Does NICE have a cost‐effectiveness threshold and what other factors influence its decisions? A binary choice analysis

The decisions made by the National Institute for Clinical Excellence (NICE) give rise to two questions: how is cost‐effectiveness evidence used to make judgements about the ‘value for money’ of health technologies? And how are factors other than cost‐effectiveness taken into account? The aim of this paper is to explore NICE's cost‐effectiveness threshold(s) and the tradeoffs between cost effectiveness and other factors apparent in its decisions. Binary choice analysis is used to reveal the preferences of NICE and to consider the consistency of its decisions. For each decision to accept or reject a technology, explanatory variables include: the cost per life year or per QALY gained; uncertainty regarding cost effectiveness; the net cost to the NHS; the burden of disease; the availability (or not) of alternative treatments; and specific factors indicated by NICE. Results support the broad notion of a threshold, where the probability of rejection increases as the cost per QALY increases. Cost effectiveness, together with uncertainty and the burden of disease, explain NICE decisions better than cost effectiveness alone. The results suggest a threshold somewhat higher than NICEs stated ‘range of acceptable cost effectiveness’ of £20 000–£30 000 per QALY – although the exact meaning of a ‘range’ in this context remains unclear. Copyright © 2004 John Wiley & Sons, Ltd.     

The Influence of Cost‐Effectiveness and Other Factors on Nice Decisions

The National Institute for Health and Care Excellence (NICE) emphasises that cost‐effectiveness is not the only consideration in health technology appraisal and is increasingly explicit about other factors considered relevant but not the weight attached to each. The objective of this study is to investigate the influence of cost‐effectiveness and other factors on NICE decisions and whether NICE's decision‐making has changed over time. We model NICE's decisions as binary choices for or against a health care technology in a specific patient group. Independent variables comprised of the following: clinical and economic evidence; characteristics of patients, disease or treatment; and contextual factors potentially affecting decision‐making. Data on all NICE decisions published by December 2011 were obtained from HTAinSite [ www.htainsite.com ]. Cost‐effectiveness alone correctly predicted 82% of decisions; few other variables were significant and alternative model specifications had similar performance. There was no evidence that the threshold has changed significantly over time. The model with highest prediction accuracy suggested that technologies costing £40 000 per quality‐adjusted life‐year (QALY) have a 50% chance of NICE rejection (75% at £52 000/QALY; 25% at £27 000/QALY). Past NICE decisions appear to have been based on a higher threshold than £20 000–£30 000/QALY. However, this may reflect consideration of other factors that cannot be easily quantified. © 2014 The Authors. Health Economics published by John Wiley & Sons Ltd.     

SOCIETAL VIEWS ON NICE,CANCER DRUGS FUND AND VALUE‐BASED PRICING CRITERIA FOR PRIORITISING MEDICINES: A CROSS‐SECTIONAL SURVEY OF 4118 ADULTS IN GREAT BRITAIN

The criteria used by the National Institute for Health and Clinical Excellence (NICE) for accepting higher incremental cost‐effectiveness ratios for some medicines over others, and the recent introduction of the Cancer Drugs Fund (CDF) in England, are assumed to reflect societal preferences for National Health Service resource allocation. Robust empirical evidence to this effect is lacking. To explore societal preferences for these and other criteria, including those proposed for rewarding new medicines under the future value‐based pricing (VBP) system, we conducted a choice‐based experiment in 4118 UK adults via web‐based surveys. Preferences were determined by asking respondents to allocate fixed funds between different patient and disease types reflecting nine specific prioritisation criteria. Respondents supported the criteria proposed under the VBP system (for severe diseases, address unmet needs, are innovative—provided they offered substantial health benefits, and have wider societal benefits) but did not support the end‐of‐life premium or the prioritisation of children or disadvantaged populations as specified by NICE, nor the special funding status for treatments of rare diseases, nor the CDF. Policies introduced on the basis of perceived—and not actual—societal values may lead to inappropriate resource allocation decisions with the potential for significant population health and economic consequences. Copyright © 2012 John Wiley & Sons, Ltd.     

