Variation in access and prescription of vedolizumab and ustekinumab in paediatric patients with inflammatory bowel disease: A UK-wide study
Variation in access and prescription of vedolizumab and ustekinumab in paediatric patients with inflammatory bowel disease: A UK-wide study
Background: therapeutic options for paediatric inflammatory bowel disease (IBD) are limited, especially for younger children. Unlike in adults, vedolizumab and ustekinumab are not licensed for paediatric use in the UK. We aimed to understand the real-world access to, and use of, these therapies in the paediatric population.
Methods: we surveyed UK IBD centres to assess the incident use of vedolizumab and ustekinumab from 1 January 2021 to 31 December 2021. We collected information on funding, dose escalations and therapeutic drug monitoring.
Results: 18 of 21 centres responded, covering an estimated 5260 patients. One hundred and thirteen were started on vedolizumab, prescription incidence 2.2%, median prescriptions per centre was 4 (range 1–20). Considering ustekinumab, 73 patients were commenced, prescription incidence 1.4%. Median prescription per centre was 3.5 (range 1–13). Prescription rates at each centre were not predicted by patient number cared for at that centre (p=0.2). Dose escalation was common in vedolizumab (66.7% centres) and ustekinumab (55.5%).
Funding strategies varied substantially, and multiple funding sources were used; 12 of 18 centres (66.7%) reported funding through routine National Health Service (NHS) England/Scottish arrangements. There was local NHS trust funding in 8 of 18 centres (44.4%). Individual funding requests (IFRs) were used in 5 of 18 (27.8%), although IFRs are reserved for patients with unique additional characteristics. Four centres were unable to achieve funding in pre-pubescent children.
Conclusions: there is widespread use of vedolizumab and ustekinumab across the UK, although practice is highly variable. Access to therapy appeared to differ substantially. There is a growing disparity between international guidelines and real-world practice. Establishing early and effective therapy in all patients remains a priority.
Gastroenterology, Healthcare Disparities
994-998
Auth, Marcus Karl-Heinz
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Ashton, James John
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Jones, Kelsey D.J.
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Rodrigues, Astor
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Thangarajah, Dhamyanthi
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Devadason, David
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Lee, Gemma
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Ayaz, Mashhood
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Lee, Huey Miin
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Kammermeier, Jochen
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4 August 2023
Auth, Marcus Karl-Heinz
c7cb4b0f-8bfc-477b-9aa1-5dbcaa7fb9f7
Ashton, James John
03369017-99b5-40ae-9a43-14c98516f37d
Jones, Kelsey D.J.
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Rodrigues, Astor
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Thangarajah, Dhamyanthi
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Devadason, David
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Lee, Gemma
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Ayaz, Mashhood
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Lee, Huey Miin
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Kammermeier, Jochen
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Auth, Marcus Karl-Heinz, Ashton, James John, Jones, Kelsey D.J., Rodrigues, Astor, Thangarajah, Dhamyanthi, Devadason, David, Lee, Gemma, Ayaz, Mashhood, Lee, Huey Miin and Kammermeier, Jochen
(2023)
Variation in access and prescription of vedolizumab and ustekinumab in paediatric patients with inflammatory bowel disease: A UK-wide study.
Archives of Disease in Childhood, 108 (12), , [325236].
(doi:10.1136/archdischild-2022-325236).
Abstract
Background: therapeutic options for paediatric inflammatory bowel disease (IBD) are limited, especially for younger children. Unlike in adults, vedolizumab and ustekinumab are not licensed for paediatric use in the UK. We aimed to understand the real-world access to, and use of, these therapies in the paediatric population.
Methods: we surveyed UK IBD centres to assess the incident use of vedolizumab and ustekinumab from 1 January 2021 to 31 December 2021. We collected information on funding, dose escalations and therapeutic drug monitoring.
Results: 18 of 21 centres responded, covering an estimated 5260 patients. One hundred and thirteen were started on vedolizumab, prescription incidence 2.2%, median prescriptions per centre was 4 (range 1–20). Considering ustekinumab, 73 patients were commenced, prescription incidence 1.4%. Median prescription per centre was 3.5 (range 1–13). Prescription rates at each centre were not predicted by patient number cared for at that centre (p=0.2). Dose escalation was common in vedolizumab (66.7% centres) and ustekinumab (55.5%).
Funding strategies varied substantially, and multiple funding sources were used; 12 of 18 centres (66.7%) reported funding through routine National Health Service (NHS) England/Scottish arrangements. There was local NHS trust funding in 8 of 18 centres (44.4%). Individual funding requests (IFRs) were used in 5 of 18 (27.8%), although IFRs are reserved for patients with unique additional characteristics. Four centres were unable to achieve funding in pre-pubescent children.
Conclusions: there is widespread use of vedolizumab and ustekinumab across the UK, although practice is highly variable. Access to therapy appeared to differ substantially. There is a growing disparity between international guidelines and real-world practice. Establishing early and effective therapy in all patients remains a priority.
Text
26_06_23_Variation in access and prescription_(accepted version)
- Accepted Manuscript
More information
Accepted/In Press date: 20 July 2023
e-pub ahead of print date: 4 August 2023
Published date: 4 August 2023
Additional Information:
Funding Information:
JJA is funded by an NIHR clinical lectureship. This research received no specific grant from any funding agency in the public, commercial or not-for-profit sectors.
Publisher Copyright:
© Author(s) (or their employer(s)) 2023. No commercial re-use. See rights and permissions. Published by BMJ.
Keywords:
Gastroenterology, Healthcare Disparities
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Local EPrints ID: 483075
URI: http://eprints.soton.ac.uk/id/eprint/483075
ISSN: 0003-9888
PURE UUID: c58d86e1-89ca-4647-8601-d6b30bfd7ebd
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Date deposited: 23 Oct 2023 16:32
Last modified: 14 Aug 2024 01:53
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Contributors
Author:
Marcus Karl-Heinz Auth
Author:
Kelsey D.J. Jones
Author:
Astor Rodrigues
Author:
Dhamyanthi Thangarajah
Author:
David Devadason
Author:
Gemma Lee
Author:
Mashhood Ayaz
Author:
Huey Miin Lee
Author:
Jochen Kammermeier
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