OBJECTIVES: Cerebral palsy is one of the most common cause of sialorrhea, an unintentional loss of saliva from the mouth considered pathological if present after 3 years of age. Complications of sialorrhea include aspiration and recurrent pulmonary infections and have a negative impact on quality of life.

The objective is to assess the cost-effectiveness of a novel liquid formulation of glycopyrronium bromide, for the management of severe sialorrhea in children and adolescents aged 3 years and older with cerebral palsy in Poland.

METHODS: A Markov model was built considering 5 mutually exclusive heath states to perform a cost-utility analysis comparing glycopyrronium bromide with no treatment. The model consisted of a hypothetical cohort of patients transiting between severity-based health states, defined according to the Drooling Severity and Frequency Scale (DSFS), in 2-weekly cycles over 1 year.

The efficacy and distribution of patients between sialorrhea severity states were modeled using a placebo-controlled randomized trial (Zeller et al. 2012) and a single-arm clinical trial (Zanon et al. 2021). Only direct healthcare costs were considered from the public payer perspective. The primary outcome measure was the incremental cost-effectiveness ratio (ICER) representing cost per quality-adjusted life-year (QALY). Probabilistic and deterministic sensitivity analyses were conducted.

RESULTS: Compared to no treatment, glycopyrronium bromide generated 0.08 QALY at an additional cost of PLN 13,604.55. ICER was estimated at 174,524.90 PLN per QALY and was below the applicable willingness to pay threshold of PLN 190,380/QALY. The wide variety of sensitivity and scenario analyses performed showed that the cost-effectiveness results were sensitive to some of the model inputs, including baseline age, utility values and dosing.

CONCLUSIONS: The analysis showed that glycopyrronium bromide, the first therapy dedicated to children and adolescents with severe sialorrhea in cerebral palsy in Poland, is cost-effective compared to no treatment, generating additional health benefits at an acceptable therapy cost.