Is There Anything Nicer than NICE? A Question the Conservative Shadow Health Team Is Right to Ask

Access to life-saving treatments, and the role played by the National Institute for Health and Clinical Excellence (NICE) in reaching decisions, continues to represent an important part of modern health policy. High profile cases and critical media coverage have sharpened public interest in this issue.
In November 2008, the Conservative Party published detailed proposals on NICE outlining policies for improving the systems and processes for making decisions about NHS drug availability. The Conservatives clearly state their support for NICE, but highlight six areas to improve its configuration, structure and efficiency. These areas are consistent with the Conservative commitment to focus on health outcomes rather than central targets.
A "NICE Charter" to codify the Institute's roles and responsibilities; scrapping the current system of Ministerial referral; allowing appraisals to commence at the time of drug licensing; and increasing the use of risk-sharing schemes are among the headline pledges.
The policy document also makes clear the need for pharmaceutical companies to better demonstrate product clinical value by shifting the burden of proof from NICE to the manufacturer. Improved cooperation between industry and NICE is promised through the creation of a steering committee. Furthermore, a clear commitment to evaluate wider social costs and benefits is provided.
The Conservative proposals make clear that there are no easy solutions to tackle the basic health economic problem of how to best allocate finite NHS resources to satisfy all healthcare needs. However, the proposals offer a solid blueprint for focused reform moving forward.     

Reimbursement and value‐based pricing: stratified cost‐effectiveness analysis may not be the last word

During recent discussions, it has been argued that stratified cost‐effectiveness analysis has a key role in reimbursement decision‐making and value‐based pricing (VBP). It has previously been shown that when manufacturers are price‐takers, reimbursement decisions made in reference to stratified cost‐effectiveness analysis lead to a more efficient allocation of resources than decisions based on whole‐population cost‐effectiveness analysis. However, we demonstrate that when manufacturers are price setters, reimbursement or VBP based on stratified cost‐effectiveness analysis may not be optimal. Using two examples – one considering the choice of thrombolytic treatment for specific patient subgroups and the other considering the extension of coverage for a cancer treatment to include an additional indication – we show that combinations of extended coverage and reduced price can be identified that are advantageous to both payers and manufacturers. The benefits of a given extension in coverage and reduction in price depend both upon the average treatment benefit in the additional population and its size relative to the original population. Negotiation regarding trade‐offs between price and coverage may lead to improved outcomes both for health‐care systems and manufacturers compared with processes where coverage is determined conditional simply on stratified cost‐effectiveness at a given price. Copyright © 2010 John Wiley & Sons, Ltd.     

How should cost-effectiveness analysis be used in health technology coverage decisions? Evidence from the National Institute for Health and Clinical Excellence approach

BACKGROUND: In the National Health Service in England and Wales, technology coverage decisions are taken by the National Institute for Health and Clinical Excellence (NICE). The intention formally to apply cost-effectiveness analysis to the decision-making process distinguishes NICE from most other bodies making similar policy recommendations. We carried out a case study of the NICE Appraisals Committee to explore the influence and use of economic evaluation in the decision-making process. METHODS: Qualitative case study methodology. This involved analysis of all relevant secondary sources, observations of Appraisals Committee deliberations and interviews with a cross-section of Committee members. FINDINGS: Economic evaluation is integrated into the Committee's work. There are two main ways in which the use of economic analysis is understood by Committee members: an ordinal approach, whereby cost-effectiveness is only considered if the technology has passed a clinical effectiveness hurdle; and a framework approach, whereby the economic evaluation and model provide a structure for considering the decision problem and the evidence. These two approaches appear to operate simultaneously but are, in essence, inconsistent. CONCLUSIONS: The NICE 'experiment' has seen cost-effectiveness analysis move to the centre-ground of UK national policy deliberations regarding technology coverage. However, our case study implies that there may be room for further refinement of the appraisal process in order to resolve the observed tension between two different ways of incorporating cost-effectiveness analysis in NICE's decision-making.     

