. 2023 Mar 18;17(2):249-258.

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 Croft¹, Lissy de Ridder², Anne M Griffiths³, Jeffrey S Hyams⁴, Frank M Ruemmele^{5

6}, Dan Turner⁷, Katharine Cheng⁸, Irja Lutsar⁹, Marco Greco¹⁰, Zuzanna Gołębiewska¹¹, Floriane Laumond¹², Maria Cavaller-Bellaubi¹³, Adam Elgreey¹⁴, Tara A Altepeter¹⁵, Chrissi Pallidis¹⁶, Koen Norga¹⁷, Robert Nelson⁸, Wallace Crandall¹⁸, Gilles Vassal¹⁹

Affiliations

¹ Blizard Institute, Faculty of Medicine and Dentistry, Queen Mary University of London, London, UK.
² Department of Pediatric Gastroenterology, Erasmus MC Sophia Children's Hospital, Rotterdam, The Netherlands.
³ Inflammatory Bowel Disease Centre, The Hospital for Sick Children, University of Toronto, Canada.
⁴ Connecticut Children's Medical Center, Hartford, CT, USA.
⁵ Université de Paris, Faculté de Médecine, Paris, France.
⁶ Assistance Publique-Hôpitaux de Paris, Hôpital Universitaire Necker Enfants Malades, Service de Gastroentérologie Pédiatrique, Paris, France.
⁷ Shaare Zedek Medical Center, the Hebrew University of Jerusalem, Jerusalem, Israel.
⁸ Johnson and Johnson, New Brunswick, Canada.
⁹ Institute of Medical Microbiology, University of Tartu, Tartu, Estonia.
¹⁰ European Patients' Forum (EPF), Chaussée d'Etterbeek, Brussels, Belgium.
¹¹ J-elita, Polish Society for the Support of People with Inflammatory Bowel Disease, Warsaw, Poland.
¹² Aupetit François Association (AFA) Crohn RCH, Paris, France.
¹³ EURORDIS-Rare Diseases Europe, Paris, France.
¹⁴ Crohn and Colitis Foundation Israel (CCFI), Tel Aviv, Israel.
¹⁵ Division of Gastroenterology, Office of Immunology and Inflammation, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
¹⁶ Paediatric Medicines, European Medicines Agency, The Netherlands.
¹⁷ Paediatric Committee (PDCO), European Medicines Agency, The Netherlands.
¹⁸ Pediatric Immunology, Eli Lilly and Company, Indianapolis, IN, USA.
¹⁹ Department of Pediatric and Adolescent Oncology, Gustave Roussy Comprehensive Cancer Center and Paris Saclay University, Villejuif, France.

PMID: 36130314
PMCID: PMC10024546
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 et al. J Crohns Colitis. 2023.

. 2023 Mar 18;17(2):249-258.

doi: 10.1093/ecco-jcc/jjac135.

Authors

Affiliations

¹ Blizard Institute, Faculty of Medicine and Dentistry, Queen Mary University of London, London, UK.
² Department of Pediatric Gastroenterology, Erasmus MC Sophia Children's Hospital, Rotterdam, The Netherlands.
³ Inflammatory Bowel Disease Centre, The Hospital for Sick Children, University of Toronto, Canada.
⁴ Connecticut Children's Medical Center, Hartford, CT, USA.
⁵ Université de Paris, Faculté de Médecine, Paris, France.
⁶ Assistance Publique-Hôpitaux de Paris, Hôpital Universitaire Necker Enfants Malades, Service de Gastroentérologie Pédiatrique, Paris, France.
⁷ Shaare Zedek Medical Center, the Hebrew University of Jerusalem, Jerusalem, Israel.
⁸ Johnson and Johnson, New Brunswick, Canada.
⁹ Institute of Medical Microbiology, University of Tartu, Tartu, Estonia.
¹⁰ European Patients' Forum (EPF), Chaussée d'Etterbeek, Brussels, Belgium.
¹¹ J-elita, Polish Society for the Support of People with Inflammatory Bowel Disease, Warsaw, Poland.
¹² Aupetit François Association (AFA) Crohn RCH, Paris, France.
¹³ EURORDIS-Rare Diseases Europe, Paris, France.
¹⁴ Crohn and Colitis Foundation Israel (CCFI), Tel Aviv, Israel.
¹⁵ Division of Gastroenterology, Office of Immunology and Inflammation, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
¹⁶ Paediatric Medicines, European Medicines Agency, The Netherlands.
¹⁷ Paediatric Committee (PDCO), European Medicines Agency, The Netherlands.
¹⁸ Pediatric Immunology, Eli Lilly and Company, Indianapolis, IN, USA.
¹⁹ Department of Pediatric and Adolescent Oncology, Gustave Roussy Comprehensive Cancer Center and Paris Saclay University, Villejuif, France.

