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In the developed world, the demand for healthcare is rising faster than supply, so that governments are faced with the need to allocate limited resources for maximum benefit. Many governments are responding to these pressures by developing health technology assessment agencies, which evaluate the cost effectiveness of new pharmaceutical and medical products relative to existing interventions. In England and Wales, the agency concerned with health technology assessment is the National Institute for Clinical Excellence (NICE). NICE brings together evidence of clinical and cost effectiveness to judge the value of the treatment relative to alternative uses of National Health Service (NHS) resources and makes recommendations on use of the treatment by the NHS in England and Wales. NICE evaluates technologies where they may result in significant impact on NHS resources or key healthcare policy. The health technology assessment includes a review of the clinical effectiveness, cost effectiveness and service impact of the technology under consideration. This health technology assessment report, together with submissions from the technology manufacturer, and patient and healthcare professionals groups, is then considered by an appraisal committee that formulates guidance to the NHS and is ultimately published by NICE.A number of countries have formal guidelines on the use of outcome measures and economic evaluations in the submissions required for health technology assessment prior to market access. These guidelines vary in both the detail and level of mandatory requirement to be followed by technology manufacturers. NICE has recently updated its guidance to technology manufacturers on their submissions. These guidelines, developed in consultation with the healthcare industry, provide detailed specification of the requirements of NICE for health outcomes data and economic evaluation. These details are described in more detail in this paper. [ABSTRACT FROM AUTHOR]

Background: Chest low-dose computed tomography (LDCT) is a promising technology for population-based screening because it is non-invasive, relatively inexpensive, associated with low radiation and highly sensitive to lung cancer. To improve the cost-effectiveness of lung cancer screening, simultaneous screening for other diseases could be considered. This systematic review was conducted to analyse studies that published evidence on the cost-effectiveness of chest LDCT screening programs for different diseases. Methods: Scopus and PubMed were searched for English publications (1 January 2011–22 July 2022) using search terms related to screening, computed tomography and cost-effectiveness. An additional search specifically searched for the cost-effectiveness of screening for lung cancer, chronic obstructive pulmonary disease or cardiovascular disease. Included publications should present a full health economic evaluation of population screening with chest LDCT. The extracted data included the disease screened for, model type, country context of screening, inclusion of comorbidities or incidental findings, incremental costs, incremental effects and the resulting cost-effectiveness ratio amongst others. Reporting quality was assessed using the 2022 Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. Results: The search yielded 1799 unique papers, of which 43 were included. Most papers focused on lung cancer screening (n = 40), and three were on coronary calcium scoring. Microsimulation was the most commonly applied modelling type (n = 16), followed by life table analysis (n = 10) and Markov cohort models (n = 10). Studies reflected the healthcare context of the US (n = 15), Canada (n = 4), the UK (n = 3) and 13 other countries. The reported incremental cost-effectiveness ratio ranged from US$10,000 to US$90,000/quality-adjusted life year (QALY) for lung cancer screening compared to no screening and was US$15,900/QALY–US$45,300/QALY for coronary calcium scoring compared to no screening. Discussion: Almost all health economic evaluations of LDCT screening focused on lung cancer. Literature regarding the health economic benefits of simultaneous LDCT screening for multiple diseases is absent. Most studies suggest LDCT screening is cost-effective for current and former smokers aged 55–74 with a minimum of 30 pack-years of smoking history. Consequently, more evidence on LDCT is needed to support further cost-effectiveness analyses. Preferably evidence on simultaneous screening for multiple diseases is needed, but alternatively, on single-disease screening. Registration of systematic review: Prospective Register of Ongoing Systematic Reviews registration CRD42021290228 can be accessed https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=290228. [ABSTRACT FROM AUTHOR]

