National Institute For Health And Clinical Excellence 's Decisions Research Articles

Decision making by NICE: examining the influences of evidence, process and context

The National Institute for Health and Clinical Excellence (NICE) provides guidance to the National Health Service (NHS) in England and Wales on funding and use of new technologies. This study examined the impact of evidence, process and context factors on NICE decisions in 2004-2009. A data set of NICE decisions pertaining to pharmaceutical technologies was created, including 32 variables extracted from published information. A three-category outcome variable was used, defined as the decision to 'recommend', 'restrict' or 'not recommend' a technology. With multinomial logistic regression, the relative contribution of explanatory variables on NICE decisions was assessed. A total of 65 technology appraisals (118 technologies) were analysed. Of the technologies, 27% were recommended, 58% were restricted and 14% were not recommended by NICE for NHS funding. The multinomial model showed significant associations (p ⩽ 0.10) between NICE outcome and four variables: (i) demonstration of statistical superiority of the primary endpoint in clinical trials by the appraised technology; (ii) the incremental cost-effectiveness ratio (ICER); (iii) the number of pharmaceuticals appraised within the same appraisal; and (iv) the appraisal year. Results confirm the value of a comprehensive and multivariate approach to understanding NICE decision making. New factors affecting NICE decision making were identified, including the effect of clinical superiority, and the effect of process and socio-economic factors.

Dabigatran for the Prevention of Stroke and Systemic Embolism in Atrial Fibrillation: A NICE Single Technology Appraisal

The National Institute for Health and Clinical Excellence (NICE) invited the manufacturer of dabigatran etexilate (Boehringer Ingelheim Ltd, UK) to submit evidence for the clinical and cost-effectiveness of this drug for the prevention of stroke and systemic embolism in patients with non-valvular atrial fibrillation (AF) as part of the NICE single technology appraisal process. The Centre for Reviews and Dissemination and the Centre for Health Economics at the University of York were commissioned to act as the evidence review group (ERG). This article presents a summary of the manufacturer's submission, the ERG report and the subsequent development of NICE guidance for the use of dabigatran within the UK National Health Service. Dabigatran was granted marketing authorisation by the European Medicines Agency for a sequential dosing regimen (DBG sequential), in which patients under 80years are treated with dabigatran 150mg twice daily (DBG150) and patients 80years and over are given dabigatran 110mg twice daily (DBG110). NICE decisions are bound by the marketing authorisation; therefore, the decision problem faced by the committee was whether the DBG sequential regimen was effective and cost-effective compared with warfarin or aspirin for patients with non-valvular AF and one or more risk factors. The RE-LY trial, a large multi-centre non-inferiority randomised clinical trial, was the primary source of clinical evidence. DBG150 was shown to be non-inferior, and subsequently superior to warfarin, for the primary outcome of all stroke/systemic embolism. DBG110 was found to be non-inferior to warfarin. Results were presented for a post hoc subgroup analysis for patients under and over 80years of age, where DBG110 showed a statistically significant reduction of haemorrhagic stroke and intracranial haemorrhage in comparison to warfarin in patients over 80years of age. This post hoc subgroup analysis by age was the basis for the licensed DBG sequential regimen. The economic evaluation compared the costs and outcomes of DBG110, DBG150 and DBG sequential against warfarin, aspirin, and aspirin plus clopidogrel. Across the three dosing regimens, dabigatran was associated with greater costs and better health outcomes than warfarin; however, DBG150 offered the most benefits and dominated DBG110 and DBG sequential (i.e. less costly and more effective). The cost-effectiveness of DBG150 was less favourable for patients well controlled on warfarin. In the first appraisal meeting, the committee issued a 'minded no' decision until additional analyses on the licensed DBG sequential regimen were presented by the manufacturer. These additional analyses indicated that the incremental cost-effectiveness ratio (ICER) of the DBG sequential regimen compared with warfarin ranged from £8,388 to £18,987 per quality-adjusted life year (QALY) gained depending on the level of monitoring costs assumed for warfarin. Patients on warfarin would need to be within therapeutic range 83-85% of the time for the ICER to exceed £30,000 per additional QALY. Following consideration of the additional evidence and the responses from a large number of consultees and commentators, the committee recommended dabigatran as DBG sequential as an option for the prevention of stroke and systemic embolism in people with non-valvular AF with one or more risk factors for ischaemic stroke.

Drug reimbursement recommendations by the National Institute for Health and Clinical Excellence: Have they impacted the National Health Service budget?

