SURMOUNT-1 demonstrated significant weight reduction and improvement in cardiometabolic risk factors with tirzepatide treatment, but data beyond 72 weeks are currently unavailable for people with overweight and obesity without T2DM. This study uses a patient-level simulation (PLS) to estimate long-term clinical outcomes with tirzepatide versus relevant comparators.

Health economic models of type 2 diabetes are increasingly being used to guide formulary decision making in the US. Models informing such decisions must be clinically credible and valid for the populations of interest. The aim of the present analysis was to evaluate the impact of using risk equations derived from different populations on modeled long-term outcomes for a US population with type 2 diabetes. Long-term projections of clinical outcomes were made for a US cohort with type 2 diabetes receiving two hypothetical interventions to reduce HbA1c and BMI (treatment A was superior to treatment B in terms of HbA1c and BMI improvements). In the first scenario, the risk of complications and mortality was modeled using equations from the BRAVO model (derived from a US population) and in a second scenario United Kingdom Prospective Diabetes Study Outcomes Model 2 (UKPDS OMS2, derived from a UK population) equations were used. All other simulation settings and parameter inputs were identical. Future clinical benefits were discounted at 3% annually. Differences in mortality were observed with incremental life expectancy of approximately 0.09 years using BRAVO equations (11.91 versus 11.82 years) compared with 0.03 years using UKPDS OM2 (12.56 versus 12.53 years). Incremental quality adjusted life expectancy estimates were 0.16 QALYs with BRAVO equations (7.52 versus 7.36 QALYs) versus 0.08 QALYs with UKPDS OM2 (7.36 versus 7.28 QALYs). Small differences between treatments were projected in the cumulative incidence of most diabetes-related complications, but overall complication rates were lower with UKPDS OM2 than with BRAVO equations. Different risk equations in an economic model of a US cohort with type 2 diabetes produces different long-term outcomes, which could have a notable influence on the cost-effectiveness of interventions.

To estimate T2DM and CV-related health care resource utilization (HCRU) and costs among patients with T2DM and cardiovascular disease (CVD; e.g., stroke) or CV risk factors (CV risk; e.g., hypertension) in the United States. Patients with ≥24 months of continuous enrollment were selected from MarketScan Commercial and Medicare databases from 1/1/2014-9/30/2018. Months 1-12 designated the baseline period and months 13-24 designated the follow-up period. Patients were required to have evidence of T2DM during the baseline period. From the overall T2DM population, two cohorts were created based on baseline diagnoses: patients with CVD (CVD cohort), and patients with CV risk but no CVD (CV risk cohort). Annual all-cause, T2DM-, and CVD-related HCRU and costs were reported during baseline. The proportion of all-cause costs attributable to T2DM and CVD were reported. The overall T2DM population included 1,106,716 patients (224,018 CVD cohort, 812,144 CV risk cohort; 70,554 neither cohort). Any hospitalization during the baseline period was higher in the CVD cohort (32.4%), compared with CV risk (6.3%) and overall T2DM (11.5%). T2DM-related (9.6%) and CVD-related (7.7%) emergency department visits were lowest in the CV risk cohort, compared to the overall T2DM cohort (11.4%; 9.7%) and CVD cohort (18.3%; 19.0%). During baseline, all-cause ($38,985), CVD-related ($16,208) and T2DM-related ($18,842) costs were highest for the CVD cohort, compared with the overall T2DM ($18,296; $7,368; $5,921) and CV risk ($13,207; $5,226; $2,754) cohorts. During baseline, 40.3%, 41.6%, and 39.6% of all-cause costs were T2DM-related and 32.4%, 48.3%, and 20.9% were CVD-related in the overall T2DM, CVD, and CV risk cohorts. Hospital admission and emergency department visits were highest among patients with CVD, compared with overall T2DM and CV risk patients. All-cause costs were also higher for CVD patients, with a greater proportion being CVD-related compared to other cohorts.

Galcanezumab-gnlm is a newly approved calcitonin gene-related (CGRP) inhibitor indicated for the prevention of migraine in adults. This study aimed to estimate the budget impact of adding galcanezumab-gnlm as a preventive treatment for patients with migraine in a US Commercial population. A budget impact model with a 1 to 5-year time horizon was developed to evaluate the financial impact of including galcanezumab-gnlm as a preventive treatment for prevention-eligible patients with migraine. The population was based on a hypothetical one million commercially insured members. The current scenario included CGRP inhibitors, antiepileptics, beta-blockers, antidepressants, and onabotulinumtoxinA (chronic migraine only). The new scenario included galcanezumab-gnlm as a new treatment, without change to market size or total predicted CGRPs uptake. Calculations relied on estimated epidemiology, market share, drug and administration costs. Deterministic sensitivity analyses were conducted, including the impact of healthcare resource use, to identify key drivers and assess uncertainties. Over a 3-year period, the analysis estimated 53,090 patients with migraine would be eligible for preventive treatment, with 0.8%, 2.40%, and 4.8% of them prescribed galcanezumab-gnlm in the first, second, and third year, respectively. Overall, the addition of galcanezumab-gnlm resulted in no change to a plan budget. The cost per member per month was estimated to remain at $1.22 for both the scenario with or without galcanezumab-gnlm. The lack of budgetary change was attributable to treatment cost parity across CGRPs over 3 years and no change in the CGRP market size with the addition of galcanezumab-gnlm. Sensitivity analyses suggested uptake and drug costs of galcanezumab-gnlm and other CGRPs were key drivers of results. When compared to a current scenario that includes CGRP use and assuming no increased market size, the introduction of galcanezumab-gnlm as a preventive treatment of migraine will not lead to an increase of a US commercial payer budget.

A phase 3 trial demonstrated superior outcomes for CPX-351 versus conventional daunorubicin/cytarabine (3+7) in patients aged 60–75 years with newly diagnosed secondary AML (high-risk AML). Understanding the economics of CPX-351 in the substantial fraction of this population aged <60 years is important. A systematic literature review (SLR) of differences in clinical outcomes in younger high-risk patients was employed, and findings were used to assess the cost-effectiveness of CPX-351 versus 3+7 among patients aged <60 years in the UK. An SLR was conducted to identify potential effects of age on clinical outcomes (response, transplant rate, overall survival, progression-free survival) of patients with 3+7 treatment. The findings from this review were incorporated into an existing economic model of CPX-351 versus 3+7 treatment for high-risk AML, assuming the effects of age applied equally regardless of treatment, to project lifetime health and cost outcomes. A survival-partition approach was used, with distinct survival curves for cohorts defined by treatment pathways (response and transplantation). Model outputs included survival, quality-adjusted life-years (QALYs), and costs. Limited evidence was identified in the literature for an effect of age. Four studies reported a difference in response rate by age, with the largest and most relevant reporting an OR of 1.9. Applying an age-dependent response rate and age-adjusted background mortality in the model resulted in a higher predicted transplant rate in the younger versus older populations (56% vs 50% among responders receiving CPX-351) and longer mean survival over the course of the model (4.1 vs 2.6 years with CPX-351). Consequently, a more favorable incremental cost-effectiveness ratio (ICER) was computed for CPX-351 versus 3+7 in patients with high-risk AML aged <60 years (£30,450/QALY) versus >60 years (£51,421/QALY). An economic model of CPX-351 versus 3+7 for high-risk AML estimates a lower ICER among younger versus older patients.