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Objectives: In many countries, future unrelated medical costs occurring during life-years gained are excluded from economic evaluation, and benefits of unrelated medical care are implicitly included, leading to life-extending interventions being disproportionately favored over quality of life-improving interventions. This article provides a standardized framework for the inclusion of future unrelated medical costs and demonstrates how this framework can be applied in England and Wales.Methods: Data sources are combined to construct estimates of per-capita National Health Service spending by age, sex, and time to death, and a framework is developed for adjusting these estimates for costs of related diseases. Using survival curves from 3 empirical examples illustrates how our estimates for unrelated National Health Service spending can be used to include unrelated medical costs in cost-effectiveness analysis and the impact depending on age, life-years gained, and baseline costs of the target group.Results: Our results show that including future unrelated medical costs is feasible and standardizable. Empirical examples show that this inclusion leads to an increase in the ICER of between 7% and 13%.Conclusions: This article contributes to the methodology debate over unrelated costs and how to systematically include them in economic evaluation. Results show that it is both important and possible to include future unrelated medical costs. [ABSTRACT FROM AUTHOR]

Introduction: The appraisal of medicines is often a complex and iterative process. We compared the health technology assessment (HTA) process in England and France taking as a case study the example of ixazomib for multiple myeloma. Methods: We undertook an analysis of eight relevant published documents identifed from the websites of the French and English HTA bodies (HAS and NICE, respectively). We analyse patients' availability of ixazomib resulting in the different stages of the appraisal process. Results: We identified differences in the assessment, one of these being the use of an appraisal scope in England allowing the differentiation of populations and comparators according to previously approved treatments. Ixazomib became available earlier in France as part of an early access programme, but the availability was soon discontinued for newly eligible patients following an HAS determination that Ixazomib yielded no additional benefit. This opinion resulted in long pricing discussions. In England, despite the absence of an early access programme and following a process that included cost-effectiveness evaluation combined with pricing discussions, the medicine was fairly rapidly recommended for use. Conclusions: Differences in the HTA process may result in appreciable differences in time from marketing authorisation to health service adoption of newly licensed drugs. [ABSTRACT FROM AUTHOR]

Objective Diagnostic Assessment Reviews are part of the work programme of the National Institute of Health and Care Excellence (NICE) in England to evaluate emergent medical technologies and so ensure timely and consistent adoption within the National Health Service (NHS). New diagnostics have the potential to improve health outcomes and impact positively on health service resource use. This study sought to assess the quality of economic evidence informing Diagnostic Assessment Reviews. Methods We reviewed 27 Diagnostic Assessment Reviews that had been published by NICE as of 30 May 2017 by summarizing and interpreting the evidence that was used to carry out cost-effectiveness analyses. Common issues and challenges of the assessment process were illustrated. Findings: DARs differed in the methods and assumptions used to construct economic models, and linkage of economic model and diagnostic findings. Even though some diagnostic technologies were estimated to be cost-effective, they were not always adopted for routine use in the NHS. The majority of Diagnostic Assessment Review economic models relied heavily on assumptions and expert opinion, with considerable uncertainty about the cost-effectiveness of diagnostic testing. Conclusions Diagnostic Assessment Reviews appraisals as commissioned by NICE typically feature varying evidence for diagnostic performance and limited evidence for resource implications and quality of life, often leading to recommendations for further research. Given the process opportunity cost, a two-stage topic selection process, with initial assessment specifying further research and proceeding to full assessment when adequate evidence is available may be more appropriate and help signal to diagnostics companies the type of research evidence required. [ABSTRACT FROM AUTHOR]

