Economic and Social Burden of Pulmonary Arterial Hypertension in Italy: A Cost-of-Illness Study

INTRODUCTION: Pulmonary arterial hypertension (PAH) is a rare, chronic and progressive disease with a significant clinical, social, and economic impact. Despite available therapies, none address the underlying cause but rather focus on symptom management. OBJECTIVES: This study aims to estimate the economic and social burden of PAH in Italy, including direct healthcare costs, direct nonhealthcare costs, and indirect costs related to productivity loss. METHODS: A bottom-up prevalence-based cost-of-illness model was developed using epidemiological data and healthcare resource consumption from national and international literature, validated by a panel of expert clinicians with extensive experience in the management of the condition across different regions of Italy. The analysis was conducted from a societal perspective over a 1-year horizon. The economic burden included direct healthcare costs (hospitalization, pharmaceuticals, and specialist care), direct nonhealthcare costs, and indirect costs related to productivity loss. RESULTS: In Italy, the prevalent population of patients with PAH is estimated between 2100 and 3500 individuals, with 2-5% in functional class (FC) I, 28-31% in FC II, 53-57% in FC III, and 7-11% in FC IV. The total annual expenditure for treatment and management is estimated between 263 million and 438 million euros, with 74% attributable to direct healthcare costs, 9% to direct nonhealthcare costs, and 17% to indirect costs. The mean annual cost per patient is approximately 125,000 € and increases with disease severity, ranging from 46,303 € for FC I to 252,176 € for FC IV. CONCLUSIONS: PAH has a substantial economic burden, increasing with disease severity. Early diagnosis and targeted interventions could improve patient outcomes, reduce complications, and optimize resource allocation for the National Health Service and society.