Deliberating Tarceva: A case study of how British NHS managers decide whether to purchase a high-cost drug in the shadow of NICE guidance

This paper examines audio-recorded data from meetings in which NHS managers decide whether to fund high-cost drugs for individual patients. It investigates the work of a Welsh individual patient commissioning (IPC) panel responsible for sanctioning the purchase of 'un-commissioned' treatments for exceptional cases. The case study presented highlights the changing rationales used for approving or denying a cancer drug, Tarceva, during a period when NICE first suggested it was not cost effective, but then changed its position in a final technology appraisal recommending use when the cost did not exceed that of an alternative product. Our data show how decisions taken in the shadow of NICE guidance remain complex and subject to local discretion. Guidance that takes time to prepare, is released in stages, and relates to particular disease stages, must be interpreted in the context of particular cases. The case-based IPC panel discourse stands in tension with the standardised population-based recommendations in guidance. Panel members, who based their decisions on the central notions of 'efficacy' and 'exceptionality', often struggled to apply NICE information on cost-effectiveness to their deliberations on efficacy (clinical effectiveness). The case study suggests that the complex nature of decision making makes standardization of outcomes very difficult to achieve, so that local professional judgement is likely to remain central to health care rationing at this level.     

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. Copyright © 2014 John Wiley & Sons, Ltd.     

Seeing the NICE side of cost-effectiveness analysis: a qualitative investigation of the use of CEA in NICE technology appraisals

Resource scarcity is the raison d'être for the discipline of economics. Thus, the primary purpose of economic analysis is to help decision-makers when addressing problems arising due to the scarcity problem. The research reported here was concerned with how cost-effectiveness information is used by the National Institute for Health & Clinical Excellence (NICE) in national technology coverage decisions in the UK, and how its impact might be increased. The research followed a qualitative case study methodology with semi-structured interviews, supported by observation and analysis of secondary sources. Our research highlights that the technology appraisal function of NICE represents an important progression for the UK health economics community: new cost-effectiveness work is commissioned for each technology and that work directly informs national health policy. However, accountability in policy decisions necessitates that the information upon which decisions are based (including cost-effectiveness analysis, CEA) is accessible. This was found to be a serious problem and represents one of the main ongoing challenges. Other issues highlighted include perceived weaknesses in analysis methods and the poor alignment between the health maximisation objectives assumed in economic analyses and the range of other objectives facing decision-makers in reality.     

Running along parallel lines: how political reality impedes the evaluation of public health interventions. A case study of exercise referral schemes in England

Political decisions about the way that public health initiatives are implemented have a significant impact on the ability to evaluate their effectiveness. However, the influence of the political imperative has been little explored. This case study of key research, policy and practice events concerning one initiative, exercise referral schemes (ERSs), demonstrates that these schemes were encouraged to expand by the Department of Health (DH) before DH-funded evaluations had reported their findings and with little reference to National Institute for Health and Clinical Excellence (NICE) recommendations. Policy evolved in parallel rather than in conjunction with the development of evidence, and experimental evaluations in England are now unlikely. This is due to the comprehensive coverage of schemes, widespread assumptions of effectiveness, likely difficulties in obtaining research funding, indirect adverse consequences of dismantling schemes and lack of appropriate process and outcome data. Embedding a commitment to robust evaluation prior to universal adoption of new initiatives has been shown to be feasible by policy-makers in the international setting. This is required to prevent the establishment of public health interventions that do not work and may cause harm or widen health inequalities.     

The lag between effectiveness and cost-effectiveness evidence of new drugs

A new drug is approved for use if its efficacy and safety have been demonstrated. However, healthcare decision makers may also require data on the cost-effectiveness of new drugs if they are to make informed decisions about their place in therapy. Cost-effectiveness evidence may lag behind the effectiveness data in terms of its availability. We explored the timeliness of delivering cost-effectiveness information about new drugs with established effectiveness and significant financial impact. Drugs were identified, based on guidance documents and reports published by the UK National Institute for Clinical Excellence (NICE), and the following data were collected: dates of publication of first effectiveness and cost-effectiveness evidence, methodology of the cost-effectiveness analysis, quality scores of the clinical studies. Eighteen guidance documents on the use of new drugs/drug groups published by NICE by October 2001 covered 30 health technologies, which were included in the analysis. The analysis of the evidence showed that their effectiveness had been demonstrated in the last 12 years, with only two exceptions. However, cost-effectiveness evidence had been published for 21 (70%) of the technologies. The cost-effectiveness was estimated in 52.4% of cases using models. The good quality effectiveness evidence lagged behind the first effectiveness evidence by 1.40 years (95% CI 0.57–2.23), while the mean lag between the first effectiveness evidence and the first cost-effectiveness publications was estimated as 3.20 years (95% CI 1.76–4.65). Cost-effectiveness evidence thus often lags behind the effectiveness evidence. As a result healthcare decision makers are sometimes in a position of having to take decisions without having adequate cost-effectiveness data at their disposal.     

