Acthar Gel in African Americans versus Non-African Americans with Symptomatic Sarcoidosis: Physician Assessment of Patient Medical Records.

Introduction: Sarcoidosis is common among African Americans in the United States. Acthar® Gel is a viable option for the treatment of advanced symptomatic sarcoidosis. This study examined patient characteristics, Acthar Gel utilization, co-medication use, and treatment response based on physicians' assessments among African Americans versus non-African Americans with advanced symptomatic sarcoidosis. Methods: Data from the medical charts of patients were used. During data collection, patients had either completed ≥1 course or received treatment with Acthar Gel for ≥6 months. Results: This study comprised 168 African Americans and 104 non-African Americans. On average, the time since the first diagnosis of sarcoidosis was slightly longer among African Americans than non-African Americans (5.2 versus 4.3 years). Skin, heart, eyes, and joints were the most common extrapulmonary sites involved among both race groups. Shortness of breath, fatigue, bone and joint pain, and wheezing/coughing were the most frequent symptoms among both race groups. A higher proportion of African Americans versus non-African Americans were first-time Acthar Gel users and had not completed treatment during data collection. Patients in both race groups with higher starting doses of Acthar Gel therapy had a shorter treatment duration and vice-versa. A significantly lower proportion of patients among both race groups were on any co-medication after Acthar Gel initiation (p<0.0001). Further, a higher proportion of African Americans versus non-African Americans had a reduction in any co-medication use after Acthar Gel initiation. The mean daily dose of prednisone decreased among African Americans (18.5 to 10.1 mg) and non-African Americans (17.6 to 10.0 mg) after Acthar Gel initiation. Improvement in patient health status and overall symptoms was similar for both race groups. Conclusion: Findings suggest that Acthar Gel improves health outcomes for patients with sarcoidosis, which could help to alleviate health disparities among African Americans, who are disproportionately affected by this disease.

Cost-Effectiveness of Acthar Gel versus Standard of Care for the Treatment of Advanced Symptomatic Sarcoidosis.

Introduction: Sarcoidosis is a multisystem, inflammatory, systemic granulomatous disease with unknown etiology. Despite the current standard of care (SoC), there is an unmet need for the treatment of advanced symptomatic sarcoidosis. This study assessed the cost-effectiveness of Acthar® Gel (repository corticotropin injection) versus SoC in patients with advanced symptomatic sarcoidosis from the United States (US) payer and societal perspectives over 2 and 3 years. Methods: A probabilistic cohort-level state-transition approach was used for this cost-effectiveness analysis. Patients were monitored at the end of a 3-month cycle for the attainment of partial or complete response. Patients in the partial, complete, or no-response state were allowed to transition in each of these states at each 3-month cycle. Following the attainment of response, patients could have a durable response or relapse to a no-response state. Patients in a no-response state received treatment and could transition into a response or no-response state based on the probability of treatment success with the respective treatment. Clinical parameters and health utility data were sourced from the Acthar Gel in Participants with Pulmonary Sarcoidosis (PULSAR) trial (NCT03320070) and healthcare utilization, costs, and disutilities were sourced from the published literature. Base case analysis considered a payer perspective over 2 years. Results: From a payer perspective, Acthar Gel versus SoC results in an incremental cost-effectiveness ratio (ICER) of $134,796 per quality-adjusted life-year (QALY) and $39,179 per QALY over 2 and 3 years, respectively. From a societal perspective, Acthar Gel versus SoC results in an ICER of $117,622 per QALY and $21,967 per QALY over 2 and 3 years, respectively. Sensitivity analysis findings were consistent with the base case. Conclusion: The results from this cost-effectiveness analysis indicate that Acthar Gel is a cost-effective, value-based treatment option for advanced symptomatic sarcoidosis compared to the SoC from the US payer and societal perspectives.

Treatment-Related Cost Analysis of Terlipressin for Adults with Hepatorenal Syndrome with Rapid Reduction in Kidney Function.

