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

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. This article is protected by copyright. All rights reserved.

Original languageEnglish
JournalArthritis and Rheumatology
DOIs
Publication statusE-pub ahead of print - Jul 16 2019

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Juvenile Arthritis
Arthritis
Rheumatology
Clinical Trials
Adolescent Medicine
Patient Advocacy
United States Food and Drug Administration
Pharmaceutical Preparations
Research Personnel
Organizations
Pediatrics
Research

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Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis. / Schanberg, Laura E; Ramanan, Athimalaipet V; De Benedetti, Fabrizio; Beukelman, Timothy; Eakin, Guy S; Del Gaizo, Vincent; Ringold, Sarah; Vesely, Richard; Schrandt, Suzanne; Jaki, Thomas; Bili, Androniki; Chung, James B; De Bono, Stephanie; Douglass, Wendy; Enejosa, Jeffrey V; Kanik, Keith S; Knobe, Karin; Kunder, Rebecca; Leite-Schnell, Juliana C; Suehiro, Ricardo Maisse; Wong, Robert L; Mieszkalski, Kelly L; Marrow, Laura C; Siebenaler, Kristin; Fraulo, Elizabeth; Kimura, Yukiko.

In: Arthritis and Rheumatology, 16.07.2019.

Research output: Contribution to journalArticle

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 2019, 'Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis', Arthritis and Rheumatology. https://doi.org/10.1002/art.41043
Schanberg, Laura E ; Ramanan, Athimalaipet V ; De Benedetti, Fabrizio ; Beukelman, Timothy ; Eakin, Guy S ; Del Gaizo, Vincent ; Ringold, Sarah ; Vesely, Richard ; Schrandt, Suzanne ; Jaki, Thomas ; Bili, Androniki ; Chung, James B ; De Bono, Stephanie ; Douglass, Wendy ; Enejosa, Jeffrey V ; Kanik, Keith S ; Knobe, Karin ; Kunder, Rebecca ; Leite-Schnell, Juliana C ; Suehiro, Ricardo Maisse ; Wong, Robert L ; Mieszkalski, Kelly L ; Marrow, Laura C ; Siebenaler, Kristin ; Fraulo, Elizabeth ; Kimura, Yukiko. / Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis. In: Arthritis and Rheumatology. 2019.
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title = "Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis",
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. This article is protected by copyright. All rights reserved.",
author = "Schanberg, {Laura E} and Ramanan, {Athimalaipet V} and {De Benedetti}, Fabrizio and Timothy Beukelman and Eakin, {Guy S} and {Del Gaizo}, Vincent and Sarah Ringold and Richard Vesely and Suzanne Schrandt and Thomas Jaki and Androniki Bili and Chung, {James B} and {De Bono}, Stephanie and Wendy Douglass and Enejosa, {Jeffrey V} and Kanik, {Keith S} and Karin Knobe and Rebecca Kunder and Leite-Schnell, {Juliana C} and Suehiro, {Ricardo Maisse} and Wong, {Robert L} and Mieszkalski, {Kelly L} and Marrow, {Laura C} and Kristin Siebenaler and Elizabeth Fraulo and Yukiko Kimura",
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T1 - Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis

AU - Schanberg, Laura E

AU - Ramanan, Athimalaipet V

AU - De Benedetti, Fabrizio

AU - Beukelman, Timothy

AU - Eakin, Guy S

AU - Del Gaizo, Vincent

AU - Ringold, Sarah

AU - Vesely, Richard

AU - Schrandt, Suzanne

AU - Jaki, Thomas

AU - Bili, Androniki

AU - Chung, James B

AU - De Bono, Stephanie

AU - Douglass, Wendy

AU - Enejosa, Jeffrey V

AU - Kanik, Keith S

AU - Knobe, Karin

AU - Kunder, Rebecca

AU - Leite-Schnell, Juliana C

AU - Suehiro, Ricardo Maisse

AU - Wong, Robert L

AU - Mieszkalski, Kelly L

AU - Marrow, Laura C

AU - Siebenaler, Kristin

AU - Fraulo, Elizabeth

AU - Kimura, Yukiko

N1 - This article is protected by copyright. All rights reserved.

PY - 2019/7/16

Y1 - 2019/7/16

N2 - 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. This article is protected by copyright. All rights reserved.

AB - 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. This article is protected by copyright. All rights reserved.

U2 - 10.1002/art.41043

DO - 10.1002/art.41043

M3 - Article

C2 - 31313532

JO - Arthritis and Rheumatology

JF - Arthritis and Rheumatology

SN - 2326-5191

ER -