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Identifying Attributes for a Value Assessment Framework in China: A Qualitative Study
Abstract
Background:
Value assessment frameworks (VAFs) are promising tools for measuring the value of health technologies and informing coverage policymaking; however, most published VAFs were developed for high-income countries. This study aimed to identify value attributes as part of the development of a VAF in China.
Methods:
We used the qualitative description approach. Specifically, we conducted open-ended semi-structured interviews with Chinese stakeholders, as well as a review and analysis of publicly available government documents related to health technology assessment (HTA) and coverage policies in China. Conventional content analysis and the constant comparison technique were used to generate value attributes. Multiple criteria were used to determine the inclusion of a value attribute, with response levels of included attributes finalized via consensus meetings among the research team.
Results:
Thirty-four stakeholders living or working in China completed the semi-structured interview. These stakeholders included policymakers (n = 4), healthcare providers (n = 8), HTA researchers (n = 6), patients and members of the general public (n = 9), and industry representatives (n = 7). In addition, 16 government documents were included for analysis. Twelve value attributes grouped in eight categories are included in the VAF: (1) severity of disease; (2) health benefit, including survival, clinical outcomes, and patient-reported outcomes; (3) safety; (4) economic impact, including budget impact to payer and patients, and cost effectiveness; (5) innovation; (6) organizational impact; (7) health equity; and (8) quality of evidence.
Conclusion:
These 12 value attributes were identified for the development of a VAF to support health technologies' value assessment and coverage policymaking in China.
... Furthermore, spine loss and progressive changes of mushroom spines in CA1 hippocampal neurons were observed in ex vivo hippocampal slices prepared from 7-or 14-day-old mice that were kept in culture for 15 or 20 days [41]. It is therefore likely that the findings indicate the development of the processes of mushroom into thin dendritic spines conversion in hippocampal CA1 neurons of 5xFAD-M mice, reflecting the dendritic spine shrinkage with decreased synaptic plasticity revealed in 5xFAD mice [42][43][44]. ...
Cognitive impairments are closely related to synaptic loss in Alzheimer’s disease (AD). Functional changes in synaptic contacts are reflected in dendritic spine morphology. Visualization of neurons for morphological studies in vivo is complicated by the fixed brain slice staining or expensive adeno-associated virus injections. We created a transgenic 5xFAD-M line of mice with AD-associated mutations and expressed GFP protein in single neurons of the brain. This mouse model of AD is a useful tool for the simplified visualization of the hippocampal neurons’ morphology in vivo without additional staining manipulations. The progressive elimination of mushroom spines was demonstrated in 5xFAD-M mice between 4 and 5 months of age. Five-month-old 5xFAD-M male and female mice showed change both in the total density and the mushroom spines number compared to sex-matched control. We conclude 5xFAD-M mice can be a useful AD model for studying the mechanisms of synaptic pathology under neurodegenerative conditions and evaluating the effects of potential therapeutic agents on spine morphology as crucial aspect of memory loss in AD.
Background
China launched a new round of healthcare-system reform in 2009 and proposed the goal of equal and guaranteed essential medical and health services for all by 2020. We aimed to investigate the changes in China’s health resources over the past ten years after the healthcare reform.
Methods
Data were collected from the China Statistical Yearbook and China Health Statistics Yearbook from 2009 to 2018. Four categories and ten indicators of health resources were analyzed. A descriptive analysis was used to present the overall condition. The Health Resource Density Index was applied to showcase health-resource distribution in demographic and geographic dimensions. The global and local Moran’s I were used to assess the spatial autocorrelation of health resources. Concentration Index (CI) was used to quantify the equity of health-resource distribution. A Geo-Detector model and Geographic Weighted Regression (GWR) were applied to assess the association between gross domestic product (GDP) per capita and health resources.
Results
Health resources have increased over the past ten years. The global and local Moran’s I suggested spatial aggregation in the distribution of health resources. Hospital beds were concentrated in wealthier areas, but this inequity decreased yearly (from CI=0.0587 in 2009 to CI=0.0021 in 2018). Primary medical and health institutions (PMHI) and their beds were concentrated in poorer areas (CI remained negative). Healthcare employees were concentrated in wealthier areas (CI remained positive). In 2017, the q-statistics indicated that the explanatory power of GDP per capita to beds, health personnel, and health expenditure was 40.7%, 50.3%, and 42.5%, respectively. The coefficients of GWR remained positive with statistical significance, indicating the positive association between GDP per capita and health resources.
Conclusions
From 2009 to 2018, the total amount of health resources in China has increased substantially. Spatial aggregation existed in the health-resources distribution. Health resources tended to be concentrated in wealthier areas. When allocating health resources, the governments should take economic factors into account.
Background
Medicine purchasing in Chinese public hospitals is decided by the hospital Pharmacy Management Committee (PMC), that is complex, subjective and requires efficient approaches to ensure transparency and consistency for the factors being considered. This study aimed to use the Evidence and Value: Impact on Decision Making (EVIDEM) framework to assess medicine in these hospitals. In this study anti-diabetic drugs DPP-4 inhibitors, which work by inhibiting the activation of the Dipeptidyl Peptidase 4 (DPP-4) inhibitors, were appraised.
Methods
Following EVIDEM methodology (EVIDEM-10th), we convened an appraisal group and asked each individual to express their perspectives by assigning weights to each criterion. A systematic literature search for information of each criterion of five DPP-4 inhibitors was completed. Then the appraisal group scored for each criterion of the five DPP-4 inhibitors. The estimated value of the five DPP-4 inhibitors was obtained by Multi-Criteria Decision Analysis (MCDA) which combined individual weighting of each criterion with individual scoring for each intervention in each criterion.
Results
By assigning weights, the most important criterion was the quality of evidence (4.01±0.52), and that the comparative cost consequences-non-medical cost was the least important criterion (2.87±1.03). Criteria included disease severity, size of the affected population, comparative effectiveness, type of therapeutic/preventive benefit and cost of intervention, all of which were assigned the same weight of 3.58. After MCDA, the overall value orders for each DPP-4 inhibitor included Sitagliptin (0.45), Linagliptin (0.44), Vildagliptin (0.43), Alogliptin (0.42) and Saxagliptin (0.40).
