Ausgabe Sonderheft 3/2014
Inhalt (16 Artikel)
Introduction to a Supplement on Innovative Approaches to Studying Health Outcomes in Rare Diseases
Aaron S. Kesselheim, Joshua J. Gagne
Highly Effective Cystic Fibrosis Clinical Research Teams: Critical Success Factors
George Z. Retsch-Bogart, Jill M. Van Dalfsen, Bruce C. Marshall, Cynthia George, Joseph M. Pilewski, Eugene C. Nelson, Christopher H. Goss, Bonnie W. Ramsey
Quantifying a Rare Disease in Administrative Data: The Example of Calciphylaxis
Sagar U. Nigwekar, Craig A. Solid, Elizabeth Ankers, Rajeev Malhotra, William Eggert, Alexander Turchin, Ravi I. Thadhani, Charles A. Herzog
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, J. R. Mann, O. D. Ozturk, J. Bolen
The Rare Diseases Clinical Research Network’s Organization and Approach to Observational Research and Health Outcomes Research
Jeffrey P. Krischer, Rashmi Gopal-Srivastava, Stephen. C. Groft, David J. Eckstein
Orphan Therapies: Making Best Use of Postmarket Data
Judith C. Maro, Jeffrey S. Brown, Gerald J. Dal Pan, Lingling Li
Utilization of Health Care Services and Satisfaction with Care in Adults Affected by Disorders of Sex Development (DSD)
Ute Thyen, Anke Lux, Martina Jürgensen, Olaf Hiort, Birgit Köhler
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
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, Joseph Beyene, Marjorie Mclimont, Victor Blanchette, Brian M. Feldman
Application of a Policy Framework for the Public Funding of Drugs for Rare Diseases
Eric Winquist, Doug Coyle, Joe T. R. Clarke, Gerald A. Evans, Christine Seager, Winnie Chan, Janet Martin
RD-Connect: An Integrated Platform Connecting Databases, Registries, Biobanks and Clinical Bioinformatics for Rare Disease Research
Rachel Thompson, Louise Johnston, Domenica Taruscio, Lucia Monaco, Christophe Béroud, Ivo G. Gut, Mats G. Hansson, Peter-Bram A. ’t Hoen, George P. Patrinos, Hugh Dawkins, Monica Ensini, Kurt Zatloukal, David Koubi, Emma Heslop, Justin E. Paschall, Manuel Posada, Peter N. Robinson, Kate Bushby, Hanns Lochmüller
A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases
Laura P. Forsythe, Victoria Szydlowski, Mohammad Hassan Murad, Stanley Ip, Zhen Wang, Tarig A. Elraiyah, Rachael Fleurence, David H. Hickam
Patient-Reported Outcomes in Clinical Trials of Rare Diseases
Ethan Basch, Antonia V. Bennett
Important Role of Translational Science in Rare Disease Innovation, Discovery, and Drug Development
Anne R. Pariser, William A. Gahl
A Lesson in Participatory Research for a Rare Mutation of Cystic Fibrosis
Emily Kramer-Golinkoff