ASSESSMENT OF THE CLINICAL AND ECONOMIC IMPACT OF DELAYED SOTATERCEPT INITIATION AS ADD-ON TO BACKGROUND THERAPY IN ADULT PATIENTS WITH SYMPTOMATIC PULMONARY ARTERIAL HYPERTENSION IN ITALY
Author(s)
Murvin Jootun, -1, Martina Paoletti, -2, Virginia Scansetti, -2, Chiara Bini, -2, Rosita van den Puttelaar, -3, Gijs van de Wetering, -3, Danilo Di Laura, -4, Paolo Sciattella, -2.
1MSD (UK) Limited, London, United Kingdom, 2Economic Evaluation and HTA (CEIS - EEHTA), University of Rome “Tor Vergata”, Rome, Italy, 3OPEN Health, Rotterdam, Netherlands, 4MSD, Rome, Italy.
1MSD (UK) Limited, London, United Kingdom, 2Economic Evaluation and HTA (CEIS - EEHTA), University of Rome “Tor Vergata”, Rome, Italy, 3OPEN Health, Rotterdam, Netherlands, 4MSD, Rome, Italy.
OBJECTIVES: Pulmonary Arterial Hypertension (PAH) is a rare and progressive disease associated with high morbidity and mortality affecting an estimated population of between 2,100 and 3,500 individuals in Italy. The phase 3 STELLAR trial evaluated the efficacy of sotatercept added to background therapy (BGT) versus placebo plus BGT, where BGT included combinations of Endothelin Receptor Antagonists, Phosphodiesterase-5 Inhibitors, soluble Guanylate Cyclase stimulators, prostacyclin analogues, and prostacyclin receptor agonists. This study aimed to compare the clinical and economic outcomes of immediate versus delayed initiation of sotatercept plus BGT in Italy.
METHODS: A societal perspective and lifetime time horizon were adopted. A six-state Markov model was developed to simulate disease progression in a hypothetical cohort of symptomatic PAH patients eligible for sotatercept in combination with BGT in Italy. Health states included four disease-risk categories, post lung/heart transplantation, and death. Transition probabilities were derived from the STELLAR clinical trial, while risk-stratified mortality data were obtained from the European PAH registry (COMPERA database). Quality-of-life inputs were based on STELLAR trial data while disability weights were derived from literature estimates using the Global Burden of Disease methodology. Direct costs included treatment acquisition costs, PAH-related hospitalization costs, lung/heart transplant costs, and disease management and end-of-life care costs. Indirect costs were estimated in terms of productivity loss for both patients and caregivers.
RESULTS: The model suggests that delaying initiation of sotatercept by two years after treatment with BGT alone, compared with immediate initiation of sotatercept at model start, is associated with a reduction in life expectancy and quality-adjusted life years, as well as an increase in disability-adjusted life years. Earlier introduction of sotatercept in combination with BGT is associated with a lower incidence of clinical events, including PAH-related hospitalizations and the need for transplantation.
CONCLUSIONS: Earlier sotatercept treatment may improve clinical outcomes.
METHODS: A societal perspective and lifetime time horizon were adopted. A six-state Markov model was developed to simulate disease progression in a hypothetical cohort of symptomatic PAH patients eligible for sotatercept in combination with BGT in Italy. Health states included four disease-risk categories, post lung/heart transplantation, and death. Transition probabilities were derived from the STELLAR clinical trial, while risk-stratified mortality data were obtained from the European PAH registry (COMPERA database). Quality-of-life inputs were based on STELLAR trial data while disability weights were derived from literature estimates using the Global Burden of Disease methodology. Direct costs included treatment acquisition costs, PAH-related hospitalization costs, lung/heart transplant costs, and disease management and end-of-life care costs. Indirect costs were estimated in terms of productivity loss for both patients and caregivers.
RESULTS: The model suggests that delaying initiation of sotatercept by two years after treatment with BGT alone, compared with immediate initiation of sotatercept at model start, is associated with a reduction in life expectancy and quality-adjusted life years, as well as an increase in disability-adjusted life years. Earlier introduction of sotatercept in combination with BGT is associated with a lower incidence of clinical events, including PAH-related hospitalizations and the need for transplantation.
CONCLUSIONS: Earlier sotatercept treatment may improve clinical outcomes.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE570
Topic
Clinical Outcomes, Economic Evaluation
Disease
Rare & Orphan Diseases