Conclusions
Based on EVIDEM framework and MCDA, we found that overall value of five DPP-4 inhibitors was similar. It is feasible to use the EVIDEM framework and MCDA in purchasing medicine for Chinese public hospitals.
Multiple Criteria Decision Analysis (MCDA) has emerged as a likely methodology for Health Technology Assessment (HTA). However limited empirical evidence is available on its use by decision-makers and only as part of single-setting exercises, without cross-county studies available. This pilot study applies the Advance Value Framework (AVF), an MCDA methodology for HTA based on multi-attribute value theory, through a series of case studies with decision-makers in four countries, to explore its feasibility and compare their value preferences and results. The AVF was applied in the evaluation of three drugs for metastatic, castrate resistant, prostate cancer (abiraterone, cabazitaxel and enzalutamide in the post-chemotherapy indication). Decision conferences were organised in four European countries in collaboration with their HTA or health insurance organisations by engaging relevant assessors and experts: Sweden (TLV), Andalusia/Spain (AETSA), Poland (AOTMiT) and Belgium (INAMI-RIZIV). Participant value preferences, including performance scoring and criteria weighting, were elicited through a facilitated decision-analysis modelling approach using the MACBETH technique. Between 6 and 11 criteria were included in the value model of each country, allocated across four criteria domains; Therapeutic Benefit criteria conistently ranked first across countries in their relative importance. Consistent drug rankings were observed in all settings, with enzalutamide generating the highest overall weighted preference value (WPV) score, followed by abiraterone and cabazitaxel; dividing drugs' overall WPV scores by their costs produced the lowest "cost-per-unit of value" for enzalutamide, followed for abiraterone and cabazitaxel. These results contrast the HTA recommendations and pricing decisions in real life.
Objectives: Economic growth and rapid development of health technology in China have created opportunities to strengthen health technology assessment (HTA) capacity. Over the time, HTA institutions have been established to conduct HTA related work. This study reviewed the current status of HTA in China and analysed the challenges of HTA development in the context of health reform under “new normal” economy.
Methods: Literature review and webpage searches were used to document the development of HTA in China. An institutional survey has also been conducted to collect information on the HTA research institutions in China.
Results: The number of articles and research projects on HTA were rising and are continuing to rise. HTA development has made substantial progress in China in terms of growing number of research institutions and qualified HTA workforce. However, HTA has notable weaknesses such as low capacity for conducting HTA research, limited experience in HTA researchers, and lack of knowledge translation.
Conclusion: Currently, the translation of HTA findings to policy-making is limited and the integration of HTA in the policy-making processes is still in its infancy. The HTA development in China has had opportunities due to demands of health care, health insurance, and health technology as a result of health reform. Capacity building and institutionalization of HTA are urgently needed for further development of HTA in China.
Background
Value assessment frameworks have emerged as tools to assist healthcare decision makers in the United States in assessing the relative value of healthcare services and treatments. As more healthcare decision makers in the United States—including state government agencies, pharmacy benefit managers, employers, and health plans—publicly consider the adoption of value frameworks, it is increasingly important to critically evaluate their ability to accurately measure value and reliably inform decision making.
Objective
To examine the evolution of the value assessment landscape in the past two years, including new entrants and updated frameworks, and assess if these changes successfully advance the field of value assessment.
Methods
We analyzed the progress of the three currently active value assessment frameworks developed by the Institute for Clinical and Economic Review, the Innovation and Value Initiative, and the National Comprehensive Cancer Network, against six key areas of concern.
Results
Value assessment frameworks are moving closer to meeting the challenge of accurately measuring value and reliably informing healthcare decisions. Each of the six concerns has been addressed in some way by at least one framework.
Conclusions
Although value assessments are potential inputs that can be considered for healthcare decision making, none of them should be the sole input for these decisions. Considering the limitations, they should, at most, be only one of many tools in the toolbox.
Background:
Orphan medicines show some characteristics that hinder the evaluation of their clinical added value. The often low level of evidence available for orphan drugs, together with a high budget impact and an incremental cost-effectiveness ratio many times higher than drugs used for non-orphan diseases, represent challenges in their appraisal and effective access to clinical use. In order to explore how to handle these hurdles, the Catalan Health Service (CatSalut) began an initiative on a multidimensional assessment of drugs value during the appraisal process. Reflective multicriteria decision analysis (MCDA) using analytical methods was chosen, since it may help to standardise and contextualize all the relevant data related with the drug that could contribute to a decision. The aim of the study was to determine whether the implementation of reflective MCDA methodology could support the decision-making process about orphan medicines in the context of CatSalut.
Methods:
The assessment and decision-making process for orphan drugs in the Programa d'Harmonització Farmacoterapeutica (PHF) of CatSalut was prioritized to test the implementation of the reflective MCDA both a qualitative and quantitatively. A staged approach was used with the following main steps: selection and structuration of quantitative criteria (Core Model) and qualitative criteria (Contextual Tool), framework scoring and assessment of three orphan drug case studies. This proof-of-concept would grant a continued refinement of the methodology and, if and when validated, its potential integration to other therapeutic areas of the PHF.
Results:
The final framework was composed by 10 quantitative criteria (Core Model) and 4 qualitative criteria (Contextual Tool) according to the PHF goals being the most important criteria "disease severity", "unmet need", "comparative effectiveness" and "comparative safety /tolerability". The matrix developed for the case studies served as a guide for the selection of the essential information that the decision-makers were expected to include in a framework. The reflective discussion was considered the most relevant phase of the approach to support inputs for health decision-making processes reflecting both drug value and place in therapy.
Conclusions:
The study showed that reflective MCDA methodology could be implemented to complement the decision-making process in CatSalut, as an aid to determine the clinical added value for orphan medicines. MCDA provided transparency and a structured discussion during the committee meetings, thus increasing transparency and predictability of the relevant items supporting the agreements adopted on orphan drugs access.
Background: Choosing a suitable sample size in qualitative research is an area of conceptual debate and practical
uncertainty. That sample size principles, guidelines and tools have been developed to enable researchers to set,
and justify the acceptability of, their sample size is an indication that the issue constitutes an important marker of
the quality of qualitative research. Nevertheless, research shows that sample size sufficiency reporting is often poor,
if not absent, across a range of disciplinary fields.
