Pegcetacoplan Associated With Substantial Cost Savings for Patients With Paroxysmal Nocturnal Hemoglobinuria

Results of a recent study in the Journal of Comparative Effectiveness Research suggest that pegcetacoplan is a cost-effective alternative for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in complement treatment-naive adults when compared with eculizumab and ravulizumab.

Investigators employed a de novo cost-effectiveness model that utilized a Markov cohort structure to assess lifetime (55-year) PNH costs and outcomes. The model incorporated 6-month cycles to reflect the follow-up period of the PRINCE trial (NCT04085601), an open-label study comparing pegcetacoplan with eculizumab in patients with PNH who had not received complement treatment before. Data from PRINCE were used to inform various aspects of the model, including clinical efficacy, safety profiles, and health-related quality of life outcomes.

Pegcetacoplan was associated with substantial lifetime cost savings, amounting to US $1,176,808 in comparison with eculizumab. When compared with ravulizumab, pegcetacoplan resulted in a cost savings of US $213,062. “These substantial cost savings for pegcetacoplan versus both comparators were largely attributed to reduced drug costs (−US $981,242 and −US $86,352, respectively),” the investigators wrote.

Furthermore, pegcetacoplan was associated with an additional 0.25 quality-adjusted life years (QALYs) when compared with eculizumab. When compared against ravulizumab, pegcetacoplan was associated with an additional 0.24 QALYs.

“In treatment-naïve patients with PNH, the base-case cost-effectiveness, scenario analysis, and sensitivity analysis showed both lifetime cost savings and increased QALYs associated with pegcetacoplan compared with eculizumab or ravulizumab, hence potentially offering substantial clinical value within the early PNH treatment paradigm in [the United States],” the investigators concluded.