ObjectiveDetermine whether reimbursement restrictions recommended by the National Institute for Health and Clinical Excellence (NICE) have impacted the United Kingdom (UK) National Health Service (NHS) budget. MethodsData were abstracted from NICE guidance documents and costing statements through March 2011. Estimated maximum and adjusted potential budget impact (PBI) on the NHS was derived using estimates of the UK marketing-approved population and the annual cost for the new drug. Descriptive and logistic analyses were used to estimate the correlation between the degree of restrictions on reimbursement recommended by NICE for each new drug indication and the PBI controlling for clinical effectiveness and cost-effectiveness. ResultsPBI was significantly correlated with the degree of reimbursement restrictions. In descriptive analysis, the adjusted PBI for drugs that were recommended without restrictions was £20.3million (SD=22.2) compared with £49.8million (SD=90.8) for those recommended with restrictions and £71.1million (SE=99.9) for those not recommended. In logistic analysis, the odds ratio for less restrictive reimbursement was 0.848 (95% CI, 0.762–0.945) for each £20million increase in the adjusted PBI. Results were similar using the maximum PBI. ConclusionsAfter controlling for clinical effectiveness and cost-effectiveness, the degree of reimbursement restriction recommended by NICE remains significantly correlated with the PBI, despite that fact that the NICE decision process does not consider budget impact. This correlation might be due to NICE consideration of effectiveness and cost-effectiveness for subgroups of the approved population.

PHP116 Clincal and Economic Evidence Bases for Health Technology Assessment: A Comparison of Three Jurisdictions

To understand how different evidence bases may contribute to health technology assessment (HTA) across jurisdictions, we reviewed the evidence considered by three HTA agencies supporting reimbursement recommendations for nine drugs. We selected nine drugs for which the Canadian Common Drug Review (CDR), the Australian Pharmaceutical Benefits Advisory Committee (PBAC), and the National Institute for Health and Clinical Excellence (NICE) had each provided recent reimbursement recommendations. We reviewed the clinical and economic evidence considered for each decision and evaluated whether different evidence bases could have contributed to different HTA decisions. The three HTA agencies agreed (recommended to reimburse) for four drugs and reached different recommendations for five drugs. In both categories, somewhat different evidence bases were used by each agency. For a given drug, different comparators were sometimes considered by different agencies. Even when comparators were common across agencies, there was variability regarding which clinical trials were considered. All agencies considered data from direct, randomized trials, but PBAC and NICE accepted indirect comparisons, whereas CDR did not. Regarding economic outcomes, all NICE decisions made use of cost-effectiveness (mostly cost-utility) analyses, but cost-minimization approaches were considered by CDR and PBAC for several drugs. Overall, NICE provided the most transparent reporting on decision making, and CDR was generally the least transparent of the three. HTA drug decisions across the three countries lack good agreement, and considerable variability exists in the clinical and economic bases considered by CDR, PBAC, and NICE. The reluctance of CDR to accept indirect clinical comparisons, and the propensity of NICE to heed expert advice when analyses were inconclusive may contribute to dissimilar decisions being reached for some drugs. Greater transparency and harmonization of HTA methods have the potential to improve efficiency in health care decision-making, and further research analyzing additional HTA drug decisions is warranted.

The NHS: assessing new technologies, NICE and value for money

The healthcare system in the UK, essentially the NHS, is an open economic system subject to the same pressures as any other economic system. The pressures concern limited resources coupled with powerful drivers for increasing spending: invention, demography and inflation. There have only ever been three types of economic system: steady state (everything, as in a feudal system, stays as it was the year before), market capitalism (supply and demand are allowed to find their own equilibrium) and some version of central planning. In healthcare, most advanced countries favour the last of the three. This is for three reasons: distribution (not only are the poor less able to pay for sickness, but sickness exacerbates poverty), information (markets operate poorly when providers can easily outsmart customers) and externalities (it is in the interest of everyone that infectious diseases and the other knock-on consequences of ill health are ameliorated). So in the UK, the state, with a good deal of cross-party consensus, directs most of health service supply. This system has become more complex over the decades since the formation of the NHS in 1948. A notable element of the complexity is the regulation of the introduction of new technologies. A key element of the regulatory system has been the National Institute for Health and Clinical Excellence (NICE), and a key aspect of NICE's decisions has been not just value, but also value for money. This has not been without controversy.