Background: This paper describes the first evaluation of the construct validity and performance of the newly developed preference-based measure of health, the SF-6D version 2 (SF-6Dv2). Method: Utilising data from the Multi-Instrument Comparison (MIC) project (n = 7932), we explored the descriptive differences in utility values between the SF-6Dv2 and the SF-6D and evaluated the known group validity of both measures by testing the statistical significance of differences in utility values and calculating the effect sizes across known groups. The convergent validity of the SF-6Dv2 was explored by examining whether the SF-6Dv2 is related to alternative validated measures, including the EQ-5D-5L and AQoL-8D. Results: Differences between the utility values of the SF-6Dv2 and SF-6D were evident; utilities were generally lower for the SF-6Dv2, with larger standard deviations resulting in larger absolute differences between groups. The SF-6Dv2 performed well in terms of known-group validity and successfully distinguished disease severity and between the disease and healthy groups, outperforming the SF-6D in some but not all groups. Convergent validity analyses indicated strong associations between the SF-6Dv2 and the SF-6D, EQ-5D-5L and AQoL-8D utilities. Conclusions: The psychometric performance of the SF-6Dv2 is favourable with respect to known group validity and convergent validity, but does not seem to have improved, compared with the SF-6D. However, the new method of valuation has had a substantial impact on the size of absolute differences in utility values, which could impact quality-adjusted life-year results. The economic evaluation of health interventions may therefore be influenced by the choice of the SF-6Dv2 over the SF-6D. [ABSTRACT FROM AUTHOR]

The UK's National Institute for Health and Care Excellence (NICE) recently launched a consultation on the methods it uses to evaluate new health technologies, and has highlighted the issue of how 'modifiers', including equity weights, should be incorporated into its processes. The practice of applying equity weights to specific population subgroups, as a means for increasing the effective cost-effectiveness threshold for some new health technologies, is well established in health technology assessment. It is also the subject of extensive discussion in the academic literature. In this paper, we demonstrate that NICE's current approach to equity weighting has the effect of reducing both population health and equity-weighted population health, a fundamental problem that appears to place NICE in contravention of its principles and obligations. We consider two potential methods for modifying NICE's current approach to address this problem. We also consider the merits of NICE abandoning its current approach to equity weighting and adopting a standard 'net benefit' approach in its place. We find that adopting a standard 'net benefit' approach is the most desirable option, as it provides for the most transparency while avoiding specific issues that arise when attempting to modify NICE's current approach. Regardless of the approach NICE uses for equity weighting, we find that protecting the health of National Health Service patients requires that some new technologies be evaluated using an effective cost-effectiveness threshold lower than the 'supply-side' cost-effectiveness threshold. This poses a particular challenge for NICE, given its obligations under the 2019 'Voluntary Scheme' between the UK pharmaceutical industry, the National Health Service, and the UK Government. We conclude by making some recommendations as to how NICE can move forward with the use of 'modifiers' in its decision making. [ABSTRACT FROM AUTHOR]

