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Jas Bindra,1 Ishveen Chopra,2 Kyle Hayes,3 John Niewoehner,3 Mary Prince Panaccio,3 George J Wan3 1Falcon Research Group, North Potomac, MD, USA; 2Manticore Consultancy, Bethesda, MD, USA; 3Mallinckrodt Pharmaceuticals, Bridgewater, NJ, USACorrespondence: George J Wan, Mallinckrodt Pharmaceuticals, 440 Route 22 East, Bridgewater, NJ, USA, Email george.wan@mnk.comIntroduction: 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.Keywords: Acthar® Gel, African Americans, medical chart review, sarcoidosis, treatment utilization, treatment response

Jas Bindra,1 Ishveen Chopra,2 Kyle Hayes,3 John Niewoehner,3 Mary Panaccio,3 George J Wan3 1Falcon Research Group, North Potomac, MD, USA; 2Manticore Consultancy, Bethesda, MD, USA; 3Mallinckrodt Pharmaceuticals, Bridgewater, NJ, USACorrespondence: George J Wan, Mallinckrodt Pharmaceuticals, 440 Route 22 East, Bridgewater, NJ, USA, Email george.wan@mnk.comIntroduction: 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 &dollar;134,796 per quality-adjusted life-year (QALY) and &dollar;39,179 per QALY over 2 and 3 years, respectively. From a societal perspective, Acthar Gel versus SoC results in an ICER of &dollar;117,622 per QALY and &dollar;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.Keywords: Acthar® Gel, cost-effectiveness analysis, incremental cost-effectiveness ratio, repository corticotropin injection, sarcoidosis, quality-adjusted life-years

George J Wan, John Niewoehner, Kyle Hayes Mallinckrodt Pharmaceuticals, Bridgewater, NJ, USACorrespondence: George J Wan, Mallinckrodt Pharmaceuticals, 440 Route 22 East, Bridgewater, NJ, 08807, USA, Tel +1 (908) 238-6600, Email George.Wan@mnk.comAbstract: Acthar® Gel (repository corticotropin injection [RCI]) is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides used to treat patients with serious and rare inflammatory and autoimmune conditions. This narrative review summarizes the key clinical and economic findings among 9 indications: infantile spasms (IS), multiple sclerosis (MS) relapses, rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), dermatomyositis and polymyositis (DM/PM), ocular inflammatory diseases (primarily uveitis and severe keratitis), symptomatic sarcoidosis, and proteinuria in nephrotic syndrome (NS). Key studies of clinical efficacy and healthcare resource utilization and cost from 1956 to 2022 are discussed. Evidence supports the efficacy of RCI across all 9 indications. RCI is recommended as first-line treatment for IS and is associated with improved outcomes for the other 8 indications, including increased recovery rates in MS relapse; improved disease control in RA, SLE, and DM/PM; real-world effectiveness in patients with uveitis and severe keratitis; improved lung function and reduced corticosteroid use in symptomatic sarcoidosis; and increased rates of partial remission of proteinuria in NS. For many indications, RCI may improve clinical outcomes during exacerbations or when conventional treatments have failed to show a benefit. RCI is also associated with a reduction in the use of biologics, corticosteroids, and disease-modifying antirheumatic drugs. Economic data suggest RCI is a cost-effective, value-based treatment option for MS relapse, RA, and SLE. Other economic benefits have been demonstrated for IS, MS relapses, RA, SLE, and DM/PM, including reduced hospitalizations, lengths of stay, inpatient and outpatient services, and emergency department visits. RCI is considered safe and effective and features economic benefits for numerous indications. Its ability to control relapse and disease activity makes RCI an important nonsteroid treatment option that could help preserve functioning and well-being among patients with inflammatory and autoimmune conditions.Keywords: dermatomyositis and polymyositis, multiple sclerosis relapse, nephrotic syndrome, rheumatoid arthritis, sarcoidosis, systemic lupus erythematosus

Samuel F Hunter,1 Jas Bindra,2 Ishveen Chopra,3 John Niewoehner,4 Mary P Panaccio,4 George J Wan4 1Advanced Neurosciences Institute, Franklin, TN, USA; 2Falcon Research Group, North Potomac, MD, USA; 3Manticore Consultancy, Bethesda, MD, USA; 4Mallinckrodt Pharmaceuticals, Hampton, NJ, USACorrespondence: George J WanMallinckrodt Pharmaceuticals, 53 I-78 Frontage Road, Hampton, NJ, 08827, USAEmail george.wan@mnk.comBackground: 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.Keywords: Acthar® Gel, cost-effectiveness analysis, acute exacerbation, multiple sclerosis, quality-adjusted life-year, repository corticotropin injection

