Pegcetacoplan Found to Be Cost-Effective Approach to Treating PNH

Pegcetacoplan is a cost-effective option for producing a good to complete response to paroxysmal nocturnal hemoglobinuria (PNH) treatment in patients who had suboptimal responses to eculizumab, according to a study published in Hematology Reports.

PNH is a rare blood disorder that causes fatigue, thrombosis, hemolytic anemia, and peripheral blood cytopenias and needs lifelong treatment. Eculizumab, ravulizumab, and pegcetacoplan have been approved for the treatment of PNH, so clinicians must weigh the costs and outcomes of each medicine when choosing among them. Because pegcetacoplan had been approved as a replacement for patients who did not respond to eculizumab, this study aimed to determine average cost per responder who had used eculizumab before switching to pegcetacoplan.

Red blood cells circulating in the blood vessels | Image credit: Design Cells - stock.adobe.com

European Society for Blood and Marrow Transplantation (EBMT) response categorization analyses of the PEGASUS, PADDOCK, PALOMINO, and PRINCE clinical trials were used to collect post hoc data on costs per responder. REDBOOK was used to collect drug and administration costs. The PEGASUS trial included adults with PNH who had had suboptimal responses to eculizumab treatment. Patients were randomized to monotherapy with eculizumab or pegcetacoplan for 16 weeks after a run-in period of 4 weeks. Patients then had an open-label period where those who received eculizumab in the first 16 weeks could receive a mix of eculizumab and pegcetacoplan for 4 weeks before switching to solely pecgetacoplan through the remainder of the study.

Researchers separated the patients into groups of complete response, major response, good response, partial response, minor response, and no response. Costs were calculated from the beginning of the trial through week 16, which included the run-in period. This included average drug costs per patient through 16 weeks, average drug costs per patient per week, average drug costs per responder, and percentage of drug costs in total in patients who had partial or no response or had discontinued or missing status.

There were 41 patients who used pegcetacoplan and 39 who used eculizumab in the PEGASUS trial. The average drug cost per patient was $186,411 for pegcetacoplan and $177,313 for eculizumab, including the run-in costs of eculizumab. Patients with a complete response to pegcetacoplan paid an average of $477,679 over 16 weeks; this was not calculated for eculizumab as no patients had a complete response to the treatment. Complete responders in pegcetacoplan had a number needed to treat (NNT) of 2.6, with the average drug cost per responder and NNT much lower in pegcetacoplan compared with eculizumab when responses were good and good to complete. Incremental cost of pegcetacoplan was $23,315 for each additional complete response.

The total cost of pegcetacoplan was $2.0 million for patients with partial to no response in 16 weeks compared with $6.5 million for patients who used eculizumab in those 16 weeks. A total of 27% of the total drug costs for patients who had partial to no response in the pegcetacoplan group whereas 95% of the total drug costs for eculizumab were for patients with partial to no response.

Average cost per responder was lower with pegcetacoplan for good to complete responders and for partial to no responders compared with eculizumab. The average drug cost per good to complete response was lower at week 48 ($15,459 per week) compared with week 16 ($15,923) in patients with suboptimal response to eculizumab who switched.

There were some limitations to this study. Ravulizumab was not included in this study due to lack of data. The population was small due to the rarity of PNH. Other treatment-related costs outside of the third-party payer were not accounted for in this analysis, including lost productivity due to PNH, and the costs of switching from ravulizumab to pegcetacoplan were also not accounted for. Data for drug dosage and treatment response were not available. These results are not generalizable to patients who are naïve to eculizumab, and the prices came from publicly available wholesale acquisition costs rather than health care providers.

The researchers concluded that switching from eculizumab to pegcetacoplan is cost-effective and can improve responses in patients with PNH who do not have optimal responses to eculizumab. These results apply to patients who had suboptimal results with eculizumab alone, which invites future research in other demographics.

Reference

Fishman J, Anderson S, Talbird SE, Dingli D. Analysis of costs per responder in US adults with paroxysmal nocturnal hemoglobinuria with a suboptimal response to prior eculizumab treatment. Hematol Rep. 2023;15:578-591. doi:10.3390/hematolrep15040060