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Background: Motor neurone disease (MND) places a significant burden on patients, their carers, and healthcare systems.Objectives: To estimate health utilities and costs of MND within the UK setting.Methods: Patients with MND, recruited via 22 regional clinics, completed a postal questionnaire of a cost and quality-of-life survey. Health outcome assessment included the EuroQoL (EQ)-5D-5L, EQ-5D-visual analogue scale, Amyotrophic Lateral Sclerosis Utility Index, and the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised. Clinical staging was based on the Kings and Milano-Torino (MiToS) systems. The questionnaire asked about patients' use of primary, secondary, and community care services in the previous 3 months. Variability in total costs was examined using regression models.Results: 595 patients were included in the health utility analysis, of whom 584 patients also completed a resource use questionnaire. Mean health utility decreased and costs increased between consecutive Kings stages, from 0.76 (95% CI 0.71-0.80) and £1096 (£757-£1240) in Kings stage 1, to 0.50 (0.45-0.54) and £3311 (£2666-£4151) in stage 4, respectively. The changes by MiToS stages were from 0.71 (0.69-0.73) and £1115 (£937-£1130) in MiToS stage 0, to 0.25 (0.07-0.42) and £2899 (£2190-£3840) in stage 2. Kings stages 3 and 4 and MiToS stages 1 and 2, respectively, were significant in explaining variability in total costs.Conclusions: The impact of MND on health utilities and costs differs by disease severity. The data provided here can be used in cost-effectiveness analyses and to inform decision-making regarding healthcare provision for people with MND. [ABSTRACT FROM AUTHOR]

Introduction and Objective: The ConcePTION project aims to improve the way medication use during pregnancy is studied. This includes exploring the possibility of developing a distributed data processing and analysis infrastructure using a common data model that could form a foundational platform for future surveillance and research. A prerequisite would be that data from various data access providers (DAPs) can be harmonised according to an agreed set of standard rules concerning the structure and content of the data. To do so, a reference framework of core data elements (CDEs) recommended for primary data studies on drug safety during pregnancy was previously developed. The aim of this study was to assess the ability of several public and private DAPs using different primary data sources focusing on multiple sclerosis, as a pilot, to map their respective data variables and definitions with the CDE recommendations framework. Methods: Four pregnancy registries (Gilenya, Novartis; Aubagio, Sanofi; the Organization of Teratology Information Specialists [OTIS]; Aubagio, Sanofi; the Dutch Pregnancy Drug Register, Lareb), two enhanced pharmacovigilance programmes (Gilenya PRIM, Novartis; MAPLE-MS, Merck Healthcare KGaA) and four Teratology Information Services (UK TIS, Jerusalem TIS, Zerifin TIS, Swiss TIS) participated in the study. The ConcePTION primary data source CDE includes 51 items covering administrative functions, the description of pregnancy, maternal medical history, maternal illnesses arising in pregnancy, delivery details, and pregnancy and infant outcomes. For each variable in the CDE, the DAPs identified whether their variables were: identical to the one mentioned in the CDE; derived; similar but with a divergent definition; or not available. Results: The majority of the DAP data variables were either directly taken (85%, n = 305/357, range 73–94% between DAPs) or derived by combining different variables (12%, n = 42/357, range 0–24% between DAPs) to conform to the CDE variables and definitions. For very few of the DAP variables, alignment with the CDE items was not possible, either because of divergent definitions (1%, n = 3/357, range 0–2% between DAPs) or because the variables were not available (2%, n = 7/357, range 0–4% between DAPs). Conclusions: Data access providers participating in this study presented a very high proportion of variables matching the CDE items, indicating that alignment of definitions and harmonisation of data analysis by different stakeholders to accelerate and strengthen pregnancy pharmacovigilance safety data analyses could be feasible. [ABSTRACT FROM AUTHOR]

Background: The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is the preferred measure of health outcome in clinical trials in motor neuron disease (MND). It, however, does not provide a preference-based health utility score required for estimating quality-adjusted life-years in economic evaluations for health technology assessments.Objectives: To develop algorithms for mapping from measures used in MND clinical studies to allow for future prediction of the five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) utility in populations of patients with MND when utility data have not been collected.Methods: Direct mapping models were developed using ordinary least squares and Tobit regression analyses to estimate EQ-5D-5L utilities (based on English tariffs), with ALSFRS-R total, domain, and item scores used as explanatory variables, using patient-level data from a UK cohort study. Indirect mapping models were also used to map EQ-5D-5L domains, using the same variables, along with the Neuropathic Pain Scale and the Hospital and Anxiety Depression Scale for MND using multinomial logistic regression analysis. Goodness of fit was assessed along with predicted values for each mapping model.Results: The best-performing model predicting EQ-5D-5L utilities used five items of the ALSFRS-R items as explanatory variables in a stepwise ordinary least squares regression. The mean squared error was 0.0228, and the mean absolute error was 0.1173. Prediction was good, with 55.4% of estimated values within 0.1 and 91.4% within 0.25 of the observed EQ-5D-5L utility value. Indirect mapping using the Neuropathic Pain Scale and the Hospital and Anxiety Depression Scale for MND provided less predictive power than direct mapping models.Conclusions: This is the first study to present mapping algorithms to crosswalk between ALSFRS-R and EQ-5D-5L. This analysis demonstrates that the ALSFRS-R can be used to estimate EQ-5D-5L utilities when they have not been collected directly within a trial. [ABSTRACT FROM AUTHOR]

What do people consciously experience when they read? There has been almost no rigorous research on this question, and opinions diverge radically among both philosophers and psychologists. We describe three studies of the phenomenology of reading and its relationship to memory of textual detail and general cognitive abilities. We find three main results. First, there is substantial variability in reports about reading experience, both within and between participants. Second, reported reading experience varies with passage type: passages with dialogue prompted increased reports of inner speech, while passages with vivid visual detail prompted increased reports of visual imagery. Third, reports of visual imagery experiences, inner speech experiences, and experiences of conscious visual perception of the words on the page were at best weakly related to general cognitive abilities and memory of visual and auditory details. [ABSTRACT FROM AUTHOR]