J Crohns Colitis. 2022 Sep 21;jjac135. doi: 10.1093/ecco-jcc/jjac135. Online ahead of print.

Nicholas M Croft ¹, Lissy de Ridder ², Anne M Griffiths ³, Jeffrey S Hyams ⁴, Frank M Ruemmele ^5 6, Dan Turner ⁷, Katharine Cheng ⁸, Irja Lutsar ⁹, Marco Greco ¹⁰, Zuzanna Golebiewska ¹¹, Floriane Laumond ¹², Maria Cavaller-Bellaubi ¹³, Adam Elgreey ¹⁴, Tara A Altepeter ¹⁵, Chrissi Pallidis ¹⁶, Koen Norga ¹⁷, Robert Nelson ⁸, Wallace Crandall ¹⁸, Gilles Vassal ¹⁹

Author information

¹Blizard Institute, Barts and the London School of Medicine, Queen Mary University of London, London, UK.

²Department of Pediatric Gastroenterology, Erasmus MC Sophia Children's Hospital, Rotterdam, The Netherlands.

³Inflammatory Bowel Disease Centre, The Hospital for Sick Children, University of Toronto, Canada.

⁴Connecticut Children's Medical Center, Hartford, Connecticut, United States.

⁵Université de Paris, Faculté de Médecine, Paris, France.

⁶Assistance Publique-Hôpitaux de Paris, Hôpital Universitaire Necker Enfants Malades, Service de Gastroentérologie pédiatrique, Paris, France.

⁷Shaare Zedek Medical Center, the Hebrew University of Jerusalem, Jerusalem, Israel.

⁸Johnson and Johnson, New Brunswick, United States.

⁹Institute of Medical Microbiology, University of Tartu, Tartu, Estonia.

¹⁰European Patients' Forum (EPF), Chaussée d'Etterbeek, Brussels, Belgium.

¹¹J-elita, Polish Society for the Support of People with Inflammatory Bowel Disease, Warsaw, Poland.

¹²Aupetit François Association (AFA) Crohn RCH, Paris, France.

¹³EURORDIS-Rare Diseases Europe, Paris, France.

¹⁴Crohn and Colitis Foundation Israel (CCFI), Tel Aviv, Israel.

¹⁵Division of Gastroenterology, Office of Immunology and Inflammation, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.

¹⁶Paediatric Medicines, European Medicines Agency, Netherlands.

¹⁷Paediatric Committee (PDCO), European Medicines Agency, Netherlands.

¹⁸Pediatric Immunology, Eli Lilly and Company, Indianapolis, Indiana, United States.

¹⁹Department of Pediatric and Adolescent Oncology, Gustave Roussy Comprehensive Cancer Center and Paris Saclay University, Villejuif, France.

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. There are many potential new therapies being developed for adult and paediatric inflammatory bowel disease. Moreover, regulatory agencies in both European Union and United States 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 seven-year gap, or longer, between authorisation of new inflammatory bowel disease drugs for adults and children.

Methods: A 2-day virtual meeting was held April 14-15, 2021 for multi-stakeholders (clinical academics, patient community, pharmaceutical companies, and regulators) to discuss their perspectives on paediatric drug development for inflammatory bowel disease.

Results: The multi-stakeholder group presented, discussed and proposed actions to achieve expediting the approval of new drugs in development for paediatric inflammatory bowel disease.

Conclusions: Collaborative action points for all stakeholders are required to make progress and facilitate new drug development for children with inflammatory bowel disease.