A new version of the EQ-5D, the EQ-5D-5L, is available. The aim of this study is to produce a value set to support use of EQ-5D-5L data in decision-making. The study design followed an international research protocol. Randomly selected members of the English general public completed 10 time trade-off and 7 discrete choice experiment tasks in face-to-face interviews. A 20-parameter hybrid model was used to combine time trade-off and discrete choice experiment data to generate values for the 3,125 EQ-5D-5L health states. Valuation data are available for 996 respondents. Face validity of the data has been demonstrated, with more severe health states generally given lower values. Problems with pain/discomfort and anxiety/depression received the greatest weight. Compared to the existing EQ-5D-3L value set, there are considerably fewer "worse than dead" states (5.1%, compared with over one third), and the minimum value is higher. Values range from -0.285 (extreme problems on all dimensions) to 0.950 (for health states 11211 and 21111). Results have important implications for users of the EQ-5D-5L both in England and internationally. Quality-adjusted life year gains from interventions seeking to improve very poor health may be smaller using this value set and may previously have been overestimated. [ABSTRACT FROM AUTHOR]

In 2016 the National Institute for Health and Care Excellence (NICE) published a guideline on oral health for adults in care homes In England. The author was a co-opted memberof the NICE oral health for adults in care homes public health advisory committee. This article reviews the NICE guideline as it applies to care homes, and relates it to the results of a survey of oral care practice undertaken in a large core home organisation and the available research literature from the past 20 years. The literature and survey results suggest that, if translated into practice, the NICE guideline could do much to improve oral health for adults in care homes. The survey highlighted that 85% of residents required support from carers to undertake mouth care. It also found that care homes experienced significant difficulties in accessing dental services for residents. The author concludes that providers need to equip staff with the necessary knowledge and skills to undertake mouth care and to give this area of personal care greater priority. Finally, the author suggests that the Care Quality Commission could ensure that the NICE guideline is translated into practice in care homes. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to compare reimbursement recommendations by the British National Institute for Health and Care Excellence (NICE) and the French National Authority for Health (Haute Autorité de Santé; HAS) for oncology new medicines indicated for the treatment of solid tumors., Methods: Public assessment reports published on NICE and HAS Web sites between January 1, 2015, and December 31, 2021, describing appraisals for public reimbursement for oncology new medicines indicated for the treatment of solid tumors were searched and systematically reviewed. Biosimilars and generic drugs were excluded from the analysis., Results: Overall, 119 public assessment reports published by NICE and 134 by HAS were identified, with 101 interventions assessed by both agencies. Of 101, 38 (38%) interventions received similar recommendations, 38 (38%) were recommended for different populations, and 25 (25%) were endorsed by one agency but rejected by the other. The main reason NICE would not recommend a drug was due to lack of cost-effectiveness, whereas HAS would primarily reject a drug on the grounds of insufficient clinical evidence., Conclusion: The major divergence between agencies is the key criterion used for reimbursement recommendations. NICE mainly considers cost-effectiveness, whereas HAS primarily focuses on the clinical value of interventions. This contributes to the variability in reimbursement recommendations and leads to differential access to oncology new medicines indicated for the treatment of solid tumors among patients in France, and England and Wales., Highlights: Both the National Institute for Health and Care Excellence (NICE) and the National Authority for Health (Haute Autorité de Santé; HAS) have established formal health technology assessment (HTA) processes and offer universal public health care coverage. However, both agencies diverge in the weight given to different elements of evidence during HTA evaluations. NICE uses cost-effectiveness as key criterion for recommendations on drug reimbursement, while HAS mostly limits its assessment to clinical value.For oncology new medicines indicated for treating solid tumors between 2015 and 2021, recommendations differed 62% of the time between NICE and HAS, primarily due to the distinct key decision-making criteria each HTA agency uses.For 4 interventions not endorsed by NICE, HAS saw these drugs as providing a substantial enhancement in clinical value over existing treatments, potentially providing an edge in price negotiations. Conversely, NICE deemed these drugs as not delivering adequate value for money in comparison with current therapies.A key difference between the 2 agencies is HAS's insistence on methodological rigor in efficacy results, compared with NICE's more flexible approach, considering descriptive efficacy results in cost-effectiveness modeling., Competing Interests: The authors declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Both authors were employed by Pierre Fabre Laboratories at the time of the development of the study. The authors disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: Financial support for this study was provided by Pierre Fabre Laboratories. The funding agreement ensured the authors’ independence in designing the study, interpreting the data, writing, and publishing the report. All authors were employed by the sponsor at the time of the development of the study.