INTRODUCTION: Hepatorenal syndrome (HRS), a special form of acute kidney failure, is a rare, acute, life-threatening complication of cirrhosis and has a very poor prognosis. Terlipressin (TERLIVAZ®) is the first and only pharmacological treatment approved by Food and Drug Administration (September 2022) to improve kidney function for adults with HRS with rapid reduction in kidney function. We constructed a decision analytic economic model to estimate the cost per complete response/HRS reversal of terlipressin + albumin from a United States hospital perspective. METHODS: A decision analytic model was developed to estimate the HRS treatment-related cost per response over an HRS hospitalization (assuming 14 days). Patients can experience either HRS reversal (complete response) or no HRS reversal (partial/no response) upon receipt of treatment. The efficacy, safety, and treatment duration data were from published head-to-head randomized international trials. Total treatment cost comprised drug acquisition and treatment-related costs (intensive care unit [ICU], dialysis [intermittent or continuous], pulse oximetry monitoring for terlipressin, and adverse events) sourced from the published literature. Cost per response, defined as the total treatment cost per HRS reversal was estimated for each treatment. The number needed to treat (NNT), defined as the number of patients treated to achieve HRS reversal in 1 additional patient, was estimated. RESULTS: Cost per response of terlipressin + albumin was lower than midodrine and octreotide + albumin (M&O) (US$85,315 vs. $467,794) and norepinephrine + albumin ($81,614 vs. $139,324). NNT for HRS reversal was 2 patients with terlipressin + albumin vs. M&O + albumin and 4 patients with terlipressin + albumin vs. norepinephrine + albumin, respectively. CONCLUSIONS: The analysis shows that terlipressin is a cost-effective treatment due to its higher efficacy and administration in the non-ICU setting. Terlipressin is a value-based treatment option for appropriate adults with HRS with rapid reduction in kidney function.

Hepatorenal syndrome, a functional, progressive kidney failure, is a life-threatening complication of cirrhosis. It is important to improve kidney function in patients who are hospitalized with hepatorenal syndrome considering the cost of treatment. This study assessed the cost per complete response/ hepatorenal syndrome reversal of terlipressin + albumin from a United States hospital perspective. This study shows that terlipressin improves kidney function with lower intensive care unit and dialysis costs compared with unapproved treatments. Terlipressin is a cost-effective, value-based treatment option for appropriate adults with hepatorenal syndrome with rapid reduction in kidney function.

Cost-Effectiveness of Acthar Gel Versus Standard of Care for the Treatment of Exacerbations in Moderate-to-Severe Systemic Lupus Erythematosus.

INTRODUCTION: Despite current standard of care (SoC), there is an unmet need for the treatment of active systemic lupus erythematosus (SLE). The study assessed the cost-effectiveness of Acthar® Gel (repository corticotropin injection) versus SoC treatment in patients with active, moderate-to-severe SLE from the US payer and societal perspectives over 2 and 3 years. METHODS: Cost-effectiveness model was developed using a probabilistic cohort-level state-transition approach. Patients received Acthar Gel in an exacerbation state, and the outcomes were assessed at the end of a 3-month cycle for response achievement based on the probability of treatment success with Acthar Gel. Patients may sustain the response or experience an exacerbation. For the base case scenario, moderate-to-severe SLE was defined as British Isles Lupus Assessment Group (BILAG)-2004 ≥ 20 or SLE Disease Activity Index 2000 (SLEDAI-2K) ≥ 10 and clinical response was based on SLE responder index (SRI)-4. Clinical response, productivity loss, and utility were derived from a phase 4 SLE trial; cost and disutility estimates were sourced from the literature. RESULTS: From a payer perspective, Acthar Gel versus SoC resulted in an incremental cost-effectiveness ratio (ICER) of $133,110 per quality-adjusted life-year (QALY) and $94,818 per QALY over 2 and 3 years, respectively. From a societal perspective, Acthar Gel versus SoC results in an ICER of $70,827 per QALY and $32,525 per QALY over 2 and 3 years, respectively. Results from the sensitivity and scenario analyses are consistent with those of the base case model. CONCLUSIONS: Acthar Gel is a cost-effective, value-based treatment option for appropriate patients with moderate-to-severe SLE at a willingness-to-pay threshold of $150,000 over 2-3 years from the US payer and societal perspectives. Acthar Gel results in the reduction of direct medical and indirect costs.

Understanding Predictors of Response to Repository Corticotropin Injection Treatment Among Patients With Advanced Symptomatic Sarcoidosis.

Background: Sarcoidosis, an inflammatory systemic granulomatous disease, affects multiple organs and has a diverse clinical course. Repository corticotropin injection (RCI) is an effective treatment for advanced symptomatic sarcoidosis. Since sarcoidosis affects patients differently, treatment response may vary by patient demographic, clinical, and treatment-related characteristics and physician specialty. However, there is a paucity of literature regarding predictors of sarcoidosis treatment response. Objectives: This study investigated predictors of response to RCI treatment. Methods: Post-hoc analysis was conducted using data from a previously published retrospective cross-sectional chart review study among symptomatic sarcoidosis patients ≥18 years of age previously treated with RCI. Outcome improvement 3 months post-RCI treatment was based on the clinician's subjective evaluation and analyzed using adjusted logistic regression. The most influential predictors for each outcome were based on statistical significance (P<.05) and the strength of the relationship assessed by the standardized ß coefficients. Results: The top predictors of outcome improvements were as follows. Global health assessment: (1) improvement in current health status influenced by complete RCI compliance, moderate overall symptom severity, and presence of extrapulmonary sites; and (2) improvement in overall symptoms influenced by age, shorter duration since sarcoidosis diagnosis, and complete RCI compliance. Clinical outcomes: (1) lung function improvement influenced by mild weight loss, mild wheezing/coughing, and non-African American race; (2) reduction in pulmonary fibrosis influenced by moderate overall symptom severity, mild wheezing/coughing, and mild weight loss; and (3) reduction in inflammation influenced by physician specialty, completing a course of RCI treatment, and moderate-to-severe night sweats. Patient-related outcomes: (1) reduction in fatigue influenced by physician specialty and moderate-to-severe fatigue; and (2) improvement in quality-of-life influenced by shorter duration since sarcoidosis diagnosis, moderate-to-severe wheezing/coughing, and complete RCI compliance. Corticosteroid discontinuation/reduction was influenced by physician specialty, moderate-to-severe shortness of breath, and comedication use before RCI. Conclusions: RCI may be a better treatment option for patients with more severe disease, primarily those presenting with symptoms. Complete compliance with RCI treatment may improve patients' health and quality of life. Understanding factors that influence RCI effectiveness across different treatment outcomes in real-world clinical practice is important for designing optimal sarcoidosis treatment strategies.

