Designing clinical trials in paediatric inflammatory bowel diseases: a PIBDnet commentary

Dan Turner; Anne M Griffiths; David Wilson; Diane R Mould; Robert N Baldassano; Richard K Russell; Marla Dubinsky; Melvin B Heyman; Lissy de Ridder; Jeffrey Hyams; Javier Martin de Carpi; Laurie Conklin; William A Faubion; Sibylle Koletzko; Athos Bousvaros; Frank M Ruemmele

doi:10.1136/gutjnl-2018-317987

Designing clinical trials in paediatric inflammatory bowel diseases: a PIBDnet commentary

Dan Turner, Anne M Griffiths, David Wilson, Diane R Mould, Robert N Baldassano, Richard K Russell, Marla Dubinsky, Melvin B Heyman, Lissy de Ridder, Jeffrey Hyams, Javier Martin de Carpi, Laurie Conklin, William A Faubion, Sibylle Koletzko, Athos Bousvaros, Frank M Ruemmele

Deanery of Clinical Sciences

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INTRODUCTION: The optimal trial design for assessing novel therapies in paediatric IBD (PIBD) is a subject of intense ongoing global discussions and debate among the different stakeholders. However, there is a consensus that the current situation in which most medications used in children with IBD are prescribed as off-label without sufficient paediatric data is unacceptable. Shortening the time lag between adult and paediatric approval of drugs is of the upmost importance. In this position paper we aimed to provide guidance from the global clinical research network (Pediatric Inflammatory Bowel Disease Network, PIBDnet) for designing clinical trials in PIBD in order to facilitate drug approval for children.

METHODS: A writing group has been established by PIBDnet and topics were assigned to different members. After an iterative process of revisions among the writing group and one face-to-face meeting, all statements have reached consensus of >80% as defined a priori. Next, all core members of PIBDnet voted on the statements, reaching consensus of >80% on all statements. Comments from the members were incorporated in the text.

RESULTS: The commentary includes 18 statements for guiding data extrapolation from adults, eligibility criteria to PIBD trials, use of placebo, dosing, endpoints and recommendations for feasible trials. Controversial issues have been highlighted in the text.

CONCLUSION: The viewpoints expressed in this paper could assist planning clinical trials in PIBD which are both of high quality and ethical, while remaining pragmatic.

Original language	English
Journal	Gut
Early online date	12 Apr 2019
DOIs	https://doi.org/10.1136/gutjnl-2018-317987
Publication status	E-pub ahead of print - 12 Apr 2019

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10.1136/gutjnl-2018-317987

Industry trial advisory Revised to Gut 9.2.19 WilsonAccepted author manuscript, 415 KB

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Turner, D., Griffiths, A. M., Wilson, D., Mould, D. R., Baldassano, R. N., Russell, R. K., Dubinsky, M., Heyman, M. B., de Ridder, L., Hyams, J., Martin de Carpi, J., Conklin, L., Faubion, W. A., Koletzko, S., Bousvaros, A., & Ruemmele, F. M. (2019). Designing clinical trials in paediatric inflammatory bowel diseases: a PIBDnet commentary. Gut. Advance online publication. https://doi.org/10.1136/gutjnl-2018-317987

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title = "Designing clinical trials in paediatric inflammatory bowel diseases: a PIBDnet commentary",

abstract = "INTRODUCTION: The optimal trial design for assessing novel therapies in paediatric IBD (PIBD) is a subject of intense ongoing global discussions and debate among the different stakeholders. However, there is a consensus that the current situation in which most medications used in children with IBD are prescribed as off-label without sufficient paediatric data is unacceptable. Shortening the time lag between adult and paediatric approval of drugs is of the upmost importance. In this position paper we aimed to provide guidance from the global clinical research network (Pediatric Inflammatory Bowel Disease Network, PIBDnet) for designing clinical trials in PIBD in order to facilitate drug approval for children.METHODS: A writing group has been established by PIBDnet and topics were assigned to different members. After an iterative process of revisions among the writing group and one face-to-face meeting, all statements have reached consensus of >80% as defined a priori. Next, all core members of PIBDnet voted on the statements, reaching consensus of >80% on all statements. Comments from the members were incorporated in the text.RESULTS: The commentary includes 18 statements for guiding data extrapolation from adults, eligibility criteria to PIBD trials, use of placebo, dosing, endpoints and recommendations for feasible trials. Controversial issues have been highlighted in the text.CONCLUSION: The viewpoints expressed in this paper could assist planning clinical trials in PIBD which are both of high quality and ethical, while remaining pragmatic.",

