Exclusive human milk diet for very preterm babies in England: protocol for a cost-effectiveness and budget impact analysis

Introduction: Babies born before 30 weeks’ gestation are at increased risk of major clinical complications and have greater nutritional requirements. Where nutritional requirements cannot be sufficiently provided for by the mother’s own milk (MOM), routine care in England uses cow milk-derived fortifiers and formulas. However, the use of cow milk in the diets of preterm babies has been associated with adverse health outcomes. Clinical trials have shown that an exclusive human milk diet (EHMD) – where MOM is supplemented by donor human milk-derived formulas and fortifiers – has the potential to be clinically beneficial and reduce the risk of complications. Objectives: This study has two key objectives: 1) estimate the cost-effectiveness of an EHMD for babies born before 30 weeks’ gestation, relative to routine care; 2) estimate the budget impact of adopting EHMDs in practice in England. Methods : The analysis will use a modelling approach based on the most relevant data available. The population will consist of babies born in England before 30 weeks’ gestation. Babies in the intervention arm will be simulated to represent outcomes associated with babies fed an EHMD, and those in the comparator arm to receive routine care. Model parameters will be drawn from three sources: i) a recently completed randomised clinical trial, ii) the National Neonatal Research Database, and iii) published literature. The model will adopt a time horizon of two years following initial admission to a neonatal unit. The primary outcome for the cost-effectiveness analysis will be the incremental cost per life-year gained (if observed) associated with the intervention, relative to the comparator. We will also present disaggregated outcomes in a cost-consequence analysis. The primary outcome for the budget impact analysis will be the total cost associated with EHMD compared with current practice from the perspective of the English National Health Service (NHS).