Children are not mini-adults, and thus require studies to be conducted in the population of interest to inform decisions about their care. The paucity of such studies for clinical efficacy lead them to be termed ‘therapeutic orphans’. Following the introduction of the ‘fourth hurdle’ of reimbursement approval on the basis of cost-utility analysis, utility data is now a key requirement for patients to access treatments in England and many other countries. This special report considers whether a paucity of utility valuation studies in children may have made them ‘economic orphans’ as well and presents results of a review of NICE appraisals as a window on this problem over time. [ABSTRACT FROM PUBLISHER]

A source of debate in the field of health care priority setting is whether health gains should be weighted differently for different groups of patients. The debate has recently focused on the relative value of life extensions for patients with short life expectancy. However, few studies have examined empirically whether society is prepared to fund life-extending end-of-life treatments that would not meet the reimbursement criteria used for other treatments. A web-based discrete choice experiment was conducted in 2012 using a sample of 3969 members of the general public in England and Wales. The study design was informed by the National Institute for Health and Care Excellence's supplementary policy for the appraisal of life-extending end-of-life treatments. The choice tasks involved asking respondents which of two hypothetical patients they would prefer to treat, assuming that the health service has enough funds to treat only one of them. Conditional logit regressions were used for modelling. Choices about which patient to treat were influenced more by the sizes of treatment gains than by patients' life expectancy without treatment. Some respondents appear to support a health-maximisation type objective throughout, whilst a small minority always seek to treat those who are worse off without treatment. The majority of respondents, however, seem to advocate a mixture of the two approaches. Overall, we find little evidence that members of the general public prefer to give higher priority to life-extending end-of-life treatments than to other types of treatment. When asked to make decisions about the treatment of hypothetical patients with relatively short life expectancies, most people's choices are driven by the size of the health gains offered by treatment. [ABSTRACT FROM AUTHOR]

Abstract: Clinical practice guidelines produced by NICE – the National Institute for Health and Care Excellence – are seen as key mechanisms to regulate and standardise UK healthcare practice, but their development is known to be problematic, and their adoption and uptake variable. Examining what a guideline or health policy means to different audiences, and how it means something to those communities, provides new insight about interpretive discourses. In this paper we present a micro-analysis of the response of healthcare professionals to publication of a single NICE guideline in 2009 which proposed a re-organisation of professional services for chronic non-specific low back pain. Adopting an interpretive approach, we seek to understand both the meaning of the guideline and the socio-political events associated with it. Drawing on archived policy documents related to the development and publication of the guideline, texts published in professional journals and on web-sites, and semi-structured interview data from professionals associated with the debate, we identify a key discourse that positions the management of chronic non-specific low back pain within physician jurisdiction. We examine the emergence of this discourse through policy-related symbolic artifacts taking the form of specific languages, objects and acts. This discourse effectively resisted and displaced the service reorganisation proposed by the guideline and, in so doing, ensured medical hegemony within practice and professional organisations concerned with the management of non-specific low back pain. [Copyright &y& Elsevier]

The National Institute for Health and Care Excellence (NICE) is the independent organisation in England responsible for developing national guidance, standards, and information on providing high-quality health and social care, and preventing and treating ill health. Editing plays a major role in this process, by helping to ensure that published guidance from NICE lacks mistakes, omissions, and ambiguities and that it is easy to understand for both healthcare professionals and the public. [ABSTRACT FROM AUTHOR]

