Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis.
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.
Schanberg, LE; Ramanan, AV; De Benedetti, F; Beukelman, T; Eakin, GS; Del Gaizo, V; Ringold, S; Vesely, R; Schrandt, S; Jaki, T; Bili, A; Chung, JB; De Bono, S; Douglass, W; Enejosa, JV; Kanik, KS; Knobe, K; Kunder, R; Leite-Schnell, JC; Suehiro, RM; Wong, RL; Mieszkalski, KL; Marrow, LC; Siebenaler, K; Fraulo, E; Kimura, Y
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