Health Technology Assessment–Informed Decision Making by the Federal Joint Committee/Institute for Quality and Efficiency in Health Care in Germany and the National Institute for Health and Care Excellence in England: The Role of Budget Impact

ObjectivesThis study aimed to test (official) evaluation criteria including the potential role of budget impact (BI) on health technology assessment (HTA) outcomes published by the Federal Joint Committee (Gemeinsamer Bundesausschuss [GBA]) and the Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen [IQWiG]) in Germany as well as the National Institute for Health and Care Excellence (NICE) in England.MethodsData were extracted from all publicly available GBA decisions and IQWiG assessments as well as NICE single technology appraisals between January 2011 and June 2018, and information with regard to evaluation criteria used by these agencies was collected. Data were analyzed using logistic regression to estimate the effect of the BI on the HTA outcomes while controlling for criteria used by GBA/IQWiG and NICE.ResultsNICE recommendations are largely driven by the incremental cost-effectiveness ratio and, if applicable, by end-of-life criteria (P < .01). While IQWiG assessments are significantly affected by the availability of randomized controlled trials and patient-relevant endpoints (P < .01), GBA appraisals primarily focus on endpoints (P < .01). The BI correlated with NICE single technology appraisals (inverted-U relationship, P < .1) and IQWiG recommendations (increasing linear relationship, P < .05), but not with GBA decisions (P > .1). Nevertheless, given that IQWiG assessments seem to be more rigorous than GBA appraisals regarding the consideration of evidence-based evaluation criteria, decisions by GBA might be negatively associated with the BI.ConclusionsResults reveal that GBA/IQWiG and NICE follow their official evaluation criteria consistently. After controlling for all significant variables, the BI seems to have an (independent) effect on HTA outcomes as well.     

Data analysis in evaluation of health education: toward standardization of procedures and terminology

Although data analysis is both the culmination as well as thereward for one's labor in evaluating a health education program,to date there have been few standardized data analytic proceduresfor estimating the program's need or impact. Such proceduresare necessary to make informed decisions about the relativemerits of any health education program. The current paper offersa systematic and integrated framework for calculating programneed, reach, coverage, impact, efficacy, effectiveness, cost,efficiency, cost-effectiveness, benefits, cost-benefit, income,net gain or loss, start-up cost, operating cost, operating costeffectivenessand benefit. Data analysis from two different health educationprograms provide detailed examples of the calculations.     

Confronting the "gray zones" of technology assessment: evaluating genetic testing services for public insurance coverage in Canada

We describe an evaluation model to guide public coverage of new predictive genetic tests in Ontario, Canada. The model confronts common "gray zones" in evaluation and coverage policy for challenging new technologies. Analysis addresses three domains of the evaluation picture. The first specifies evaluative criteria (purpose, effectiveness, additional effects, unit cost, demand, cost-effectiveness). The second induces or deduces acceptable cutoffs for each criterion. The third domain addresses the need to make decisions under uncertainty and to respond to "gray" evaluations with conditional-coverage decisions. The evaluation criteria should be applied within sound decision-making processes.     

Regulating pharmaceutical markets: improving efficiency and controlling costs in the UK

UK government policy on pharmaceuticals is broadly integrated across the whole of health care policy. In the early 1990s, cost containment was emphasized, through budget holding by doctors to ensure clinical acceptability. From 2000 onwards, increased government funding for the NHS has allowed expansion of services and prescribing in areas of public health importance, but has been coupled with increased accountability and ambitious targets for the process of care and health outcomes. Standards for care are set in national guidelines including those from the National Institute for Clinical Excellence (NICE). NICE recommends or rejects new technologies to the NHS for their clinical value and cost effectiveness. Although following its advice is mandatory, evidence so far suggests that it has been only partly successful at improving services and eliminating variations. GP prescribing is monitored by Primary Care Organisations (PCO) which also hold the medicines budget. They may provide incentives to GPs for meeting targets in quality or expenditure. The UK government regulates the prices of generics but not of branded medicines; instead it regulates the profitability of the pharmaceutical industry. This arrangement seems to have been successful both at maintaining a major employer and export earner, and in limiting high drug expenditure.     

