Broadening cost-effectiveness analysis beyond the traditional payer perspective had a significant impact on the result and should be considered in order to capture all treatment benefits and costs of societal relevance.

Introduction: Mobility impairments have substantial physical and mental health consequences, resulting in diminished quality of life. Whereas most studies on the health economic consequences of mobility limitations focus on short-term implications, this study examines the long-term value of improving mobility in older adults. Methods: Our six-step approach used clinical trial data to calibrate mobility improvements and estimate health economic outcomes using a microsimulation model. First, we measured improvement in steps per day calibrated with clinical trial data examining hylan G-F20 viscosupplementation treatment. Second, we created a cohort of osteoarthritis patients aged ≥51. In the third step, we estimated their baseline quality of life (QoL). Fourth, we translated steps per day improvements to changes in QoL using estimates from the literature. Fifth, we calibrated QoL in this cohort to match those in the trial. Lastly, we incorporated these data and parameters into The Health Economic Medical Innovation Simulation (THEMIS) model to estimate how mobility improvements affect functional status limitations, medical expenditures, nursing home utilization, employment, and earnings between 2012 and 2030. Results: In our sample of 12.6 million patients, 66.7% were female and 70% had BMI>25 kg/m2. Our model predicted that a 554-steps-per-day increase in mobility would reduce functional status limitations by 5.9%, total medical expenditures by 0.9%, and nursing home utilization by 2.8%, and increase employment by 2.9%, earnings by 10.3% and monetized QoL by 3.2% over this 18-year period. Conclusions: Interventions that improve mobility are likely to reduce long-run medical expenditures and nursing home utilization, and increase employment.

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Aim: This study examines how chimeric antigen receptor T-cell (CAR-T) therapy’s incremental effectiveness and cost–effectiveness profile fits into the recent history of anticancer treatments. Materials & methods: We conducted graphical and multivariable analyses using data from the Cost–Effectiveness Analysis Registry of the Tufts Medical Center and the Institute for Clinical and Economic Review’s analysis of CAR-T therapies. We collected additional information including the US FDA approval years for pharmacologic innovations. Results: CAR-T provided 5.03 (95% CI: 3.88–6.18) more incremental quality-adjusted life-years than the average pharmaceutical intervention and 4.61 (95% CI: 1.67–7.56) more than the average nonpharmaceutical intervention, while retaining similar cost–effectiveness. There was evidence of worsening cost–effectiveness by approval year for pharmaceutical interventions. Limitations: Analysis is limited to anticancer treatments studied in cost–utility analyses, estimated to cover approximately 60% of FDA-approved antineoplastic agents. Conclusion: CAR-T therapy breaks a pattern of stagnant efficacy growth in pharmaceutical innovation and demonstrates significantly greater incremental effectiveness and similar cost–effectiveness to prior innovations.

Aims: Model how moving from current disease-modifying drug (DMD) prescribing patterns for relapsing-remitting multiple sclerosis (RRMS) observed in the United Kingdom (UK) to prescribing patterns based on patient preferences would impact health outcomes over time. Materials and methods: A cohort-based Markov model was used to measure the effect of DMDs on long-term health outcomes for individuals with RRMS. Data from a discrete choice experiment were used to estimate the market shares of DMDs based on patient preferences (i.e. preference shares). These preference shares and real-world UK market shares were used to calculate the effect of prescribing behavior on relapses, disability progression, and quality-adjusted life-years (QALYs). The incremental benefit of patient-centered prescribing over current practices for the UK RRMS population was then estimated; scenario and sensitivity analyses were also conducted. Results: Compared to current prescribing practices, when UK patients with RRMS were treated following patient preferences, health outcomes were improved. This population was expected to experience 501,690 relapses and gain 1,003,263 discounted QALYs over 50 years under patient-centered prescribing practices compared to 538,417 relapses and 958,792 discounted QALYs under current practices (À6.8% and þ4.6%, respectively). Additionally, less disability progression was observed when prescribed treatment was based on patient preferences. In a scenario analysis where only oral treatments were considered, the results were similar, although the magnitude of benefit was smaller. Number of relapses was most sensitive to how the annualized relapse rate was modeled; disability progression was most sensitive to mortality rate assumptions. Limitations: Treatment efficacy estimates applied to various models in this study were based on data derived from clinical trials, rather than real-world data; the impact of patient-centered prescribing on treatment adherence and/or switching was not modeled. Conclusions: The population of UK RRMS patients may experience overall health gains if patient preferences are better incorporated into prescribing practices.