Methods: A systematic analysis of single-interview-per-participant designs within three health-related journals from
the disciplines of psychology, sociology and medicine, over a 15-year period, was conducted to examine whether
and how sample sizes were justified and how sample size was characterised and discussed by authors. Data pertinent
to sample size were extracted and analysed using qualitative and quantitative analytic techniques.
Results: Our findings demonstrate that provision of sample size justifications in qualitative health research is limited; is
not contingent on the number of interviews; and relates to the journal of publication. Defence of sample size
was most frequently supported across all three journals with reference to the principle of saturation and to
pragmatic considerations. Qualitative sample sizes were predominantly – and often without justification – characterised
as insufficient (i.e., ‘small’) and discussed in the context of study limitations. Sample size insufficiency was seen
to threaten the validity and generalizability of studies’ results, with the latter being frequently conceived in nomothetic
terms.
Conclusions: We recommend, firstly, that qualitative health researchers be more transparent about evaluations of their
sample size sufficiency, situating these within broader and more encompassing assessments of data adequacy.
Secondly, we invite researchers critically to consider how saturation parameters found in prior methodological
studies and sample size community norms might best inform, and apply to, their own project and encourage
that data adequacy is best appraised with reference to features that are intrinsic to the study at hand. Finally,
those reviewing papers have a vital role in supporting and encouraging transparent study-specific reporting.
Background
Multiple technologies, procedures and programs call for fairly-based decisions for prioritization of healthcare interventions. There is a diversity of perspectives of what constitutes a legitimate decision, which depends on both the process and the reasoning applied. Current approaches focus on technical aspects while methods to support alignment of decisions with the compassionate impetus of healthcare systems is lacking.
Methods
The framework was developed based on an analysis of the foundations of healthcare systems, the reasoning underlying decisions and fair processes. The concept of reflective multicriteria was created: it assumes that decisionmakers guided by a generic interpretative frame rooted in the compassionate impetus of healthcare systems, can sharpen their reasoning, raise awareness of their motivation and increase legitimacy of decisions. The initial framework was made available through a not for profit organization (the EVIDEM Collaboration, 2006–2017) to stimulate its development with thought leaders and stakeholders in an open source philosophy. Development was tailored to the real-life needs of decisionmakers and drew on several domains of knowledge including healthcare ethics, evidenced-based medicine, health economics, health technology assessment and multicriteria approaches.
Results
The 10th edition framework builds on four dimensions: (1) the universal impetus of healthcare systems, (2) reasoning, values and ethics, (3) evidence and knowledge on interventions, and (4) a transformative process. Mathematical aspects of the framework are designed to help clarify, express and share individual reasoning; this non-conventional use of numbers requires a cultural change and needs to be phased in slowly. The framework includes four tools for easy adaptation and operationalization: (a) concepts and operationalization, (b) adapt and pilot, (c) evidence matrix, (d) mathematical representation of reasoning. Application is useful throughout all types of healthcare interventions, for all levels of decision, and across the globe.
Conclusion
By clarifying their reasoning while keeping decisionmakers aware of the impetus of healthcare systems, reflective multicriteria provides an effective approach to increase the legitimacy of decisions. Beyond a tool, reflective multicriteria pioneered by EVIDEM is geared to transform our vision of the value of healthcare interventions and how they might contribute to relevant, equitable and sustainable healthcare systems.
Electronic supplementary material
The online version of this article (10.1186/s12962-018-0116-9) contains supplementary material, which is available to authorized users.
A qualitative description design is particularly relevant where information is required directly from those experiencing the phenomenon under investigation and where time and resources are limited. Nurses and midwives often have clinical questions suitable to a qualitative approach but little time to develop an exhaustive comprehension of qualitative methodological approaches. Qualitative description research is sometimes considered a less sophisticated approach for epistemological reasons. Another challenge when considering qualitative description design is differentiating qualitative description from other qualitative approaches. This article provides a systematic and robust journey through the philosophical, ontological, and epistemological perspectives, which evidences the purpose of qualitative description research. Methods and rigor issues underpinning qualitative description research are also appraised to provide the researcher with a systematic approach to conduct research utilizing this approach. The key attributes and value of qualitative description research in the health care professions will be highlighted with the aim of extending its usage.
Saturation has attained widespread acceptance as a methodological principle in qualitative research. It is commonly taken to indicate that, on the basis of the data that have been collected or analysed hitherto, further data collection and/or analysis are unnecessary. However, there appears to be uncertainty as to how saturation should be conceptualized, and inconsistencies in its use. In this paper, we look to clarify the nature, purposes and uses of saturation, and in doing so add to theoretical debate on the role of saturation across different methodologies. We identify four distinct approaches to saturation, which differ in terms of the extent to which an inductive or a deductive logic is adopted, and the relative emphasis on data collection, data analysis, and theorizing. We explore the purposes saturation might serve in relation to these different approaches, and the implications for how and when saturation will be sought. In examining these issues, we highlight the uncertain logic underlying saturation—as essentially a predictive statement about the unobserved based on the observed, a judgement that, we argue, results in equivocation, and may in part explain the confusion surrounding its use. We conclude that saturation should be operationalized in a way that is consistent with the research question(s), and the theoretical position and analytic framework adopted, but also that there should be some limit to its scope, so as not to risk saturation losing its coherence and potency if its conceptualization and uses are stretched too widely.
In 2014, balanced assessment systems (BAS) were proposed as a resource-conscious, ‘fit-for-purpose’ form of health technology assessment for middle-income countries which lack resources and competences necessary for resource-intensive health technology assessment models. BAS has undergone extensive policy debate in the period since its publication but it has not been critically assessed in a structured form yet. This article aims to describe both the contributions and the weak spots of the original framework and to reflect on them with the intention of further developing the model.
Escalating drug prices have catalysed the generation of numerous “value frameworks” with the aim of informing payers, clinicians and patients on the assessment process of new medicines for the purpose of coverage and treatment selection decisions. Although this is an important step towards a more inclusive Value Based Assessment (VBA) approach, aspects of these frameworks are based on weak methodologies and could potentially result in misleading recommendations or decisions.
