Hospital mangers need information for decision making (1). Hospital-based health technology assessment (HTA) methods were tested out to support the budget planning of investments for a new building to be constructed for diagnostic and teaching units at a publicly funded tertiary care university hospital. The hospital board nominated an ad hoc working group to reassess all investment proposals for devices, equipment and furniture for the diagnostic or teaching units that intended to move into the new building. The need for assessment was obliged when the submitted proposals of the units exceeded two-fold the initially allocated investment budget.

Depending on the level of expenditure, all proposals were assessed by one of the following processes: (i) Proposals over EUR250,000 were evaluated by three to five person expert groups using multi-domain assessment adapting Hospital-based HTA-principles; (ii) Proposals between EUR50,000 and EUR250,000 were returned to the units for miniHTA-assessments by clinicians who submitted the initial proposals and (iii) All proposals below EUR50,000 were prioritized by the units to cut the expenditure by at least 25 percent, with a special emphasis on synergistic use of devices and equipment among the units.

The expert groups suggested significant reductions to the proposals, including the withdrawal of a Magnetic Resonance Imaging (MRI)-unit considered to be suboptimally located. Furthermore, the need for a new scanner was declined by promoting adherence to evidence-based diagnostic guidelines and more efficient utilization of existing scanners. Self-assessed MiniHTAs revealed proposals that were unnecessary or the specifications for devices needed re-adjustments. Prioritization revealed excess numbers of devices, for instance the number of cold storage appliances could be reduced. Altogether, the investment proposals were cut by over EUR3.8 million to reach the initial budgetary allocation.

Innovative and flexible usage of hospital-based HTA methodology can be applied to budget planning and evaluation of investment proposals to support decision making. Based on encouraging results, hospital-based HTA was accepted to become a part of hospital strategy as a tool for the annual investment planning.

To summarize the key methodological challenges identified by health technology assessment (HTA) agencies assessing gene therapy (GT) and consideration of broad elements of value.

Economic evaluations (EEs) of voretigene neparvovec (VN) in RPE65-mediated inherited retinal disease (IRD) published in English were selected. HTA evaluations from Australia, Canada, Ireland, Scotland, England, and the United States were reviewed. An existing methodological framework was used to identify the challenges and considerations.

Eight unique EEs were identified of which six were evaluated by HTA agencies. Incremental cost-effectiveness ratios ranged from $68,951 to $643,813 per quality-adjusted life-years (QALY) gained (healthcare perspective) and dominant to $480,130 per QALY gained (societal perspective). The key challenges were the lack of validated surrogate outcome, utility values and indirect costs from IRD patients, and limited evidence of the long-term treatment effect. Two HTA agencies reviewed a range of novel broader elements of value and whether they were associated with VN while other agencies discussed some elements of broader value. Caregiver disutility was included in some, but not all, evaluations.

The methodological challenges were consistent with innovative interventions for rare diseases and managed using standard methods. Broader value was important to decision-makers but inconsistently applied across agencies. Possible reasons are limitations in the evidence available of the broader benefits that VN offers and how to incorporate these within an EE. A need exists for greater guidance and consistency across jurisdictions regarding the consideration of broader value that considers latest best practice.

Principal component analysis (PCA) is used for dimension reduction and data summary. However, principal components (PCs) cannot be easily interpreted. To interpret PCs, this study compares two methods to approximate PCs. One uses the PCA loadings to understand how input variables are projected to PCs. The other uses forward-stepwise regression to determine the proportions of PC variances explained by input variables.

Two data sets derived from the Canadian Health Measures Survey (CHMS) were used to test the concept of PC approximation: a spirometry subset with the measures from the first trial of spirometry; and, full data set that contained representative variables. Variables were centered and scaled. PCA were conducted with 282 and twenty-three variables respectively. PCs were approximated with two methods.

The first PC (PC1) could explain 12.1 percent and 50.3 percent of total variances in respective data sets. The leading variables explained 89.6 percent and 79.0 percent of the variances of PC1 in respective data sets. It required one and two variables to explain more than 80 percent of the variances of PC1, respectively. Measures related to physical development were the leading variables to approximate PC1 and lung function variables were leading to approximate PC2 in the full data set. The leading variable to approximate PC1 of the spirometry subset were forced expiratory volume (FEV) 0.5/forced vital capacity (FVC) (percent) and FEV1/FVC (percent).

