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Abstract
While the incidence of IBD is rising, therapeutic options for paediatric IBD are limited, especially for younger children. Unlike in adults, vedolizumab and ustekinumab are not generally licenced for paediatric use in the UK. We aimed to obtain data on the real-world access to, and use of, these therapies in the paediatric population.
We surveyed all 21 designated UK IBD centres to assess the incident use of vedolizumab and ustekinumab from 1/1/2021–31/12/2021. We collected information on funding, dose escalations and therapeutic drug monitoring (TDM) in an online questionnaire.
Covering an estimated 5710 patients, 19/21 centres responded,. One-hundred-and-thirteen were started on vedolizumab (figure 1A) , prescription incidence 2%, median prescriptions per centre was 3 (range 0–20). Considering ustekinumab (figure 1B) , 74 patients were commenced, prescription incidence 1.3%. Median prescriptions per centre was 3 (range 1–13). Prescription rates at each centre were not predicted by patient number cared for at that centre (p=0.34). Dose escalation was common in vedolizumab (66.7% centres) and ustekinumab (52.6%).
Funding strategies varied substantially, and multiple funding sources were used; 13/19 centres (68.5%) reported funding through routine NHS England/Scottish arrangements. There was local NHS trust funding in 8/19 centres (42.1%). Individual funding requests (IFRs) were used in 5/19 (26.3%), although IFRs are reserved for patients with unique additional characteristics. Four centres unable to achieve funding in pre-pubescent children.
Our survey shows that 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 real-world practice/feasibility and international guidelines/benchmarking. Establishing effective and early therapy in all eligible patients remains a priority.
