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1.

Objectives

To test the hypothesis that the “severity effect”—the preference for more than utility-maximizing expenditure on severe health states—may be the result of, or exacerbated by, the uncertainty associated with the chance of contracting the illness that causes the severe health state.

Methods

Survey respondents were asked to imagine that they will contract one of two illnesses and asked to allocate a budget between two insurance policies, each of which provides services for the treatment of one of the illnesses. A person’s final health state varied with the amount of insurance purchased for the illness that occurred and therefore with the level of treatment. The relative cost of the two policies was altered and the selected levels of insurance compared with the levels that would be provided by a health authority that sought to maximize total utility or quality-adjusted life-years from its own budget.

Results

Respondents selected more than utility-maximizing insurance for protection against severe health states. A number of psychological factors that affect measurement under uncertainty do not affect utility as currently measured. This difference may explain the present results and also explain the “severity paradox” that personal preferences as presently measured imply less expenditure on severe health states than do “social preferences” for the treatment of strangers.

Conclusions

Uncertainty alters preferences. Incorporating these preferences in decision making would result in greater spending on severe health states.  相似文献   

2.

Background

Expert elicitation is required to inform decision making when relevant “better quality” data either do not exist or cannot be collected. An example of this is to inform decisions as to whether to screen for melanoma. A key input is the counterfactual, in this case the natural history of melanoma in patients who are undiagnosed and hence untreated.

Objectives

To elicit expert opinion on the probability of disease progression in patients with melanoma that is undetected and hence untreated.

Methods

A bespoke webinar-based expert elicitation protocol was administered to 14 participants in the United Kingdom, Australia, and New Zealand, comprising 12 multinomial questions on the probability of progression from one disease stage to another in the absence of treatment. A modified Connor-Mosimann distribution was fitted to individual responses to each question. Individual responses were pooled using a Monte-Carlo simulation approach. Participants were asked to provide feedback on the process.

Results

A pooled modified Connor-Mosimann distribution was successfully derived from participants’ responses. Feedback from participants was generally positive, with 86% willing to take part in such an exercise again. Nevertheless, only 57% of participants felt that this was a valid approach to determine the risk of disease progression. Qualitative feedback reflected some understanding of the need to rely on expert elicitation in the absence of “hard” data.

Conclusions

We successfully elicited and pooled the beliefs of experts in melanoma regarding the probability of disease progression in a format suitable for inclusion in a decision-analytic model.  相似文献   

3.

Objective

To develop and validate a tool for assessing dietitians' self-efficacy with using mobile health (mHealth) apps in dietetic practice.

Design and Participants

A tool for measuring mHealth app self-efficacy among dietitians was constructed based on a literature review, consultation with experts, and Bandura's self-efficacy scales development guide. Senior dietetic students and new graduate dietitians in Australia (n?=?100) tested the tool.

Outcome Measures and Analysis

Validation of the tool was conducted using factor analysis. Reliability testing was undertaken to examine internal consistency and repeatability.

Results

Construct validity was demonstrated through extraction of 4 factors with significant loadings accounting for 68% of variance: efficiency and effectiveness of nutrition care, training and support, integration into dietetic work systems, and familiarity with apps. Internal consistency reliability overall was 0.90; 3 of 4 constructs had Cronbach α?>?.70. Weighted kappas for test-retest reliability indicated moderate to substantial agreement (0.43–0.76).

Conclusions and Implications

This tool will be useful to researchers and dietetic associations interested in measuring dietitians' levels of mHealth app self-efficacy, developing interventions to enhance self-efficacy, and/or evaluating changes in self-efficacy after professional training. Testing of the tool in a more diverse sample of dietitians and among other health professional groups could enhance the applicability of the tool.  相似文献   

4.

Background

Differences in payer evaluation and coverage of pharmaceuticals and medical procedures suggest that coverage may differ for medications and procedures independent of their clinical benefit. We hypothesized that coverage for medications is more restricted than corresponding coverage for nonmedication interventions.

