The cost-effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting.

Aim: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis. We evaluated, the cost-effectiveness of pegcetacoplan, a novel proximal C3 inhibitor, versus ravulizumab in patients with PNH and hemoglobin levels <10.5 g/dl despite eculizumab treatment in the UK healthcare and social services setting. Materials & methods: A Markov cohort framework model, based on the data from the pivotal trial of pegcetacoplan (PEGASUS/NCT03500549), evaluated lifetime costs and outcomes. Patients transitioned through 3 PNH hemoglobin level/red blood cell transfusion health states. Results: Pegcetacoplan provides lower lifetime costs/greater quality-adjusted life years (£6,409,166/14.694QALYs, respectively) versus ravulizumab (£6,660,676/12.942QALYs). Conclusion: Pegcetacoplan is associated with enhanced anemia control, greater QALYs and reduced healthcare costs versus ravulizumab in the UK healthcare and social services setting.

Cost analysis of a growth guidance system compared with traditional and magnetically controlled growing rods for early-onset scoliosis: a US-based integrated health care delivery system perspective.

PURPOSE: Treating early-onset scoliosis (EOS) with traditional growing rods (TGR) is effective but requires periodic surgical lengthening, risking complications. Alternatives include magnetically controlled growing rods (MCGR) that lengthen noninvasively and the growth guidance system (GGS), which obviate the need for active, distractive lengthenings. Previous studies have reported promising clinical effectiveness for GGS; however the direct medical costs of GGS compared to TGR and MCGR have not yet been explored. METHODS: To estimate the cost of GGS compared with MCGR and TGR for EOS an economic model was developed from the perspective of a US integrated health care delivery system. Using dual-rod constructs, the model estimated the cumulative costs associated with initial implantation, rod lengthenings (TGR, MCGR), revisions due to device failure, surgical-site infections, device exchange, and final spinal fusion over a 6-year episode of care. Model parameters were from peer-reviewed, published literature. Medicare payments were used as a proxy for provider costs. Costs (2016 US$) were discounted 3% annually. RESULTS: Over a 6-year episode of care, GGS was associated with fewer invasive surgeries per patient than TGR (GGS: 3.4; TGR: 14.4) and lower cumulative costs than MCGR and TGR, saving $25,226 vs TGR. Sensitivity analyses showed that results were sensitive to changes in construct costs, rod breakage rates, months between lengthenings, and TGR lengthening setting of care. CONCLUSION: Within the model, GGS resulted in fewer invasive surgeries and deep surgical site infections than TGR, and lower cumulative costs per patient than both MCGR and TGR, over a 6-year episode of care. The analysis did not account for family disruption, pain, psychological distress, or compromised health-related quality of life associated with invasive TGR lengthenings, nor for potential patient anxiety surrounding the frequent MCGR lengthenings. Further analyses focusing strictly on current generation technologies should be considered for future research.

Paper #38: Cost analysis of a growth guidance system compared with magnetically controlled and traditional growing rods for early-onset scoliosis in the US: an integrated health care delivery system perspective.

Traditional growing rods (TGR), magnetically controlled growing rods (MCGR) and growth guidance systems (GGS) have demonstrated comparable radiographic outcomes in the treatment of early- onset scoliosis (EOS). The purpose of this study was to compare direct medical costs of GGS compared to TGR and MCGR using an economic model. Over a 6-year episode of care GGS had lower cumulative costs than MCGR and TGR, saving an estimated $25,226 vs TGR (16% decrease) and $29,916 vs MCGR (18% decrease).