Background: Venous thromboembolism (VTE) is a major cause of morbidity and mortality in hospitalised patients. VTE prevention has been identified as a major health need internationally to improve patient safety. A National Institute for Health and Clinical Excellence (NICE) guideline was issued in February 2010. Its key priorities were to assess patients for risk of VTE on admission to hospital, assess patients for bleeding risk and evaluate the risks and benefits of prescribing VTE prophylaxis. The aim of this study was to evaluate the implementation of NICE guidance and its impact on patient safety. Methods: A before-after observational design was used to investigate changes in VTE risk assessment documentation and inappropriate prescribing of prophylaxis between the year prior to (2009) and the year following (2010) the implementation of NICE guidance, using data from a 3-week period during each year. A total of 408 patients were sampled in each year across four hospitals in the NHS South region.Results: Implementation strategies such as audit, education and training were used. The percentage of patients for whom a VTE risk assessment was documented increased from 51.5% (210/408) in 2009 to 79.2% (323/408) in 2010; difference 27.7% (95% CI: 21.4% to 33.9%; p < 0.001). There was little evidence of change in the percentage who were prescribed prophylaxis amongst patients without a risk assessment (71.7% (142/198) in 2009 and 68.2% (58/85) in 2010; difference −3.5% (95% CI: -15.2% to 8.2%; p =0.56) nor the percentage who were prescribed low molecular weight heparin amongst patients with a contraindication (14% (4/28) in 2009 and 15% (6/41) in 2010; RD = 0.3% (95% CI: -16.5% to 17.2%; p =0.97). Conclusions: The documentation of risk assessment improved following the implementation of NICE guidance; it is questionable, however, whether this led to improved patient safety with respect to prescribing appropriate prophylaxis. [ABSTRACT FROM AUTHOR]

Abstract: Background: Hip fracture is a common injury with associated high mortality. Recent drives by the Department of Health have sought to prioritise these patients’ care. In April 2010, the Best Practice Tariff was introduced in England and Wales. This offers financial incentives to institutions that provide holistic care and surgery within 36h for hip fracture patients. The England and Wales National Institute for Health and Clinical Excellence (NICE) published its first guidance on hip fracture management in June 2011, and emphasised the need for surgery on the day or day after admission. In spite of the emphasis placed on this injury, the predictors of in-hospital mortality remain ill-defined. In particular the effect of the timing of surgery remains contentious. Objective: To address the issues raised by NICE around surgical timing and examine whether surgery before a 36h watershed improves survival. In addition, to examine survival outcomes for each 12h watershed following admission. Materials and methods: Prospectively collected data on 2056 patients presenting to our unit with hip fractures between February 2008 and May 2011 were retrospectively reviewed. Multivariate regression analysis was used to correct for confounders, and so determine the effect of various parameters on in-patient mortality. Results: Age (p <0.0001), male-gender (p <0.0001), source of admission (p <0.05), ASA-grade (p <0.0001) and delay of surgery (p <0.01) were associated with an increased risk of in-hospital mortality. The adjusted odds of in-hospital mortality were 1.58 (p <0.05) times higher in those undergoing surgery after 36h compared to surgery before this time. Early surgery (within 24h) resulted in reduced in-hospital mortality when compared to the 36h watershed. Similarly ultra-early surgery (within 12h) was even better still (adjusted odds ratio 3.9 p <0.05). Conclusions: Expeditious surgery is associated with improved patient survival. Other predictors of in-hospital mortality include age, gender, in-hospital fracture and ASA-grade. Ultra-early surgery (within 12h) reduces risk of in-hospital mortality. [Copyright &y& Elsevier]

New medicines represent exciting possibilities to individual patients for improved futures through reduced morbidity, as well as the potential for disappointment where promised outcomes are not forthcoming. For healthcare systems these technologies can enable enhanced effectiveness as well as spiralling costs, depending on value-for-money offered. Amidst uncertainty over efficacy and pricing, institutions such as the National Institute for Health and Clinical Excellence have been set up to regulate the cost-effectiveness of new products. The increased transparency of this new regulation, combined with inescapable uncertainties, leads to the heightened politicisation of decision-making and an insidious subjectivity beneath a veneer of rational-bureaucracy. This paper develops a theoretical framework for understanding the influence of hope within such contexts. Conceptualised here as ideological, affective and prone to lapsing into trust, hope is considered to shape the lifeworld-background of regulatory decisions and mask uncertainty around effectiveness. These processes challenge regulator legitimacy and effectiveness within polycentric regimes, through the distortion of communication and the manipulation of blame. [ABSTRACT FROM AUTHOR]

