학술논문
Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis.
Document Type
Academic Journal
Author
Schanberg LE; Duke University Medical Center, Durham, North Carolina.; Ramanan AV; University Hospitals Bristol NHS Foundation Trust, Bristol Medical School, University of Bristol, Bristol, UK.; De Benedetti F; Ospedale Pediatrico Bambino Gesù, Rome, Italy.; Beukelman T; University of Alabama at Birmingham.; Eakin GS; Arthritis Foundation, Atlanta, Georgia.; Del Gaizo V; Childhood Arthritis and Rheumatology Research Alliance, Milwaukee, Wisconsin.; Ringold S; Seattle Children's Hospital, Seattle, Washington.; Vesely R; European Medicines Agency, London, UK.; Schrandt S; Arthritis Foundation, Atlanta, Georgia.; Jaki T; Lancaster University, Lancaster, UK.; Bili A; Janssen Immunology R&D, Spring House, Pennsylvania.; Chung JB; Amgen Inc., Thousand Oaks, California.; De Bono S; Eli Lilly, Basingstoke, UK.; Douglass W; Roche, Basel, Switzerland.; Enejosa JV; AbbVie Inc., North Chicago, Illinois.; Kanik KS; Global Product Development, Pfizer Inc., New London, Connecticut.; Knobe K; Sobi, Stockholm, Sweden.; Kunder R; Gilead Sciences, Inc., Foster City, California.; Leite-Schnell JC; AbbVie Inc., North Chicago, Illinois.; Suehiro RM; Pfizer Inc, Collegeville, Pennsylvania.; Wong RL; Bristol-Myers Squibb, Princeton, New Jersey.; Mieszkalski KL; Childhood Arthritis and Rheumatology Research Alliance, Milwaukee, Wisconsin.; Marrow LC; Arthritis Foundation, Atlanta, Georgia.; Siebenaler K; Duke Clinical Research Institute, Durham, North Carolina.; Fraulo E; Duke University Medical Center, Durham, North Carolina.; Kimura Y; Hackensack University Medical Center, Hackensack, New Jersey.
Source
Publisher: Wiley Country of Publication: United States NLM ID: 101623795 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2326-5205 (Electronic) Linking ISSN: 23265191 NLM ISO Abbreviation: Arthritis Rheumatol Subsets: MEDLINE
Subject
Language
English
Abstract
A meeting was organized to bring together multiple stakeholders involved in the testing and authorization of new medications for juvenile idiopathic arthritis (JIA) to discuss current issues surrounding clinical trials and access to new medications for children and adolescents with JIA. The Childhood Arthritis and Rheumatology Research Alliance invited representatives of regulatory agencies (Food and Drug Administration and European Medicines Agency), and major pharmaceutical companies with JIA-approved products or products in development, patient and parent representatives, representatives of an advocacy organization (Arthritis Foundation), and pediatric rheumatology clinicians/investigators to a 1-day meeting in April 2018. The participants engaged in discussion regarding issues in clinical trials. As the pharmacologic options to treat inflammatory arthritis rapidly expand, registration trial designs to test medications in JIA patients must adapt. Many methodologies successfully used in the recent past are no longer feasible. The pool of patients meeting entry criteria who are willing to participate is shrinking while the number of medications to be tested is growing. Suggested solutions included proposing innovative clinical trial methods to regulatory agencies, as well as open discussions among stakeholders. Ensuring that new medications are authorized in a timely manner to meet the needs of JIA patients worldwide is critical. Approaches should include open dialog between regulatory agencies, pharmaceutical companies, and other stakeholders to develop and implement novel study designs, including patient and clinician perspectives to define meaningful trial outcomes, and changing existing study plans.
(© 2019, American College of Rheumatology.)
(© 2019, American College of Rheumatology.)