RÉSUMÉ
BACKGROUND: Neonatal respiratory distress syndrome (RDS) is one of the most common problems for preterm infants, and symptoms include tachypnoea, grunting, retractions and cyanosis, which occur immediately after birth. Treatment with surfactants has reduced morbidity and mortality rates associated with neonatal RDS. OBJECTIVE: The objective of this review is to describe the treatment costs, healthcare resource utilization (HCRU) and economic evaluations of surfactant use in the treatment of neonates with RDS. METHODS: A systematic literature review (SLR) was performed to identify available economic evaluations and costs associated with neonatal RDS. Electronic searches were conducted in Embase, MEDLINE, MEDLINE In-Process, NHS EED, DARE and HTAD to identify studies published between 2011 and 2021. Supplementary searches of reference lists, conference proceedings, websites of global health technology assessment bodies and other relevant sources were conducted. Publications were screened by two independent reviewers for inclusion and followed the population, interventions, comparators and outcomes framework eligibility criteria. Quality assessment of the identified studies was performed. RESULTS: Eight publications included in this SLR met all eligibility criteria: three conference abstracts and five peer-reviewed original research articles. Four of these publications evaluated costs/HCRU, and five (three abstracts and two peer-reviewed articles) investigated economic evaluations (two from Russia, and one each from Italy, Spain and England). The main cost drivers and causes of increased HCRU were invasive ventilation, duration of hospitalization and RDS-associated complications. There were no significant differences in neonatal intensive care unit (NICU) length of stay or NICU total costs between infants treated with beractant (Survanta®), calfactant (Infasurf®) or poractant alfa (Curosurf®). However, treatment with poractant alfa was associated with reduced total costs compared with no treatment, continuous positive airway pressure (CPAP) alone or calsurf (Kelisu®), due to shorter duration of hospitalization and fewer complications. Early use of the surfactant after birth was more clinically effective and cost-effective than late intervention in infants with RDS. Poractant alfa was found to be cost-effective and cost-saving compared to beractant for the treatment of neonatal RDS in two Russian studies. CONCLUSION: There were no significant differences in NICU length of stay or NICU total costs between surfactants evaluated for treating neonates with RDS. However, early use of surfactant was found to be more clinically effective and cost-effective than late treatment. Treatment with poractant alfa was found to be cost-effective versus beractant and cost-saving compared with CPAP alone or beractant or CPAP in combination with calsurf. Limitations included the small number of studies, the geographic scope of the studies and the retrospective study design of the cost-effectiveness studies.
RÉSUMÉ
BACKGROUND: In patients with asthma that is uncontrolled by a medium- or high-dose inhaled corticosteroid (ICS) plus long-acting ß2-agonist (LABA), a maintenance therapy option is the addition of a long-acting muscarinic agonist, either via multiple inhalers, or single-inhaler triple therapy (SITT). One SITT is the extrafine formulation of beclometasone dipropionate/formoterol fumarate/glycopyrronium (BDP/FF/G). We used data from two 52-week clinical trials (TRIMARAN and TRIGGER), both conducted in adults with asthma uncontrolled by ICS/LABA, to investigate the cost-effectiveness of BDP/FF/G. METHODS: A Markov cohort state transition model (focusing on exacerbations) was used to investigate the cost-effectiveness of medium- or high-dose BDP/FF/G vs medium- or high-dose BDP/FF, and high-dose BDP/FF/G vs high-dose BDP/FF + tiotropium. The model analysed cost, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER), and was developed from the England National Health Service perspective (2020 costs). Uncertainty of the inputs was estimated using one-way and probabilistic sensitivity analyses. RESULTS: Both medium- and high-dose BDP/FF/G were cost-effective vs BDP/FF, with ICERs of £12,224 and £15,587 per QALY gained. High-dose BDP/FF/G was dominant vs BDP/FF + tiotropium, as it was both cheaper and gained QALYs. Sensitivity analyses were consistent with the base model: medium- and high-dose BDP/FF/G had 94.3% and 88.3% likelihoods to be cost-effective vs BDP/FF; high-dose BDP/FF/G had 100% likelihood to be a dominant strategy vs BDP/FF + tiotropium. CONCLUSIONS: Both medium- and high-dose BDP/FF/G were cost-effective vs medium- and high-dose BDP/FF in adults with asthma that was uncontrolled by ICS/LABA. In addition, high-dose BDP/FF/G was a dominating strategy to high-dose BDP/FF + tiotropium. CLINICALTRIALS: GOV: NCT02676076 and NCT02676089.