Mallinckrodt plc announced recently shared findings from Mallinckrodt's latest health economics outcomes research on Acthar Gel (repository corticotropin injection) for patients with advanced symptomatic sarcoidosis and nephrotic syndrome (NS) at the Academy of Managed Care Pharmacy (AMCP) Nexus 2023 in Orlando, FLOctober 16-19, 2023. Acthar is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides. Acthar Gel is approved by the U.S. Food and Drug Administration (FDA) for the treatment of several autoimmune disorders and medical conditions known to cause inflammation.

In a poster (D20) titled "Cost-Effectiveness of Acthar Gel versus Standard of Care for the Treatment of Advanced Symptomatic Sarcoidosis," results were presented from a cost-effectiveness analysis of Acthar Gel and the standard of care (SoC) treatments over two and three years in patients with advanced symptomatic sarcoidosis in the U.S. In this analysis, a probabilistic cohort-level state-transition approach was used. Patients were monitored at the end of a three-month cycle for a partial or complete response. Clinical parameters and health utility data were sourced from the PULSAR trial (NCT03320070) and healthcare utilization, costs, and disutilities were sourced from the published literature.

From the payer perspective ? including treatment and direct medical costs ? the findings of this analysis showed that Acthar Gel versus SoC resulted in an incremental cost-effectiveness ratio (ICER) of $134,796 per quality-adjusted life-year (QALY) and $39,179 per QALY over two and three years, respectively.

From a societal perspective ? including treatment, direct medical and indirect (caregiving, productivity loss, work-related training) costs ? Acthar versus SoC resulted in an ICER of $117,622 per QALY over two years and $21,967 per QALY over three years.

Model inputs used in the analysis were derived or extrapolated from the PULSAR clinical trial or published literature, which may not reflect real-world experience or may result in under- or over-estimation of results. The simplified care paradigm used for the model may not capture the complexity of sarcoidosis. Clinical response was based on composite sarcoidosis treatment scores, which might result in variation in cost-effectiveness estimates.

Additionally, a poster (N1) titled "Nephrotic Syndrome: Patient Characteristics, Treatment Patterns, and Related Outcomes After Treatment with Acthar Gel or Comparable Standard of Care in a Large Administrative Claims Database" detailed results from a retrospective, observational cohort study characterizing patients with NS who initiated Acthar or other therapies used after corticosteroids or calcineurin inhibitors. In this study, treatment for NS with Acthar for proteinuria and other treatments (azathioprine, chlorambucil, cyclophosphamide, mycophenolate mofetil, or rituximab) was analyzed via a large commercial claims database (Symphony Health). Patients had a confirmed diagnosis for NS, were 18 years of age or older, and had 12 months of continuous enrollment pre- and post-index.

In this analysis, after treatment with Acthar Gel, there was a significant reduction in the proportion of patients (n=315) taking corticosteroids (66%; n=209 vs. 51%; n=162, p<0.001), patients on extended use (=60 days) of corticosteroids (37%; n=117 to 25%; n=80, p<0.001), and the average daily patient dose of corticosteroids (32.1 ± 21.3 to 21.7 ± 21.1, p=0.001) compared to baseline. The Acthar Gel cohort had an increase in patients on dialysis (6%; n=20 to 14%; n=45, p<0.001), but no change was reported in renal transplants (10%; n=30 to 10%; n=32, p=0.774) or transplant complications (6%; n=18 to 6%; n=19, p=1.000).

The comparator cohort of patients taking other therapies (n=6,812) had fewer patients on dialysis (16%; n=1,105 to 12%; n=823, p<0.001), but an increase in renal transplants (19%; n=1,315 to 22%; n=1,475, p<0.001) and transplant complications (8%; n=547 to 10%; n=681, p<0.001).