Cost analysis of the impact of caplacizumab in the treatment of acquired thrombotic thrombocytopenic purpura from a US hospital perspective.

AIM: This study aimed to model the financial impact of caplacizumab with therapeutic plasma exchange (TPE) + immunosuppression for patients experiencing an acute acquired thrombotic thrombocytopenic purpura (aTTP) episode versus TPE + immunosuppression, from a US hospital's perspective. METHODS AND MATERIALS: We developed an economic model to estimate the impact of caplacizumab on a US hospital's budget. Cost offsets from caplacizumab utilization targeted inpatient general ward days, intensive care unit (ICU) days, and TPE utilization. Costs and event probabilities were estimated from primary data analyses of the phase 3 HERCULES trial and peer-reviewed literature or other public sources. Plan reimbursement was obtained from 2019 Medicare Fee Schedules and adjusted to represent reimbursement from different US payers. Cost of ICU and general ward utilization were estimated from Medicare Provider Analysis and Review data analyses capturing hospital discharges. RESULTS: The model results indicate that caplacizumab leads to hospitalization cost savings of over $8,000 ($23,148 versus $14,904) along with TPE cost savings of over $14,000 ($37,150 versus $23,033) per patient. When the cost of caplacizumab and plan reimbursement are incorporated into the results, the per-patient cost of TPE + immunosuppression is $23,120 versus $70,068 for caplacizumab with TPE + immunosuppression, an incremental cost of $46,948. The model was robust to several scenario analyses; however, when limited to Medicare fee-for-service (FFS), the incremental cost of caplacizumab per patient was reduced to $4,852 due to add-on payments. CONCLUSIONS: Caplacizumab with TPE + immunosuppression is associated with an increase in costs; however, the increase is nominal among payers who provide an add-on payment consistent with that of Medicare FFS.

Incremental burden of mental health conditions in adult patients with focal seizures.

INTRODUCTION: Clinical research has consistently established mental health conditions (MHCs) as frequent comorbidities of epilepsy. However, the extent of economic burden of comorbid MHC in patients with focal seizures has not been systematically investigated. This retrospective cohort analysis of health plan claims compared healthcare use and costs among adult patients with focal seizures with and without comorbid MHC. METHODS: We utilized the Inovalon Medical Outcomes Research for Effectiveness and Economics (MORE2) Registry, longitudinal data from over 150 commercial, Medicare Advantage, and managed Medicaid health plans for the analysis, and identified a cohort of patients with focal (partial-onset) seizure with relevant ICD9/10 diagnosis codes with and without MHC. Mental health conditions were defined as diagnoses for anxiety, bipolar condition/mania, attention-deficit conduct condition, major depression, schizophrenia, and other psychotic conditions, and patients without MHC were propensity score-matched to patients with preexisting MHC on baseline patient characteristics. The assessment examined a series of outcomes, including 1) direct healthcare resource utilization and 2) total provider reimbursement. RESULTS: Patients with preexisting MHC were more likely to receive adjunctive epilepsy therapy as well as broad-spectrum antiepileptic drugs/antiseizure medications (ASMs). Additionally, patients with focal seizures and MHC were significantly more likely to utilize high-cost healthcare services. The presence of MHC was associated with approximately 50% greater utilization of emergency department (ED), physician, and inpatient services. Consequently, healthcare expenditures were significantly higher among patients with MHC ($17,596 vs. $10,857; 62% higher, p < 0.001), with the trend consistent across all care settings. CONCLUSIONS: This analysis illustrates the health service utilization and cost implications of MHC among patients with focal seizures. The data suggest that patients with MHC have a greater overall clinical burden, which may be associated with higher healthcare resource use and expenditures. Because of the potential burden and costs associated with MHC, neurologists should consider screening patients with focal seizures for mental health disorders to identify and initiate treatment for comorbid mental health disorders.

A ready-to-use liquid glucagon for treatment of severe hypoglycemia demonstrates reduced healthcare payer costs in a budget impact model.

Research aim: To model the annual value of a novel ready-to-use, room-temperature stable liquid glucagon rescue pen and prefilled syringe (GRP, G-PFS; Xeris Pharmaceuticals, Inc.) for treatment of severe hypoglycemia events (SHE) versus current lyophilized powder glucagon emergency kits (GEK). GRP is a prefilled auto-injector designed to promptly administer concentrated liquid glucagon in a simple two-step process. G-PFS is a stable liquid formulation of glucagon in a prefilled syringe. In simulated emergencies, GRP and G-PFS demonstrated high functional efficacy, where 99% of users successfully administered a full-dose of drug. Studies with currently available injectable GEK suggest very low success rates (6-31%). The high functional efficacy of GRP and G-PFS significantly reduces user errors and may reduce utilization across emergency medical services (EMS), emergency departments (ED), and inpatient and outpatient costs for SHE.Methods: To estimate the economic impact of GRP and G-PFS, we developed a one-year budget impact model from a US commercial health plan perspective. Cost offsets from successful glucagon administration incorporated EMS, ED, inpatient, and outpatient utilization. Diabetes prevalence and event probabilities were estimated from publicly-available sources and clinical expert opinion. Costs (US$) were obtained from the 2018 Medicare Fee Schedules and adjusted to represent commercial payer costs.Results: GRP and G-PFS led to fewer EMS, ED, inpatient, and outpatient costs compared to GEK and no kit, resulting in total per-patient SHE costs of $2,564, $3,606, and $3,849, respectively. Costs for 1 million covered lives were 8.2 million following the introduction of GRP and G-PFS compared to almost 9 million before GRP and G-PFS.Limitations: The model is limited by reliance on assumptions based on expert opinion for key variables, primarily the probability of: (1) ambulance calls, (2) ambulance transport to the ED, and (3) non-ambulance transport to the ED.Conclusions: A budget impact model suggests GRP and G-PFS can lead to significant annual cost savings for US commercial payers.

Controlling for drug dose in systematic review and meta-analysis: a case study of the effect of antidepressant dose.

PURPOSE: To describe a method for quantitatively dealing with drug dose in comparative effectiveness reviews. Second-generation antidepressants are used as an example to illustrate this method and to determine whether dose influences conclusions on comparative effectiveness. METHODS: Studies previously identified in a systematic review of second-generation antidepressants were included if data on drug dose were available. The usual dosing range for each drug was defined and then used to create 2- and 3-level dose categories. Placebo-controlled data were used to calculate overall effect sizes for the drug class and effect sizes stratified by drug dose. Meta-regression tested the impact of dose on effect size. Weighted mean differences and risk ratios were calculated for comparative studies, stratifying by whether compared doses were equivalent. RESULTS: The dose classification method was able to identify dose-response trends in the context of meta-analysis. Compared to low-dose studies, medium- and high-dose studies had a 1- to 2-point greater differential in mean Hamilton Depression Rating Scale (HAM-D) change (P < 0:001). Dose was not a statistically significant predictor of categorical HAM-D response. Among comparative trials with nonequivalent doses, trends favored higher dose categories but generally were not statistically significant. CONCLUSIONS: A structured method for quantitatively dealing with drug dose in comparative effectiveness reviews is described, with application to the second-generation antidepressants. Dose-dependent reductions in HAM-D scores were identified, although differences did not translate into better response rates for higher doses. Dose equivalency was not a significant factor among comparative studies in second-generation antidepressants.