Healthcare Resource Allocation Decisions Research Articles

'Real-world' health care priority setting using explicit decision criteria: a systematic review of the literature.

BackgroundHealth care decision making requires making resource allocation decisions among programs, services, and technologies that all compete for a finite resource pool. Methods of priority setting that use explicitly defined criteria can aid health care decision makers in arriving at funding decisions in a transparent and systematic way. The purpose of this paper is to review the published literature and examine the use of criteria-based methods in ‘real-world’ health care allocation decisions.MethodsA systematic review of the published literature was conducted to find examples of ‘real-world’ priority setting exercises that used explicit criteria to guide decision-making.ResultsWe found thirty-three examples in the peer-reviewed and grey literature, using a variety of methods and criteria. Program effectiveness, equity, affordability, cost-effectiveness, and the number of beneficiaries emerged as the most frequently-used decision criteria. The relative importance of criteria in the ‘real-world’ trials differed from the frequency in preference elicitation exercises. Neither the decision-making method used, nor the relative economic strength of the country in which the exercise took place, appeared to have a strong effect on the type of criteria chosen.ConclusionsHealth care decisions are made based on criteria related both to the health need of the population and the organizational context of the decision. Following issues related to effectiveness and affordability, ethical issues such as equity and accessibility are commonly identified as important criteria in health care resource allocation decisions.Electronic supplementary materialThe online version of this article (doi:10.1186/s12913-015-0814-3) contains supplementary material, which is available to authorized users.

How Well Do the Generic Multi-attribute Utility Instruments Incorporate Patient and Public Views Into Their Descriptive Systems?

Multi-attribute utility instruments (MAUIs) are increasingly being used to generate utility data, which can be used to calculate quality-adjusted life-years (QALYs). These QALY data can then be incorporated into a cost-utility analysis as part of an economic evaluation, to inform health care resource allocation decisions. Many health care decision-making bodies around the world, such as the National Institute for Health and Care Excellence, require the use of generic MAUIs. Recently, there has been a call for greater input of patients into the development of patient-reported outcome measures, and this is now actively encouraged. By incorporating the views of patients, greater validity of an instrument is expected and it is more likely that patients will be able to self-complete the instrument, which is the ideal when obtaining information about a patient's health-related quality of life. This paper examines the stages of MAUI development and the scope for patient and/or public involvement at each stage. The paper then reviews how much the main generic MAUIs have incorporated the views of patients/the public into the development of their descriptive systems at each of these stages, and the implications of this. The review finds that the majority of MAUIs had very little input from patients/the public. Instead, existing literature and/or the views of experts were used. If we wish to incorporate patient/public views into future development of MAUIs, qualitative methods are recommended.

Transparency in Canadian public drug advisory committees

BackgroundTransparency in health care resource allocation decisions is a criterion of a fair process. We used qualitative methods to explore transparency across 11 Canadian drug advisory committees. MethodsWe developed seven criteria to assess transparency (disclosure of members’ names, disclosure of membership selection criteria, disclosure of conflict of interest guidelines and members’ conflicts, public posting of decisions not to fund drugs, public posting of rationales for decisions, stakeholder input, and presence of an appeals mechanism) and two sub-criteria for when rationales were posted (direct website link and readability). We interviewed a purposeful sample of key informants who were conversant in English and a current or past member of either a committee or a stakeholder group. We analyzed data using a thematic approach. Interviewing continued until saturation was reached. ResultsWe examined documents from 10 committees and conducted 27 interviews. The median number of criteria addressed by committees was 2 (range 0–6). Major interview themes included addressing: (1) accessibility issues, including stakeholders’ degree of access to the decision making process and appeal mechanisms; (2) communication issues, including improving internal and external communication and public access to information; and (3) confidentiality issues, including the use of proprietary evidence. ConclusionMost committees have some mechanisms to address transparency but none had a fully transparent process. The most important ways to improve transparency include creating formal appeal mechanisms, improving communication, and establishing consistent rules about the use of, and public access to, proprietary evidence.

Cost-effectiveness analysis of interventions for tuberculosis control: DALYs versus QALYs.

