OBJECTIVES: To evaluate the prevalence of primary progressive multiple sclerosis (PPMS) in Italy and to describe the healthcare utilization resources and related costs for National Health Service (NHS).

METHODS: A cross-sectional analysis of real-world data collected in the ARNO Observatory database, covering >10 million Italian inhabitants was performed. Starting from a cohort of patients affected by multiple sclerosis (MS) in the 2013 (identified through all available administrative databases), PPMS subjects were defined by the concomitant presence of the following criteria: (i) presence of MS exemption code, (ii) utilization of rehabilitation ambulatory services, (iii) no prescription of Disease-Modifying-Drugs labelled for relapsed remitting MS. For each subject the healthcare utilization in terms of drug prescriptions, outpatient services and hospitalizations was analyzed. The overall average cost per person was estimated by integrating all expenditure items for NHS.

RESULTS: Out of 14,971 patients with MS, a cohort of 941 (6.9%) subjects affected by PPMS was selected, with a prevalence of 9.1 per 100,000 inhabitants. Among these, 24.9% received baclofen, 11.3% azathioprine and 10.4% oxybutynin. Moreover, 8.1% of patients was admitted due to MS complications and 98,2% used at least an outpatient service (excluded rehabilitation therapy). PPMS patients generated an average cost of €4,283 per person. The main cost-driver was hospitalizations, accounting for 55.3% of overall expenditure (€2,370 per person), followed by outpatient services (€1,229 per person, 28.7%) and drug prescriptions (€684 per person, 16.0%).

CONCLUSIONS: This study provided real-world data of PPMS in Italy, depicting the actual burden of disease with related healthcare utilization and costs. These findings could be useful to estimate the target population of incoming therapies addressed to PPMS that, to date, represents an unmet clinical need.