Approximating PCs with input variables were highly feasible and helpful for the interpretation of PCs, especially for the first PCs. This method is also useful to identify major or unique sources of variances in data sets. The variables related to physical development are the variables related to the most variations in the full data set. The leading variable in the spirometry subset, FEV0.5/FVC (percent), is not well studied for its application in clinical use.

Music therapy (MT) is a complementary creative arts treatment aimed at maintaining, restoring, and furthering physical, emotional, and mental health. This systematic review aimed to assess the effectiveness of MT for the treatment of autism spectrum disorder, dementia, depression, insomnia, and schizophrenia. In addition, the MT methods used for these indications were analyzed.

For this update of five Cochrane reviews, four databases (Medline, Embase, The Cochrane Library, and PsycINFO) were systematically searched for studies published from 2013 to 2020. Two review authors independently performed the study selection and data extraction. The methodological quality of the included trials was assessed using the Risk of Bias in Non-randomised Studies - of Interventions (ROBINS-I) tool and the Cochrane Risk of Bias tool for randomized controlled trials.

Ten RCTs (1,248 patients) met the inclusion criteria. For schizophrenia, no study could be included. MT improved the following: behavior, social communication, and the parent-child relationship in patients with autism; mood for patients with depression; and sleep quality for patients with insomnia. In patients with dementia, MT enhanced mood, behavior (severe disease stage), and cognitive function, whereas cognition was unchanged. Memory was improved only in the mild disease stage. None of the studies observed any significant long-term effects of MT in these patient groups. Both active (playing music) and receptive (listening to music) methods were used for dementia, whereas active methods were applied for autism spectrum disorder and depression. For insomnia, only receptive methods were used.

The findings of this update of reviews provides evidence that MT may help patients diagnosed with an autism spectrum disorder, dementia, depression, insomnia, or schizophrenia. It is crucial to focus on patient-related evidence-based health care. MT improves physical, psychological, and social aspects, but more research investigating the long-term effects of MT in these patient groups is needed as it is crucial to know how long the effects of MT last.

Patient-reported outcome measures (PROMs) provide a way to measure the impact of a disease and its associated treatments on the quality of life from the patients’ perspective. The aim of this review was to identify PROMs that have been developed and/or validated in patients with carotid artery disease (CAD) undergoing revascularization, and to assess their psychometric properties and examine suitability for research and clinical use.

Eight electronic databases including MEDLINE and CINAHL were searched from inception to May 2015 and updated in the MEDLINE database to February 2017. A two-stage search approach was used to identify studies reporting the development and/or validation of relevant PROMs in patients with CAD undergoing revascularization. Supplementary citation searching and hand-searching reference lists of included studies were also undertaken. The Consensus-based standards for the selection of health measurement instruments (COSMIN) and Oxford criteria were used to assess the methodological quality of the included studies, and the psychometric properties of the PROMs were evaluated using established assessment criteria.

Six PROMs, reported in five studies, were identified: 36-Item Short Form Health Survey (SF-36), Euro-QoL-5-Dimension Scale (EQ-5D), Hospital Anxiety and Depression Scale (HADS), Dizziness Handicap Inventory (DHI), Quality of life for CAD scale by Ivanova 2015 and a disease-specific PROM designed by Stolker 2010. The rigour of the psychometric assessment of the PROMs were variable with most only attempting to assess a single psychometric criterion. No study reported evidence on criterion validity and test-retest reliability. The overall psychometric evaluation of all included PROMs was rated as poor.

This review highlighted a lack of evidence in validated PROMs used for patients undergoing carotid artery revascularization. As a result, the development and validation of a new PROM for this patient population is warranted in order to provide data which can supplement traditional clinical outcomes (stroke >30 days post-procedural, myocardial infarction and death), and capture changes in health status and quality of life in patients to help inform treatment decisions.

Qualitative methods allow in-depth exploration of patient experiences and can provide context for healthcare decision making. Frameworks for patient-based evidence in health technology assessment (HTA) are expanding; yet, how extensively qualitative methods are currently used is unclear. This review characterized the extent and quality of qualitative data submitted to National Institute for Health and Care Excellence (NICE) and Canadian Agency for Drugs and Technologies in Health (CADTH) for HTA.

NICE and CADTH submissions from September 2019 to August 2021 were reviewed. Submission characteristics and features of patient-based evidence included within submissions were extracted. The quality of qualitative reporting was assessed using the CASP checklist.