The National Institute for Health and Care Excellence (NICE), as part of the institute's single technology appraisal (STA) process, invited the company that makes obinutuzumab (Roche Products Limited) to submit evidence of the clinical and cost effectiveness of the drug in combination with chemotherapy, with or without obinutuzumab as maintenance therapy for adult patients with untreated, advanced follicular lymphoma (FL) in the UK. Kleijnen Systematic Reviews Ltd (KSR), in collaboration with Erasmus University Rotterdam, was commissioned to act as the Evidence Review Group (ERG). This paper describes the company's submission, the ERG review, and NICE's subsequent decisions. The clinical evidence was derived from two phase III, company-sponsored, randomised, open-label studies. Most evidence on obinutuzumab was based on the GALLIUM trial that compared obinutuzumab in combination with chemotherapy as induction followed by obinutuzumab maintenance monotherapy with rituximab in combination with chemotherapy as induction followed by rituximab maintenance monotherapy in previously untreated patients with FL (grades 1-3a). Long-term clinical evidence was based on the PRIMA trial, studying the benefit of two years of rituximab maintenance after first-line treatment in patients with FL. The cost-effectiveness evidence submitted by the company relied on a partitioned survival cost-utility model, implemented in Microsoft® Excel. The base-case incremental cost-effectiveness ratio (ICER) presented in the company submission was <£20,000 per quality-adjusted life-year (QALY) gained. Although the ERG concluded that the economic model met the NICE reference case to a reasonable extent, some errors were identified and several assumptions made by the company were challenged. A new base-case scenario produced by the ERG suggested an ICER that was higher than the company base case, but still below £30,000 per QALY gained. However, some ERG scenario analyses were close to or even above the threshold. This was the case in particular for assuming a treatment effect that did not extend beyond trial follow-up. These results led to an initial negative recommendation by the appraisal committee. Subsequently, the company submitted a revised base case focusing on patients at intermediate or high risk of premature mortality. Simultaneously, a further price discount for obinutuzumab was granted. In addition to the company's revised base case, the ERG suggested a restriction of the treatment effect to 5 years and implemented biosimilar uptake and cheaper prices for rituximab. All of these adjustments did not exceed £30,000 per QALY gained and therefore the use of obinutuzumab for patients with advanced FL and a Follicular Lymphoma International Predictive Index (FLIPI) score of two or more could be recommended. [ABSTRACT FROM AUTHOR]

The National Institute for Health and Care Excellence (NICE) invited Servier, the company manufacturing trifluridine and tipiracil (T/T; trade name: Lonsurf®), to submit evidence for the clinical and cost effectiveness of T/T compared with best supportive care (BSC) for metastatic colorectal cancer (third-line or later). Kleijnen Systematic Reviews Ltd (KSR), in collaboration with Maastricht University Medical Center, was commissioned as the Evidence Review Group (ERG). This paper presents a summary of the company's submission (CS), the ERG report and the development of the NICE guidance for the use of this drug in England and Wales by the appraisal committee (AC). The ERG produced a critical review of the clinical and cost effectiveness of T/T based upon the CS. In the CS, pooled evidence of two trials (a phase II trial and RECOURSE) showed that T/T resulted in a significant increase in overall survival [OS; hazard ratio (HR) 0.67, 95% CI 0.58-0.78] and progression-free survival (PFS; HR 0.46, 95% CI 0.40-0.53). The AC considered the survival benefit of T/T clinically meaningful although relatively small. The ERG highlighted that none of the participants in the phase II trial and approximately half of the RECOURSE participants (394 of 800) were from Europe, which might limit the applicability of the study findings to the NHS. Moreover, the ERG's critical assessment of the company's economic evaluation highlighted a number of concerns that resulted in 11 adjustments to the company's base-case analysis. The ERG adjustments that had the largest impact were using the RECOURSE trial data only (instead of the pooled evidence), fixing errors and violations and using the utilities from the CORRECT trial (identified in the literature review) only. The ERG preferred to use the RECOURSE trial data only given the suboptimal methodology used by the company to pool the evidence. However, since there were no fundamental arguments to prevent the two trials from being pooled, the ERG also presented its base-case analysis based on the pooled effectiveness estimates. The company base-case resulted in an incremental cost effectiveness ratio (ICER) of £44,032 per QALY gained while the ERG base-case resulted in ICERs of £52,695 and £49,392 per QALY gained based on the RECOURSE trial only and pooled evidence, respectively. Since the AC concluded that the most plausible ICER was £49,392 per QALY gained, and that T/T meets end-of-life criteria, T/T was recommended as a cost effective use of NHS resources. [ABSTRACT FROM AUTHOR]