The Economics of the NHS Cancer Drugs Fund

In their 2010 election campaign, the Conservative party pledged to create a fund to provide access to cancer drugs that are recommended by doctors but that would not normally be reimbursed by the English NHS, either because the National Institute for Health and Clinical Excellence (NICE) recommended against their reimbursement, or because they have not yet been appraised by NICE. Elected to government in May 2010, the Conservatives, in coalition with the Liberal Democrats, have honoured their pledge and are increasing funding for cancer care. An initial fund of £50 million was introduced between October 2010 and April 2011, and a further £200 million per year has been allocated from April 2011 to 2014.[1] This is an interim measure until a 'value-based pricing' mechanism is introduced in 2014. Preferential funding of disease areas such as cancer without evidence of the efficiency may be politically advantageous, but it is likely to be inefficient, depriving potential patients of care from which they could benefit. One explanation of this special funding increase has been the use of comparative international tables of survival in cancer, which showed the NHS as lagging behind the achievements of other European countries.[2] During the Blair administration, NHS funding grew on average by 7% annually.[3] Some of these additional funds were targeted at areas where performance was deemed to be poor. Cancer was one of these areas. The current Coalition Government criticises the achievements of the Blair funding increase as failing to improve performance. Cancer mortality rates in the UK continue to be worse than many other European countries.[2] However, as highlighted by Appleby,[2] the focus should not be on absolute rates alone but on rates of change. He has shown that the Blair funding increases have taken time to impact but trends are clearly established and, for instance, in the next year, UK cancer mortality rates will be better than those of France. Cancer, for many, is a chronic condition in which good management can result in many additional years of good-quality life. Treatments, especially newer pharmaceuticals, are often expensive, as industry innovates and requires high prices for products that often offer only marginal improvements in outcomes for patients. Consequently, some of these products are not recommended by NICE as their benefits do not justify their costs. This is despite NICE already having a higher cost-per-QALY threshold for 'end-of-life' drugs.[4] The Cancer Drugs Fund not only undermines NICE decision making and weakens incentives for companies to price their products at a level that is affordable and justified by health improvements, but also singles out a particular disease for favourable treatment in an essentially arbitrary manner. The emotive power of this disease led politicians to capture support by singling it out for preference. Why favour cancer when there are so many other life-threatening and disabling diseases (such as heart disease, multiple sclerosis, respiratory disease, dementia and diabetes) that, through lobbies (often funded by sympathetic and commercially dependent pharmaceutical companies), clamour for more funding? One way of rationing scarce resources and prioritizing investments in a healthcare system is to focus on political advantage. …

An Analysis of NICEʼs ‘Restricted’ (or ‘Optimized’) Decisions

A common way of describing UK National Institute for Health and Clinical Excellence (NICE) decisions is to distinguish between cases where NICE recommended use of a healthcare technology by all relevant patients ('yes'); those where it did not recommend use ('no'); and those where its decisions are a mixture of 'yes' to some patient subgroups, and 'no' to others. Over half of NICE's decisions are of this mixed type, which involve restricting (or 'optimizing') patient use in some way. To report an attempt to develop a robust and defensible means of measuring and describing the degree of patient access in mixed NICE decisions. A list of mixed decisions made from 2006 to the end of 2009 was identified using HTAinSite™. The following calculation was used: M = (p/P) × 100, where M is a measure of the level of patient access (0 = no access, 100 = full access), P is the set of patients considered in the guidance as Potential candidates for treatment (given the licensed use and the scope of NICE's appraisal), and p is a subset of those patients, for whom NICE did recommend treatment. M can be estimated either for a specific product or for a group of technologies (Multiple Technology Appraisals). Both product-specific and overall M were estimated, using estimates of p obtained from NICE costing templates. These data are subject to some important limitations, so the results should be regarded as illustrative. Of the 69 medicines that have received a mixed decision since January 2006, 34 included details that allowed the estimation of M. Of these 34 decisions, 24 (71%) had a product-specific M ≤50, 16 (47%) M ≤25 and 11 (32%) M ≤10. That is, in just under three-quarters of the mixed decisions for which P and p were available, NICE recommended use for less than half of patients for whom the medicine is licensed, and in nearly one-third of these sorts of decisions, NICE recommended use in ≤10% of potential patients. The estimates of M for groups of technologies provide a slightly different picture: for example, grouped M was ≤10 in <20% of decisions. The measure of patient access, M, proposed here has the potential to provide a more informative way of reporting all NICE decisions, particularly 'restricted' (or 'optimized') decisions.

050 News media coverage of NICE's decisions on new health technologies

ObjectiveThis project aims to: (1) describe the frequency of news coverage in mass media related to the National Institute for Health and Clinical Excellence (NICE) draft or final guidance; (2)...

