Background: According to the Chinese guidelines for lipid management (2023), evolocumab in combination with statins was recommended as secondary prevention of cardiovascular disease. However, because of the variation in the price of evolocumab and its different methods of confirming clinical efficacy, it was necessary to explore its economics and the impact of different methods of confirming efficacy on its economic studies.
Objective: The purpose of this paper was to assess the cost-effectiveness of evolocumab with statins versus statins alone for patients with acute myocardial infarction(AMI) in China and to investigate the impact of different clinical effectiveness modeling approaches on economic outcomes.
Methods: A Markov cohort state-transition model was used to estimate the incremental cost-effectiveness ratio (ICER) based on Chinese observational data on cardiovascular event rates, efficacy from the Asian subgroup of the FOURIER trial, cost and utility from the Chinese Yearbook of Health Statistics, health insurance data, and published studies conducted in China. This study conducted subgroup analyses for different populations and dosing regimens; sensitivity analyses for parameters such as cost, utility, and cardiovascular event rates; and scenario analyses on hospital hierarchy, time horizon, starting age, and price for statins.
Results: ICERs ranged from 27423 to 214777 Chinese yuan(CNY) per QALY gained, all below the willingness-to-pay threshold of CNY 257094. Only when the time horizon became small, the ICERs were greater than the willingness-to-pay. The probabilities that adding evolocumab to statins was cost-effective ranged from 76 to 98%. When the time horizon became small, i.e. evolocumab was discontinued before the age of 75 (after conversion), the corresponding ICERs were almost always greater than the willingness-to-pay. ICERs for modelling approaches based on clinical endpoints were 1.34 to 1.95 times higher than ICERs for modelling approaches based on reduced LDL-C levels.
Conclusions: From the Chinese healthcare and private payer perspectives, adding evolocumab to statin therapy in AMI patients is more likely to be a cost-effective treatment option at the current list price of CNY 283.8. However, evolocumab may not be cost-effective if used for shorter periods of time. The results based on different clinical effectiveness modeling approaches were significantly different.
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http://dx.doi.org/10.1186/s12962-023-00501-4 | DOI Listing |
Background: Risk-based analyses are increasingly popular for understanding heterogeneous treatment effects (HTE) in clinical trials. For time-to-event analyses, the assumption that high-risk patients benefit most on the clinically important absolute scale when hazard ratios (HRs) are constant across risk strata might not hold. Absolute treatment effects can be measured as either the risk difference (RD) at a given time point or the difference in restricted mean survival time (ΔRMST) which aligns more closely with utilitarian medical decision-making frameworks.
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Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA
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J Inherit Metab Dis
January 2025
Department of Pediatrics, Medical School, University of Minnesota, Minneapolis, Minnesota, USA.
Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal storage disorder leading to deleterious brain effects. While animal models suggested that MPS I severely affects white matter (WM), whole-brain diffusion tensor imaging (DTI) analysis was not performed due to MPS-related morphological abnormalities. 3T DTI data from 28 severe (MPS IH, treated with hematopoietic stem cell transplantation-HSCT), 16 attenuated MPS I patients (MPS IA) enrolled under the study protocol NCT01870375, and 27 healthy controls (HC) were analyzed using the free-water correction (FWC) method to resolve macrostructural partial volume effects and unravel differences in DTI metrics accounting for microstructural abnormalities.
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Children's Mercy Kansas City, Endocrinology, Kansas City, Missouri, USA.
To use electronic health record (EHR) data to develop a scalable and transferrable model to predict 6-month risk for diabetic ketoacidosis (DKA)-related hospitalization or emergency care in youth with type 1 diabetes (T1D). To achieve a sharable predictive model, we engineered features using EHR data mapped to the T1D Exchange Quality Improvement Collaborative's (T1DX-QI) data schema used by 60+ U.S.
View Article and Find Full Text PDFFood Environ Virol
January 2025
School of Environmental and Natural Sciences, Bangor University, Bangor, Gwynedd, LL57 2UW, UK.
Capsid Integrity qPCR (CI-qPCR) assays offer a promising alternative to cell culture-based infectivity assays for assessing pathogenic human virus viability in wastewater. This study compared three CI-qPCR methods: two novel (Crosslinker, TruTiter) and one established (PMAxx dye). These methods were evaluated on heat-inactivated and non-heat-inactivated 'live' viruses spiked into phosphate-buffered saline (PBS) and wastewater, as well as on viruses naturally present in wastewater samples.
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