The emergence of multi-drug-resistant tuberculosis (MDR-TB) in the European region and the high costs (nearly <euro>536 million) generated by the nearly 72,000 notified TB cases in the EU are the factors driving the need for development and implementation of new tools against TB. In this context, cost-effectiveness analyses applying quality-adjusted life-years (QALYs) or disability-adjusted life-years (DALYs) as outcome measures for economic evaluation of improved approaches to TB control are increasingly important. While the methodology applied to derive the effectiveness data is commonly reported, less information is given regarding the derivation of utility weights in the calculation of QALYs for TB treatment. To date, despite the particular complexities of the disease, TB health effects have not been fully measured and there is no agreement on how disutility of TB disease should be accounted for. Consequently, disutility values in published studies vary considerably, and often appear to lack empirical evidence. As the need for a solid heath-economics rationale for investment in new tools against TB grows, adequate and comprehensive methods for assessing the impairments caused by different types of TB must be developed. Focusing on the assessment of DALYs as a measure of outcome in economic evaluation, we have built an exemplary model calculation applying the original TB data for Germany as reported to the Robert Koch Institute. Our work demonstrates that the use of standard equations provided in the scientific literature probably results in an underestimation of lost DALYs compared with probabilistic techniques. Providing distributions around epidemiological averages, coupled with Monte Carlo simulation to address uncertainty, may result in more realistic values. In line with a previous recommendation by the World Health Organization, it appears worthwhile to consider this more intricate approach to providing healthcare resource allocation decisions, particularly for TB.

PHP94 - Basic Attitude Towards Health Care Resource Allocation Decision Making in Japanese People -Utilitarianism or Egalitarianism?

PHP94 - Basic Attitude Towards Health Care Resource Allocation Decision Making in Japanese People -Utilitarianism or Egalitarianism?

RESOURCE ALLOCATION FOR THE TREATMENT OF RARE DISEASES

IntroductionDrugs for rare diseases are often associated with extremely high costs which can be a barrier to patients' accessibility. Due to scarce healthcare resources, funding for expensive orphan therapies is...

Can the NICE "End-of-Life Premium" Be Given a Coherent Ethical Justification?

In 2009 the UK National Institute for Health and Clinical Excellence (NICE) announced that its health technology appraisal committees would henceforth give special additional weight to health gains from life-extending end-of-life treatments. This was a response to mounting concern from NICE's stakeholders that effective new drugs for end-stage cancer often fail NICE's standard test of cost effectiveness. This change of policy may be justifiable on procedural grounds as the result of a democratic political process responding to stakeholder concerns. However, according to the "accountability for reasonableness" framework proposed by the philosopher Norman Daniels and endorsed by NICE, there also needs to be transparency about the substantive ethical grounds for public health care resource allocation decisions. In that spirit, I analyze eleven potentially relevant justifications for the NICE "end-of-life premium," drawn from the economics and philosophy literature: (1) rule of rescue, (2) fair chances, (3) ex post willingness to pay, (4) caring externality, (5) financial protection, (6) symbolic value, (7) diminishing marginal value of future life years, (8) concentration of benefits, (9) dread, (10) time to set your affairs in order, and (11) severity of illness. I conclude that none of them yields a coherent ethical justification for the NICE end-of-life premium.

Interview: Setting up NICE International

Kalipso Chalkidou is the founding director of the NICE's international program helping governments build technical and institutional capacity for using evidence to inform health policy. She is interested in how comparative effectiveness evidence, combined with local expertise and local institutions, can drive scientific and legitimate healthcare resource allocation decisions. She has been involved in the Chinese rural health reform and also in national health reform projects in Colombia, Turkey and the Middle East, working with the World Bank, Pan American Health Organization (PAHO), Department For International Development (DFID) and the Inter-American Development Bank, as well as national governments. She holds a doctorate on the molecular biology of prostate cancer from the University of Newcastle (UK), an MD with Honors from the University of Athens (Greece) and is an honorary senior lecturer at the London School of Hygiene and Tropical Medicine (UK), a senior advisor on international policy at the Center for Medical Technology Policy (MD, USA) and a visiting faculty member at the Johns Hopkins Berman Institute for Bioethics (MD, USA). Between 2007 and 2008, she spent a year at the Johns Hopkins School of Public Health as a Harkness Fellow in Health Policy and Practice, studying how comparative effectiveness research can inform policy and US government drug pricing policies.

