Volume 29, Issue 3 Supplement, August 2014

In this issue (16 articles)

1. Editorial

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

   Aaron S. Kesselheim M.D., J.D., M.P.H… Pages 709-711

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2. Editorial

   Preface to the AHRQ Supplement

   Scott R. Smith M.S.P.H, Ph.D. Pages 712-713

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3. Original Research

   Highly Effective Cystic Fibrosis Clinical Research Teams: Critical Success Factors

   George Z. Retsch-Bogart MD, Jill M. Van Dalfsen BS… Pages 714-723

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4. Original Research

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

   Sagar U. Nigwekar M.D., M.M.Sc.… Pages 724-731

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5. Original Research

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

   J. A. Royer, J. W. Hardin, S. McDermott, L. Ouyang… Pages 732-738

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6. Original Research

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

   Jeffrey P. Krischer Ph.D.… Pages 739-744

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7. Original Research

   Orphan Therapies: Making Best Use of Postmarket Data

   Judith C. Maro Ph.D., Jeffrey S. Brown Ph.D.… Pages 745-751

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8. Original Research

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

   Ute Thyen MD, Anke Lux MS, Martina Jürgensen PhD… Pages 752-759

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9. Original Research

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

   Jiat-Ling Poon PhD, Jason N. Doctor PhD… Pages 760-766

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10. Original Research

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

    Lusine Abrahamyan MD MPH PhD, Andrew R. Willan PhD… Pages 767-773

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11. Original Research

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

    Eric Winquist MD, MSc, Doug Coyle PhD… Pages 774-779

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12. Review Article

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

    Rachel Thompson M.Chem., Louise Johnston Ph.D.… Pages 780-787

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13. Review Article

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

    Laura P. Forsythe PhD, MPH, Victoria Szydlowski BS… Pages 788-800

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14. Perspective

    Patient-Reported Outcomes in Clinical Trials of Rare Diseases

    Ethan Basch MD, Antonia V. Bennett PhD Pages 801-803

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15. Perspective

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

    Anne R. Pariser MD, William A. Gahl MD, PhD Pages 804-807

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16. Healing Arts

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

    Emily Kramer-Golinkoff BA, MBE Pages 808-809

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