeutics

    Volume 92, Issue 2, August 2012, Pages: 258–261, L J Lesko

    Version of Record online : 27 JUN 2012, DOI: 10.1038/clpt.2012.80

  13. What is known about the cost-effectiveness of orphan drugs? Evidence from cost-utility analyses

    Journal of Clinical Pharmacy and Therapeutics

    Volume 40, Issue 3, June 2015, Pages: 304–307, E. Picavet, D. Cassiman and S. Simoens

    Version of Record online : 20 APR 2015, DOI: 10.1111/jcpt.12271

  14. Orphan Drugs

    Principles and Practice of Pharmaceutical Medicine, Third Edition

    Lionel D. Edwards, Anthony W. Fox, Peter D. Stonier, Pages: 264–274, 2010

    Published Online : 9 NOV 2010, DOI: 10.1002/9781444325263.ch22

  15. You have free access to this content
    Delayed access to treatments for rare diseases: Who's to blame?

    Respirology

    Volume 20, Issue 3, April 2015, Pages: 361–369, Karen Feltmate, Peter M. Janiszewski, Sheena Gingerich and Michael Cloutier

    Version of Record online : 26 FEB 2015, DOI: 10.1111/resp.12498

  16. Ethical Considerations in Orphan Drug Approval and Use

    Clinical Pharmacology & Therapeutics

    Volume 92, Issue 2, August 2012, Pages: 153–155, A S Kesselheim

    Version of Record online : 27 JUN 2012, DOI: 10.1038/clpt.2012.92

  17. A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?

    The Journal of Clinical Pharmacology

    Volume 56, Issue 3, March 2016, Pages: 257–259, Charles Oo and Lorraine M. Rusch

    Version of Record online : 18 SEP 2015, DOI: 10.1002/jcph.599

  18. You have free access to this content
    RESEARCH POSTER ABSTRACTS

    Value in Health

    Volume 13, Issue 7, November 2010, Pages: A250–A480,

    Version of Record online : 11 OCT 2010, DOI: 10.1111/j.1524-4733.2010.00791_2.x

  19. Strategies for Postmarketing Surveillance of Drugs for Rare Diseases

    Clinical Pharmacology & Therapeutics

    Volume 95, Issue 3, March 2014, Pages: 265–268, A S Kesselheim and J J Gagne

    Version of Record online : 5 NOV 2013, DOI: 10.1038/clpt.2013.218

  20. The Challenges of Orphan Drugs and Orphan Diseases: Real and Imagined

    Clinical Pharmacology & Therapeutics

    Volume 92, Issue 2, August 2012, Pages: 151–153, I Hudson and A Breckenridge

    Version of Record online : 27 JUN 2012, DOI: 10.1038/clpt.2012.72