ObjectivesTo assess the economic impact of introducing into clinical practice in the UK the soluble fms‐like tyrosine kinase (sFlt‐1) to placental growth factor (PlGF) ratio test for guiding the management of pre‐eclampsia.MethodsWe used an economic model estimating the incremental value of information, from a UK National Health Service payer's perspective, generated by the sFlt‐1/PlGF ratio test, compared with current diagnostic procedures, in guiding the management of women with suspected pre‐eclampsia. The economic model estimated costs associated with the diagnosis and management of pre‐eclampsia in pregnant women between 24 + 0 and 36 + 6 weeks' gestation, managed in either a ‘test’ scenario in which the sFlt‐1/PlGF test is used in addition to current diagnostic procedures, or a ‘no‐test’ scenario in which clinical decisions are based on current diagnostic procedures alone. Test characteristics and resource use were derived from PROGNOSIS, a non‐interventional study in women presenting with clinical suspicion of pre‐eclampsia. The main outcome measure from the economic model was the cost per patient per episode of care, from first suspicion of pre‐eclampsia to birth.ResultsIntroduction of the sFlt‐1/PlGF ratio test into clinical practice is expected to result in cost savings of £344 per patient compared with a no‐test scenario. Savings are generated primarily through an improvement in diagnostic accuracy and subsequent reduction in unnecessary hospitalization.ConclusionsIntroducing the sFlt‐1/PlGF ratio test into clinical practice in the UK was shown to be cost‐saving by reducing unnecessary hospitalization of women at low risk of developing pre‐eclampsia. In addition, the test ensures that those women at higher risk are identified and managed appropriately. © 2016 Authors. Ultrasound in Obstetrics & Gynecology published by John Wiley & Sons Ltd on behalf of International Society of Ultrasound in Obstetrics and Gynecology.

BackgroundLess than one-third of patients who are estimated to be infected with multidrug-resistant tuberculosis (MDR-TB) receive MDR-TB treatment regimens, and only 48% of those who received treatment have successful outcomes. Despite current regimens, newer, more effective and cost-effective approaches to treatment are needed. The aim of the study was to project health outcomes and impact on healthcare resources of adding bedaquiline to the treatment regimen of MDR-TB in selected high burden countries: Estonia, Russia, South Africa, Peru, China, the Philippines, and India.MethodsThis study adapted an existing Markov model to estimate the health outcomes and impact on total healthcare costs of adding bedaquiline to current MDR-TB treatment regimens. A price threshold analysis was conducted to determine the price range at which bedaquiline would be cost-effective.ResultsAdding bedaquiline to the background regimen (BR) resulted in increased disability-adjusted life years (DALYs) averted, and reduced total healthcare costs (excluding treatment acquisition costs) compared with BR alone in all countries analyzed. Addition of bedaquiline to BR resulted in savings to healthcare costs compared with BR alone in all countries analyzed, with the highest impact expected in Russia (US$194 million) and South Africa (US$43 million). The price per regimen at which bedaquiline would be cost-effective ranged between US$23,904-US$203,492 in Estonia, Russia, Peru, South Africa, and China (high and upper middle-income countries) and between US$6,996-US$20,323 in the Philippines and India (lower middle-income countries); however, these cost-effective prices do not necessarily address concerns about affordability.ConclusionsAdding bedaquiline to BR provides improvements in health outcomes and reductions in healthcare costs in high MDR-TB burden countries. The range of prices per regimen for which bedaquiline would be cost-effective varied between countries.Electronic supplementary materialThe online version of this article (doi:10.1186/s12913-016-1931-3) contains supplementary material, which is available to authorized users.

Objective The objective of this study was to assess long-term survival outcomes for nivolumab and everolimus in renal cell carcinoma predicted by three model structures, a partitioned survival model (PSM) and two variations of a semi-Markov model (SMM), for use in cost-effectiveness analyses. Methods Three economic model structures were developed and populated using parametric curves fitted to patient-level data from the CheckMate 025 trial. Models consisted of three health states: progression-free, progressed disease, and death. The PSM estimated state occupancy using an area under-the-curve approach from overall survival (OS) and progression-free survival (PFS) curves. The SMMs derived transition probabilities to calculate patient flow between health states. One SMM assumed that post-progression survival (PPS) was independent of PFS duration (PPS Markov); the second SMM assumed differences in PPS based on PFS duration (PPS-PFS Markov). Results All models provide a reasonable fit to the observed OS data at 2 years. For estimating cost effectiveness, however, a more relevant comparison is between estimates of OS over the modeling horizon, because this will likely impact differences in costs and quality-adjusted life-years. Estimates of the incremental mean survival benefit of nivolumab versus everolimus over 20 years were 6.6 months (PSM), 7.6 months (PPS Markov), and 7.4 months (PPS-PFS Markov), reflecting non-trivial differences of + 14% and + 11%, respectively, compared with PSM. Conclusions The evidence from this study and previous work highlights the importance of the assumptions underlying any model structure, and the need to validate assumptions regarding survival and the application of treatment effects against what is known about the characteristics of the disease.

