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.
| Original language | English (US) |
|---|---|
| Journal | Gut |
| DOIs | |
| State | Published - Jan 1 2019 |
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Keywords
- clinical trials
- IBD clinical
- paediatric gastroenterology
ASJC Scopus subject areas
- Gastroenterology
Cite this
Designing clinical trials in paediatric inflammatory bowel diseases : A PIBDnet commentary. / Turner, Dan; Griffiths, Anne M.; Wilson, David; Mould, Diane R.; Baldassano, Robert N.; Russell, Richard K.; Dubinsky, Marla; Heyman, Melvin B.; De Ridder, Lissy; Hyams, Jeffrey; Martin De Carpi, Javier; Conklin, Laurie; Faubion, William Alvis; Koletzko, Sibylle; Bousvaros, Athos; Ruemmele, Frank M.
In: Gut, 01.01.2019.Research output: Contribution to journal › Article
}
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 Alvis
AU - Koletzko, Sibylle
AU - Bousvaros, Athos
AU - Ruemmele, Frank M.
PY - 2019/1/1
Y1 - 2019/1/1
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.
KW - clinical trials
KW - IBD clinical
KW - paediatric gastroenterology
UR - http://www.scopus.com/inward/record.url?scp=85064261952&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85064261952&partnerID=8YFLogxK
U2 - 10.1136/gutjnl-2018-317987
DO - 10.1136/gutjnl-2018-317987
M3 - Article
AN - SCOPUS:85064261952
JO - Gut
JF - Gut
SN - 0017-5749
ER -