Abstract: 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. [ABSTRACT FROM AUTHOR]

Aims: To explore health technology assessment (HTA) outcomes of matched drug pairs by national agencies in Germany (Gemeinsamer Bundesausschuss, GBA), France (Haute Autorité de Santé, HAS) and England and Wales (NICE). Methods: We considered published GBA decisions, HAS reports and NICE guidance from January 2011 to June 2018. HTAs of matched pairs were compared overall, and for non-cancer and cancer drugs separately. We further analyzed the role of additional attributes related to cancer therapies. Results: Matched pairs show higher concordance for GBA/HAS than for GBA/NICE and HAS/NICE. Overall, NICE evaluated technologies more favorably than GBA and HAS. GBA appraisals of cancer drugs, however, tended to be more positive than cancer-related recommendations by NICE and HAS. Conclusion: The findings indicate substantial variations in HTAs, although cancer-related outcomes seem to diverge less than non-cancer results.

Introduction: Ocrelizumab is the first approved drug for primary progressive multiple sclerosis. Following appraisal by health technology assessment (HTA) bodies, this medicine has not been widely covered across European countries. We have compared the HTA process in England and France., Area Covered: We undertook an analysis of relevant documents that were published by the two HTA bodies. We analyzed patients' availability of Ocrelizumab at the different stages of the process., Expert Opinion: We identified differences in the assessment, one being the use of a different population of the pivotal trial, which has resulted in the consideration of distinct clinical effectiveness estimates. Ocrelizumab became available earlier in France as part of an early access program. However, rapid access was discontinued for newly eligible patients following an opinion concluding that Ocrelizumab yielded no additional benefit over placebo. This opinion was not compatible with the criteria allowing reimbursement in France.In England, there was no early access program and following an appraisal that included cost-effectiveness evaluation combined with pricing agreements, medicine was finally recommended. In conclusion, differences in the HTA process may result in appreciable differences in timing and outcome from marketing authorization to the adoption of newly licensed drugs.

Background: Treatment options at third-line and beyond for patients with late-line metastatic colorectal cancer (mCRC) are limited, and outcomes are poor with best supportive care (BSC). This study investigated the cost-effectiveness of trifluridine/tipiracil and regorafenib relative to BSC alone in patients with mCRC who have been previously treated with, or are not considered candidates for, standard chemotherapies., Materials and Methods: A partitioned survival model was constructed to assess the lifetime costs and benefits accrued by patients. Clinical data were derived from the pivotal phase III (Randomized, Double-Blind, Phase 3 Study of TAS-102 plus Best Supportive Care [BSC] versus Placebo plus BSC in Patients with Metastatic Colorectal Cancer Refractory to Standard Chemotherapies [RECOURSE]) and supporting phase II (J003-10040030) randomized controlled trial of trifluridine/tipiracil + BSC versus placebo + BSC, as well as the phase III Colorectal Cancer Treated With Regorafenib or Placebo After Failure of Standard Therapy (CORRECT) randomized controlled trial of regorafenib, and were extrapolated to estimate lifetime outcomes. Costs were taken from published sources, and health effects sourced from previous mCRC studies., Results: Trifluridine/tipiracil was associated with a 0.27 incremental life year versus BSC alone, which corresponds to a 0.17 quality-adjusted life year gain. The incremental cost of treatment with trifluridine/tipiracil was £8,479, resulting in an incremental cost-effectiveness ratio of £51,194 per quality-adjusted life year gained. Trifluridine/tipiracil was shown to dominate regorafenib (improve outcomes with reduced costs). Sensitivity analyses showed principal areas of uncertainty were survival estimates and patient utility., Conclusions: The results show that trifluridine/tipiracil is more clinically and cost-effective than regorafenib, with clinical outcomes greatly exceeding those for patients treated with BSC alone. Based on the results of the analysis, trifluridine/tipiracil offers an important new treatment option for patients with mCRC maintaining good performance status at the end of life., (Copyright © 2017 The Authors. Published by Elsevier Inc. All rights reserved.)