The cost-effectiveness of nivolumab versus docetaxel in patients with previously treated non-small cell lung cancer (NSCLC) was estimated in a cohort-based, partitioned survival model with three health states (progression-free, progressed disease, and death) and a time horizon of 15 years. The base-case model was developed using extrapolations of progression-free survival (PFS) and overall survival (OS) data from the CheckMate 017 and 057 randomized trials, and 2015 Swedish unit costs. An annual discount rate of 3% was applied. Base-case time-on-treatment was based on PFS (CheckMate 017) or time-to-treatment discontinuation (CheckMate 057), depending on whether PFS was a close proxy for time-on-treatment. Data extrapolations from CheckMate 017 and 057 were validated against external trial and registry data. Model utilities were derived from CheckMate 017 and 057 with UK weights (base-case) and Swedish weights (scenario analysis). Uncertainty was assessed using sensitivity analyses adjusted for clinical, utility, and cost data. Outcomes included incremental cost per quality-adjusted life-year (QALY) gained. The base-case model showed that nivolumab was associated with QALY gains of 0.72 (squamous) and 0.81 (non-squamous) versus docetaxel at an incremental cost of 734,573 SEK (€69,174) and 999,032 SEK (€94,078), respectively. This resulted in an incremental cost per QALY gained for nivolumab versus docetaxel of 1,013,697 SEK (€95,459) and 1,231,664 SEK (€115,985) in squamous and non-squamous NSCLC, respectively. Scenario analysis utilizing Swedish utility weights resulted in slightly lower incremental cost per QALY gained of 855,505 SEK (€80,562) (squamous) and 1,165,401 SEK (€109,745) (non-squamous). Deterministic sensitivity analysis showed that utility weights, treatment costs, discount rates, and body weight were key drivers of cost-effectiveness. Overall, the model showed that cost-effectiveness was driven by nivolumab price, but nivolumab remained cost-effective in squamous and non-squamous NSCLC in accordance with previous appraisals by the Dental and Pharmaceutical Benefits Agency (Tandvårds- och läkemedelsförmånsverket) and New Therapies Council in Sweden. Published: Online December 2019.

Aims: Nivolumab has been approved for advanced squamous and non-squamous non-small cell lung cancer (NSCLC) following platinum-based chemotherapy in both Canada and Sweden. We aimed to determine the value-for-money of nivolumab versus docetaxel in a Canadian and Swedish setting based on 5-year data. Methods: These cost effectiveness analyses used partitioned survival models with three mutually exclusive health states: progression-free, progressed disease, and death. All clinical parameters were derived from two registration phase 3 randomized trials, CheckMate 017 and CheckMate 057, with a minimum follow-up of 5 years. Treatment duration was based on time-on-treatment data from the clinical trials. Costs were derived from published sources. The primary outcomes of the analyses were quality-adjusted life-years (QALYs), life-years gained, and incremental cost-effectiveness ratios (ICERs). The model input parameters for each analysis were chosen in line with guidance from the respective HTA authorities. Results: From a Canadian payer perspective, the ICERs were CAN$140,753 per QALY in the squamous population, and CAN$173,804 per QALY in the non-squamous population, assuming a 10-year time horizon and a 5% discount rate for both costs and outcomes. Sensitivity analyses demonstrated that changes to the discount rates for outcomes had the highest impact on the ICERs. In the Swedish analysis, the ICERs were SEK568,895 per QALY in the squamous population and SEK662,991 per QALY in the non-squamous population, assuming a 15-year time horizon, a 3% discount rate, and a 2-year maximum treatment duration for nivolumab. Sensitivity analyses demonstrated that the ICERs were most sensitive to changes in the discount rate for outcomes. Conclusion: These updated analyses, based on more mature trial data with a minimum follow-up of 5 years, generate more favorable ICERs versus the previously submitted HTA assessments that resulted in approval of nivolumab for patients with previously treated NSCLC in Canada and Sweden.