Patient-based evidence was included in 83/107 NICE and 119/124 CADTH submissions. A small proportion described qualitative data collection (NICE=14; CADTH=24) and analysis (NICE=6; CADTH=9) methods. One-to-one interviews were the most common data collection method, and thematic analysis was exclusively used. Thirty-three percent of NICE submissions scored >7 yes responses on CASP, versus 78 percent of CADTH submissions.

Although patient-based evidence was common in the submissions reviewed, only 14/107 NICE and 24/124 CADTH submissions involved formal qualitative data collection. Use of formal analysis methods was even rarer and reporting tended to be brief. At present, there is little guidance about qualitative evidence most likely to be informative and therefore to potentially impact decision making. Ensuring, however, that qualitative data are collected and analyzed in a systematic, rigorous way will maximize their usefulness and ensure that patient voices are clearly heard.

Ningún sistema de salud cuenta con los recursos necesarios para evaluar todas las tecnologías. Contar con un proceso claro para priorizar qué tecnologías serán evaluadas por las agencias de evaluación de tecnologías sanitarias (ETESA) constituye un principio de buena práctica reconocido a nivel internacional. El objetivo del Foro de Políticas en Latino América (LatamPF) 2020 de Health Technology Assessment International fue explorar cómo puede mejorarse la forma en que las agencias de ETESA de Latino América identifican y priorizan las tecnologías a ser evaluadas.

Este manuscrito está basado en un documento base, una encuesta, y en el trabajo deliberativo realizado por los miembros (cuarenta y seis participantes, once países) que participaron del LatamPF, a través de la metodología design-thinking.

Los participantes coincidieron en que la falta de mecanismos claros de priorización trae como consecuencia una falta de legitimidad de las decisiones y procesos de ETESA, que son percibidos como poco transparentes y demasiado expuestos a presiones políticas o de grupos de interés. También se identificaron barreras y acciones para mejorar los mecanismos de priorización de ETESA en América Latina. Los criterios identificados como más importantes para ser tenidos en cuenta por las agencias de ETESA de la región al momento de priorizar una tecnología para ser evaluada fueron la carga de enfermedad, el potencial beneficio clínico, la alineación con prioridades de salud nacionales, el potencial impacto en la equidad, ausencia de otras alternativas para los pacientes, y el potencial impacto económico.

Los participantes del Foro coincidieron en que el establecimiento de procesos transparentes de priorización es un elemento clave para todos los sistemas de salud. Las mejoras en este proceso fortalecerán la ETESA en Latino América y darán mayor legitimidad a sus decisiones.

Health Technology Assessment (HTA) aims at providing decision makers with relevant data, matching different perspectives, with an evidence-based approach. The most common framework used is the European Network for Health Technology Assessment (EUnetHTA) Core Model (1): HTA may be further supported by a Multi-Criteria Decision Analysis (MCDA) (2,3), leading to a final quantitative synthesis, facilitating the appraisal phase.

This project presents a multi-dimensional comparison of the technologies available for the treatment of diabetic macular edema (Ranibizumab, Aflibercept, Dexamethasone implant and off-label Bevacizumab), comparing three Italian Regions: Lombardy, Liguria and Veneto.

The nine EUnetHTA dimensions were first prioritized by seventeen multidisciplinary evaluators. Thereafter a further nine professionals attributed a 3-level rating score (from “1” not performant, to “3” most performant) to each dimension and sub-dimension, after carefully assessing the three HTA reports. In conclusion, the investigation of statistically significant differences between the attributed scores of the evaluators was conducted, using a multi-variate analysis.

No statistically significant differences were reported in the prioritization of each dimension, except for the equity (more important in Liguria and in Lombardy) and the economic financial dimensions (more relevant in Veneto and in Lombardy).

Notwithstanding the evaluators’ different professional titles, job roles, center size, and various Regional contexts, they attributed similar scores to the HTA dimensions during the appraisal phase (even though conducted in different years, in 2015 and 2016). This finding demonstrates the robustness of both the evaluations and the final MCDA results: i) no statistically inter-regional significant differences emerged regarding Ranibizumab and Aflibercept (p-value >.05); ii) no statistically significant inter-regional differences emerged regarding Dexamethasone, except for the assessments in the clinical dimensions (p-value = .026), since in Lombardy Region the evaluation was carried out earlier in the technology's life-cycle.

Dexamethasone was consistently attributed a higher total score, considering the final normalised weight derived from the MCDA approach (p-value =.001).