Impact of one or two visits strategy on hypertension burden estimation in HYDY, a population-based cross-sectional study: implications for healthcare resource allocation decision making

ContextThe prevalence of hypertension in developing countries is coming closer to values found in developed countries. However, surveys usually rely on readings taken at a single visit, the option to...

Elicitation of Informed General Population Health State Utility Values: A Review of the Literature

Objective Within a health-care decision-making context, whose health state utility values (HSUVs) should be used is constantly debated. This discussion is important because patient and general population utilities can differ. These discrepancies may be due to the general population not being informed about the health states. This article investigates approaches used to inform the general population about the health states that they are valuing. Methods Studies reporting methods to obtain informed general population HSUVs were identified, outlined, and critically appraised. Results Fourteen studies were identified: seven used information sessions, two used simulation methods, two used reflection and deliberation techniques, and three used adaptation exercises. Conclusions This review demonstrated a range of approaches to elicit informed general population HSUVs. The majority of the studies indicated that informing the respondents significantly affected their opinions of the health states and hence their HSUVs. This suggests that the utilities that are currently used to guide health-care resource allocation decisions may not represent the general population's preferences for specific health states. This could result in decisions that do not maximize societal health benefits.

Challenges in Health State Valuation in Paediatric Economic Evaluation

With the growth in the use of health economic evaluation to inform healthcare resource allocation decisions, the challenges in applying standard methods to child health have become apparent. A unique limitation is the paucity of child-specific preference-based measures. A single, valid, preference-based measure of utility that can be used in children of all ages does not exist. Thus, the ability to derive a QALY for use in cost-utility analysis (CUA) is compromised. This paper presents and discusses existing and novel options for deriving utilities for paediatric health states for use in CUAs. While a direct elicitation may be preferred, a child's ability to complete a standard gamble or time trade-off task is hampered by cognitive and age limitations. The abstract notions contained in indirect instruments such as the EQ-5D and Health Utilities Index may also pose challenges for young children. Novel approaches to overcome these challenges include the development of age-appropriate instruments such as the EQ-5D-Y, the development of new child-specific utility instruments such as the Child Health Utility-9D and the re-calibration of existing adult instruments to derive preference weights for health states from children themselves. For children aged <6 years, researchers have little choice but to use a proxy reporter such as parents. While parents may be reliable reporters for physical activity limitations and externally manifest symptoms, their ability to accurately report on subjective outcomes such as emotion is questionable. Catalogues of utility weights for a range of conditions are increasingly becoming available but retain many of the same limitations as valuing health states from children or from proxies. Given the dynamic relationship in quality of life (QOL) between family members when a child is ill, it seems appropriate to consider a 'family perspective' rather than an individual perspective in child health state valuation. In a collective approach, health state utilities derived from multiple family members may be combined mathematically. Alternatively, in a unitary approach, a single utility estimate may be determined to represent the family's perspective. This may include deriving utilities through parent-child dyad estimation or by using a household model that combines the utility weights of the patient and family members, incorporating reciprocal QOL effects. While these various approaches to child health state valuation represent novel research developments, the measurement challenges and threats to validity persist. Given the importance of non-health benefits to child health, especially in the domains of education and public policy, it may be worthwhile to consider an approach that allows incorporation of externalities to produce a cost-benefit analysis. The use of discrete-choice methods to assess willingness to pay for novel child health interventions holds promise as a means to produce meaningful economic evidence. Regardless of the approach taken, the highest degree of methodological rigour is essential. The increasing attention being paid by health economic researchers to the measurement challenges of paediatric health state valuation can only increase the value of child health economic evidence for decision making.

