Rights statement: This is the peer reviewed version of the following article: Schanberg, L. E., Ramanan, A. V., De Benedetti, F. , Beukelman, T. , Eakin, G. S., Del Gaizo, V. , Ringold, S. , Vesely, R. , Schrandt, S. , Jaki, T. , Bili, A. , Chung, J. B., De Bono, S. , Douglass, W. , Enejosa, J. V., Kanik, K. S., Knobe, K. , Kunder, R. , Leite‐Schnell, J. C., Suehiro, R. M., Wong, R. L., Mieszkalski, K. L., Marrow, L. C., Siebenaler, K. , Fraulo, E. and Kimura, Y. (2019), Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis. Arthritis Rheumatol. doi:10.1002/art.41043 which has been published in final form at https://onlinelibrary.wiley.com/doi/full/10.1002/art.41043 This article may be used for non-commercial purposes in accordance With Wiley Terms and Conditions for self-archiving.
Accepted author manuscript, 336 KB, PDF document
Available under license: CC BY-NC: Creative Commons Attribution-NonCommercial 4.0 International License
Final published version
Research output: Contribution to Journal/Magazine › Journal article › peer-review
Research output: Contribution to Journal/Magazine › Journal article › peer-review
}
TY - JOUR
T1 - Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis
AU - Schanberg, L.E.
AU - Ramanan, A.V.
AU - De Benedetti, F.
AU - Beukelman, T.
AU - Eakin, G.S.
AU - Del Gaizo, V.
AU - Ringold, S.
AU - Vesely, R.
AU - Schrandt, S.
AU - Jaki, T.
AU - Bili, A.
AU - Chung, J.B.
AU - De Bono, S.
AU - Douglass, W.
AU - Enejosa, J.V.
AU - Kanik, K.S.
AU - Knobe, K.
AU - Kunder, R.
AU - Leite-Schnell, J.C.
AU - Suehiro, R.M.
AU - Wong, R.L.
AU - Mieszkalski, K.L.
AU - Marrow, L.C.
AU - Siebenaler, K.
AU - Fraulo, E.
AU - Kimura, Y.
N1 - This is the peer reviewed version of the following article: Schanberg, L. E., Ramanan, A. V., De Benedetti, F. , Beukelman, T. , Eakin, G. S., Del Gaizo, V. , Ringold, S. , Vesely, R. , Schrandt, S. , Jaki, T. , Bili, A. , Chung, J. B., De Bono, S. , Douglass, W. , Enejosa, J. V., Kanik, K. S., Knobe, K. , Kunder, R. , Leite‐Schnell, J. C., Suehiro, R. M., Wong, R. L., Mieszkalski, K. L., Marrow, L. C., Siebenaler, K. , Fraulo, E. and Kimura, Y. (2019), Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis. Arthritis Rheumatol. doi:10.1002/art.41043 which has been published in final form at https://onlinelibrary.wiley.com/doi/full/10.1002/art.41043 This article may be used for non-commercial purposes in accordance With Wiley Terms and Conditions for self-archiving.
PY - 2019/12/1
Y1 - 2019/12/1
N2 - 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.
AB - 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.
U2 - 10.1002/art.41043
DO - 10.1002/art.41043
M3 - Journal article
VL - 71
SP - 1976
EP - 1984
JO - Arthritis and Rheumatology
JF - Arthritis and Rheumatology
SN - 2326-5191
IS - 12
ER -