MLD is an ultra-rare neurodegenerative disease which leads to progressive motor and cognitive decline. The GMFC-MLD is a validated measure of motor dysfunction in MLD, which represents all clinically relevant stages from normal (GMFC-MLD 0) to loss of all gross motor function (GMFC-MLD 6). Each of the seven GMFC-MLD levels are associated with healthcare costs necessary to manage the condition. This study aimed to determine and compare the average cost of care by GMFC-MLD health state across nine countries.

Delphi panel methodology was employed using a bottom-up approach to determine the total healthcare resource use (HCRU) by GMFC-MLD health state in the UK. European clinical experts also validated the HCRU. The unit costs for each item of HCRU were from a variety of sources including national databases and official tariffs specific to each country. The price power parities (PPP) ratios derived from OECD 2021 data were used to convert local currency into USD.

Clinical experts from the countries confirmed that the pathway of care does not significantly differ between countries. Consequently, the difference in cost of care between countries is driven by the local pricing and reimbursement context. HCRU included drugs, medical tests, healthcare professional visits, hospitalisations, GP/emergency visits, healthcare equipment, and social services. The average monthly medical costs across all countries for each GMFC-MLD health state from 0 through to 6 were $102, $1,592, $1,865, $2,478, $3,118, $3,453, and $9,559, respectively. The minimum and maximum monthly medical costs were observed in the Netherlands and Finland, respectively.

Monthly medical costs increase with disease severity. In the absence of claims data analysis and chart review, using Delphi panel data with the relevant clinical expert input and a bottom-up micro-costing approach could provide a useful approximation of the cost of care for MLD in the appropriate country of interest.