Methods

We included top-selling medications and highly utilized procedures. For each intervention–indication pair, we classified value in terms of cost-effectiveness (incremental cost per quality-adjusted life-year), as reported by the Tufts Medical Center Cost-Effectiveness Analysis Registry. For each intervention–indication pair and for each of 10 large payers, we classified coverage, when available, as either “more restrictive” or as “not more restrictive,” compared with a benchmark. The benchmark reflected the US Food and Drug Administration label information, when available, or pertinent clinical guidelines. We compared coverage policies and the benchmark in terms of step edits and clinical restrictions. Finally, we regressed coverage restrictiveness against intervention type (medication or nonmedication), controlling for value (cost-effectiveness more or less favorable than a designated threshold).

Results

We identified 392 medication and 185 procedure coverage decisions. A total of 26.3% of the medication coverage and 38.4% of the procedure coverage decisions were more restrictive than their corresponding benchmarks. After controlling for value, the odds of being more restrictive were 42% lower for medications than for procedures. Including unfavorable tier placement in the definition of “more restrictive” greatly increased the proportion of medication coverage decisions classified as “more restrictive” and reversed our findings.

Conclusions

Therapy access depends on factors other than cost and clinical benefit, suggesting potential health care system inefficiency.  相似文献   

5.

Background

A broad literature on health state utility values exists, but compared with chronic health states (HSs), issues surrounding the valuation of temporary health states (THSs) have been poorly explored.

Objectives

To assess the methods used by previous studies to value HSs that are considered temporary so as to determine the strengths and limitations associated with various approaches and to inform future study designs.

Methods

A systematic review was undertaken to explore the methods used, assess how the valuation was conducted for diseases that might lead to HSs deemed as temporary, and identify the challenges encountered in the valuation of THSs.

Results

Of the 36 relevant studies, 22 were explicit that the HS being valued was temporary. Most of the studies used more than one technique (often incorporating both conventional and adapted approaches). In using adapted techniques, the primary challenge was identifying an appropriate intermediate “anchor” HS and the possibility of negative utilities.

Conclusions

There is no agreement on the most methodologically robust approach to THS valuation. Valuation is complex and important issues relating to the validity, practicality, and reliability of the techniques used were not adequately covered by most of the studies identified.  相似文献   

6.

Background

The Functional Assessment of Cancer Therapy–Kidney Symptom Index Disease-Related Symptoms (FKSI-DRS) is important to gauge clinical benefit in metastatic renal cell carcinoma (mRCC).

Objectives

To estimate important difference (ID) in FKSI-DRS scores that is considered to be meaningful when comparing treatment effect between groups, using mRCC trial data.

Methods

Data were derived from two pivotal phase III mRCC trials comparing sunitinib versus interferon alfa (N = 750) in first-line mRCC, and axitinib versus sorafenib (N = 723) in second-line mRCC. The change from baseline in FKSI-DRS score was examined as a function of a set of anchors using the repeated-measures model. Several anchors were evaluated: FKSI item “I am bothered by side effects of treatment,” EuroQol five-dimensional questionnaire utility score, and adverse events.

Results

When the “I am bothered by side effects of treatment” score was used as an anchor, the ID ranged between 1.2 and 1.3 points. When change in the EuroQol five-dimensional questionnaire utility score was used as an anchor, the FKSI-DRS ID ranged between 0.62 and 0.63 points. Selecting the adverse events that corresponded to a maximum worsening in the FKSI-DRS score in either trial, the ID ranged between 0.62 and 0.74 points.

Conclusions

Among patients undergoing treatment for mRCC, between-group differences in FKSI-DRS scores as low as 1 point might be meaningful.  相似文献   

7.

Objectives

The aim of this article was to provide practical guidance in setting up patient registries to facilitate real-world data collection for health care decision making.

Methods

This guidance was based on our experiences and involvement in setting up patient registries in oncology in the Netherlands. All aspects were structured according to 1) mission and goals (“the Why”), 2) stakeholders and funding (“the Who”), 3) type and content (“the What”), and 4) identification and recruitment of patients, data handling, and pharmacovigilance (“the How”).

Results

The mission of most patient registries is improving patient health by improving the quality of patient care; monitoring and evaluating patient care is often the primary goal (“the Why”). It is important to align the objectives of the registry and agree on a clear and functional governance structure with all stakeholders (“the Who”). There is often a trade off between reliability, validity, and specificity of data elements and feasibility of data collection (“the What”). Patient privacy should be carefully protected, and address (inter-)national and local regulations. Patient registries can reveal unique safety information, but it can be challenging to comply with pharmacovigilance guidelines (“the How”).