Cost-Effectiveness of Repository Corticotropin Injection for the Treatment of Acute Exacerbations in Multiple Sclerosis.

BACKGROUND: Relapses are common among patients with multiple sclerosis (MS) despite treatment with disease-modifying therapies. Repository corticotropin injection (RCI, Acthar® Gel), plasmapheresis (PMP), and intravenous immunoglobulin (IVIg) are alternative therapies for MS relapse. There is a dearth of economic assessments of these therapies for the acute exacerbations of MS. This study estimated the cost-effectiveness of RCI compared to PMP or IVIg. METHODS: A Markov state-transition model compared outcomes (costs, relapses, remission, and utilities) with RCI versus PMP or IVIg for the acute exacerbations in MS. The model was developed from the United States (US) payer and societal perspectives over one to three years. Patients initiated on alternative therapies were evaluated in one-day increments for the first 30 days during treatment. The model assumes the natural history of MS after treatment in the first month, adjusting for the effect of treatment. Incremental cost-effectiveness ratios (ICERs) were estimated as cost per quality-adjusted life-year (QALY) gained. The uncertainty in model parameters was evaluated in probabilistic sensitivity analyses. RESULTS: In the base case, RCI has an ICER of USD 42,078 per QALY compared to PMP over one year from the payer perspective and is dominant over two and three years; RCI is dominant compared to PMP from the societal perspective over all three years. Compared to IVIg, RCI is a dominant strategy from both payer and societal perspectives over all three years. Probabilistic sensitivity analysis supports the base case findings, suggesting that RCI may be cost-effective versus PMP and IVIg for acute exacerbations in MS. CONCLUSION: RCI is a cost-effective alternative treatment for MS relapses compared to PMP and IVIg from the US payer and societal perspectives.

Cost-Effectiveness of Repository Corticotropin Injection versus Standard of Care for the Treatment of Active Rheumatoid Arthritis.

INTRODUCTION: Patients with active rheumatoid arthritis (RA) often have inadequately controlled symptoms and are unable to achieve remission or low disease activity despite aggressive treatment. This results in irreversible joint damage, adversely affecting patients' physical and social functioning. The objective was to estimate the cost-effectiveness of repository corticotropin injection (RCI) versus standard of care (SoC) in patients with active RA from the United States (US) payer and societal perspectives over two to three years. METHODS: An individual-level microsimulation was developed to generate individual trajectories for patients with RA, using data from a published Phase 4 trial of RCI. These trajectories report a patient's disease pathway and associated cost and quality-of-life outcomes. The incremental cost-effectiveness ratio (ICER) of RCI versus SoC was assessed using the literature-derived direct medical and indirect costs, and quality-adjusted life-years (QALY) derived from a Phase 4 trial of RCI. The uncertainty in base case estimates of the parameters was evaluated in the sensitivity analysis. RESULTS: Over two years, RCI has an incremental QALY gain of 1.591 and incremental cost of $183,965 and $117,443 from payer and societal perspective, respectively, resulting in an ICER of $115,629/QALY and $73,817/QALY compared to SoC. Over three years, RCI has an incremental QALY gain of 2.336 and incremental cost of $202,315 and $104,506 from payer and societal perspective, respectively, resulting in an ICER of $86,607/QALY and $44,737/QALY compared to SoC. Results from the probabilistic sensitivity analysis are consistent with those of the base case model. CONCLUSION: RCI is a cost-effective strategy for patients with persistently active RA who are previously treated with disease-modifying anti-rheumatic drugs and corticosteroids compared to SoC over two to three years from the payer and societal perspectives at a US willingness-to-pay threshold of $150,000/QALY. Further, the economic benefit of RCI is realized with improvement in a patient's clinical and health outcomes.