Since 1999, the National Institute for Health and Clinical Excellence (NICE) Technology Appraisal Programme has been charged with producing guidance for the NHS in England and Wales on the appropriate use of new and existing healthcare programmes. Guidance is based on an assessment of a number of factors, including cost effectiveness. The identification, measurement and valuation of costs are important components of any cost-effectiveness analysis. However, working through these steps raises a number of important methodological questions. For example, how should ‘future’ resource use be estimated, and is there a need to consider all ‘future’ costs? Given that NICE produces national guidance, should national unit cost data be used to value resources or should local variations in negotiated prices be taken into account? This paper was initially prepared as a briefing paper as part of the process of updating NICE’s 2004 Guide to the Methods of Technology Appraisal for a workshop on ‘costs’. It outlines the issues that were raised in the original briefing paper and the subsequent questions that were discussed at the workshop. [ABSTRACT FROM AUTHOR]

Previous application of value-of-information methods to optimal clinical trial design have predominantly taken a societal decision-making perspective, implicitly assuming that healthcare costs are covered through public expenditure and trial research is funded by government or donation-based philanthropic agencies. In this paper, we consider the interaction between interrelated perspectives of a societal decision maker (e.g. the National Institute for Health and Clinical Excellence [NICE] in the UK) charged with the responsibility for approving new health interventions for reimbursement and the company that holds the patent for a new intervention. We establish optimal decision making from societal and company perspectives, allowing for trade-offs between the value and cost of research and the price of the new intervention. Given the current level of evidence, there exists a maximum (threshold) price acceptable to the decision maker. Submission for approval with prices above this threshold will be refused. Given the current level of evidence and the decision maker's threshold price, there exists a minimum (threshold) price acceptable to the company. If the decision maker's threshold price exceeds the company's, then current evidence is sufficient since any price between the thresholds is acceptable to both. On the other hand, if the decision maker's threshold price is lower than the company's, then no price is acceptable to both and the company's optimal strategy is to commission additional research. The methods are illustrated using a recent example from the literature. [ABSTRACT FROM AUTHOR]

The National Institute for Health and Clinical Excellence (NICE) invited the manufacturer of eltrombopag (GlaxoSmithKline) to submit evidence for the clinical and cost effectiveness of this drug for the treatment of patients with chronic immune or idiopathic thrombocytopenic purpura (ITP), as part of the their Single Technology Appraisal (STA) process. The Aberdeen Technology Assessment Review (TAR) Group, commissioned to act as the evidence review group (ERG), critically reviewed and supplemented the submitted evidence. This paper describes the company submission, the ERG review and NICE's subsequent decisions. The ERG critically appraised the clinical and cost-effectiveness evidence submitted by the manufacturer, independently searched for relevant literature, conducted a critical appraisal of the submitted economic models and explored the impact of altering some of the key model assumptions as well as combining relevant sensitivity analyses. Three trials were used to inform the safety and efficacy aspects of this submission; however, one high-quality randomized controlled trial (RAISE study) was the principal source of evidence and was used to inform the economic model. Eltrombopag had greater odds of achieving the primary outcome of a platelet count between 50 × 10^9/L and 400 × 10^9/L during the 6-month treatment period than placebo (odds ratio [OR] 8.2, 99% CI 3.6, 18.7). In the eltrombopag group, 50/83 (60%) of non-splenectomized patients and 18/49 (37%) of splenectomized patients achieved this outcome. The median duration of response was 10.9 weeks for eltrombopag (splenectomized 6 and non-splenectomized 13.4) compared with 0 for placebo. Eltrombopag patients required less rescue medication and had lower odds of bleeding events for both the splenectomized and the non-splenectomized patients. For a watch-and-rescue strategy of care, the comparator was placebo and the ERG found that substantial reductions in the cost of eltrombopag are needed before the incremental cost per QALY is less than d30 000. There was significant uncertainty, with the incremental cost-effectiveness ratio (ICER) reported varying from d33 561 to d103 500 per QALY (splenectomized) and d39 657 to d150 245 per QALY (non-splenectomized). All costs are presented in d, year 2008 values, as this was the costing year for the manufacturer's model. Other than bleeding, no adverse events were modelled. In relation to the long-term treatment model, the ERG questioned the robustness of the use of non-randomized non-comparative data. The base-case results restricting the time horizon to 2 years and prescribing eltrombopag as second-line treatment post-rituximab were found to be favourable towards eltrombopag. As rituximab is not a licensed treatment for ITP, the ERG were concerned that its inclusion may not be reflective of clinical practice. None of the treatment sequences resulted in an ICER approaching the recommended threshold of d30 000 per QALY gained. Eltrombopag appears to be a safe treatment for ITP (although longterm follow-up studies are awaited) and has short-term efficacy. However, NICE found based on the evidence submitted and reviewed that there was no robust evidence on the long-term efficacy or cost effectiveness of eltrombopag and a lack of direct evidence for eltrombopag tested against other relevant comparators. [ABSTRACT FROM AUTHOR]