author = "Dan Turner and Griffiths, {Anne M} and David Wilson and Mould, {Diane R} and Baldassano, {Robert N} and Russell, {Richard K} and Marla Dubinsky and Heyman, {Melvin B} and {de Ridder}, Lissy and Jeffrey Hyams and {Martin de Carpi}, Javier and Laurie Conklin and Faubion, {William A} and Sibylle Koletzko and Athos Bousvaros and Ruemmele, {Frank M}",

note = "{\textcopyright} Author(s) (or their employer(s)) 2019. No commercial re-use. See rights and permissions. Published by BMJ.",

year = "2019",

month = apr,

day = "12",

doi = "10.1136/gutjnl-2018-317987",

language = "English",

journal = "Gut",

issn = "0017-5749",

publisher = "BMJ Publishing Group",

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TY - JOUR

T1 - Designing clinical trials in paediatric inflammatory bowel diseases

T2 - a PIBDnet commentary

AU - Turner, Dan

AU - Griffiths, Anne M

AU - Wilson, David

AU - Mould, Diane R

AU - Baldassano, Robert N

AU - Russell, Richard K

AU - Dubinsky, Marla

AU - Heyman, Melvin B

AU - de Ridder, Lissy

AU - Hyams, Jeffrey

AU - Martin de Carpi, Javier

AU - Conklin, Laurie

AU - Faubion, William A

AU - Koletzko, Sibylle

AU - Bousvaros, Athos

AU - Ruemmele, Frank M

PY - 2019/4/12

Y1 - 2019/4/12

N2 - INTRODUCTION: The optimal trial design for assessing novel therapies in paediatric IBD (PIBD) is a subject of intense ongoing global discussions and debate among the different stakeholders. However, there is a consensus that the current situation in which most medications used in children with IBD are prescribed as off-label without sufficient paediatric data is unacceptable. Shortening the time lag between adult and paediatric approval of drugs is of the upmost importance. In this position paper we aimed to provide guidance from the global clinical research network (Pediatric Inflammatory Bowel Disease Network, PIBDnet) for designing clinical trials in PIBD in order to facilitate drug approval for children.METHODS: A writing group has been established by PIBDnet and topics were assigned to different members. After an iterative process of revisions among the writing group and one face-to-face meeting, all statements have reached consensus of >80% as defined a priori. Next, all core members of PIBDnet voted on the statements, reaching consensus of >80% on all statements. Comments from the members were incorporated in the text.RESULTS: The commentary includes 18 statements for guiding data extrapolation from adults, eligibility criteria to PIBD trials, use of placebo, dosing, endpoints and recommendations for feasible trials. Controversial issues have been highlighted in the text.CONCLUSION: The viewpoints expressed in this paper could assist planning clinical trials in PIBD which are both of high quality and ethical, while remaining pragmatic.

AB - INTRODUCTION: The optimal trial design for assessing novel therapies in paediatric IBD (PIBD) is a subject of intense ongoing global discussions and debate among the different stakeholders. However, there is a consensus that the current situation in which most medications used in children with IBD are prescribed as off-label without sufficient paediatric data is unacceptable. Shortening the time lag between adult and paediatric approval of drugs is of the upmost importance. In this position paper we aimed to provide guidance from the global clinical research network (Pediatric Inflammatory Bowel Disease Network, PIBDnet) for designing clinical trials in PIBD in order to facilitate drug approval for children.METHODS: A writing group has been established by PIBDnet and topics were assigned to different members. After an iterative process of revisions among the writing group and one face-to-face meeting, all statements have reached consensus of >80% as defined a priori. Next, all core members of PIBDnet voted on the statements, reaching consensus of >80% on all statements. Comments from the members were incorporated in the text.RESULTS: The commentary includes 18 statements for guiding data extrapolation from adults, eligibility criteria to PIBD trials, use of placebo, dosing, endpoints and recommendations for feasible trials. Controversial issues have been highlighted in the text.CONCLUSION: The viewpoints expressed in this paper could assist planning clinical trials in PIBD which are both of high quality and ethical, while remaining pragmatic.

U2 - 10.1136/gutjnl-2018-317987

DO - 10.1136/gutjnl-2018-317987

M3 - Article

C2 - 30979718

SN - 0017-5749

JO - Gut

JF - Gut

ER -

Designing clinical trials in paediatric inflammatory bowel diseases: a PIBDnet commentary

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