OBJECTIVES: Health economic evaluations for paediatric diseases are often characterised by high uncertainty, in part due to small study sizes, the lack of long-term clinical effectiveness data, age-specific utility values and accurate indirect costs. This study sought to review how economic evaluations of paediatric diseases were received by NICE in the last 5 years.

METHODS: We identified medicines for paediatric populations assessed by NICE between June 2018 and June 2023. Publicly available NICE appraisal documents were screened with regards to decision outcome, alignment with licensed indication, utility values, ICER, and any other uncertainties around modelling inputs and outcomes.

RESULTS: In total, 37 published technology appraisals of treatments with a paediatric indication were identified. The majority (26/37) were for indications in mixed/adult populations. All 37 medicines were recommended for use, though only 6 within their marketing authorisation. Approximately one third (13/37) of the recommended medicines were above the cost-effective threshold for use in the NHS. Nonetheless, additional factors and arrangements not easily captured by modelling were considered in order to alleviate uncertainties in estimates.

CONCLUSIONS: High uncertainty surrounding the cost-effectiveness of paediatric treatments assessed by NICE is often mitigated by a wide range of factors such as rarity and severity of the disease as well as benefits such as the effect on the quality of life of siblings of the patient, which are not easily captured by health economic evaluations.