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Exploring the benefits, harms and costs of genomic newborn screening for rare diseases
The prospect of genomic screening in newborns attracts both hype and criticism, but carefully designed, large-scale, prospective research studies are the only way to generate the data needed for its rigorous e...
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Article
Open AccessOvercoming challenges in the economic evaluation of interventions to optimise antibiotic use
Bacteria are becoming increasingly resistant to antibiotics, reducing our ability to treat infections and threatening to undermine modern health care. Optimising antibiotic use is a key element in tackling the...
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Open AccessQALYs and rare diseases: exploring the responsiveness of SF-6D, EQ-5D-5L and AQoL-8D following genomic testing for childhood and adult-onset rare genetic conditions in Australia
Genomic testing transforms the diagnosis and management of rare conditions. However, uncertainty exists on how to best measure genomic outcomes for informing healthcare priorities. Using the HTA-preferred meth...
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Open AccessImportant Considerations for Signal Detection and Evaluation
Safety clinicians have a wealth of resources describing how to perform signal detection. Nevertheless, there are some nuances concerning approaches taken by regulatory authorities and statistical consideration...
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Open AccessRetrospective file review shows limited genetic services fail most patients – an argument for the implementation of exome sequencing as a first-tier test in resource-constrained settings
Exome sequencing is recommended as a first-line investigation for patients with a developmental delay or intellectual disability. This approach has not been implemented in most resource-constraint settings, in...
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Open AccessGParareal: a time-parallel ODE solver using Gaussian process emulation
Sequential numerical methods for integrating initial value problems (IVPs) can be prohibitively expensive when high numerical accuracy is required over the entire interval of integration. One remedy is to inte...
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Open AccessFinancing and Reimbursement Models for Personalised Medicine: A Systematic Review to Identify Current Models and Future Options
The number of healthcare interventions described as ‘personalised medicine’ (PM) is increasing rapidly. As healthcare systems struggle to decide whether to fund PM innovations, it is unclear what models for fi...
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Open AccessWhat Aspects of Illness Influence Public Preferences for Healthcare Priority Setting? A Discrete Choice Experiment in the UK
Decisions on funding new healthcare technologies assume that all health improvements are valued equally. However, public reaction to health technology assessment (HTA) decisions suggests there are health attri...
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Open AccessPreferences for Medical Consultations from Online Providers: Evidence from a Discrete Choice Experiment in the United Kingdom
In the UK, consultations for prescription medicines are available via private providers such as online pharmacies. However, these providers may have lower thresholds for prescribing certain drugs. This is a pa...
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Open AccessWhy do hospital prescribers continue antibiotics when it is safe to stop? Results of a choice experiment survey
Deciding whether to discontinue antibiotics at early review is a cornerstone of hospital antimicrobial stewardship practice worldwide. In England, this approach is described in government guidance (‘Start Smar...
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A Framework for Safety Evaluation Throughout the Product Development Life-Cycle
Evaluation of the safety profile of medicines is moving from a more reactive approach, where safety experts and statisticians have been primarily focusing on the review of clinical trial data and spontaneous r...
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A Review of Health Economic Studies Comparing Traditional and Massively Parallel Sequencing Diagnostic Pathways for Suspected Genetic Disorders
Genetic disorders are clinically diverse and genetically heterogeneous, and are traditionally diagnosed based on an iterative phenotype-guided genetic assessment. However, such diagnostic approaches are long (...
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Open AccessUse of Decision Modelling in Economic Evaluations of Diagnostic Tests: An Appraisal and Review of Health Technology Assessments in the UK
Diagnostic tests play an important role in the clinical decision-making process by providing information that enables patients to be identified and stratified to the most appropriate treatment and management s...
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Open AccessA Review of the Challenges of Using Biomedical Big Data for Economic Evaluations of Precision Medicine
There is potential value in incorporating biomedical big data (BBD)—observational real-world patient-level genomic and clinical data in multiple sub-populations—into economic evaluations of precision medicine....
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Open AccessCorrection to: The Impact of Hospital Costing Methods on Cost-Effectiveness Analysis: A Case Study
The Open Access license, which previously read.
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Open AccessEvaluating the Outcomes Associated with Genomic Sequencing: A Roadmap for Future Research
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Open AccessThe Impact of Hospital Costing Methods on Cost-Effectiveness Analysis: A Case Study
Several methods exist to cost hospital contacts when estimating the cost effectiveness of a new intervention. However, the implications of choosing a particular approach remain unclear. We compare the use of t...
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Open AccessUsing Genomic Information to Guide Ibrutinib Treatment Decisions in Chronic Lymphocytic Leukaemia: A Cost-Effectiveness Analysis
Genomic tests may improve the stratification of patients to receive new therapies in several disease areas. However, the use of expensive targeted therapies can impact on the cost effectiveness of these tests....
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Open AccessErratum to: Methods for evaluating medical tests and biomarkers
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Open AccessMethods for Evaluating Medical Tests and Biomarkers