HOSPITALIZATION BURDEN IN PULMONARY ARTERIAL HYPERTENSION: EVIDENCE FROM SYSTEMATIC REVIEWS OF RCTS AND RWS
Author(s)
Amélie Beaudet, MSc, PharmD1, Stacy N. Davis2, Daniela Gonçalves-Bradley, PhD3, Tanja Babic, PhD4, Moez Ahmed, MSc5;
1Janssen, Allschwill, Switzerland, 2Horsham, PA, USA, 3Stratenym Inc., Principal, Toronto, Canada, 4Stratenym Inc., Ottawa, ON, Canada, 5Stratenym Inc., Brampton, ON, Canada
1Janssen, Allschwill, Switzerland, 2Horsham, PA, USA, 3Stratenym Inc., Principal, Toronto, Canada, 4Stratenym Inc., Ottawa, ON, Canada, 5Stratenym Inc., Brampton, ON, Canada
OBJECTIVES: Pulmonary arterial hypertension (PAH) is a life‑threatening disease characterized by elevated pulmonary vascular resistance. Despite therapeutic advances, PAH treatment remains suboptimal with high hospitalization rates. Studies show that hospitalization for worsening PAH is a strong clinical marker for increased mortality and that hospitalization is associated with substantial economic burden. This study evaluates hospitalization rates reported in randomized controlled trials (RCTs) versus real‑world studies (RWS).
METHODS: Systematic literature reviews of clinical and economic evidence in PAH were performed in accordance with established methodological standards.
RESULTS: Among 29 synthesized RCTs, two reported hospitalization rates. The all-cause rates were 30-38 events and 28-42 events per 100 patient-years (PYs), in AMBITION and SERAPHIN, respectively. SERAPHIN also reported PAH-related hospitalization, which was lower at 11-22 events per 100 PYs. Rates amongst the 23 RWS reporting hospitalization (out of 114 studies identified) were mostly higher than in RCTs, although highly variable. The mean all‑cause hospitalization rate across RWS was 134 events per 100 PYs (median 106; range 20-804), while PAH‑related hospitalization averaged 69 events per 100 PYs (median 57; range 1.5-220). Heterogeneity in study design, patient populations, and clinical practice across RWS likely accounts for observed variance, with analyses varying by patient cohorts (comorbidity status, geographic location), timing (pre- vs. post-diagnosis), and treatment approach (drug combinations). Comparative evidence with non‑PAH populations was scarce; however, two studies reported significantly lower hospitalization rates among non-PAH controls, highlighting the elevated burden observed in PAH patients.
CONCLUSIONS: Hospitalization burden remains high in PAH, with rates in RWS often several-fold higher than those reported in RCTs. These findings reinforce hospitalization as a meaningful clinical indicator and highlight the urgent need for optimized treatment combinations that can reduce hospitalization rates and associated economic and clinical impact.
METHODS: Systematic literature reviews of clinical and economic evidence in PAH were performed in accordance with established methodological standards.
RESULTS: Among 29 synthesized RCTs, two reported hospitalization rates. The all-cause rates were 30-38 events and 28-42 events per 100 patient-years (PYs), in AMBITION and SERAPHIN, respectively. SERAPHIN also reported PAH-related hospitalization, which was lower at 11-22 events per 100 PYs. Rates amongst the 23 RWS reporting hospitalization (out of 114 studies identified) were mostly higher than in RCTs, although highly variable. The mean all‑cause hospitalization rate across RWS was 134 events per 100 PYs (median 106; range 20-804), while PAH‑related hospitalization averaged 69 events per 100 PYs (median 57; range 1.5-220). Heterogeneity in study design, patient populations, and clinical practice across RWS likely accounts for observed variance, with analyses varying by patient cohorts (comorbidity status, geographic location), timing (pre- vs. post-diagnosis), and treatment approach (drug combinations). Comparative evidence with non‑PAH populations was scarce; however, two studies reported significantly lower hospitalization rates among non-PAH controls, highlighting the elevated burden observed in PAH patients.
CONCLUSIONS: Hospitalization burden remains high in PAH, with rates in RWS often several-fold higher than those reported in RCTs. These findings reinforce hospitalization as a meaningful clinical indicator and highlight the urgent need for optimized treatment combinations that can reduce hospitalization rates and associated economic and clinical impact.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
CO43
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory)