Social Decision-Making Analysis: A General Approach to Inform Decisions on Resources in the Public Sector.

OBJECTIVES: Public expenditure aims to achieve social objectives by improving a range of socially valuable attributes of benefit (arguments in a social welfare function). Public expenditure is typically allocated to public sector budgets, where budget holders are tasked with meeting a subset of social objectives. METHODS: Decision makers require an evidence-based assessment of whether a proposed investment is likely to be worthwhile given existing levels of public expenditure. However, others also require some assessment of whether the overall level and allocation of public expenditure are appropriate. This article proposes a more general theoretical framework for economic evaluation that addresses both these questions. RESULTS: Using a stylized example of the economic evaluation of a new intervention in a simplified UK context, we show that this more general framework can support decisions beyond the approval or rejection of single projects. It shows that broader considerations about the level and allocation of public expenditure are possible and necessary when evaluating specific investments, which requires evidence of the range of benefits offered by marginal changes in different types of public expenditure and normative choices of how the attributes of benefit gained and forgone are valued. CONCLUSIONS: The proposed framework shows how to assess the value of a proposed investment and whether and how the overall level of public expenditure and its allocation across public sector budgets might be changed. It highlights that cost-benefit analysis and cost-effectiveness analysis can be viewed as special cases of this framework, identifying the weakness with each.

Achieving dynamic efficiency in pharmaceutical innovation: Identifying the optimal share of value and payments required.

It has been argued that cost-effectiveness analysis of branded pharmaceuticals only considers static efficiency, neglects dynamic effects and undermines incentives for socially valuable innovation. We present a framework for designing pharmaceutical pricing policy to achieve dynamic efficiency. We develop a coherent framework that identifies the long-term static and dynamic benefits and costs of offering manufacturers different levels of reward. The share of value that would maximise long-term population health depends on how the quantity and quality of innovation responds to payment. Using evidence of the response of innovation to payment, the optimal share of value of new pharmaceuticals to offer to manufacturers is roughly 20% (range: 6%-51%). Reanalysis of a sample of NICE technology appraisals suggests that, in most cases, the share of value offered to manufacturers and the price premium paid by the English NHS were too high. In the UK, application of optimal shares would offer considerable benefits under both a public health objective and a broader view of social welfare. We illustrate how an optimal share of value can be delivered through a range of payment mechanisms including indirect price regulation via the use of different approval norms by an HTA body.

Does public long-term care expenditure improve care-related quality of life of service users in England?

Public long-term care (LTC) systems provide services to support people experiencing difficulties with their activities of daily living. This study investigates the marginal effect of changes in public LTC expenditure on care-related quality of life (CRQoL) of existing service users in England. The public LTC program for people aged 18 or older in England is called Adult Social Care (ASC) and it is provided and managed by local authorities. We collect data on the outcomes and characteristics of public ASC users, on public ASC expenditure, and on the characteristics of local authorities across England in 2017/18. We employ an instrumental variable approach using conditionally exogenous elements of the public funding system to estimate the effect of public ASC expenditure on user CRQoL. Our findings show that by increasing public ASC expenditure by £1000 per user, on average, local authorities increase user CRQoL by 0.0030. These results suggest that public ASC is effective in increasing users' quality of life but only to a relatively small extent. When combined with the other potential effects of LTC expenditure (e.g., on informal carers, mortality), this study can inform policy makers in the United Kingdom and internationally about whether social care provides good value for money.

Estimating the shares of the value of branded pharmaceuticals accruing to manufacturers and to patients served by health systems.

Previous studies have estimated that patients served by health systems accrue 59-98% of the value generated by new pharmaceuticals. This has led to questions about whether sufficient returns accrue to manufacturers to incentivize socially optimal levels of R&D. These studies have not, however, fully reflected the health opportunity costs imposed by payments for branded pharmaceuticals. We present a framework for estimating how the value generated by new branded pharmaceuticals is shared. We quantify value in net health effects and account for benefits and health opportunity costs in the patent period and post-patent period when generic/biosimilar products become available. We apply the framework to 12 National Institute for Health and Care Excellence appraisals and show that realized net health effects range from losses of 160%, to gains of 94%, of the potential net health benefits available. In many cases, even in the long run, the benefits of new medicines are not sufficient to offset the opportunity costs of payments to manufacturers, and approval is expected to reduce population health. This cannot be dynamically efficient as it incentivizes future innovation at prices which will also reduce population health. Further work should consider how to reflect these findings in reimbursement policies.

Estimating the Marginal Productivity of the English National Health Service From 2003 to 2012.

OBJECTIVE: Estimates of the marginal productivity of the health sector are required for a wide range of resource allocation decisions. Founding these estimates on robust empirical analysis can inform these decisions and improve allocative efficiency. This article estimates the annual marginal productivity of the English NHS over a 10-year period (between 2003 and 2012). METHODS: Data on expenditure and mortality by program budget category are used in conjunction with socioeconomic and demographic variables from the censuses for 2001 and 2011. This article applies an econometric strategy that employs an established instrumental variable approach, which is then subjected to a number of sensitivity analyses. The results of the econometric analysis, along with additional data on the burden of disease, are used to generate an estimate of the marginal productivity for each of the study years. RESULTS: We find that an additional unit of health benefit has cost between £5000 and £15 000 per quality-adjusted life-year from 2003 to 2012. Over this period these estimates (all in current prices) have increased at a faster rate than NHS price inflation, suggesting an increase in real terms. CONCLUSIONS: These results are discussed in the context of the existing literature, and the potential policy implications for decisions about resource allocation are explored.

Resolving the "Cost-Effective but Unaffordable" Paradox: Estimating the Health Opportunity Costs of Nonmarginal Budget Impacts.

Considering whether or not a proposed investment (an intervention, technology, or program of care) is affordable is really asking whether the benefits it offers are greater than its opportunity cost. To say that an investment is cost-effective but not affordable must mean that the (implicit or explicit) "threshold" used to judge cost-effectiveness does not reflect the scale and value of the opportunity costs. Existing empirical estimates of health opportunity costs are based on cross-sectional variation in expenditure and mortality outcomes by program budget categories (PBCs) and do not reflect the likely effect of nonmarginal budget impacts on health opportunity costs. The UK Department of Health regularly updates the needs-based target allocation of resources to local areas of the National Health Service (NHS), creating two subgroups of local areas (those under target allocation and those over). These data provide the opportunity to explore how the effects of changes in health care expenditure differ with available resources. We use 2008-2009 data to evaluate two econometric approaches to estimation and explore a range of criteria for accepting subgroup specific effects for differences in expenditure and outcome elasticities across the 23 PBCs. Our results indicate that health opportunity costs arising from an investment imposing net increases in expenditure are underestimated unless account is taken of likely nonmarginal effects. They also indicate the benefits (reduced health opportunity costs or increased value-based price of a technology) of being able to "smooth" these nonmarginal budget impacts by health care systems borrowing against future budgets or from manufacturers offering "mortgage" type arrangements.

Experiences of Structured Elicitation for Model-Based Cost-Effectiveness Analyses.

BACKGROUND: Empirical evidence supporting the cost-effectiveness estimates of particular health care technologies may be limited, or it may even be missing entirely. In these situations, additional information, often in the form of expert judgments, is needed to reach a decision. There are formal methods to quantify experts' beliefs, termed as structured expert elicitation (SEE), but only limited research is available in support of methodological choices. Perhaps as a consequence, the use of SEE in the context of cost-effectiveness modelling is limited. OBJECTIVES: This article reviews applications of SEE in cost-effectiveness modelling with the aim of summarizing the basis for methodological choices made in each application and recording the difficulties and challenges reported by the authors in the design, conduct, and analyses. METHODS: The methods used in each application were extracted along with the criteria used to support methodological and practical choices and any issues or challenges discussed in the text. Issues and challenges were extracted using an open field, and then categorised and grouped for reporting. RESULTS: The review demonstrates considerable heterogeneity in methods used, and authors acknowledge great methodological uncertainty in justifying their choices. Specificities of the context area emerging as potentially important in determining further methodological research in elicitation are between- expert variation and its interpretation, the fact that substantive experts in the area may not be trained in quantitative subjects, that judgments are often needed on various parameter types, the need for some form of assessment of validity, and the need for more integration with behavioural research to devise relevant debiasing strategies. CONCLUSIONS: This review of experiences of SEE highlights a number of specificities/constraints that can shape the development of guidance and target future research efforts in this area.

The impact of NHS expenditure on health outcomes in England: Alternative approaches to identification in all-cause and disease specific models of mortality.

Several recent studies have estimated the responsiveness of mortality to English National Health Service spending. Although broadly similar, the studies differ in how they identify the outcome equation. One approach uses conventional socio-economic variables as instruments for endogenous health care expenditure, whereas the other exploits exogenous elements in the resource allocation formula for local budgets. The former approach has usually been applied to specific disease areas (e.g., for cancer and circulatory disease), whereas the other has only been applied to all-cause mortality. In this letter, we compare the two approaches by using them to estimate the direct all-cause elasticity as well as disease-specific elasticities. We also calculate the implied all-cause elasticity associated with the disease-specific results. We find that the "funding rule" approach to identification can be successfully replicated and applied to disease area models. This is important because disease area models reduce the danger of aggregation bias present in all-cause analysis, and they offer the opportunity to link estimated mortality effects to more complete measures of health outcome that reflect what is currently known about the survival and morbidity disease burden in different programmes.

