Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents

Nicholas M. Croft; Lissy de Ridder; Anne M. Griffiths; Jeffrey S. Hyams; Frank M. Ruemmele; Dan Turner; Katharine Cheng; Irja Lutsar; Marco Greco; Zuzanna Gołębiewska; Floriane Laumond; Maria Cavaller-Bellaubi; Adam Elgreey; Tara A. Altepeter; Chrissi Pallidis; Koen Norga; Robert Nelson; Wallace Crandall; Gilles Vassal

doi:10.1093/ecco-jcc/jjac135

Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents

Nicholas M. Croft^*, Lissy de Ridder, Anne M. Griffiths, Jeffrey S. Hyams, Frank M. Ruemmele, Dan Turner, Katharine Cheng, Irja Lutsar, Marco Greco, Zuzanna Gołębiewska, Floriane Laumond, Maria Cavaller-Bellaubi, Adam Elgreey, Tara A. Altepeter, Chrissi Pallidis, Koen Norga, Robert Nelson, Wallace Crandall, Gilles Vassal

^*Corresponding author for this work

Pediatrics

Research output: Contribution to journal › Article › Academic › peer-review

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Abstract

BACKGROUND AND AIMS: Despite recent approvals for new drugs to treat adults with Crohn's disease or ulcerative colitis, there are only two approved advanced treatment options [infliximab and adalimumab] for children with inflammatory bowel disease [IBD]. There are many potential new therapies being developed for adult and paediatric IBD. Moreover, regulatory agencies in both the European Union and USA have processes in place to support the early planning and initiation of paediatric studies. Nevertheless, unacceptable delays in approvals for use of drugs in children persist, with an average 7-year gap, or longer, between authorization of new IBD drugs for adults and children. METHODS: A 2-day virtual meeting was held during April 14-15, 2021 for multi-stakeholders [clinical academics, patient community, pharmaceutical companies and regulators] to discuss their perspectives on paediatric drug development for IBD. RESULTS: The multi-stakeholder group presented, discussed and proposed actions to achieve expediting the approval of new drugs in development for paediatric IBD. CONCLUSIONS: Collaborative action points for all stakeholders are required to make progress and facilitate new drug development for children with IBD.

Original language	English
Pages (from-to)	249-258
Number of pages	10
Journal	Journal of Crohn's & colitis
Volume	17
Issue number	2
DOIs	https://doi.org/10.1093/ecco-jcc/jjac135
Publication status	Published - Feb 2023

Bibliographical note

Funding:
This work was supported by the Collaborative Network for
European Clinical Trials for Children [conect4children; c4c],
including medical writing support, and has received funding
from the Innovative Medicines Initiative 2 Joint Undertaking
under grant agreement No. 777389. The Joint Undertaking
receives support from the European Union’s Horizon 2020 research and innovation programme and European Federation
of Pharmaceutical Industries and Associations.

Publisher Copyright:
© The Author(s) 2022. Published by Oxford University Press on behalf of European Crohn’s and Colitis Organisation.

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Croft, N. M., de Ridder, L., Griffiths, A. M., Hyams, J. S., Ruemmele, F. M., Turner, D., Cheng, K., Lutsar, I., Greco, M., Gołębiewska, Z., Laumond, F., Cavaller-Bellaubi, M., Elgreey, A., Altepeter, T. A., Pallidis, C., Norga, K., Nelson, R., Crandall, W., & Vassal, G. (2023). Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents. Journal of Crohn's & colitis, 17(2), 249-258. https://doi.org/10.1093/ecco-jcc/jjac135

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title = "Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents",

abstract = "BACKGROUND AND AIMS: Despite recent approvals for new drugs to treat adults with Crohn's disease or ulcerative colitis, there are only two approved advanced treatment options [infliximab and adalimumab] for children with inflammatory bowel disease [IBD]. There are many potential new therapies being developed for adult and paediatric IBD. Moreover, regulatory agencies in both the European Union and USA have processes in place to support the early planning and initiation of paediatric studies. Nevertheless, unacceptable delays in approvals for use of drugs in children persist, with an average 7-year gap, or longer, between authorization of new IBD drugs for adults and children. METHODS: A 2-day virtual meeting was held during April 14-15, 2021 for multi-stakeholders [clinical academics, patient community, pharmaceutical companies and regulators] to discuss their perspectives on paediatric drug development for IBD. RESULTS: The multi-stakeholder group presented, discussed and proposed actions to achieve expediting the approval of new drugs in development for paediatric IBD. CONCLUSIONS: Collaborative action points for all stakeholders are required to make progress and facilitate new drug development for children with IBD.",

author = "Croft, {Nicholas M.} and {de Ridder}, Lissy and Griffiths, {Anne M.} and Hyams, {Jeffrey S.} and Ruemmele, {Frank M.} and Dan Turner and Katharine Cheng and Irja Lutsar and Marco Greco and Zuzanna Go{\l}{\c e}biewska and Floriane Laumond and Maria Cavaller-Bellaubi and Adam Elgreey and Altepeter, {Tara A.} and Chrissi Pallidis and Koen Norga and Robert Nelson and Wallace Crandall and Gilles Vassal",