From rationing to rational: the evolving status of NICE.

The National Institute for Health and Clinical Excellence (NICE) underwent a Health Select Committee (HSC) Inquiry in April 2007, the second since its inception in 1999. The inquiry was set up to examine the reasons why NICE's decisions have witnessed increasing challenges and whether public confidence in NICE has waned. Consequently the focus was on the technology appraisal programme and its mandatory recommendations, although its clinical guidelines programme came under scrutiny too. We appraise the challenges now facing NICE in light of the HSC's conclusions,1 the subsequent government response to the HSC's recommendations2 published in March 2008 and the recent Darzi report.3 The HSC report is exhaustive, running to over 100 pages, and draws upon submissions from over 116 different groups with witness testimony from over 30 representatives including patients, clinical and non clinical health professionals, senior NICE representatives, Department of Health representatives and a Health Minister. The task of the HSC was formidable – to investigate the function of what is essentially a regulatory agency by evaluating feedback from those whom it regulates. The challenges facing such an inquiry are obvious – the submissions are from self-selected groups, many of whom are complainants, and the inquiry aspired to report objective findings to improve NICE processes. Despite these challenges, the inquiry has largely succeeded in identifying and differentiating the ‘special pleading’ of vested interests from genuine issues. We review the findings of the inquiry in relation to (1) the current role of NICE, (2) the role of health care professionals and their representative organizations, (3) the role of patient interest groups, (4) the role of the pharmaceutical and devices industry, (5) NICE, the media and public perception and (6) guidance development and implementation.

Revised NICE guidance on drugs for Alzheimer's disease and its impact on patients and carers

In November 2006 the National Institute for Health and Clinical Excellence (NICE) published the final review of its guidance on the use of anticholinesterase drugs and memantine hydrochloride for the treatment of Alzheimer's disease. Recommendations included a reduction in the use of anticholinesterase drugs, limiting them to the treatment of patients in the moderate-to-severe stages of the disease and an end to the use of memantine for newly diagnosed patients. This article examines the impact of this NICE decision on those people suffering from Alzheimer's disease and their carers and families.

The National Institute for Health and Clinical Excellence (NICE) and Drug Treatment for Alzheimer???s Disease

Britain's National Institute for Health and Clinical Excellence (NICE) has recently issued guidance that restricts the use of cholinesterase inhibitors and memantine for the treatment of Alzheimer's disease in the National Health Service. This stance contains lessons for designers of trials, drug regulators, health economists and those developing clinical guidelines for dementia care. The debates that took place around and within NICE were about identifying the benefits of these medicines and the beneficiaries, clarifying the costs of the medication and whom bears them, the methods of weighing benefit against cost, and the consequences of using different approaches to cost-benefit analysis. This article discusses each of these themes and outlines the changes in research and clinical practice and policy making that might flow from NICE's decisions on medication use. Outcome measures that capture changes in dementia syndromes need further development. Cost-benefit analysis needs refinement with better tools than quality-adjusted life-years, and the policy implications of restricting treatments in a progressive neurodegenerative disorder need more careful consideration.

“Yes”, “No” or “Yes, but”? Multinomial modelling of NICE decision-making

The National Institute for Health and Clinical Excellence (NICE) issues mandatory guidance on health technologies to the UK NHS, based on clinical evidence, cost-effectiveness and other considerations. However, the exact factors considered, their relative importance and tradeoffs between them are not made explicit. Previous research modelled NICE decisions as a binary choice (accept/reject) dependent on cost-effectiveness, amongst other variables. This paper proposes and tests an alternative model of decision-making that may better represent the “yes, but…” nature of many NICE decisions. Decisions were categorised as “recommended for routine use”, “recommended for restricted use” or “not recommended”. The NICE appraisal process was modelled as a single decision between the three categories. Multinomial logistic regression techniques were used to evaluate the impact of: quantity/quality of clinical evidence; cost-effectiveness; decision date; existence of alternative treatments; budget impact; technology type. Results suggest that interventions supported by more randomised trials are more likely to be recommended and endorsed for routine use. Higher cost-effectiveness ratios increased the likelihood of interventions being rejected rather than recommended for restricted use but did not significantly affect the decision between routine and restricted use. Pharmaceuticals, interventions appraised early in the NICE programme and those with more systematic reviews were also less likely to be rejected, while patient group submissions made a recommendation for routine rather than restricted use more likely. The presence of factors affecting the decision between routine and restricted use but not that between routine use and rejection suggests that modelling these three outcomes reflects NICE decision-making more closely than binary-choice analyses.