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Article
Advances in Addressing Patient Heterogeneity in Economic Evaluation: A Review of the Methods Literature
Cost-effectiveness analyses commonly use population or sample averages, which can mask key differences across subgroups and may lead to suboptimal resource allocation. Despite there being several new methods d...
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Article
Open AccessDe Novo Cost-Effectiveness Model Framework for Nonalcoholic Steatohepatitis—Modeling Approach and Validation
Nonalcoholic steatohepatitis (NASH) is a chronic liver disease associated with hepatic morbidity and mortality and extra-hepatic comorbidities. Published NASH cost-effectiveness models (CEMs) are heterogeneous...
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Article
An Investigation into the Relationship Between Choice of Model Structure and How to Adjust for Subsequent Therapies Using a Case Study in Oncology
A common challenge in health technology assessments (HTAs) of cancer treatments is how subsequent therapy use within the trial follow-up may influence cost-effectiveness model outcomes. Although overall surviv...
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Article
Open AccessIncorporating external trial data to improve survival extrapolations: a pilot study of the COU-AA-301 trial
Survival extrapolation plays a key role within cost effectiveness analysis and is often subject to substantial uncertainty. Use of external data to improve extrapolations has been identified as a key research ...
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Article
Out of Date or Best Before? A Commentary on the Relevance of Economic Evaluations Over Time
The impact of time on the applicability and relevance of historical economic evaluations can be considerable. Ignoring this may lead to the use of weak or invalid evidence to inform important research question...
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Article
Open AccessCost-Effectiveness Analysis of rFVIIIFc Versus Contemporary rFVIII Treatments for Patients with Severe Hemophilia A Without Inhibitors in the United States
A range of treatments for patients with severe hemophilia A (HA) have been developed over the last decade, allowing for reduced frequency of administration and improved outcomes (joint health and breakthrough ...
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Article
Open AccessWhat is the Impact of the Analysis Method Used for Health State Utility Values on QALYs in Oncology? A Simulation Study Comparing Progression-Based and Time-to-Death Approaches
Health state utility values (‘utilities’) are an integral part of health technology assessment. Though traditionally categorised by disease status in oncology (i.e. progression), several recent assessments hav...
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Article
Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: An Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal
The UK National Institute for Health and Care Excellence (NICE) considered evidence for voretigene neparvovec (VN; Luxturna®) for the treatment of RPE65-mediated inherited retinal dystrophies (IRD) within its hig...
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Article
Open AccessEvaluation of survival extrapolation in immuno-oncology using multiple pre-planned data cuts: learnings to aid in model selection
Due to limited duration of follow up in clinical trials of cancer treatments, estimates of lifetime survival benefits are typically derived using statistical extrapolation methods. To justify the method used, ...
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Article
Open AccessCost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data
Patients with severe hemophilia A (SHA) in Italy are routinely treated with standard half-life recombinant factor VIII (rFVIII) products. rFVIII Fc-fusion protein (rFVIIIFc) is an extended half-life rFVIII pro...
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Article
Open AccessA Review of Recent Decision-Analytic Models Used to Evaluate the Economic Value of Cancer Treatments
Cost-effectiveness analysis provides information on the potential value of new cancer treatments, which is particularly pertinent for decision makers as demand for treatment grows while healthcare budgets rema...
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Article
NICE, in Confidence: An Assessment of Redaction to Obscure Confidential Information in Single Technology Appraisals by the National Institute for Health and Care Excellence
Health technology assessment (HTA) aims to provide a transparent framework within which normative judgements can be applied for decision making. Such transparency enables the public to understand the rationale...
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Article
Open AccessCost Effectiveness of Avelumab for Metastatic Merkel Cell Carcinoma
Metastatic Merkel cell carcinoma (mMCC) is a rare and aggressive skin cancer. Until recently, there were no licensed treatment options for patients with mMCC, and prognosis was poor. A cost-effectiveness analy...
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Article
Probabilistic Sensitivity Analysis in Cost-Effectiveness Models: Determining Model Convergence in Cohort Models
Probabilistic sensitivity analysis (PSA) demonstrates the parameter uncertainty in a decision problem. The technique involves sampling parameters from their respective distributions (rather than simply using m...
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Article
Cost Effectiveness of Characterised Chondrocyte Implantation for Treatment of Cartilage Defects of the Knee in the UK
Until recently, treatment options for damage to cartilage in the knee were limited to the use of microfracture or, occasionally, mosaicplasty. The developments of autologous and characterised chondrocyte impla...