The Appropriate Elicitation of Expert Opinion in Economic Models

Decision-analytic modelling has become an indispensible method to inform healthcare resource allocation decision making. Prospective randomized economic trials can produce internally robust cost and outcome data. However, such prospective data are typically unavailable or insufficient for the purpose of guiding national-level resource allocation decisions because results are not generalizable outside the trial study population or design. Using an economic model allows a decision analyst to carefully structure the research question and assimilate all available data. Additionally, outcome data from trials with short timeframes can be extrapolated to long-term estimates. Modelling also has a key role in prioritizing and planning future trials and research. Ideally, a model should be structured to represent all clinical pathways relevant to the decision problem. Methods guidelines state that data availability should not be a reason for rejecting a model or even an overriding factor when defining model structure. However, more often than not, the model cannot be populated using published, robust trial or observational data. Alternative data sources are then needed. Expert opinion is a recognised potential source of data and is often used to fill in data gaps and/or supplement the trial and observational data. Data elicited from experts is appropriately viewed as being second best and a potential source of parameter uncertainty. This is in part due to experts’ limited experience and perspective but also, importantly, because the methods used to identify the experts and elicit their opinion is often neither conducted robustly nor reported explicitly. Guidelines for the selection, design and population of economic models are necessary to encourage the transparent and explicit reporting of methods. Previous commentators have already noted that the methods used to elicit expert opinion for health technology assessments should also be reported explicitly. Existing methods guidelines do allude to the need for explicit reporting of expert opinion elicitation methods but do not go into methodological detail about the different elicitation methods available and their application. This commentary describes the potential use of the Delphi method as an elicitation technique. It clarifies that there are different types of Delphi that can be used, with quite distinct research and policy applications and also outlines where other expert elicitation methods might be more appropriate when focussing on the primary data requirements for economic modelling.

The (Possible) Impact of Comparative Effectiveness Research on Pharmaceutical Industry Decision Making

Public and private payers in rich and emerging economies are becoming increasingly interested in using evidence to inform health-care resource allocation decisions. The National Institute for Health and Clinical Excellence (NICE) in the United Kingdom has been doing so on behalf of the National Health Service (NHS) for more than 10 years. To some, NICE-type entities are barriers to access and innovation. Increasingly, however, even NICE's critics appreciate that evidence-informed decision making, carried out in a fair, inclusive, and transparent way, is better than arbitrary government-imposed price cuts. For health-care systems in developed and developing countries around the world, faced with limited budgets, there may be no third option.

Developing a descriptive system for a new preference-based measure of health-related quality of life for children

The use of preference-based measures (PBMs) of health-related quality of life (HRQoL) is increasing in health care resource allocation decisions. Whilst there are measures widely used for this purpose in adults, research in the paediatric field is more limited. This paper reports on how the descriptive system for a new paediatric generic PBM of HRQoL was developed from dimensions identified in previous research. Existing scales from the paediatric literature were reviewed for suitability, and scales were also developed empirically, based on qualitative interview data from children, by taking adverbial phrases and confirming the ordinality by a ranking exercise with children. The resulting scales were applied to the dimensions from the previous research. No suitable scales were found in the paediatric literature, so the empirically derived scales were used resulting in seven different types. Children were successfully able to rank these to determine the ordinality, and these types were applied to the dimensions. This work has empirically developed a descriptive system for the dimensions of HRQoL identified in previous research. Further research is needed to test the descriptive system on a paediatric population and reduce the number of dimensions to be amenable to health state valuation.

Aversion to geographic inequality and geographic variation in preferences in the context of healthcare

It is now widely accepted that social values have a role to play in terms of healthcare resource allocation decisions. Furthermore, with the recent focus on decentralization, geographic variation in preferences has become a pertinent issue. However, the geographic dimension of inequality in the distribution of health gains across different regions has received little attention in the literature. To elicit the general public's preferences regarding inequality in the distribution of health gains across regions and to test the assumption of variation in preferences across regions. A questionnaire was developed to elicit social and personal preferences. The first preference questions focused on the criteria of maximization of health gain and geographic equality of health gain. Person trade-off questions were used to assess trade-offs and inequality aversion. The latter preference questions focused on attributes of healthcare delivery. The questionnaire was administered in two Portuguese municipalities. Samples from both localities were defined by quotas based on the composition of local populations. Overall, 70 respondents (half from each municipality) self-completed the questionnaire. Respondents agreed or strongly agreed that both maximization of health gain and geographic equality of health gain should be considered in resource allocation decisions; when they were asked to make trade-offs, 70-80% were prepared to do so. Of those making trade-offs, most people were willing to forego 10-30% of total health gain to keep geographic equality of health gain in return. Regarding inequality aversion, the results indicate that there was some aversion to inequality in the sample. Regarding preference variation, the results do not corroborate the hypothesis of variation in preferences across regions. These results suggest that the general public supports the principles of maximization of health gain and geographic equality in the distribution of health gain. The results further suggest the existence of aversion to inequality, subject to a maximum opportunity cost. Further research is required into preference variation; in particular, in larger samples, and in other regions of Portugal.