How to design the cost-effectiveness appraisal process of new healthcare technologies to maximise population health: A conceptual framework.

This paper presents a conceptual framework to analyse the design of the cost-effectiveness appraisal process of new healthcare technologies. The framework characterises the appraisal processes as a diagnostic test aimed at identifying cost-effective (true positive) and non-cost-effective (true negative) technologies. Using the framework, factors that influence the value of operating an appraisal process, in terms of net gain to population health, are identified. The framework is used to gain insight into current policy questions including (a) how rigorous the process should be, (b) who should have the burden of proof, and (c) how optimal design changes when allowing for appeals, price reductions, resubmissions, and re-evaluations. The paper demonstrates that there is no one optimal appraisal process and the process should be adapted over time and to the specific technology under assessment. Optimal design depends on country-specific features of (future) technologies, for example, effect, price, and size of the patient population, which might explain the difference in appraisal processes across countries. It is shown that burden of proof should be placed on the producers and that the impact of price reductions and patient access schemes on the producer's price setting should be considered when designing the appraisal process.

Social value and individual choice: The value of a choice-based decision-making process in a collectively funded health system.

Evidence about cost-effectiveness is increasingly being used to inform decisions about the funding of new technologies that are usually implemented as guidelines from centralized decision-making bodies. However, there is also an increasing recognition for the role of patients in determining their preferred treatment option. This paper presents a method to estimate the value of implementing a choice-based decision process using the cost-effectiveness analysis toolbox. This value is estimated for 3 alternative scenarios. First, it compares centralized decisions, based on population average cost-effectiveness, against a decision process based on patient choice. Second, it compares centralized decision based on patients' subgroups versus an individual choice-based decision process. Third, it compares a centralized process based on average cost-effectiveness against a choice-based process where patients choose according to a different measure of outcome to that used by the centralized decision maker. The methods are applied to a case study for the management of acute coronary syndrome. It is concluded that implementing a choice-based process of treatment allocation may be an option in collectively funded health systems. However, its value will depend on the specific health problem and the social values considered relevant to the health system.

A Cost-Effectiveness Analysis of Intradiscal Electrothermal Therapy Compared with Circumferential Lumbar Fusion.

STUDY DESIGN: Cost-effectiveness analysis. OBJECTIVE: To evaluate the cost-effectiveness of intradiscal electrothermal therapy (IDET) relative to circumferential lumbar fusion with femoral ring allograft (FRA) in the United Kingdom. SUMMARY OF BACKGROUND DATA: Circumferential lumbar fusion is an established treatment for discogenic low back pain. However, IDET could be a cost-effective treatment alternative as it can be carried out as a day case. METHODS: Patient-level data were available for patients with discogenic low back pain treated with FRA (n = 37) in a randomized trial of FRA vs. titanium cage, and for patients recruited to a separate study evaluating the use of IDET (n = 85). Both studies were carried out at a single institution in the United Kingdom. Patients were followed-up for 24 months, with data collected on low back disability (Oswestry Disability Index), back and leg pain (visual analog scale), quality of life (Short Form 36), radiographic evaluations, and U.K. National Health Service (NHS) resource use. Cost-effectiveness was measured by the incremental cost per quality-adjusted life year (QALY) gained. RESULTS: Both treatments produced statistically significant improvements in outcome at 24-month follow-up. NHS costs were significantly lower with IDET due to a shorter mean procedure time (377.4 minutes vs. 49.9 minutes) and length of stay (7 days vs. 1.2 days). At a threshold of £20,000 per QALY, the probability that IDET is cost effective is high. CONCLUSIONS: Both treatments led to significant improvements in patient outcomes that were sustained for at least 24 months. Costs were lower with IDET, and for appropriate patients IDET is an effective and cost-effective treatment alternative.

Developing a Value Framework: The Need to Reflect the Opportunity Costs of Funding Decisions.

