OBJECTIVES: Atopic dermatitis (AD) is a chronic, systemic, type-2 inflammatory disease caused by an increased production/signaling of IL-4/IL-13 associated with long-term negative effects and variable disease course. Dupilumab is the first-in-class targeted therapy (anti-IL4/IL-13) approved and reimbursed in Portugal for the treatment of children/adolescent with AD. We aimed to evaluate cost-utility of dupilumab versus best supportive care (BSC) in children/adolescents (6-17 years) with severe AD candidates for systemic therapy in Portugal.

METHODS: We developed a decision tree followed by Markov model with treatment response evaluated at week 16 and 52 (improvement of at least 50% in EASI and at least 6 points in DLQI): responders stay on treatment; non-responders discontinue to BSC. Effectiveness was derived from double-blind RCT. EQ-5D-3L trial data was converted to utilities based on Portuguese tariffs. Direct medical costs (drug acquisition/administration, disease management, adverse events) were based on literature, expert opinion and official sources. Base-case analysis assumed Portuguese NHS perspective, no cost with BSC, patients followed until 18 years-old, 4% discount rate in costs and consequences. Costs were expressed in 2021 euros. Sensitivity analyses were performed including impact on caregiver’s quality of life.

RESULTS: Over a time-horizon of up to 18 years-old, dupilumab was more effective than BSC leading to a gain of +1.28 QALY at an additional overall cost of €34,827/patient (€2,900/patient/year). Drug acquisition cost was modestly offset by less need of other healthcare resources. Incremental cost-utility ratio (ICUR) was €27,303 per QALY gained. When including impact of severe AD in caregiver’s quality of life, ICUR decreases to €21,363.

CONCLUSIONS: Our results showed that dupilumab is cost-effective versus BSC in treating severe AD in children/adolescents considering a willingness-to-pay threshold of €30,000/QALY gained. Dupilumab may free up some healthcare resources for other diseases. Impact of severe AD in caregivers reinforces cost-effectiveness of dupilumab in this young population.