QALYs: The Basics

The aim of this article is to review the concept of the qualityadjusted life-year (QALY), a widely used measure of health improvement that is used to guide health-care resource allocation decisions. The QALY was originally developed as a measure of health effectiveness for cost-effectiveness analysis, a method intended to aid decision-makers charged with allocating scarce resources across competing health-care programs [1–3]. We refer to this original concept of the QALY, as defined in the early literature, as the “conventional” QALY, recognizing that alternative conceptual models have been proposed, including but not limited to so-called “equity-weighted” QALYs. The US Panel on Cost-Effectiveness in Health and Medicine [4] and the National Institute of Health and Clinical Excellence (NICE) in Britain have both endorsed the conventional QALY for their “reference case,” i.e., a standardized methodological approach to promote comparability in cost-effectiveness analyses of different health-care interventions. In using QALYs, we assume that a major objective of decisionmakers is to maximize health or health improvement across the population subject to resource constraints. The use of QALYs further assumes that health or health improvement can be measured or valued based on amounts of time spent in various health states. The conventional QALY is therefore a valuation of health benefit. We note, however, that decision-makers may also have other objectives such as equity, fairness, and political goals, all of which currently must be handled outside the conventional Special Issue [5] addresses some of these variations on the conventional QALY. The QALY was not initially developed to aid individual patient decision-making, although its use has sometimes been extended into clinical decision analyses for this purpose. The core concept of the conventional QALY is grounded in decision science and expected utility theory. The basic construct is that individuals move through health states over time and that each health state has a value attached to it. Health, which is what we are seeking to maximize, is defined as the value-weighted time—life-years weighted by their quality—accumulated over the relevant time horizon to yield QALYs. Health states must be valued on a scale where the value of being dead must be 0, because the absence of life is considered to be worth 0 QALYs. By convention, the upper end of the scale is defined as perfect health, with a value of 1. To permit aggregation of QALY changes, the value scale should have interval scale properties such that, for example, a gain from 0.2 to 0.4 is equally valuable as a gain from 0.6 to 0.8. States worse than dead can exist and they would have a negative value and subtract from the number of QALYs. These conditions, along with an assumption of risk neutrality over life-years, are sufficient to ensure that the QALY is a useful representation of health state preferences.

A Difficult Balancing Act: Policy Actors' Perspectives on Using Economic Evaluation to Inform Health-Care Coverage Decisions under the Universal Health Insurance Coverage Scheme in Thailand

Objectives In Thailand, policymakers have come under increasing pressure to use economic evaluation to inform health-care resource allocation decisions, especially after the introduction of the Universal Health Insurance Coverage (UC) scheme. This article presents qualitative findings from research that assessed a range of policymakers' perspectives on the acceptability of using economic evaluation for the development of health-care benefit packages in Thailand. The policy analysis examined their opinions about existing decision-making processes for including health interventions in the UC benefit package, their understanding of health economic evaluation, and their attitudes, acceptance, and values relating to the use of the method. Methods Semistructured interviews were conducted with 36 policy actors who play a major role or have some input into health resource allocation decisions within the Thai health-care system. These included 14 senior policymakers at the national level, 5 hospital directors, 10 health professionals, and 7 academics. Results and Conclusions Policy actors thought that economic evaluation information was relevant for decision-making because of the increasing need for rationing and more transparent criteria for making UC coverage decisions. Nevertheless, they raised several difficulties with using economic evaluation that would pose barriers to its introduction, including distrust in the method, conflicting philosophical positions and priorities compared to that of “health maximization,” organizational allegiances, existing decision-making procedures that would be hard to change, and concerns about political pressure and acceptability.