A growing number of health care systems internationally use formal economic evaluation methods to support health care funding decisions. Recently, a range of organizations have been advocating forms of analysis that have been termed "value frameworks." There has also been a push for analytical methods to reflect a fuller range of benefits of interventions through multicriteria decision analysis. A key principle that is invariably neglected in current and proposed frameworks is the need to reflect evidence on the opportunity costs that health systems face when making funding decisions. The mechanisms by which opportunity costs are realized vary depending on the system's financial arrangements, but they always mean that a decision to fund a specific intervention for a particular patient group has the potential to impose costs on others in terms of forgone benefits. These opportunity costs are rarely explicitly reflected in analysis to support decisions, but recent developments to quantify benefits forgone make more appropriate analyses feasible. Opportunity costs also need to be reflected in decisions if a broader range of attributes of benefit is considered, and opportunity costs are a key consideration in determining the appropriate level of total expenditure in a system. The principles by which opportunity costs can be reflected in analysis are illustrated in this article by using the example of the proposed methods for value-based pricing in the United Kingdom.

Characterising Uncertainty in the Assessment of Medical Devices and Determining Future Research Needs.

Decisions about the adoption of medical interventions are informed by evidence on their costs and effects. For a range of reasons, evidence relating to medical devices may be limited. The decision to adopt a device early in its life cycle when the evidence base is least mature may impact on the prospects of acquiring further evidence to reduce uncertainties. Equally, rejecting a device will result in no uptake in practice and hence no chance to learn about performance. Decision options such as 'only in research' or 'approval with research' can overcome these issues by allowing patients early access to promising new technologies while limiting the risks associated with making incorrect decisions until more evidence or learning is established. In this paper, we set out the issues relating to uncertainty and the value of research specific to devices: learning curve effects, incremental device innovation, investment and irrecoverable costs, and dynamic pricing. We show the circumstances under which an only in research or approval with research scheme may be an appropriate policy choice. We also consider how the value of additional research might be shared between the manufacturer and health sector to help inform who might reasonably be expected to conduct the research needed. © 2017 The Authors. Health Economics published by John Wiley & Sons, Ltd.

Country-Level Cost-Effectiveness Thresholds: Initial Estimates and the Need for Further Research.

BACKGROUND: Cost-effectiveness analysis can guide policymakers in resource allocation decisions. It assesses whether the health gains offered by an intervention are large enough relative to any additional costs to warrant adoption. When there are constraints on the health care system's budget or ability to increase expenditures, additional costs imposed by interventions have an "opportunity cost" in terms of the health foregone because other interventions cannot be provided. Cost-effectiveness thresholds (CETs) are typically used to assess whether an intervention is worthwhile and should reflect health opportunity cost. Nevertheless, CETs used by some decision makers-such as the World Health Organization that suggested CETs of 1 to 3 times the gross domestic product (GDP) per capita-do not. OBJECTIVES: To estimate CETs based on opportunity cost for a wide range of countries. METHODS: We estimated CETs based on recent empirical estimates of opportunity cost (from the English National Health Service), estimates of the relationship between country GDP per capita and the value of a statistical life, and a series of explicit assumptions. RESULTS: CETs for Malawi (the country with the lowest income in the world), Cambodia (with borderline low/low-middle income), El Salvador (with borderline low-middle/upper-middle income), and Kazakhstan (with borderline high-middle/high income) were estimated to be $3 to $116 (1%-51% GDP per capita), $44 to $518 (4%-51%), $422 to $1967 (11%-51%), and $4485 to $8018 (32%-59%), respectively. CONCLUSIONS: To date, opportunity-cost-based CETs for low-/middle-income countries have not been available. Although uncertainty exists in the underlying assumptions, these estimates can provide a useful input to inform resource allocation decisions and suggest that routinely used CETs have been too high.

A Comprehensive Algorithm for Approval of Health Technologies With, Without, or Only in Research: The Key Principles for Informing Coverage Decisions.

BACKGROUND: The value of evidence about the performance of a technology and the value of access to a technology are central to policy decisions regarding coverage with, without, or only in research and managed entry (or risk-sharing) agreements. OBJECTIVES: We aim to outline the key principles of what assessments are needed to inform "only in research" (OIR) or "approval with research" (AWR) recommendations, in addition to approval or rejection. METHODS: We developed a comprehensive algorithm to inform the sequence of assessments and judgments that lead to different types of guidance: OIR, AWR, Approve, or Reject. This algorithm identifies the order in which assessments might be made, how similar guidance might be arrived at through different combinations of considerations, and when guidance might change. RESULTS: The key principles are whether the technology is expected to be cost-effective; whether the technology has significant irrecoverable costs; whether additional research is needed; whether research is possible with approval and whether there are opportunity costs that once committed by approval cannot be recovered; and whether there are effective price reductions. Determining expected cost-effectiveness is only a first step. In addition to AWR for technologies expected to be cost-effective and OIR for those not expected to be cost-effective, there are other important circumstances when OIR should be considered. CONCLUSIONS: These principles demonstrate that cost-effectiveness is a necessary but not sufficient condition for approval. Even when research is possible with approval, OIR may be appropriate when a technology is expected to be cost-effective due to significant irrecoverable costs.