note = "Funding: This work was supported by the Collaborative Network for European Clinical Trials for Children [conect4children; c4c], including medical writing support, and has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No. 777389. The Joint Undertaking receives support from the European Union{\textquoteright}s Horizon 2020 research and innovation programme and European Federation of Pharmaceutical Industries and Associations. Publisher Copyright: {\textcopyright} The Author(s) 2022. Published by Oxford University Press on behalf of European Crohn{\textquoteright}s and Colitis Organisation.",

year = "2023",

month = feb,

doi = "10.1093/ecco-jcc/jjac135",

language = "English",

volume = "17",

pages = "249--258",

journal = "Journal of Crohn's and Colitis",

issn = "1873-9946",

publisher = "Oxford University Press",

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Croft, NM, de Ridder, L, Griffiths, AM, Hyams, JS, Ruemmele, FM, Turner, D, Cheng, K, Lutsar, I, Greco, M, Gołębiewska, Z, Laumond, F, Cavaller-Bellaubi, M, Elgreey, A, Altepeter, TA, Pallidis, C, Norga, K, Nelson, R, Crandall, W & Vassal, G 2023, 'Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents', Journal of Crohn's & colitis, vol. 17, no. 2, pp. 249-258. https://doi.org/10.1093/ecco-jcc/jjac135

TY - JOUR

T1 - Paediatric Inflammatory Bowel Disease

T2 - A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents

AU - Croft, Nicholas M.

AU - de Ridder, Lissy

AU - Griffiths, Anne M.

AU - Hyams, Jeffrey S.

AU - Ruemmele, Frank M.

AU - Turner, Dan

AU - Cheng, Katharine

AU - Lutsar, Irja

AU - Greco, Marco

AU - Gołębiewska, Zuzanna

AU - Laumond, Floriane

AU - Cavaller-Bellaubi, Maria

AU - Elgreey, Adam

AU - Altepeter, Tara A.

AU - Pallidis, Chrissi

AU - Norga, Koen

AU - Nelson, Robert

AU - Crandall, Wallace

AU - Vassal, Gilles

N1 - Funding: This work was supported by the Collaborative Network for European Clinical Trials for Children [conect4children; c4c], including medical writing support, and has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No. 777389. The Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and European Federation of Pharmaceutical Industries and Associations. Publisher Copyright: © The Author(s) 2022. Published by Oxford University Press on behalf of European Crohn’s and Colitis Organisation.

PY - 2023/2

Y1 - 2023/2

N2 - BACKGROUND AND AIMS: Despite recent approvals for new drugs to treat adults with Crohn's disease or ulcerative colitis, there are only two approved advanced treatment options [infliximab and adalimumab] for children with inflammatory bowel disease [IBD]. There are many potential new therapies being developed for adult and paediatric IBD. Moreover, regulatory agencies in both the European Union and USA have processes in place to support the early planning and initiation of paediatric studies. Nevertheless, unacceptable delays in approvals for use of drugs in children persist, with an average 7-year gap, or longer, between authorization of new IBD drugs for adults and children. METHODS: A 2-day virtual meeting was held during April 14-15, 2021 for multi-stakeholders [clinical academics, patient community, pharmaceutical companies and regulators] to discuss their perspectives on paediatric drug development for IBD. RESULTS: The multi-stakeholder group presented, discussed and proposed actions to achieve expediting the approval of new drugs in development for paediatric IBD. CONCLUSIONS: Collaborative action points for all stakeholders are required to make progress and facilitate new drug development for children with IBD.

AB - BACKGROUND AND AIMS: Despite recent approvals for new drugs to treat adults with Crohn's disease or ulcerative colitis, there are only two approved advanced treatment options [infliximab and adalimumab] for children with inflammatory bowel disease [IBD]. There are many potential new therapies being developed for adult and paediatric IBD. Moreover, regulatory agencies in both the European Union and USA have processes in place to support the early planning and initiation of paediatric studies. Nevertheless, unacceptable delays in approvals for use of drugs in children persist, with an average 7-year gap, or longer, between authorization of new IBD drugs for adults and children. METHODS: A 2-day virtual meeting was held during April 14-15, 2021 for multi-stakeholders [clinical academics, patient community, pharmaceutical companies and regulators] to discuss their perspectives on paediatric drug development for IBD. RESULTS: The multi-stakeholder group presented, discussed and proposed actions to achieve expediting the approval of new drugs in development for paediatric IBD. CONCLUSIONS: Collaborative action points for all stakeholders are required to make progress and facilitate new drug development for children with IBD.

UR - http://www.scopus.com/inward/record.url?scp=85150665432&partnerID=8YFLogxK

U2 - 10.1093/ecco-jcc/jjac135

DO - 10.1093/ecco-jcc/jjac135

M3 - Article

C2 - 36130314

AN - SCOPUS:85150665432

SN - 1873-9946

VL - 17

SP - 249

EP - 258

JO - Journal of Crohn's and Colitis

JF - Journal of Crohn's and Colitis

IS - 2

ER -

Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents

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