Comparison of outpatient coverage in Canada: Assistive and medical devices

Outpatient technologies are important for maintaining health and overall quality of life, yet the degree of access and coverage of these technologies remains variable within and across jurisdictions. In Canada, assistive technologies are not included in universal health coverage, and are not subject to the Canada Health Act's criteria and conditions that provinces and territories must fulfill to receive the full federal cash contribution under the Canada Health Transfer. As such, the thirteen Canadian provincial and territorial governments make separate decisions on programs and coverage. Drawing on the WHO Universal Coverage Cube we compare who gets access, the types of technologies that can be accessed, and the level of coverage (total costs covered) in Canada. Overall, each Canadian jurisdiction had at least one publicly supported program. All relied on a ‘health assessment’ of an individual's need to determine eligibility. Income and eligibility for social assistance was used as eligibility criteria in 6 of the 13 jurisdictions. Mobility aids as well as audio, visual, and communication aids were included in all jurisdictions. While some programs offered full financial support for some technologies, forms of cost sharing were common. The results are discussed in the context of international experiences, demographic changes, and health system trends to highlight areas for policy learning.     

Evidence based public health: A review of the experience of the National Institute of Health and Clinical Excellence (NICE) of developing public health guidance in England

This paper describes the application of the principles of evidence based medicine to public health. It recounts the experience of the National Institute for Health and Clinical Excellence in England (NICE) which acquired a remit to develop public health guidance in 2005. Some of the history of the origins of the evidence based approach is described in the writings of Cochrane and others, and the way that this came to be a critical part of the NICE approach to developing clinical cost effectiveness is outlined. The challenge of applying these methods to an evidence base which is social and psychological as well as biomedical is considered. Key problems are identified: the breadth of the evidence base, different analytic levels of explanation, and the length of the causal chain between interventions and outcomes in public health.     

Prioritizing health technologies in a Primary Care Trust

BACKGROUND: In the English National Health Service (NHS), Primary Care Trusts (PCTs) are responsible for commissioning health-care services on behalf of their populations. As resources are finite, decisions are required as to which services best fulfil population needs. Evidence on effectiveness varies in quality and availability. Nevertheless, decisions still have to be made. METHODS: We report the development and pilot application of a multi-criteria prioritization mechanism in an English PCT, capable of accommodating a wide variety of evidence to rank six service developments. RESULTS: The mechanism proved valuable in assisting prioritization decisions and feedback was positive. Two community-based interventions were expected to save money in the long term and were ranked at the top of the list. Based on weighted benefit score and cost, two preventive programmes were ranked third and fourth. Finally, two National Institute for Health and Clinical Excellence (NICE)-approved interventions were ranked fifth and sixth. Sensitivity analysis revealed overlap in benefit scores for some of the interventions, representing diversity of opinion among the scoring panel. CONCLUSION: The method appears to be a practical approach to prioritization for commissioners of health care, but the pilot also revealed interesting divergences in relative priority between nationally mandated service developments and local health-care priorities.     

Evidence-informed evidence-making

The extent to which clinical and public health guidance developed by the National Institute for Health and Clinical Excellence (NICE) can effectively serve the public by improving quality and efficiency across the National Health Service (NHS) and the broader public sector depends largely on the quality and relevance of the available evidence which informs its decisions. There are well-established organizational and procedural links between NICE and academic and professional organizations that undertake evidence synthesis. However, there are fewer means for evidence gaps identified during the development of NICE guidance to lead to the commissioning of new prospective studies. In this paper, we discuss the importance of a publicly funded clinical and public health research agenda that includes new prospective studies aimed at addressing knowledge gaps identified by NICE. We describe the early experience of NICE and the National Institute for Health Research (NIHR) working together to articulate and commission research to inform best practice recommendations. We propose ways in which NICE can collaborate more effectively with research funders to improve the evidence base upon which it bases its recommendations.