Cost-effectiveness analysis of intensive and emerging rehabilitation therapies in children with cerebral palsy: an observational cohort study using real-world evidence and microsimulation modelling
摘要
To evaluate the short- and long-term cost-effectiveness of integrating Intensive and Emerging Rehabilitation Therapies into standard care for children with cerebral palsy (CP), compared to standard care alone, through a hybrid approach combining prospective real-world data analysis and individual-level microsimulation over a lifetime horizon.
MethodsA prospective observational cohort of 148 children with CP, stratified by Gross Motor Function Classification System levels, was followed over 12 months. Short-term incremental costs and quality-adjusted life years (QALYs) were estimated using seemingly unrelated regression equations (SURE) based on EQ-5D-Y scores. Costs were assessed from the Spanish public healthcare system perspective. To extrapolate long-term outcomes, an individual-level microsimulation model projected costs and QALYs over a 30-year horizon, applying a 3% annual discount rate.
ResultsCompared with standard treatment, Therasuit and intensive physiotherapy demonstrated the most favourable cost-effectiveness profiles. Therasuit generated 0.222 additional QALYs at an incremental cost-effectiveness ratio (ICER) of €18,830/QALY, while intensive physiotherapy generated 0.216 additional QALYs at €31,772/QALY. Other therapies, including occupational therapy and hippotherapy, were dominated by standard care. Long-term microsimulation provided additional insights beyond short-term findings by capturing delayed benefits, which in some cases led to different cost-effectiveness rankings among therapies. Therasuit produced 5.49 additional QALYs at an ICER of €12,922/QALY compared to standard care, and intensive physiotherapy produced 4.92 additional QALYs at €25,789/QALY. Homeopathy and the Petö Method were cost-effective under broader willingness-to-pay thresholds but were less efficient.
ConclusionsTherasuit and intensive physiotherapy are high-value options when added to standard care for children with CP. Findings support prioritising Intensive and Emerging Rehabilitation Therapies using real-world evidence and modelling to guide sustainable healthcare decision-making.