Background
Limited resources and expanding expectations push all countries and types of health systems to adopt new approaches in priority setting and resources allocation. Despite best efforts, it is difficult to reconcile all competing interests, and trade-offs are inevitable. This is why multi-criteria decision analysis (MCDA) has played a major role in recent uptake of value-based reimbursement. MCDA framework enables exploration of stakeholders’ preferences, as well as explicit organization of broad range of criteria on which real-world decisions are made. Assessment and appraisal of orphan drugs tend to be one of the most complicated health technology assessment (HTA) tasks. Access to market approved orphan therapies remains an issue. Early constructive dialog among rare disease stakeholders and elaboration of orphan drug-tailored decision support tools could set the scene for ongoing accumulation of evidence, as well as for proper reimbursement decision-making.
Objective
The objective of this study was to create an MCDA value measurement model to assess and appraise orphan drugs. This was achieved by exploring the preferences on decision criteria’s weights and performance scores through a stakeholder-representative survey and a focus group discussion that were both organized in Bulgaria.
Results/Conclusion
Decision criteria that describe the health technology’s characteristics were unanimously agreed as the most important group of reimbursement considerations. This outcome, combined with the high individual weight of disease severity and disease burden criteria, underlined some of the fundamental principles of health care – equity and fairness. Our study proved that strength of evidence may be a key criterion in orphan drug assessment and appraisal. Evidence is used not only to shape reimbursement decision-making but also to lend legitimacy to policies pursued. The need for real-world data on orphan drugs was largely stressed. Improved knowledge on MCDA feasibility and integration to HTA is of paramount importance, as progress in medicine and innovative health technologies should correspond to patient, health-care system, and societal values.
#### Summary points
Healthcare decision making is complex. Decision-making processes and the factors (criteria) that decision makers should consider vary for different types of decisions, including clinical recommendations, coverage decisions, and health system or public health recommendations or decisions.1 2 3 4 However, some criteria are relevant for all of these decisions, including the anticipated effects of the options being considered, the certainty of the evidence for those effects (also referred to as quality of evidence or confidence in effect estimates), and the costs and feasibility of the options. Decision makers must make judgments about each relevant factor, informed by the best evidence that is available to them.
Often, the processes that decision makers use, the criteria that they consider and the evidence that they …
Because of significant differences in institutional contexts, health technology assessment (HTA) systems that are in place in core pharmaceutical markets may not be suitable, fully or in part, for middle-income countries (MICs) and for other noncore markets. Particular challenges may arise when systems based on the economic evaluation paradigm are conceptualized and implemented in MICs, sometimes with an insufficient level of awareness of the local institutional factors that influence pricing and reimbursement decision making. Focusing on pharmaceuticals, this article investigates possible development directions for HTA systems in MICs and noncore markets bearing similar institutional characteristics, and it provides recommendations for a balanced assessment system (BAS). For this, the main paradigms of HTA have also been reviewed briefly and factors influencing HTA and pricing and reimbursement decisions in MICs and in similar noncore countries have been summarized. The proposed BAS framework takes into account available resources and capabilities and is supposed to facilitate access to new pharmaceuticals while ensuring the transparency of decision-making processes and the stability of the pharmaceutical budget.
Distributional cost-effectiveness analysis (DCEA) is a framework for incorporating health inequality concerns into the economic evaluation of health sector interventions. In this tutorial, we describe the technical details of how to conduct DCEA, using an illustrative example comparing alternative ways of implementing the National Health Service (NHS) Bowel Cancer Screening Programme (BCSP). The 2 key stages in DCEA are 1) modeling social distributions of health associated with different interventions, and 2) evaluating social distributions of health with respect to the dual objectives of improving total population health and reducing unfair health inequality. As well as describing the technical methods used, we also identify the data requirements and the social value judgments that have to be made. Finally, we demonstrate the use of sensitivity analyses to explore the impacts of alternative modeling assumptions and social value judgments.
© The Author(s) 2015.
Purpose:
Standards for reporting exist for many types of quantitative research, but currently none exist for the broad spectrum of qualitative research. The purpose of the present study was to formulate and define standards for reporting qualitative research while preserving the requisite flexibility to accommodate various paradigms, approaches, and methods.
Method:
The authors identified guidelines, reporting standards, and critical appraisal criteria for qualitative research by searching PubMed, Web of Science, and Google through July 2013; reviewing the reference lists of retrieved sources; and contacting experts. Specifically, two authors reviewed a sample of sources to generate an initial set of items that were potentially important in reporting qualitative research. Through an iterative process of reviewing sources, modifying the set of items, and coding all sources for items, the authors prepared a near-final list of items and descriptions and sent this list to five external reviewers for feedback. The final items and descriptions included in the reporting standards reflect this feedback.
Results:
The Standards for Reporting Qualitative Research (SRQR) consists of 21 items. The authors define and explain key elements of each item and provide examples from recently published articles to illustrate ways in which the standards can be met.
Conclusions:
The SRQR aims to improve the transparency of all aspects of qualitative research by providing clear standards for reporting qualitative research. These standards will assist authors during manuscript preparation, editors and reviewers in evaluating a manuscript for potential publication, and readers when critically appraising, applying, and synthesizing study findings.
This article examines the function of documents as a data source in qualitative research and discusses document analysis procedure in the context of actual research experiences. Targeted to research novices, the article takes a nuts-and-bolts approach to document analysis. It describes the nature and forms of documents, outlines the advantages and limitations of document analysis, and offers specific examples of the use of documents in the research process. The application of document analysis to a grounded theory study is illustrated.
Objectives
Resource allocation is a challenging issue faced by health policy decisionmakers requiring careful consideration of many factors. Objectives of this study were to identify decision criteria and their frequency reported in the literature on healthcare decisionmaking.
Method
An extensive literature search was performed in Medline and EMBASE to identify articles reporting healthcare decision criteria. Studies conducted with decisionmakers (e.g., focus groups, surveys, interviews), conceptual and review articles and articles describing multicriteria tools were included. Criteria were extracted, organized using a classification system derived from the EVIDEM framework and applying multicriteria decision analysis (MCDA) principles, and the frequency of their occurrence was measured.