Summary: Objectives: To determine the utility of ‘risk assessment’ in selecting Mycobacterium tuberculosis isolates for rifampin resistance or rpoB genotyping compared to ‘non-selectively’ genotyping all isolates. Secondly, we examined the association between past treatment and drug resistance. Methods: From January 2003 to December 2006, demographic, clinical, and laboratory data were prospectively collected on patients with laboratory-confirmed tuberculosis (TB). On the basis of past treatment for active TB infection or known exposure to drug-resistant TB, selected samples were sent to a mycobacterial reference laboratory for rpoB genotyping. A multivariable logistic regression model was developed to examine the association between past treatment and drug resistance, adjusted for other factors. Sensitivity, specificity, and negative and positive predictive values of past treatment as a predictor for drug resistance were determined. Results: There were 392 patient episodes of culture-proven TB. Thirty-three drug-resistant isolates were cultured from 30 patients: 29 (87.9%) were isoniazid-resistant, three (9.1%) were multidrug-resistant (MDR), and one (3.0%) was rifampin mono-resistant. One patient with isoniazid resistance developed recurrent disease, and two isolates, initially isoniazid-resistant, mutated and became MDR TB. Based on risk assessment, rpoB genotyping was performed on 19 samples, and two (10.5%) had mutations that predicted multiple drug resistance. Although for MDR TB, a past history of treatment predicted two out of three patients with acquired resistance, adjusted analysis did not demonstrate a significant association between previous treatment of active TB and drug resistance (odds ratio 1.5, 95% confidence interval (CI) 0.4–5.6). The positive predictive value of past treatment as a predictor for drug resistance was 12.0% (95% CI 2.6–31.2%). Conclusion: Although numbers of MDR TB were too small to draw meaningful conclusions, past treatment may be useful in selecting samples for rpoB genotyping. Overall, previous treatment had a low positive predictive value for drug resistance in an area bordering East London. [Copyright &y& Elsevier]

Background: The English Improving Access to Psychological Therapies (IAPT) initiative aims to make evidence-based psychological therapies for depression and anxiety disorder more widely available in the National Health Service (NHS). 32 IAPT services based on a stepped care model were established in the first year of the programme. We report on the reliable recovery rates achieved by patients treated in the services and identify predictors of recovery at patient level, service level, and as a function of compliance with National Institute of Health and Care Excellence (NICE) Treatment Guidelines., Method: Data from 19,395 patients who were clinical cases at intake, attended at least two sessions, had at least two outcomes scores and had completed their treatment during the period were analysed. Outcome was assessed with the patient health questionnaire depression scale (PHQ-9) and the anxiety scale (GAD-7)., Results: Data completeness was high for a routine cohort study. Over 91% of treated patients had paired (pre-post) outcome scores. Overall, 40.3% of patients were reliably recovered at post-treatment, 63.7% showed reliable improvement and 6.6% showed reliable deterioration. Most patients received treatments that were recommended by NICE. When a treatment not recommended by NICE was provided, recovery rates were reduced. Service characteristics that predicted higher reliable recovery rates were: high average number of therapy sessions; higher step-up rates among individuals who started with low intensity treatment; larger services; and a larger proportion of experienced staff., Conclusions: Compliance with the IAPT clinical model is associated with enhanced rates of reliable recovery., (Copyright © 2013 The Authors. Published by Elsevier Ltd.. All rights reserved.)