PMID: 36130314
PMCID: PMC10024546
DOI: 10.1093/ecco-jcc/jjac135

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.

Keywords: Multi; drug development; paediatric Crohn’s disease and ulcerative colitis; stakeholder discussion.

PubMed Disclaimer

Conflict of interest statement

AMG: Consultant to Abbvie, Amgen, BristolMyersSquibb, Janssen, Lilly, Merck, Pfizer. Speaker fees from Abbvie, Janssen, Nestle. Investigator-initiated research support from Abbvie. CP: Is an employee of the European Medicines Agency. FMR: Collaboration [advisory boards, consulting, scientific presentations] with Schering-Plough, Ferring, MSD, Johnson & Johnson, Centocor, AbbVie; Nestlé Nutrition Institute, Nestlé Health Science, Danone, MeadJohnson, Takeda, Celgene, Biogen, Bristol Myers Squibb, Gossamer, Lilly. DT: during last 3 years, has received consultation fee, research grant, royalties or honorarium from Janssen, Pfizer, Hospital for Sick Children, Ferring, AbbVie, Takeda, Atlantic Health, Shire, Celgene, Lilly, Roche, ThermoFisher, Bristol-Meyers Squibb. GV: Provided advice on paediatric oncology drug development to Astra-Zeneca, Bayer, Bristol Meyers Squib, Celgene, Hutchinson-Medi Pharma, Ipsen, Celltrion, Merck, Novartis, Pfizer, Roche/Genentech. He does not accept personal remuneration. GV is also is a board member of SIOP Europe [the European Society for Paediatric Oncology]. JSH: Advisory board for Janssen, AbbVie. Consultant to Lilly, Boehringer Ingelheim, Bristol Myers Squibb, Thetis, Takeda, Pfizer. KC: Is an employee of Janssen, UK. KN: Is an employee of the European Medicines Agency. LdR: Collaboration [such as involved in industry-sponsored studies, investigator-initiated study, consultancy] with Celltrion, AbbVie, Eli Lilly, Takeda, Pfizer. AE, FL, IL, MC-B, MG, ZG: None. NMC: Trial funding, advisory board and speakers fees paid to his institution from AbbVie, Eli Lilly, Takeda, Shire, Pfizer, Janssen. RN: Is an employee of Johnson & Johnson. TAA: Is an employee of the US Food and Drugs Administration. WC: Is an employee of Eli Llly and Company. As well as the authors declaration of interests, participants of the 2-day meeting included employees of AbbVie, AstraZeneca, Boehringer Ingelheim, Eli Lilly & Company, GSK, Janssen, Johnson & Johnson, OSE Immunoth, Pfizer, Roche Genentech and Servier.

Figures

**Figure 1.**
Issues leading to delayed paediatric approvals of innovative drugs being developed for adults with IBD.

See this image and copyright information in PMC

References

1. Johnston RD, Logan RFA.. What is the peak age for onset of IBD? Inflamm Bowel Dis 2008;14:S4–5. - PubMed
1. Benchimol EI, Bernstein CN, Bitton A, et al. The impact of inflammatory bowel disease in Canada 2018: a scientific report from the Canadian gastro-intestinal epidemiology consortium to Crohn’s and Colitis Canada. J Can Assoc Gastroenterol 2018;2:S1–5. - PMC - PubMed
1. Stulman MY, Asayag N, Focht G, et al. Epidemiology of inflammatory bowel diseases in Israel: A nationwide Epi-Israeli IBD research nucleus study. Inflamm Bowel Dis 2021. - PubMed
1. Oliveira SB, Monteiro IM.. Diagnosis and management of inflammatory bowel disease in children. BMJ 2017;357:j2083. - PMC - PubMed
1. Roberts SE, Thorne K, Thapar N, et al. A systematic review and meta-analysis of paediatric inflammatory bowel disease incidence and prevalence across Europe. J Crohns Colitis 2020;14:1119–48. - PubMed

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

Substances

Actions
Actions

Grants and funding

European Federation of Pharmaceutical Industries and Associations

LinkOut - more resources

Full Text Sources
Medical
- MedlinePlus Health Information
Miscellaneous
- NCI CPTAC Assay Portal

Save citation to file

Email citation

Add to Collections

Add to My Bibliography

Your saved search

Create a file for external citation management software

Your RSS Feed

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

Affiliations

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

Authors

Affiliations

Abstract

Conflict of interest statement

Figures

References

MeSH terms

Substances

Grants and funding

LinkOut - more resources

Full Text Sources

Medical

Miscellaneous