Results
Out of 3146 records identified, 2790 were excluded. Out of 356 articles assessed for eligibility, 40 studies included. Criteria were identified from studies performed in several regions of the world involving decisionmakers at micro, meso and macro levels of decision and from studies reporting on multicriteria tools. Large variations in terminology used to define criteria were observed and 360 different terms were identified. These were assigned to 58 criteria which were classified in 9 different categories including: health outcomes; types of benefit; disease impact; therapeutic context; economic impact; quality of evidence; implementation complexity; priority, fairness and ethics; and overall context. The most frequently mentioned criteria were: equity/fairness (32 times), efficacy/effectiveness (29), stakeholder interests and pressures (28), cost-effectiveness (23), strength of evidence (20), safety (19), mission and mandate of health system (19), organizational requirements and capacity (17), patient-reported outcomes (17) and need (16).
Conclusion
This study highlights the importance of considering both normative and feasibility criteria for fair allocation of resources and optimized decisionmaking for coverage and use of healthcare interventions. This analysis provides a foundation to develop a questionnaire for an international survey of decisionmakers on criteria and their relative importance. The ultimate objective is to develop sound multicriteria approaches to enlighten healthcare decisionmaking and priority-setting.
The knowledge and use of qualitative description as a qualitative research approach in health services research is limited.The aim of this article is to discuss the potential benefits of a qualitative descriptive approach, to identify its strengths and weaknesses and to provide examples of use.
Qualitative description is a useful qualitative method in much medical research if you keep the limitations of the approach in mind. It is especially relevant in mixed method research, in questionnaire development and in research projects aiming to gain firsthand knowledge of patients', relatives' or professionals' experiences with a particular topic. Another great advantage of the method is that it is suitable if time or resources are limited.
As a consequence of the growth in qualitative research in the health sciences, researchers sometimes feel obliged to designate their work as phenomenology, grounded theory, ethnography or a narrative study when in fact it is not. Qualitative description might be a useful alternative approach to consider.
Sampling is a very complex issue in qualitative research as there are many variations of qualitative sampling described in the literature and much confusion and overlapping of types of sampling, particularly in the case of purposeful and theoretical sampling. The terms purposeful and theoretical are viewed synonomously and used interchangeably in the literature. Many of the most frequent misinterpretations relate to the disparate meanings and usage of the terminology. It is important that the terminology is examined so that underlying assumptions be made more explicit. Lack of shared meanings and terminology in the nursing discourse creates confusion for the neophyte researcher and increases the production of studies with weak methodologies. This paper analyses critically purposeful and theoretical sampling and offers clarification on the use of theoretical sampling for nursing research. The aim is not to make prescriptive statements on sampling; rather, to enhance understanding of the differences between purposeful and theoretical sampling for nursing research.
Objectives
This study aimed to investigate how value is defined and measured in existing value assessment frameworks (VAFs) in healthcare.
Methods
We searched PubMed, Embase, the Cochrane Library, and Centre for Reviews and Dissemination from 2008 to 2019. We also performed backward citation chaining of included studies and previously published systematic reviews. Studies reporting the development of a VAF in healthcare were included. For each included framework, we extracted and compared the context, target users, intended use, methods used to identify value attributes, description of the attributes, and attribute scoring approaches.
Results
Of the 8151 articles screened, 57 VAFs were included. The value attributes included in 55 VAFs were grouped into 9 categories: health benefits (n = 53, 96%), affordability (n = 45, 82%), societal impact (n = 42, 76%), burden of disease (n = 36, 65%), quality of evidence (n = 32, 58%), cost-effectiveness (n = 31, 56%), ethics and equity (n = 27, 49%), unmet needs (n = 21, 38%), and innovation (n = 15, 27%). The remaining 2 VAFs used broad attributes or user-defined attributes. Literature review was the main approach to identify value attributes in 36 VAFs. Patient or public was engaged through the development of only 11 VAFs. Weighting has been used to score 29 VAFs, of which 19 used the methods of multicriteria decision analysis.
Conclusions
There are substantial variations in defining and measuring value. A noticeable weakness of existing VAFs is that patient or public engagement was generally very limited or missing in framework development process. Existing VAFs tend to aggregate multiple value attributes into a single index for decision making.
Objectives
The growing focus on the value of new drugs for patients and society has led to a more differentiated notion of innovation in the context of pharmaceutical products. The goal of this article is to provide an overview of the current debate about the definition and assessment of innovation and how innovation is considered in reimbursement and pricing decisions.
Methods
To compile the relevant literature, we followed a 2-step approach. First, we searched for peer-reviewed literature that deals with the definition of pharmaceutical innovation. Second, we reviewed health technology assessment (HTA) guidelines of 11 selected countries (Australia, Belgium, Canada, England, France, Germany, Italy, Japan, Norway, Sweden, and The Netherlands) regarding aspects of innovation that are currently considered as relevant by the respective HTA bodies.
Results
All countries in our sample use 1 of 2 types of reward mechanism for novel drugs that they consider provide some sort of benefit. Generally, the focus is on the therapeutic benefit of a drug, whereas, depending on the exact arrangement, other aspects can also be taken into account. A reduction in side effects and aspects of treatment convenience can be invoked in some of the countries. Mostly, however, they are not considered unless they are already captured in the clinical outcomes used to measure the therapeutic benefit.
Conclusion
Our review shows that although the health economic literature discusses a range of aspects on how innovation may generate value even without providing an immediate added therapeutic benefit (or on top of it), these are only selectively considered in the reviewed HTA guidelines. For most part, only the added therapeutic value is crucial when it comes to pricing and reimbursement decisions.
Standard cost-effectiveness models compare incremental cost increases to incremental average gains in health, commonly expressed in Quality-Adjusted Life Years (QALYs). Our research generalizes earlier models in several ways. We introduce risk aversion in Quality of Life (QoL), which leads to “willingness-to-pay” thresholds that rise with illness severity, potentially by an order of magnitude. Unlike traditional CEA analyses, which discriminate against persons with disabilities, our analysis implies that the marginal value of improving QoL rises for disabled individuals. Our model can also value the uncertain benefits of medical interventions by employing well-established analytic methods from finance. Finally, we show that traditional QALYs no longer serve as a single index of health, when consumers are risk-averse. To address this problem, we derive a generalized single-index of health outcomes—the Generalized Risk-Adjusted QALY (GRA-QALY). Earlier models of CEA that abstract from risk-aversion nest as special cases of our more general model.
Objectives
To analyze the current development of HTA in China and to identify areas for improvement, we mapped the level of HTA development in China and compared it with the level of HTA development in ten other countries using a survey instrument.
