Assessment of the Budgetary Impact of Introducing Pegcetacoplan and Ravulizumab for Treating Paroxysmal Nocturnal Hemoglobinuria (PNH) in the Kingdom of Saudi Arabia

OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is marked by the destruction of hematopoietic cells through complement activation and is treated with allogeneic hematopoietic stem cell transplant and complement inhibition with eculizumab. Pegcetacoplan and ravulizumab represent viable therapeutic choices for PNH. This study assessed the budget impact of introducing either pegcetacoplan for adult patients or ravulizumab for pediatric and adult PNH patients in the KSA.

METHODS: Budget impact analysis (BIA) was performed using population, market share, and cost data as inputs. Input data for KSA were collected by literature review and discussion/validation by experts. The total cost of introducing pegcetacoplan for treating adult patients or ravulizumab for pediatric and adult patients with PNH (with and without outcome-based managed entry agreement (MEA)) in comparison to eculizumab and blood transfusion over a five-year period was compared.

RESULTS: BIA showed that introducing ravulizumab will result in a decrease in expenditure as follows: pediatric patients: 25% (Saudi Riyal (SAR) 10.4 million) without MEA and 30% (SAR 12.7 million) with MEA; adult patients: 13% (SAR 16.2 million) without MEA and 17% (SAR 21.3 million) with MEA. The 5-year cumulative expenditure will be SAR 168,398,879 without ravulizumab, SAR 141,058, with ravulizumab without MEA, and SAR 133,590,053 with ravulizumab with MEA. Pegcetacoplan introduction will result in an increase in the 5-year cumulative expenditure by SAR 31.4 million without MEA and SAR 24.5 million with MEA. The 5-year cumulative expenditures without pegcetacoplan, with pegcetacoplan without MEA, and with pegcetacoplan with MEA will be SAR 17,319,715, SAR 48,747,504, and SAR 41,899,615, respectively.

CONCLUSIONS: Introducing ravulizumab for treating PNH will result in an overall decrease in budget across all age groups, while applying pegcetacoplan treatment will result in a budgetary increase. Drug acquisition cost is the primary determinant for the cumulative net budget impact of ravulizumab and pegcetacoplan in the KSA.