Conclusions

It is crucial to set up an efficient patient registry that serves its aims by collecting the right data of the right patient in the right way. It can be expected that patient registries will become the new standard alongside randomized controlled trials due to their unique value.  相似文献   

8.

Objectives

To summarize the costs of tuberculosis (TB) diagnosis and treatment in human immunodeficiency virus (HIV)-infected patients and to assess the methodological quality of these studies.

Methods

We included cost, cost-effectiveness, and cost-utility studies that reported primary costing data, conducted worldwide and published between 1990 and August 2016. We retrieved articles in PubMed, Embase, EconLit, CINAHL plus, and LILACS databases. The quality assessment was performed using two guidelines—the Consolidated Health Economic Evaluation Reporting Standards and the Tool to Estimate Patient’s Costs. TB diagnosis was reported as cost per positive result or per suspect case. TB treatment was reported as cost of TB drugs, TB/HIV hospitalization, and treatment. We analyzed the data per level of TB/HIV endemicity and perspective of analysis.

Results

We included 34 articles, with 24 addressing TB/HIV treatment and 10 addressing TB diagnosis. Most of the studies were carried out in high TB/HIV burden countries (82%). The cost of TB diagnosis per suspect case varied from $0.5 for sputum smear microscopy to $175 for intensified case finding. The cost of TB/HIV hospitalization was higher in low/medium TB/HIV burden countries than in high TB/HIV burden countries ($75,406 vs. $2,474). TB/HIV co-infection presented higher costs than TB from the provider perspective ($814 vs. $604 vs. $454). Items such as “choice of discount rate,” “patient interview procedures,” and “methods used for valuing indirect costs” did not achieve a good score in the quality assessment.

Conclusions

Our findings point to the need of generation of more standardized methods for cost data collection to generate more robust estimates and thus, support decision-making process.  相似文献   

9.

Background

This study investigated and analyzed the relationship between medical resources and economic conditions of OECD member countries and extracted factors for introducing imaging diagnostic equipment in each country. We also investigated the possibility of introducing diagnostic imaging equipment.

Methods

A principal component analysis was conducted from medical environment indices and economic situation indices; the results were visualized on a graph with the extracted two principal components as axes, and the target nations were categorized according to the possibility of introducing diagnostic imaging equipment. Next, with the number of CTs and MRIs as response variables, we visualized the probability results on a graph by conducting a multiple regression analysis with the indices as explanatory variables and extracting the most influential factors on the number of diagnostic imaging equipment introduced.

Results

We classified 29 countries into four groups according to medical environment and economic situation indices. By extracting from the four groups a group with a high possibility for introducing medical equipment then conducting a multiple regression analysis with CT and MRI unit counts as objective variables and other medical environment and economic situation indices as explanatory variables, it became clear that the factor with the greatest influence on CT and MRI unit counts is the number of hospital beds.

Conclusion

As topics of future studies, we would like to clarify the factors behind as well as the probability for the introduction of medical equipment in each nation by researching high-growth medical equipment markets.  相似文献   

10.

Background

As part of the UK National Institute for Health and Care Excellence (NICE) single technology appraisal process, independent evidence review groups (ERGs) critically appraise a company’s submission relating to a specific technology and indication.

Objectives

To explore the type of additional exploratory analyses conducted by ERGs and their impact on the recommendations made by NICE.

Methods

The 100 most recently completed single technology appraisals with published guidance were selected for inclusion. A content analysis of relevant documents was undertaken to identify and extract relevant data, and narrative synthesis was used to rationalize and present these data.

Results

The types of exploratory analysis conducted in relation to companies’ models were fixing errors, addressing violations, addressing matters of judgment, and the provision of a new, ERG-preferred base case. Ninety-three of the 100 ERG reports contained at least one of these analyses. The most frequently reported type of analysis in these 93 ERG reports related to the category “Matters of judgment,” which was reported in 83 reports (89%). At least one of the exploratory analyses conducted and reported by an ERG is mentioned in 97% of NICE appraisal consultation documents and 94% of NICE final appraisal determinations, and had a clear influence on recommendations in 72% of appraisal consultation documents and 47% of final appraisal determinations.