Kyle Hayes,1 Mary P Panaccio,1 Parul Houston,1 John Niewoehner,1 Mohammed Fahim,2 George J Wan,1 Bhavna Dhillon3 1Mallinckrodt Pharmaceuticals, Hampton, NJ, USA; 2KMK Consulting, Morristown, NJ, USA; 3United Rheumatology, Hauppauge, NY, USACorrespondence: Kyle HayesMallinckrodt Pharmaceuticals, Hampton, NJ, USATel +1 314-393-3531Email kyle.hayes@mnk.comPurpose: Repository corticotropin injection (RCI; Acthar® Gel) is a naturally sourced mixture of adrenocorticotropic hormone analogs and other pituitary peptides that exerts anti-inflammatory and immunomodulatory properties via melanocortin receptors. RCI is approved as a short-term adjunctive therapy for rheumatoid arthritis (RA) and is typically used in patients with refractory RA. The objective of this study was to describe real-world outcomes of RA patients treated with RCI by retrospective analysis of an electronic medical records (EMR) database.Patients and Methods: EMR data were obtained from the United Rheumatology-Normal Integrated Community Evidence (UR-NICETM) data repository for patients who used RCI for the treatment of RA. Demographics, comorbidities, disease history, medications, and laboratory evaluations 365 days prior to and 365 days after initiation of RCI were examined.Results: The patient cohort was predominantly White females with a mean age of 60 years and high RA activity prior to RCI therapy. Clinical measures of disease severity indicated that patients had high RA activity before starting RCI therapy. Clinical Disease Activity Index (CDAI) scores were significantly reduced 365 days post-initiation of RCI. Swollen and tender joint counts and patient-reported outcomes, including Routine Assessment of Patient Index Data 3 (RAPID3), Physician Global Assessment, and patient assessment of pain severity were also significantly lower. The number of patients taking conventional synthetic (cs) disease-modifying antirheumatic drugs (DMARDs), biologic (b) DMARDs, nonsteroidal anti-inflammatory drugs (NSAIDS), and opioids decreased, as did the number of drugs tried within each class for csDMARDs, bDMARDs, NSAIDs, and glucocorticoids.Conclusions: These findings suggest that RCI significantly improves clinical outcomes of RA and decreases the need for concomitant medications for up to 1 year following initiation of therapy. The study provides valuable insights into the use of RCI and management of these difficult-to-treat RA patients during routine clinical practice.Keywords: Acthar Gel, DMARDs, real-world evidence, rheumatoid arthritis, repository corticotropin injection, RCI, United Rheumatology database

Jas Bindra,1 Ishveen Chopra,2 John Niewoehner,3 Mary Panaccio,3 George J Wan3 1Falcon Research Group, North Potomac, MD, USA; 2Manticore Consultancy, Bethesda, MD, USA; 3Mallinckrodt Pharmaceuticals, Hampton, NJ, USACorrespondence: George J WanMallinckrodt Pharmaceuticals, 53 Frontage Road, Hampton, NJ, 08827, USAEmail george.wan@mnk.comIntroduction: 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.Keywords: Acthar® Gel, cost-effectiveness analysis, low-disease activity, repository corticotropin injection, rheumatoid arthritis

Urbano Sbarigia,1 Tom R Denee,1 Norris G Turner,2 George J Wan,3 Alan Morrison,4 Anna S Kaufman,4 Gary Rice,5 Geoffrey M Dusheiko6,7 1Janssen Pharmaceutica, Beerse, Belgium; 2Johnson & Johnson Health Care Systems, Inc., Titusville, NJ, 3Mallinckrodt Pharmaceuticals, St. Louis, MO, USA; 4ScribCo, Effort, PA, USA, 5Diplomat Specialty Pharmacy, Flint, MI, USA, 6The University College London Medical Institute for Liver and Digestive Health, London, UK; 7Kings College Hospital, London, UK Abstract: Hepatitis C virus infection is one of the main causes of chronic liver disease worldwide. Until recently, the standard antiviral regimen for hepatitis C was a combination of an interferon derivative and ribavirin, but a plethora of new antiviral drugs is becoming available. While these new drugs have shown great efficacy in clinical trials, observational studies are needed to determine their effectiveness in clinical practice. Previous observational studies have shown that multiple factors, besides the drug regimen, affect patient outcomes in clinical practice. Here, we provide an analytical review of published outcomes studies of the management of hepatitis C virus infection. A conceptual framework defines the relationships between four categories of variables: health care system structure, patient characteristics, process-of-care, and patient outcomes. This framework can provide a starting point for outcomes studies addressing the use and effectiveness of new antiviral drug treatments. Keywords: chronic hepatitis C, humans, treatment outcome, combination drug therapy, antiviral agents