OBJECTIVES: Estimate the budget impact to a United States (US) health plan formulary of introducing pegcetacoplan, a FDA approved C3 inhibitor administered subcutaneously for paroxysmal nocturnal hemoglobinuria (PNH) treatment in adults who 1) are currently treated with the standard of care, intravenous C5 inhibitors (C5i) eculizumab or ravulizumab or 2) are treatment naïve.
METHODS: A budget-impact model was developed using a 3-year time horizon and health plan perspective. Forecasted market shares were based on existing C5i market shares and treatment responses. The pegcetacoplan-eligible population size was estimated from the literature. The model compared the current scenario using C5i to a scenario introducing pegcetacoplan to a health plan’s formulary. The model included costs for drug-acquisition, administration, transfusion, breakthrough hemolysis (BTH), vaccines, and antibiotics, but excluded physician visits and supportive treatments which were assumed equal across treatments. Annualized transfusion and BTH rates were derived from the head-to-head Phase 3 PEGASUS trial (NCT03500549) for pegcetacoplan and eculizumab. Total budget impact, transfusion avoidance, and BTH events avoided were reported annually and cumulatively over three years in 2020 US dollars. Scenario analyses were conducted on pegcetacoplan uptake, transfusion rates, BTH rates and costs, and drug costs.
RESULTS: For a 10-million-member US health plan, 43 adults in year 1 and seven additional adults each subsequent year were eligible for pegcetacoplan treatment. In all three years, introducing pegcetacoplan reduced costs, with a cumulative budget impact of −$1.7 million, and reduced transfusions (−167) and BTH events (−19). All scenario analyses found introducing pegcetacoplan was cost-saving (range, −$1.2 to −$2.2 million). Results were most sensitive to drug-acquisition costs, market share, and percentage of patients requiring higher doses or increased dose frequencies.
CONCLUSIONS: Adding pegcetacoplan to a health plan’s formulary to treat adults with PNH is estimated to lower healthcare costs by reducing costs for drug-acquisition, administration, transfusion, and BTH.