The objective of this paper is to examine how well the QALY captures the health gains generated by cancer treatments, with particular focus on the methods for constructing QALYs preferred by the UK National Institute for Health and Clinical Excellence (NICE). Data were obtained using a keyword search of the MEDLINE database and a hand search of articles written by leading researchers in the subject area (with follow up of the references in these articles). Key arguments were discussed and developed at an oncology workshop in September 2009 at the Office of Health Economics. Three key issues emerged. First, the EQ-5D, NICE's preferred measure of health-related quality of life (QOL) in adults, has been found to be relatively insensitive to changes in health status of cancer patients. Second, the time trade-off, NICE's preferred technique for estimating the values of health states, involves making assumptions that are likely to be violated in endof- life scenarios. Third, the practice of using valuations of members of the general population, as recommended by NICE, is problematic because such individuals typically display a misunderstanding of what it is really like for patients to live with cancer. Because of the way in which it is constructed, the QALY shows important limitations in terms of its ability to accurately capture the value of the health gains deemed important by cancer patients. A research agenda for addressing these limitations is proposed. [ABSTRACT FROM AUTHOR]

This paper describes the key principles of why an assessment of uncertainty and its consequences are critical for the types of decisions that a body such as the UK National Institute for Health and Clinical Excellence (NICE) has to make. In doing so, it poses the question of whether formal methods may be useful to NICE and its advisory committees in making such assessments. Broadly, these include the following: (i) should probabilistic sensitivity analysis continue to be recommended as a means to characterize parameter uncertainty; (ii) which methods should be used to represent other sources of uncertainty; (iii) when can computationally expensive models be justified and is computation expense a sufficient justification for failing to express uncertainty; (iv) which summary measures of uncertainty should be used to present the results to decision makers; and (v) should formal methods be recommended to inform the assessment of the need for evidence and the consequences of an uncertain decision for the UK NHS? [ABSTRACT FROM AUTHOR]

The use of decision-analytic modelling for the purpose of health technology assessment (HTA) has increased dramatically in recent years. Several guidelines for best practice have emerged in the literature; however, there is no agreed standard for what constitutes a ‘good model’ or how models should be formally assessed. The objective of this paper is to identify, review and consolidate existing guidelines on the use of decision-analytic modelling for the purpose of HTA and to develop a consistent framework against which the quality of models may be assessed.The review and resultant framework are summarised under the three key themes of Structure, Data and Consistency. ‘Structural’ aspects relate to the scope and mathematical structure of the model including the strategies under evaluation. Issues covered under the general heading of ‘Data’ include data identification methods and how uncertainty should be addressed. ‘Consistency’ relates to the overall quality of the model.The review of existing guidelines showed that although authors may provide a consistent message regarding some aspects of modelling, such as the need for transparency, they are contradictory in other areas. Particular areas of disagreement are how data should be incorporated into models and how uncertainty should be assessed.For the purpose of evaluation, the resultant framework is applied to a decision-analytic model developed as part of an appraisal for the National Institute for Health and Clinical Excellence (NICE) in the UK. As a further assessment, the review based on the framework is compared with an assessment provided by an independent experienced modeller not using the framework.It is hoped that the framework developed here may form part of the appraisals process for assessment bodies such as NICE and decision models submitted to peer review journals. However, given the speed with which decision-modelling methodology advances, there is a need for its continual update. [ABSTRACT FROM AUTHOR]