Methods
We launched a nationwide survey targeting different stakeholders. For this purpose, we used a validated instrument that enables mapping HTA development in a country using eight domains. The views of the respondents regarding the overall level of HTA development and for each domain were compared with the results of a mapping study that included ten countries.
Results
In total, we received 222 responses, 33 from policy-makers, 158 from researchers, and 31 from industry, as well as health provider representatives including 8 from hospitals, centers for disease control and prevention. We calculated the mean score for the level of HTA development. The overall HTA development for China was scored at 76.4 (out of a maximum of 146). Although the total score for China was comparable to the mean score of 75.6 among the ten countries, China scored significantly lower than the mean score of 117.0 among the three developed countries. In addition, China scored significantly lower in the domain of institutionalization compared to the other ten countries.
Conclusions
China needs to tackle the issue of low HTA institutionalization to strengthen the foundation of HTA development. Future government initiatives that institutionalize HTA, for example, establishing a national HTA system or consortium, will improve the development of HTA in China.
Objectives
Patient involvement in drug evaluation decision making is increasing. The aim of the current study was to develop a multi-criteria decision analysis (MCDA) framework that would enable the inclusion of the patient perspective in the selection of appropriate criteria for MCDAs being used in the value assessments of oncologic drugs.
Methods
A literature review was conducted to identify and define criteria used in drug assessments from patient perspectives. The Evidence and Value: Impact on Decision Making methodology was used to develop a MCDA framework. Identified criteria were discussed by a sample of oncology patient association representatives who decided which criteria were important from patient perspectives. Selected criteria were rated by importance. The preliminary MCDA framework was tested through the assessment of a hypothetical oncology treatment. A discussion was carried out to agree on a final pilot MCDA framework.
Results
Twenty-two criteria were extracted from the literature review. After criteria discussion, sixteen criteria remained. The most important criteria were comparative patient reported outcomes (PRO), comparative efficacy and disease severity. After the discussion generated by the scoring of the hypothetical oncology treatment, the final pilot MCDA framework included seven quantitative criteria (“disease severity”, “unmet needs”, “comparative efficacy / effectiveness”, “comparative safety / tolerability”, “comparative PROs”, “contribution of oncological innovation”) and one contextual criterion (“population priorities and access”).
Conclusions
The present study developed a pilot reflective MCDA framework that could increase patient's capability to participate in the decision-making process by providing systematic drug assessments from the patient perspective.
China has undertaken waves of healthcare reforms to keep pace with its rapid economic growth. By 2011, universal health insurance coverage was successfully achieved through the creation of a basic social medical insurance system. Growing economic power, extensive government subsidies, and strategies for program implementation are critical to that achievement. However, the breadth and depth of coverage varies considerably across insurance schemes and localities. The disjointed insurance scheme led to inequality in coverage, accessibility, and affordability of medical services, lopsided allocation of health resources, and increasing medical expenditures, and these remain crucial challenges for healthcare insurance coverage. This paper describes societal conditions, polices, achievements and challenges in improving health insurance coverage in China. Thailand's experience in universal health insurance coverage and its implications for China's new medical reform are also discussed. Solutions including sustainable increases in government investment, transformation of payment methods, reinforcement of primary health care delivery and the referral system, and standardization of benefits packages are strongly recommended to address challenges in China's long-running medical reform.
The fifth section of our Special Task Force report identifies and discusses two aggregation issues: 1) aggregation of cost and benefit information across individuals to a population level for benefit plan decision making and 2) combining multiple elements of value into a single value metric for individuals. First, we argue that additional elements could be included in measures of value, but such elements have not generally been included in measures of quality-adjusted life-years. For example, we describe a recently developed extended cost-effectiveness analysis (ECEA) that provides a good example of how to use a broader concept of utility. ECEA adds two features-measures of financial risk protection and income distributional consequences. We then discuss a further option for expanding this approach-augmented CEA, which can introduce many value measures. Neither of these approaches, however, provide a comprehensive measure of value. To resolve this issue, we review a technique called multicriteria decision analysis that can provide a comprehensive measure of value. We then discuss budget-setting and prioritization using multicriteria decision analysis, issues not yet fully resolved. Next, we discuss deliberative processes, which represent another important approach for population- or plan-level decisions used by many health technology assessment bodies. These use quantitative information on CEA and other elements, but the group decisions are reached by a deliberative voting process. Finally, we briefly discuss the use of stated preference methods for developing "hedonic" value frameworks, and conclude with some recommendations in this area.
The third section of our Special Task Force report identifies and defines a series of elements that warrant consideration in value assessments of medical technologies. We aim to broaden the view of what constitutes value in health care and to spur new research on incorporating additional elements of value into cost-effectiveness analysis (CEA). Twelve potential elements of value are considered. Four of them-quality-adjusted life-years, net costs, productivity, and adherence-improving factors-are conventionally included or considered in value assessments. Eight others, which would be more novel in economic assessments, are defined and discussed: reduction in uncertainty, fear of contagion, insurance value, severity of disease, value of hope, real option value, equity, and scientific spillovers. Most of these are theoretically well understood and available for inclusion in value assessments. The two exceptions are equity and scientific spillover effects, which require more theoretical development and consensus. A number of regulatory authorities around the globe have shown interest in some of these novel elements. Augmenting CEA to consider these additional elements would result in a more comprehensive CEA in line with the "impact inventory" of the Second Panel on Cost-Effectiveness in Health and Medicine. Possible approaches for valuation and inclusion of these elements include integrating them as part of a net monetary benefit calculation, including elements as attributes in health state descriptions, or using them as criteria in a multicriteria decision analysis. Further research is needed on how best to measure and include them in decision making.
The potential for multi-criteria decision analysis (MCDA) to support health technology assessment (HTA) has been much discussed, and various HTA agencies are piloting or applying MCDA. Alongside these developments, good practice guidelines for the application of MCDA in health care have been developed. An assessment of current applications of MCDA to HTA in light of good practice guidelines reveals, however, that many have methodologic flaws that undermine their usefulness. Three challenges are considered: the use of additive models, a lack of connection between criteria scales and weights, and the use of MCDA in economic evaluation. More attention needs to be paid to MCDA good practice by researchers, journal editors, and decision makers and further methodologic developments are required if MCDA is to achieve its potential to support HTA. © 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR).