Accuracy of terminology and methodology in economic analyses in otolaryngology

Objective: Economic studies increasingly guide health care resource allocation decisions. Because rigorous adherence to accepted definitions and research techniques is critical to ensure accuracy, we evaluated the terminology and methods of otolaryngology economic analyses. Study design: A total of 71 articles published from 1990 to 1999 in 6 peer-reviewed otolaryngology journals with terms such as “cost-effective” in their title or representing economic analyses were reviewed for terminology and use of established methodology guidelines. Results: Over half (35 of 66) of terms such as “cost-effective” were used incorrectly, and 60% of articles (39 of 64) confused “charge” and “cost” data. Eleven percent (7 of 64) of papers specified the perspective of their analysis. About half (17 of 30) reported a summary measure such as a cost-effectiveness ratio. Only one third (23 of 63) performed sensitivity analyses. Conclusion: Adherence to accepted definitions and research methods is inconsistent, although we did note moderate improvements in making the distinction between costs and charges, defining of study perspective, and performing sensitivity analysis. Significance: Greater attention to both terminology and methodology can enhance the quality of economic analyses and ultimately improve certain resource allocation decisions. (Otolaryngol Head Neck Surg 2001;124:496-502.)

The Role of Economic Evaluation in the Diagnosis and Treatment of Helicobacter PyloriInfection

The unfolding of the H. pylori story coupled with the high prevalence and economic burden associated with upper gastrointestinal symptoms makes H. pylori-associated disorders an ideal candidate for economic evaluation. As a result of the high quality and quantity of data emerging, H. pylori eradication is cost-effective in individuals with either newly diagnosed or past PUD. The role of eradication in other areas, for example, patients with nonulcer dyspepsia and screening to prevent gastric cancer, may never be worked out to some clinicians' satisfaction. Conflicting reports and the lack of definitive clinical trials have frustrated clinicians. H. pylori diagnosis and treatment have not been integrated into everyday clinical practice. As a result, the enormous potential benefits associated with H. pylori eradication have not been achieved. Economic evaluation is not a panacea to the problems confronting medical services delivery. Assessments of medical practice are methodologically challenging, time-consuming, and expensive. Once the answers are in, it remains difficult to alter an individual provider's patterns of care and integrate the research findings into everyday practice. Further research into physician decision making is necessary to complement the advances being made in determining the value of medical interventions. The fruits of these efforts will be the more efficient delivery of health care services, which, it is hoped, ultimately will improve the health of patients. There is no reason to believe, however, that more attention to effectiveness research would lead to a reduction in health care expenditures. Increasing demands for accountability of medical interventions will propel effectiveness movements. Economic analyses, performed in concert with carefully designed clinical studies, will spur more critical review of health care resource allocation decisions. Despite the limitations, the findings generated from effectiveness research will have a major impact on physician practices, guideline development, and reimbursement decisions. The tendency of clinicians not to pay attention to economic evaluations may lead to missed clinical benefits and unnecessary expenditures. Physicians who have been reluctant to appreciate the information provided by economic evaluation must understand what other stakeholders in health care delivery already have accepted: that the rational basis for cost-effectiveness analysis is the overall improvement in the quality of health care services--not simply a tool to reduce health care costs.

Building uncertainty into cost-effectiveness rankings: portfolio risk-return tradeoffs and implications for decision rules.

Current principles of cost-effectiveness analysis emphasize the rank ordering of programs by expected economic return (eg, quality-adjusted life-years gained per dollar expended). This criterion ignores the variance associated with the cost-effectiveness of a program, yet variance is a common measure of risk when financial investment options are appraised. Variation in health care program return is likely to be a criterion of program selection for health care managers with fixed budgets and outcome performance targets. Characterizing health care resource allocation as a risky investment problem, we show how concepts of portfolio analysis from financial economics can be adopted as a conceptual framework for presenting cost-effectiveness data from multiple programs as mean-variance data. Two specific propositions emerge: (1) the current convention of ranking programs by expected return is a special case of the portfolio selection problem in which the decision maker is assumed to be indifferent to risk, and (2) for risk-averse decision makers, the degree of joint risk or covariation in cost-effectiveness between programs will create incentives to diversify an investment portfolio. The conventional normative assumption of risk neutrality for social-level public investment decisions does not apply to a large number of health care resource allocation decisions in which health care managers seek to maximize returns subject to budget constraints and performance targets. Portfolio theory offers a useful framework for studying mean-variance tradeoffs in cost-effectiveness and offers some positive predictions (and explanations) of actual decision making in the health care sector.