Assessing the cost effectiveness of a new health intervention often requires modelling to estimate the impact of the intervention on cost, survival and quality of life over the lifetime of a cohort of patients. Markov modelling is a methodology that is commonly employed to estimate these long-term costs and benefits. As commonly used, these models assume that the patients continue to get the treatments assigned regardless of the change in health states. In this paper, we describe an extension to the Markov modelling approach, called Markov decision modelling. Such a model starts with a set of health states and treatments and optimally assigns treatments to each of the health states.A Markov decision model can be used to identify the optimal treatment strategy not just for the initial disease state, but also as the disease state changes over time. We present a dynamic programming approach to identifying the optimal assignment of treatments, and illustrate this methodology using an example.The Markov decision modelling approach provides an efficient way of identifying optimal assignment of treatments to health states, but, like the standard Markov model, may be of limited use when probabilities of future events depend on past history in a complex fashion. Even with its limitations, Markov decision models offer an opportunity for health economists to inform healthcare decision-makers on how to modify current treatment pathways to incorporate new treatments as they become available. [ABSTRACT FROM AUTHOR]

The pharmaceutical industry's core business is the innovation, development and marketing of new drugs. Pharmacogenetic (PG) testing and technology has the potential to increase a drug's value in many ways. A critical issue for the industry is whether products in development should be teamed with genetic tests that could segment the total population into responders and non-responders. In this paper we use a cost-effectiveness framework to model the strategic decision-making considerations by pharmaceutical manufacturers as they relate to drug development and the new technology of PG (the science of using genetic markers to predict drug response). In a simple, static, one-period model we consider three drug development strategies: a drug is exclusively developed and marketed to patients with a particular genetic marker; no distinguishing among patients based on the expression of a genetic marker is made (traditional approach); and a strategy whereby a drug is marketed to patients both with and without the genetic marker but there is price discrimination between the two subpopulations. We developed three main principles: revenues under a strategy targeting only the responder subpopulation will never generate more revenue than that which could have been obtained under a traditional approach; total revenues under a targeted PG strategy will be less than that under a traditional approach but higher than a naive [corrected] view would believe them to be; and a traditional [corrected] approach will earn the same total revenues as a price discrimination strategy, assuming no intermarket arbitrage. While these principles relate to the singular (and quite narrow) consideration of drug revenues, they may nevertheless partially explain why PG is not being used as widely as was predicted several years ago when the technology first became available, especially in terms of pharmaceutical manufacturer-developed tests. [ABSTRACT FROM AUTHOR]

Chronic heart failure (CHF) is a modern-day epidemic in most developed countries. As such, it is both common and costly. Contrary to the impression given by clinical trial data, CHF mainly affects older individuals with approximately equal numbers of men and women and concurrent disease profiles likely to complicate or even prohibit the application of proven treatments. It is within this context that there has been an increasing interest in specific CHF-management programmes designed to limit costly hospital use in typically older individuals at high risk for poor quality of life, recurrent readmissions and premature death. This paper examines the evidence to suggest that CHF programmes involving individualised multidisciplinary post-discharge healthcare, with a major focus on specialist nurse management to ensure that the patient receives optimal treatment, are clinically and economically effective in reducing the typical burden imposed by CHF. These programmes appear to be most effective in ‘high-risk’ patients who typically have recurrent readmissions in high-cost units. Overall, the literature suggests that these programmes are able to reduce recurrent hospital stay by 30-50% relative to usual care (even in the presence of gold-standard treatment) in the short to medium term with comparable cost benefits. Recent data from a management programme involving a cohort of typically older and fragile patients with CHF in Australia showed that at 3 years post index admission, hospital utilisation costs were reduced by one-third relative to usual care. The potential for enormous cost benefits (both in terms of absolute cost savings and in terms of facilitating a more efficient healthcare system) if a specialist nurse programme of care was applied in the form of a UK-wide heart failure service was also recently examined. Based on year 2000 activity levels, it was found that for each specialist heart failure nurse appointed in the UK (with a caseload of 200-250 patients per annum), nominal savings of £49 000 per annum could be generated in order to make the healthcare system more efficient. [ABSTRACT FROM AUTHOR]

