1. Introduction to a Supplement on Innovative Approaches to Studying Health Outcomes in Rare Diseases

   • Aaron S. Kesselheim
   • Joshua J. Gagne

   Editorial 17 July 2014 Pages: 709 - 711

2. Preface to the AHRQ Supplement

   • Scott R. Smith

   Editorial 17 July 2014 Pages: 712 - 713

3. Highly Effective Cystic Fibrosis Clinical Research Teams: Critical Success Factors

   • George Z. Retsch-Bogart
   • Jill M. Van Dalfsen
   • Bonnie W. Ramsey

   Original Research 17 July 2014 Pages: 714 - 723

   

4. Quantifying a Rare Disease in Administrative Data: The Example of Calciphylaxis

   • Sagar U. Nigwekar
   • Craig A. Solid
   • Charles A. Herzog

   Original Research 17 July 2014 Pages: 724 - 731

   

5. Use of State Administrative Data Sources to Study Adolescents and Young Adults with Rare Conditions

   • J. A. Royer
   • J. W. Hardin
   • J. Bolen

   Original Research 17 July 2014 Pages: 732 - 738

6. The Rare Diseases Clinical Research Network’s Organization and Approach to Observational Research and Health Outcomes Research

   • Jeffrey P. Krischer
   • Rashmi Gopal-Srivastava
   • for the Rare Diseases Clinical Research Network

   Original Research 17 July 2014 Pages: 739 - 744

   

7. Orphan Therapies: Making Best Use of Postmarket Data

   • Judith C. Maro
   • Jeffrey S. Brown
   • Lingling Li

   Original Research 17 July 2014 Pages: 745 - 751

   

8. Utilization of Health Care Services and Satisfaction with Care in Adults Affected by Disorders of Sex Development (DSD)

   • Ute Thyen
   • Anke Lux
   • Birgit Köhler

   Original Research 17 July 2014 Pages: 752 - 759

9. Longitudinal Changes in Health-Related Quality of Life for Chronic Diseases: An Example in Hemophilia A

   • Jiat-Ling Poon
   • Jason N. Doctor
   • Michael B. Nichol

   Original Research 17 July 2014 Pages: 760 - 766

   

10. Using Value-of-Information Methods when the Disease Is Rare and the Treatment Is Expensive—The Example of Hemophilia A

    • Lusine Abrahamyan
    • Andrew R. Willan
    • for the Canadian Hemophilia Primary Prophylaxis (CHPS) Study Group

    Original Research 17 July 2014 Pages: 767 - 773

    

11. Application of a Policy Framework for the Public Funding of Drugs for Rare Diseases

    • Eric Winquist
    • Doug Coyle
    • Janet Martin

    Original Research 17 July 2014 Pages: 774 - 779

12. RD-Connect: An Integrated Platform Connecting Databases, Registries, Biobanks and Clinical Bioinformatics for Rare Disease Research

    • Rachel Thompson
    • Louise Johnston
    • Hanns Lochmüller

    Review Article 17 July 2014 Pages: 780 - 787

    

13. A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

    • Laura P. Forsythe
    • Victoria Szydlowski
    • David H. Hickam

    Review Article 22 July 2014 Pages: 788 - 800

    

14. Patient-Reported Outcomes in Clinical Trials of Rare Diseases

    • Ethan Basch
    • Antonia V. Bennett

    Perspective 17 July 2014 Pages: 801 - 803

15. Important Role of Translational Science in Rare Disease Innovation, Discovery, and Drug Development

    • Anne R. Pariser
    • William A. Gahl

    Perspective 17 July 2014 Pages: 804 - 807

16. A Lesson in Participatory Research for a Rare Mutation of Cystic Fibrosis

    • Emily Kramer-Golinkoff

    Healing Arts 17 July 2014 Pages: 808 - 809