Conclusions

These results suggest that the additional analyses undertaken by ERGs in the appraisal of company submissions are highly influential in the policy-making and decision-making process.  相似文献   

11.

Background

In addition to theoretical justifications, many statistical methods have been used for selecting covariates to include in algorithms mapping nonutility measures onto utilities. However, it is not clear whether using exploratory factor analysis (EFA) as one such method improves the predictive ability of these algorithms.

Objective

This question is addressed within the context of mapping a non–utility-based outcome, the core 23-item Women’s Health Questionnaire (WHQ-23), onto two utility instruments: five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) and the six-dimensional health state short form (derived from short form 36 health survey) (SF-6D).

Methods

Data on all three outcomes were collected from 455 women from the Australian general population participating in a study assessing attitudes toward in vitro fertilization. Statistical methods for selecting covariates included stepwise regression (SW), including all covariates (Include all), multivariable fractional polynomial (MFP), and EFA. The predictive accuracy of 108 regression models was assessed using five criteria: mean absolute error, root mean squared error, correlation, distribution of predicted utilities, and proportion of predictions with absolute errors of less than 0.0.5. Validation of “primary” models was carried out on random samples of the in vitro fertilization study.

Results

The best results for EQ-5D-5L and SF-6D predictions were obtained from models using SW, “Include all,” and MFP covariate-selection approaches. Root mean squared error (0.0762–0.1434) and mean absolute error (0.0590–0.0924) estimates for these models were within the range of published estimates. EFA was outperformed by other covariate-selection methods.

Conclusions

It is possible to predict valid utilities from the WHQ-23 using regression methods based on SW, “Include all,” and MFP covariate-selection techniques.  相似文献   

12.

Background

Studies have often stated that individual-level determinants are important drivers for the adoption of medical devices. Empirical evidence supporting this claim is, however, scarce. At the individual level, physicians’ adoption motivation was often considered important in the context of adoption decisions, but a clear notion of its dimensions and corresponding measurement scales is not available.

Objectives

To develop and subsequently validate a scale to measure the motivation to adopt medical devices of hospital-based physicians.

Methods

The development and validation of the physician-motivation-adoption (PMA) scale were based on a literature search, internal expert meetings, a pilot study with physicians, and a three-stage online survey. The data collected in the online survey were analyzed using exploratory factor analysis (EFA), and the PMA scale was revised according to the results. Confirmatory factor analysis (CFA) was conducted to test the results from the EFA in the third stage. Reliability and validity tests and subgroup analyses were also conducted.

Results

Overall, 457 questionnaires were completed by medical personnel of the National Health Service England. The EFA favored a six-factor solution to appropriately describe physicians’ motivation. The CFA confirmed the results from the EFA. Our tests indicated good reliability and validity of the PMA scale.

Conclusions

This is the first reliable and valid scale to measure physicians’ adoption motivation. Future adoption studies assessing the individual level should include the PMA scale to obtain more information about the role of physicians’ motivation in the broader adoption context.  相似文献   

13.

Background

It has been suggested that differences in health technology assessment (HTA) processes among countries, particularly within Europe, have led to inequity in patient access to new medicines.

Objectives

To provide an up-to-date snapshot analysis of the present status of HTA and reimbursement systems in select European countries, and to investigate the implications of these processes, especially with regard to delays in market and patient access.

Methods

HTA and reimbursement processes were assessed through a review of published and gray literature, and through a series of interviews with HTA experts. To quantify the impact of differences among countries, we conducted case studies of 12 products introduced since 2009, including 10 cancer drugs.

Results

In addition to the differences in HTA and reimbursement processes among countries, the influence of particular sources of information differs among HTA bodies. The variation in the time from the authorization by the European Medicines Agency to the publication of HTA decisions was considerable, both within and among countries, with a general lack of transparency as to why some assessments take longer than others. In most countries, market access for oncology products can occur outside the HTA process, with sales often preceding HTA decisions.

Conclusions

It is challenging even for those with considerable personal experience in European HTA processes to establish what is really happening in market access for new drugs. We recommend that efforts should be directed toward improving transparency in HTA, which should, in turn, lead to more effective processes.  相似文献   

14.

