Patel Minal, Shergill-Bonner Rita, Sharma Preeti, Capobianco Maria Elisa, Steeds Carolyn
Barts Health NHS Trust, London, UK.
Evelina London Children's Hospital, London, UK.
J Med Econ. 2025 Dec;28(1):1214-1225. doi: 10.1080/13696998.2025.2539644. Epub 2025 Aug 1.
This study assessed the cost-effectiveness (CE) of a specialised formula versus standard oral nutritional supplements (ONS) for the dietary management of paediatric mild-to-moderate Crohn's disease (CD), from the UK National Health Service (NHS) perspective.
We developed a Markov model with five health states (remission, mild-to-moderate, moderate-to-severe, surgery and death), using 8-week cycles and a one-year horizon. A systematic literature review identified treatment effectiveness (clinical remission rates) and health state utilities (Child Health Utility-9 Dimension). Costs were sourced from the British National Formulary and National Tariff Payment System (2024 prices) and included costs of intervention and healthcare resources such as primary and outpatient care, diagnostic tests, accident and emergency care and hospitalisations. Clinical experts validated the model structure and inputs. Deterministic and probabilistic sensitivity analyses (DSA and PSA) assessed the robustness of findings.
The base case results demonstrated that specialised formula saves £591 per patient annually while maintaining similar quality-adjusted life-years (QALYs) compared to standard ONS. Additionally, in a cohort of 1,000 patients, 552 would achieve remission with the specialised formula compared to 496 with standard ONS. DSA identified the 8-week intervention costs and utility scores as key drivers of CE. PSA indicated that at a willingness-to-pay threshold of £20,000 per QALY, specialised formula has a 78% likelihood of being CE compared with standard ONS.
The main limitation is the lack of published clinical trials comparing specialised formula with standard ONS, along with the overall scarcity of published data on standard ONS. Meta-analysis would be beneficial to validate and strengthen the comparative data; however, due to the limitations of the published data, meta-analysis is not possible at present.
Specialised formula is a less costly and more effective treatment compared to standard ONS in mild-to-moderate paediatric CD patients over one year.
本研究从英国国家医疗服务体系（NHS）的角度，评估了特殊配方奶粉与标准口服营养补充剂（ONS）在小儿轻至中度克罗恩病（CD）饮食管理中的成本效益（CE）。
我们使用8周的周期和一年的时间范围，开发了一个具有五个健康状态（缓解、轻至中度、中至重度、手术和死亡）的马尔可夫模型。系统的文献综述确定了治疗效果（临床缓解率）和健康状态效用（儿童健康效用-9维度）。成本来自英国国家处方集和国家收费支付系统（2024年价格），包括干预措施和医疗资源的成本，如初级和门诊护理、诊断测试、急诊护理和住院治疗。临床专家对模型结构和输入进行了验证。确定性和概率敏感性分析（DSA和PSA）评估了研究结果的稳健性。
基础案例结果表明，与标准ONS相比，特殊配方奶粉每年可为每位患者节省591英镑，同时维持相似的质量调整生命年（QALY）。此外，在1000名患者的队列中，特殊配方奶粉组有552人将实现缓解，而标准ONS组为496人。DSA确定8周干预成本和效用评分是CE的关键驱动因素。PSA表明，在每QALY支付意愿阈值为20000英镑时，与标准ONS相比，特殊配方奶粉具有78%的成本效益可能性。
主要局限性是缺乏比较特殊配方奶粉与标准ONS的已发表临床试验，以及关于标准ONS的已发表数据总体稀缺。荟萃分析将有助于验证和加强比较数据；然而，由于已发表数据的局限性，目前无法进行荟萃分析。
在小儿轻至中度CD患者中，特殊配方奶粉在一年时间内相比标准ONS是成本更低且更有效的治疗方法。