The general view of descriptive research as a lower level form of inquiry has influenced some researchers conducting qualitative research to claim methods they are really not using and not to claim the method they are using: namely, qualitative description. Qualitative descriptive studies have as their goal a comprehensive summary of events in the everyday terms of those events. Researchers conducting qualitative descriptive studies stay close to their data and to the surface of words and events. Qualitative descriptive designs typically are an eclectic but reasonable combination of sampling, and data collection, analysis, and re-presentation techniques. Qualitative descriptive study is the method of choice when straight descriptions of phenomena are desired.
Criteria for determining the trustworthiness of qualitative research were introduced by Guba and Lincoln in the 1980s when they replaced terminology for achieving rigor, reliability, validity, and generalizability with dependability, credibility, and transferability. Strategies for achieving trustworthiness were also introduced. This landmark contribution to qualitative research remains in use today, with only minor modifications in format. Despite the significance of this contribution over the past four decades, the strategies recommended to achieve trustworthiness have not been critically examined. Recommendations for where, why, and how to use these strategies have not been developed, and how well they achieve their intended goal has not been examined. We do not know, for example, what impact these strategies have on the completed research. In this article, I critique these strategies. I recommend that qualitative researchers return to the terminology of social sciences, using rigor, reliability, validity, and generalizability. I then make recommendations for the appropriate use of the strategies recommended to achieve rigor: prolonged engagement, persistent observation, and thick, rich description; inter-rater reliability, negative case analysis; peer review or debriefing; clarifying researcher bias; member checking; external audits; and triangulation.
© The Author(s) 2015.
Decision support is a discipline that is becoming increasingly important in health care decision making. Many jurisdictions are exploring the use of multi-criteria decision analysis (MCDA) as a decision support framework. Indeed, health care decision makers still face complex choices while being urged to provide more comprehensiveness, structure, and transparency to the existing decision-making framework.This paper documents MCDA applications in health care and aims at identifying publication patterns as well as the range of topics to which MCDA have been applied. Therefore, a bibliometric analysis was conducted on articles reporting MCDA applications in health care published from 1960 to 2011. Articles identified through a literature search of health databases were categorized by year of publication, research topics, corresponding authors, country of residence of corresponding authors, and journal titles. The analysis of citation data was conducted in Matheo Analyzer 4.062. Over the time horizon of the analysis, the number of MCDA applications in health care has shown a significant and steady increase, with health care resource allocation being the most prevalent research topic. We also found that the top ten corresponding authors were responsible for 28% of the overall articles, with corresponding authors from the United States being the most prolific. The journal 'Health Economics' ranked first among the top ten journals. The results of this bibliometric analysis are concordant with the overall publication trends of MCDA methods described in other fields. Further research is needed, within jurisdictions, to select the most appropriate MCDA method to be applied to health care.
The objective of this study is to support those undertaking a multi-criteria decision analysis (MCDA) by reviewing the approaches adopted in healthcare MCDAs to date, how these varied with the objective of the study, and the lessons learned from this experience. Searches of EMBASE and MEDLINE identified 40 studies that provided 41 examples of MCDA in healthcare. Data were extracted on the objective of the study, methods employed, and decision makers' and study authors' reflections on the advantages and disadvantages of the methods. The recent interest in MCDA in healthcare is mirrored in an increase in the application of MCDA to evaluate healthcare interventions. Of the studies identified, the first was published in 1990, but more than half were published since 2011. They were undertaken in 18 different countries, and were designed to support investment (coverage and reimbursement), authorization, prescription, and research funding allocation decisions. Many intervention types were assessed: pharmaceuticals, public health interventions, screening, surgical interventions, and devices. Most used the value measurement approach and scored performance using predefined scales. Beyond these similarities, a diversity of different approaches were adopted, with only limited correspondence between the approach and the type of decision or product. Decision makers consulted as part of these studies, as well as the authors of the studies are positive about the potential of MCDA to improve decision making. Further work is required, however, to develop guidance for those undertaking MCDA.
Objectives:
This study describes the health technology assessment (HTA) framework introduced by Regione Lombardia to regulate the introduction of new technologies. The study outlines the process and dimensions adopted to prioritize, assess and appraise the requests of new technologies.
Methods:
The HTA framework incorporates and adapts elements from the EUnetHTA Core Model and the EVIDEM framework. It includes dimensions, topics, and issues provided by EUnetHTA Core Model to collect data and process the assessment. Decision making is instead supported by the criteria and Multi-Criteria Decision Analysis technique from the EVIDEM consortium.
Results:
The HTA framework moves along three process stages: (i) prioritization of requests, (ii) assessment of prioritized technology, (iii) appraisal of technology in support of decision making. Requests received by Regione Lombardia are first prioritized according to their relevance along eight dimensions (e.g., costs, efficiency and efficacy, organizational impact, safety). Evidence about the impacts of the prioritized technologies is then collected following the issues and topics provided by EUnetHTA Core Model. Finally, the Multi-Criteria Decision Analysis technique is used to appraise the novel technology and support Regione Lombardia decision making.
Conclusions:
The VTS (Valutazione delle Tecnologie Sanitarie) framework has been successfully implemented at the end of 2011. From its inception, twenty-six technologies have been processed.
Healthcare organizations the world over are faced with having to set priorities and allocate resources within the constraint of a fixed envelope of funding. Drawing on economic principles of value for money and ethical principles of fair process, a priority setting framework was developed for Ontario's local health integration networks (LHINs) in late 2007 and early 2008. Subsequently, over an 18-month period, the framework was piloted in three LHINs. In this article, the framework and pilot implementations are described, results from a formal evaluation are outlined and recommendations for future use are highlighted.
This article is the first of a series providing guidance for use of the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) system of rating quality of evidence and grading strength of recommendations in systematic reviews, health technology assessments (HTAs), and clinical practice guidelines addressing alternative management options. The GRADE process begins with asking an explicit question, including specification of all important outcomes. After the evidence is collected and summarized, GRADE provides explicit criteria for rating the quality of evidence that include study design, risk of bias, imprecision, inconsistency, indirectness, and magnitude of effect. Recommendations are characterized as strong or weak (alternative terms conditional or discretionary) according to the quality of the supporting evidence and the balance between desirable and undesirable consequences of the alternative management options. GRADE suggests summarizing evidence in succinct, transparent, and informative summary of findings tables that show the quality of evidence and the magnitude of relative and absolute effects for each important outcome and/or as evidence profiles that provide, in addition, detailed information about the reason for the quality of evidence rating. Subsequent articles in this series will address GRADE's approach to formulating questions, assessing quality of evidence, and developing recommendations.
