Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis

Laura E. Schanberg, Athimalaipet V Ramanan, Fabrizio De Benedetti, Timothy Beukelman, Guy S. Eakin, Vincent Del Gaizo, Sarah Ringold, Richard Vesely, Suzanne Schrandt, Thomas Jaki, Androniki Bili, James B. Chung, Stephanie De Bono, Wendy Douglass, Jeffrey V. Enejosa, Keith S. Kanik, Karin Knobe, Rebecca Kunder, Juliana C. Leite-Schnell, Ricardo Maisse SuehiroRobert L. Wong, Kelly L. Mieszkalski, Laura C. Marrow, Kristin Siebenaler, Elizabeth Fraulo, Yukiko Kimura

Research output: Contribution to journalArticle (Academic Journal)peer-review

9 Citations (Scopus)

Abstract

Objective
A meeting was organized to bring together multiple stakeholders involved in the testing and authorization of new medicines for juvenile idiopathic arthritis (JIA) to discuss current issues surrounding trials and access of new medicines for children and adolescents with JIA.

Methods
The Childhood Arthritis and Rheumatology Research Alliance (CARRA) invited regulatory agencies (Food and Drug Administration [FDA] and European Medicines Agency [EMA]), major pharmaceutical companies with JIA products approved or in development, patient and parent representatives, advocacy organization (Arthritis Foundation) and pediatric rheumatology clinicians/investigators to a one‐day meeting in April 2018.

Results
The participants highlighted current issues in clinical trials. As the pharmacologic armamentarium to treat inflammatory arthritis rapidly expands, registration trial designs to test medicines in JIA patients must adapt. Many methodologies used successfully in the recent past are no longer feasible. The pool of patients meeting entry criteria who are willing to participate is shrinking at the same time that the number of medicines that need testing is growing. Solutions included proposing innovative clinical trial methods to regulatory agencies, as well as open discussion among stakeholders.

Conclusion
Ensuring new medicines are authorized in a timely manner to meet the needs of JIA patients worldwide is critical. Approaches should include: open dialogue 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.
Original languageEnglish
JournalArthritis and Rheumatology
Early online date16 Jul 2019
DOIs
Publication statusE-pub ahead of print - 16 Jul 2019

Bibliographical note

The acceptance date for this record is provisional and based upon the month of publication for the article.

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