Objectives

To determine what thresholds are most often cited in the cost-effectiveness literature for low- and middle-income countries (LMICs), given various recommendations proposed and used in the literature to date, and thereafter to assess whether studies appropriately justified their use of threshold values.

Methods

We reviewed the contents of the Tufts Medical Center Global Health Cost-Effectiveness Analysis Registry, a repository of all English language cost-per-disability-adjusted life-year averted studies indexed in PubMed. Our review included all catalogued cost-per-disability-adjusted life-year studies published from 2000 through 2015. We restricted attention to studies that investigated interventions in LMICs.

Results

Our analysis identified 381 studies (80%) focused on LMICs. Of these studies, 250 (66%) cited the World Health Organization’s 1 to 3 times gross domestic product per capita threshold. A full-text review of 60 (24%) of these articles (randomly selected) revealed that none justified use of this threshold in the particular country or countries studied beyond citing (generic) guideline documents.

Conclusions

Cost-effectiveness analysis can help inform health care spending, but its value depends on incorporating assumptions that are valid for the applicable setting. Rather than rely on commonly used, generic economic thresholds, we encourage authors to use context-specific thresholds that reflect local preferences.  相似文献   

15.

Background

The recent shift to an integrated approach to health and social care aims to provide cohesive support to those who are in need of care, but raises a challenge for resource allocation decision making, in particular for comparison of diverse benefits from different types of care across the two sectors.

Objective

To investigate the relationship of social care needs and well-being with a generic health status measure using multivariate regression.

Methods

We empirically compared responses to health and well-being measures and social care needs from a cross-sectional data set of the general population (the Health Survey for England). Multivariate regression analyses were conducted to examine whether social care needs measured by the Barthel index can be explained by health status as captured by the EuroQol five-dimensional questionnaire (EQ-5D) and two well-being measures—the Warwick Edinburgh Mental Wellbeing Scale (WEMWBS) and the General Health Questionnaire (GHQ-12).

Results

Our study found that poor overall scores for EuroQol visual analogue scale, EQ-5D index, GHQ-12, and WEMWBS indicated a need for social care. Investigation of the dimensions found that the EQ-5D dimensions self-care and pain/discomfort were statistically significantly associated with the need for social care. Two dimensions of the WEMWBS (“been feeling useful” and “had energy to spare”) were statistically significantly associated with the Barthel index, but none of the GHQ-12 dimensions were.

Conclusions

The results show that the need for social care, which is dependent on the ability to perform personal day-to-day activities, is more closely related to the EQ-5D dimensions than the well-being measures WEMWBS and GHQ-12.  相似文献   

16.

Objectives

Patient-accessible electronic health records (PAEHRs) are being implemented at international scale. Comparing policies and systems could allow countries to learn from each other to address global and nation-specific challenges. We compare national PAEHR policy (hard and soft regulation) and services in 10 countries.

Methods

PAEHR policy and system documentation was gathered from Australia, Denmark, Estonia, Finland, France, the Netherlands, New Zealand, Norway, Sweden and the United States. A basic analytic model for policy analysis was used to delimit our focus to policy content, followed by an inductive thematic analysis across countries, in which we clustered initial themes into a set of categories of PAEHR service “approaches” related to three specific content areas.

Results

Although all 10 countries ensured some patient rights to access medical records, policies and systems were highly variable, as were the technological processes arising from these. In particular, three policy areas showed great variability. Depending upon country of origin, a patient would encounter differences in: login procedures (security), access to own and other patients’ data during adolescence (user rights), and types of medical data made available to the patient (data sets).

Conclusions

Individuals encounter very different access rights to their medical data depending on where they live. Countries may be able to develop improved policies by examining how other nations have solved common problems. Harmonizing policies is also an initial step likely to be needed before cross-national PAEHRs could be possible.  相似文献   

17.

Objectives

To obtain a nationally representative Chinese three-level EuroQol five-dimensional questionnaire value set based on the time trade-off (TTO) method.

Methods

A multistage, stratified, clustered random nationally representative Chinese sample was used. The study design followed an adapted UK Measurement and Valuation of Health protocol. Each respondent valued 11 random states plus state 33333 and “unconscious” using the TTO method in face-to-face interviews. Three types of models were explored: ordinary least squares, general least squares, and weighted least squares models.

