Introduction to a Supplement on Innovative Approaches to Studying Health Outcomes in Rare Diseases Aaron S. KesselheimJoshua J. Gagne Editorial 17 July 2014 Pages: 709 - 711
Highly Effective Cystic Fibrosis Clinical Research Teams: Critical Success Factors George Z. Retsch-BogartJill M. Van DalfsenBonnie W. Ramsey Original Research 17 July 2014 Pages: 714 - 723
Quantifying a Rare Disease in Administrative Data: The Example of Calciphylaxis Sagar U. NigwekarCraig A. SolidCharles A. Herzog Original Research 17 July 2014 Pages: 724 - 731
Use of State Administrative Data Sources to Study Adolescents and Young Adults with Rare Conditions J. A. RoyerJ. W. HardinJ. Bolen Original Research 17 July 2014 Pages: 732 - 738
The Rare Diseases Clinical Research Network’s Organization and Approach to Observational Research and Health Outcomes Research Jeffrey P. KrischerRashmi Gopal-Srivastavafor the Rare Diseases Clinical Research Network Original Research 17 July 2014 Pages: 739 - 744
Orphan Therapies: Making Best Use of Postmarket Data Judith C. MaroJeffrey S. BrownLingling Li Original Research 17 July 2014 Pages: 745 - 751
Utilization of Health Care Services and Satisfaction with Care in Adults Affected by Disorders of Sex Development (DSD) Ute ThyenAnke LuxBirgit Köhler Original Research 17 July 2014 Pages: 752 - 759
Longitudinal Changes in Health-Related Quality of Life for Chronic Diseases: An Example in Hemophilia A Jiat-Ling PoonJason N. DoctorMichael B. Nichol Original Research 17 July 2014 Pages: 760 - 766
Using Value-of-Information Methods when the Disease Is Rare and the Treatment Is Expensive—The Example of Hemophilia A Lusine AbrahamyanAndrew R. Willanfor the Canadian Hemophilia Primary Prophylaxis (CHPS) Study Group Original Research 17 July 2014 Pages: 767 - 773
Application of a Policy Framework for the Public Funding of Drugs for Rare Diseases Eric WinquistDoug CoyleJanet Martin Original Research 17 July 2014 Pages: 774 - 779
RD-Connect: An Integrated Platform Connecting Databases, Registries, Biobanks and Clinical Bioinformatics for Rare Disease Research Rachel ThompsonLouise JohnstonHanns Lochmüller Review Article 17 July 2014 Pages: 780 - 787
A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases Laura P. ForsytheVictoria SzydlowskiDavid H. Hickam Review Article 22 July 2014 Pages: 788 - 800
Patient-Reported Outcomes in Clinical Trials of Rare Diseases Ethan BaschAntonia V. Bennett Perspective 17 July 2014 Pages: 801 - 803
Important Role of Translational Science in Rare Disease Innovation, Discovery, and Drug Development Anne R. PariserWilliam A. Gahl Perspective 17 July 2014 Pages: 804 - 807
A Lesson in Participatory Research for a Rare Mutation of Cystic Fibrosis Emily Kramer-Golinkoff Healing Arts 17 July 2014 Pages: 808 - 809
