PEGCETACOPLAN

The PBAC did not recommend listing pegcetacoplan for treatment of complement 3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). 

The PBAC welcomed input from individuals, health professionals and organisations. The PBAC considered that there is a high clinical need for treatments for C3G and primary IC-MPGN, which are rare conditions that impact predominantly young people and cause decline in kidney function. The PBAC acknowledged that existing treatments provide only modest improvements in maintaining kidney function. The PBAC acknowledged the impact of C3G and primary IC-MPGN on individuals who are often young adults, working, and raising or starting families. The PBAC noted the impact of chronic kidney disease, relapses and the burden of treatment on their ability to participate in daily activities and their mental health. 

The PBAC reviewed the clinical evidence comparing the effectiveness and safety of pegcetacoplan with the current standard of care for both people with native kidneys and with C3G/IC-MPGN recurrence following kidney transplantation. The PBAC considered it is possible that pegcetacoplan provides a clinical benefit for some patients in terms of slowing progression to end-stage kidney disease. However, the PBAC noted the clinical benefit was only supported by evidence of improvement in indirect measures of health benefit - reduction in proteinuria (unusually high levels of protein in urine), and slower decline in eGFR (estimated glomerular filtration rate; a test that measures how well your kidneys are filtering waste from your blood) compared to placebo. The clinical evidence also showed that pegcetacoplan was less safe than standard of care, with increased risk of infection. 

The PBAC considered that the estimated benefits relating to avoiding long-term dialysis and transplant were improbable and highly uncertain due to the limited clinical data, reliance on indirect measures of health benefit, and unsupported assumptions. The PBAC considered that the cost-effectiveness of pegcetacoplan had not been established.

In the context of a substantial price reduction for pegcetacoplan, and noting the clinical need and available evidence, the PBAC considered the cost-effectiveness may be able to be assessed using a cost per responder approach. The PBAC considered that a cost per responder approach should provide interpretation, and quantification where possible, of the clinically meaningful benefits that are expected to result from reduced proteinuria and potential reduction in decline in eGFR response, for example, avoiding or delaying dialysis and transplants.  The PBAC advised that these matters could be addressed in an early re-entry submission.