Results

In total, 5939 inhabitants aged 15 years and older were interviewed. Of these, 5503 satisfactorily interviewed participants were included in constructing models. An ordinary least squares model including 10 dummies without constant and N3 had a mean absolute error of 0.083 and a correlation coefficient of 0.899 between the predicted and mean values. Goodness-of-fit indices of two models based on split subsample were similar.

Conclusions

TTO values were higher in our study compared with those in a study carried out in urban areas, which is mirrored by the higher values in rural areas. Several other aspects, in addition to the valuation procedure, might have influenced the results, such as factors beyond demographic factors such as view on life and death and believing in an afterlife, which need further investigation. Future studies using the three-level EuroQol five-dimensional questionnaire should consider using this value set based on a nationally representative sample of the Chinese population.  相似文献   

18.

Introduction

The etiological diagnosis of infectious encephalitis is often not established 48 hours after onset. We aimed to review existing literature data before providing management guidelines.

Method

We performed a literature search on PubMed using filters such as “since 01/01/2000”, “human”, “adults”, “English or French”, and “clinical trial/review/guidelines”. We also used the Mesh search terms “encephalitis/therapy” and “encephalitis/diagnosis”.

Results

With Mesh search terms “encephalitis/therapy” and “encephalitis/diagnosis”, we retrieved 223 and 258 articles, respectively. With search terms “encephalitis and corticosteroid”, we identified 38 articles, and with “encephalitis and doxycycline” without the above-mentioned filters we identified 85 articles. A total of 210 articles were included in the analysis.

Discussion

Etiological investigations must focus on recent travels, animal exposures, age, immunodeficiency, neurological damage characteristics, and potential extra-neurological signs. The interest of a diagnosis of encephalitis for which there is no specific treatment is also to discontinue any empirical treatments initially prescribed. Physicians must consider and search for autoimmune encephalitis.  相似文献   

19.

Objective

To describe the influence of the food industry in health research, observing how funding influences health outcomes and the quality of the studies.

Method

We performed a systematic review in MEDLINE, Cochrane Library Plus and Scopus using the MESH “Food Industry”, “Food-Processing Industry”, “Biomedical Research”, “Research Support as Topic”, and the keywords “Industry Sponsorship” and “Funding Source”. The quality was assessed using the PRISMA guidelines.

Results

We revised 1,506 articles and 10 were included; two reviewed the relationship between funding-outcomes and quality-outcomes; six focused on the funding-outcomes relationship; and the other two focused on methodological quality. Six showed that funding from the food industry resulted in more favourable outcomes for their products. No differences in quality were found in relation to the funding source, but those which did not declare their funding had a worse quality.

Conclusion

Studies funded by the food industry showed favourable results for their products. However, this fact did not affect the quality of the studies.  相似文献   

20.

Background

Next-generation sequencing (NGS) technologies have seen variable adoption in the clinic. This is partly due to a lack of clinical and economic studies, with the latter increasingly challenged to examine patient preferences for health and nonhealth outcomes (e.g., false-positive rate).

Objectives

To conduct a structured review of studies valuing patients’ preference-based utility for NGS outcomes, to highlight identified methodological challenges, and to consider how studies addressed identified challenges.

Methods

We searched MEDLINE (PubMed), Embase (Ovid), and Web of Science for published studies examining outcomes from health care decisions informed by NGS. We focused our search on direct elicitations of preference-based utility. We reviewed included studies and qualitatively grouped and summarized stated challenges and solutions by theme.

Results

Eleven studies were included. Most of them (n = 6) used discrete choice experiments to value utility. We categorized challenges into four themes: 1) valuing the full range of NGS outcomes, 2) accounting for accuracy and uncertainty surrounding effectiveness, 3) allowing for simultaneous multiple and cascading risks, and 4) incorporating downstream consequences. Studies found strong evidence of utility for NGS information, regardless of health improvement. Investigators addressed challenges by simplifying complex choices, by including health outcomes alongside nonhealth outcomes, and by using multiple elicitation techniques.

Conclusions

The breadth and complexity of NGS-derived information makes the technology a unique and challenging application for utility valuation. Failing to account for the utility or disutility of NGS-related nonhealth outcomes may lead to overinvestment or underinvestment in NGS, and so there is a need for research addressing unresolved challenges.  相似文献   

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