The International Decision Support Initiative Reference Case for Economic Evaluation: An Aid to Thought.

BACKGROUND: Policymakers in high-, low-, and middle-income countries alike face challenging choices about resource allocation in health. Economic evaluation can be useful in providing decision makers with the best evidence of the anticipated benefits of new investments, as well as their expected opportunity costs-the benefits forgone of the options not chosen. To guide the decisions of health systems effectively, it is important that the methods of economic evaluation are founded on clear principles, are applied systematically, and are appropriate to the decision problems they seek to inform. METHODS: The Bill and Melinda Gates Foundation, a major funder of economic evaluations of health technologies in low- and middle-income countries (LMICs), commissioned a "reference case" through the International Decision Support Initiative (iDSI) to guide future evaluations, and improve both the consistency and usefulness to decision makers. RESULTS: The iDSI Reference Case draws on previous insights from the World Health Organization, the US Panel on Cost-Effectiveness in Health Care, and the UK National Institute for Health and Care Excellence. Comprising 11 key principles, each accompanied by methodological specifications and reporting standards, the iDSI Reference Case also serves as a means of identifying priorities for methods research, and can be used as a framework for capacity building and technical assistance in LMICs. CONCLUSIONS: The iDSI Reference Case is an aid to thought, not a substitute for it, and should not be followed slavishly without regard to context, culture, or history. This article presents the iDSI Reference Case and discusses the rationale, approach, components, and application in LMICs.

Unrelated Future Costs and Unrelated Future Benefits: Reflections on NICE Guide to the Methods of Technology Appraisal.

In this editorial, we consider the vexing issue of 'unrelated future costs' (for example, the costs of caring for people with dementia or kidney failure after preventing their deaths from a heart attack). The National Institute of Health and Care Excellence (NICE) guidance is not to take such costs into account in technology appraisals. However, standard appraisal practice involves modelling the benefits of those unrelated technologies. We argue that there is a sound principled reason for including both the costs and benefits of unrelated care. Changing this practice would have material consequences for decisions about reimbursing particular technologies, and we urge future research to understand this better. Copyright © 2016 John Wiley & Sons, Ltd.

Unifying Research and Reimbursement Decisions: Case Studies Demonstrating the Sequence of Assessment and Judgments Required.

BACKGROUND: The key principles regarding what assessments lead to different types of guidance about the use of health technologies (Only in Research, Approval with Research, Approve, or Reject) provide an explicit and transparent framework for technology appraisal. OBJECTIVE: We aim to demonstrate how these principles and assessments can be applied in practice through the use of a seven-point checklist of assessment. METHODS: The value of access to a technology and the value of additional evidence are explored through the application of the checklist to the case studies of enhanced external counterpulsation for chronic stable angina and clopidogrel for the management of patients with non-ST-segment elevation acute coronary syndromes. RESULTS: The case studies demonstrate the importance of considering 1) the expected cost-effectiveness and population net health effects; 2) the need for evidence and whether the type of research required can be conducted once a technology is approved for widespread use; 3) whether there are sources of uncertainty that cannot be resolved by research but only over time; and 4) whether there are significant (opportunity) costs that once committed by approval cannot be recovered. CONCLUSIONS: The checklist demonstrates that cost-effectiveness is a necessary but not sufficient condition for approval. Only in Research may be appropriate when a technology is expected to be cost-effective due to significant irrecoverable costs. It is only approval that can be ruled out if a technology is not expected to be cost-effective. Lack of cost-effectiveness is not a necessary or sufficient condition for rejection.

Causes for concern: is NICE failing to uphold its responsibilities to all NHS patients?

Organisations across diverse health care systems making decisions about the funding of new medical technologies face extensive stakeholder and political pressures. As a consequence, there is quite understandable pressure to take account of other attributes of benefit and to fund technologies, even when the opportunity costs are likely exceed the benefits they offer. Recent evidence suggests that NICE technology appraisal is already approving drugs where more health is likely to be lost than gained. Also, NICE recently proposed increasing the upper bound of the cost-effectiveness threshold to reflect other attributes of benefit but without a proper assessment of the type of benefits that are expected to be displaced. It appears that NICE has taken a direction of travel, which means that more harm than good is being, and will continue to be, done, but it is unidentified NHS patients who bear the real opportunity costs.