The National Institute for Clinical Excellence (NICE) responds to requests by the Department of Health for guidance on the use of selected new and established technologies in the National Health Service (NHS) in England and Wales. This paper asks whether the NICE methodological guidelines help NHS decision makers meet the objectives of maximum health improvements from NHS resources and an equitable availability of technologies. The analytical basis of the guidelines is a comparison of the costs and consequences of new and existing methods of dealing with particular conditions using the incremental cost-effectiveness ratio. We explain why information on the costs and consequences of a particular technology in isolation is insufficient to address issues of efficiency of resource use. We argue that to increase efficiency, decision makers need information on opportunity costs. We show that in the absence of such information decision makers cannot identify the efficient use of resources. Finally we argue that economics provides valid methods for identifying the maximisation of health improvements for a given allocation of resources and we describe an alternative practical approach to this problem. Drawing on the experience of Ontario, Canada where an approach similar to that proposed by NICE has been in use for almost a decade, and recent reports about the consequences of NICE decisions to date, we conclude that instead of increasing the efficiency or equity of the use of NHS resources, NICE methodological guidelines may lead to: uncontrolled increases in NHS expenditures without evidence of any increase in total health improvements; increased inequities in the availability of services; and concerns about the sustainability of public funding for new technologies. [ABSTRACT FROM AUTHOR]

This paper analyses the 32 technology appraisals completed by the National Institute for Clinical Excellence (NICE) in the UK from its establishment to the end of January 2002. It looks at why technologies have been rejected, what has happened to products reviewed at launch, evidence of rationing on cost-effectiveness grounds, and the issues raised for manufacturers and for NICE in the collection and analysis of economic data. It finds that around two-thirds of NICE appraisals have been of pharmaceuticals. Only two ‘first in class’ products have been reviewed at launch, with quite different results. There is clear evidence of the use of cost-effectiveness criteria to restrict or reject technologies, although these are not the only criteria used in decision making. While a number of concerns with the appraisal process raised by manufacturers have been addressed by NICE, and while the Department of Health is currently consulting on changes to the referral system whereby products are selected for review by NICE, manufacturers remain concerned about the timing of referrals in the product life cycle and about the quality and consistency of the reviews of evidence undertaken by academic groups for NICE. Concerns in the National Health Service centre on whether the right technologies are being referred to NICE and also on the opportunity cost of positive NICE recommendations. Given global budget constraints and the difficulty of withdrawing services, the NICE recommendations tend to pre-empt growth money that could be used for more cost-effective purposes. NICE should be asked to look at established technologies that may not be cost effective and whose discontinuance could therefore release resources for other more cost-effective treatments. [ABSTRACT FROM AUTHOR]

One of the principal roles of the National Institute for Clinical Excellence (NICE) is to appraise selected existing and emerging healthcare technologies and, as a result, produce guidance for the National Health Service (NHS) in England and Wales. A central part of this appraisal is the potential for manufacturers and sponsors to make a data submission. This paper describes the process of development of the second edition of technical guidance to manufacturers and sponsors for submission to NICE. The revision process took place during the period May 2000 and January 2001 and involved a number of key steps -- establishment of a guidance steering committee, review of current international guidelines of clinical and cost effectiveness, drafting of the guidance, detailed consultation with stakeholders, revision of the guidance and, finally, publication. The lessons learnt from revision of the NICE guidance for manufacturers and sponsors and some main issues for its future development are discussed. [ABSTRACT FROM AUTHOR]