Limb lengthening in achondroplasia: a cost-effectiveness analysis using a Markov model

Area of Science:

• Medical Economics
• Orthopedic Surgery
• Genetics

Background:

• Achondroplasia causes disproportionate short stature, impacting patient autonomy and quality of life.
• Limb lengthening surgery offers a therapeutic option to increase stature in individuals with achondroplasia.
• The study addresses the significant limitations and poorer health-related quality of life associated with achondroplasia.

Purpose of the Study:

• To evaluate the cost-effectiveness of limb lengthening surgery in achondroplasia patients.
• To estimate the economic impact of limb lengthening on the Spanish health system.
• To analyze the health gains and costs associated with surgical intervention.

Main Methods:

• A Markov model was utilized to assess cost-effectiveness.
• Quality-adjusted life years (QALYs) were calculated to measure health gains.
• Budget impact analysis was performed based on national birth rates for achondroplasia.

Main Results:

• Limb lengthening surgery resulted in an average gain of 5.954 QALYs per patient.
• The incremental cost-effectiveness ratio (ICER) was estimated at €8,310/QALY.
• Probabilistic sensitivity analysis indicated an ICER of €7,814/QALY, with a mean QALY gain of 6.287 years.

Conclusions:

• Limb lengthening surgery for achondroplasia is a cost-effective intervention.
• The procedure aligns with common willingness-to-pay thresholds in Spain and Western countries.
• The economic impact analysis suggests a significant but justifiable cost increase for the health system.