Payment systems for health care today are based on rewarding volume, not value for the money spent. Two proposed methods of payment, "episode-of-care payment" and "comprehensive care payment" (condition-adjusted capitation), could facilitate higher quality and lower cost by avoiding the problems of both fee-for-service payment and traditional capitation. The most appropriate payment systems for different types of patient conditions and some methods of addressing design and implementation issues are discussed. Although the new payment systems are desirable, many providers are not organized to accept or use them, so transitional approaches such as "virtual bundling," described in this paper, will be needed.
Looking across boarders reveals that the national immunization programs of various countries differ in their vaccination schedules and decisions regarding the implementation and funding of new vaccines. The aim of this review is to identify decision aids and crucial criteria for a rational decision-making process on vaccine introduction and to develop a theoretical framework for decision-making based on available literature.
Systematic literature search supplemented by hand-search.
We identified five published decision aids for vaccine introduction and program planning in industrialized countries. Their comparison revealed an overall similarity with some differences in the approach as well as criteria. Burden of disease and vaccine characteristics play a key role in all decision aids, but authors vary in their views on the significance of cost-effectiveness analyses. Other relevant factors that should be considered before vaccine introduction are discussed to highly differing extents. These factors include the immunization program itself as well as its conformity with other programs, its feasibility, acceptability, and equity, as well as ethical, legal and political considerations. Assuming that the most comprehensive framework possible will not provide a feasible tool for decision-makers, we suggest a stepwise procedure.
Though even the best rational approach and most comprehensive evaluation is limited by remaining uncertainties, frameworks provide at least a structured approach to evaluate the various aspects of vaccine implementation decision-making. This process is essential in making consistently sound decisions and will facilitate the public's confidence in the decision and its realization.
To guide the Ministry of Health in Ghana in the priority setting of interventions by quantifying the trade-off between equity, efficiency, and other societal concerns in health.
The study applied a multicriteria decision analytical framework. A focus group of seven policymakers identified the relevant criteria for priority setting and 63 policymakers participated in a discrete choice experiment to weigh their relative importance. Regression analysis was used to rank order a set of health interventions on the basis of these criteria and associated weights.
Policymakers in Ghana consider targeting of vulnerable populations and cost-effectiveness as the most important criteria for priority setting of interventions, followed by severity of disease, number of beneficiaries, and diseases of the poor. This translates into a general preference for interventions in child health, reproductive health, and communicable diseases.
Study results correspond with the overall vision of the Ministry of Health in Ghana, and are instrumental in the assessment of present and future investments in health. Multicriteria decision analysis contributes to transparency and accountability in policymaking.
In the preceding article, Weinstein et al. [1] explain the QALYconcept, its methods, and their underlying assumptions. Anumber of interesting themes for discussion arise from thisfactual presentation. We restrict ourselves to addressing fourissues that we deem particularly challenging.As a general background for our selection of issues, we reit-erate a basic point about valuation perspectives: StandardQALYs are meant to express the personal utility of health out-comes as judged ex ante and “on average” by the general publicfrom behind a veil of ignorance about future health (so-called“decision utility”). Standard QALYs thus express value in termsof ex ante self-interest. There are, however, possible alternatives.First, health state utilities may in principle be elicited ex postrather than ex ante, i.e., from people who have or have had directexperience with the health states that are the object of the valu-ation (so-called “experienced utility”). Second, QALYs may beconstructed to express society’s valuation of health outcomeswhen not only self-interest but also concerns for fairness aretaken into account. The choice of approach depends on thequestion one wishes to answer, and the choice of health statevaluation techniques depends on the choice of perspective.The issues we address in the following reflect the aboveplurality in possible valuation perspectives and are in part inde-pendent of each other. One issue is intermethod variation in theestimation of ex ante health state utilities. A second is the exist-ence of unwillingness to trade lifetime in elicitations of experi-enced utility. A third is the discrepancy between aggregateindividual utility of health programs on the one hand and, on theother hand, societal valuations that include concerns for fairness.A fourth is a hitherto much overlooked distinction betweenhealthy individuals’ valuations of states of illness (which
The role of economic evaluation in the efficient allocation of healthcare resources has been widely debated. Whilst economic evidence is undoubtedly useful to purchasers, it does not address the issue of affordability which is an increasing concern. Healthcare purchasers are concerned not just with maximising efficiency but also with the more simplistic goal of remaining within their annual budgets. These two objectives are not necessarily consistent.
This paper examines the issue of affordability, the relationship between affordability and efficiency and builds the case for why there is a growing need for budget impact models to complement economic evaluation. Guidance currently available for such models is also examined and it is concluded that this guidance is currently insufficient. Some of these insufficiencies are addressed and some thoughts on what constitutes best practice in budget impact modelling are suggested. These suggestions include consideration of transparency, clarity of perspective, reliability of data sources, the relationship between intermediate and final end-points and rates of adoption of new therapies. They also include the impact of intervention by population subgroups or indications, reporting of results, probability of re-deploying resources, the time horizon, exploring uncertainty and sensitivity analysis, and decision-maker access to the model. Due to the nature of budget impact models, the paper does not deliver stringent methodological guidance on modelling. The intention was to provide some suggestions of best practice in addition to some foundations upon which future research can build.
This paper is a description of inductive and deductive content analysis.
Content analysis is a method that may be used with either qualitative or quantitative data and in an inductive or deductive way. Qualitative content analysis is commonly used in nursing studies but little has been published on the analysis process and many research books generally only provide a short description of this method.
When using content analysis, the aim was to build a model to describe the phenomenon in a conceptual form. Both inductive and deductive analysis processes are represented as three main phases: preparation, organizing and reporting. The preparation phase is similar in both approaches. The concepts are derived from the data in inductive content analysis. Deductive content analysis is used when the structure of analysis is operationalized on the basis of previous knowledge.
Inductive content analysis is used in cases where there are no previous studies dealing with the phenomenon or when it is fragmented. A deductive approach is useful if the general aim was to test a previous theory in a different situation or to compare categories at different time periods.