Interventions to reduce the risk of surgically transmitted Creutzfeldt–Jakob disease: a cost-effective modelling review

Background Creutzfeldt–Jakob disease is a fatal neurological disease caused by abnormal infectious proteins called prions. Prions that are present on surgical instruments cannot be completely deactivated; therefore, patients who are subsequently operated on using these instruments may become infected. This can result in surgically transmitted Creutzfeldt–Jakob disease. Objective To update literature reviews, consultation with experts and economic modelling published in 2006, and to provide the cost-effectiveness of strategies to reduce the risk of surgically transmitted Creutzfeldt–Jakob disease. Methods Eight systematic reviews were undertaken for clinical parameters. One review of cost-effectiveness was undertaken. Electronic databases including MEDLINE and EMBASE were searched from 2005 to 2017. Expert elicitation sessions were undertaken. An advisory committee, convened by the National Institute for Health and Care Excellence to produce guidance, provided an additional source of information. A mathematical model was updated focusing on brain and posterior eye surgery and neuroendoscopy. The model simulated both patients and instrument sets. Assuming that there were potentially 15 cases of surgically transmitted Creutzfeldt–Jakob disease between 2005 and 2018, approximate Bayesian computation was used to obtain samples from the posterior distribution of the model parameters to generate results. Heuristics were used to improve computational efficiency. The modelling conformed to the National Institute for Health and Care Excellence reference case. The strategies evaluated included neither keeping instruments moist nor prohibiting set migration; ensuring that instruments were kept moist; prohibiting instrument migration between sets; and employing single-use instruments. Threshold analyses were undertaken to establish prices at which single-use sets or completely effective decontamination solutions would be cost-effective. Results A total of 169 papers were identified for the clinical review. The evidence from published literature was not deemed sufficiently strong to take precedence over the distributions obtained from expert elicitation. Forty-eight papers were identified in the review of cost-effectiveness. The previous modelling structure was revised to add the possibility of misclassifying surgically transmitted Creutzfeldt–Jakob disease as another neurodegenerative disease, and assuming that all patients were susceptible to infection. Keeping instruments moist was estimated to reduce the risk of surgically transmitted Creutzfeldt–Jakob disease cases and associated costs. Based on probabilistic sensitivity analyses, keeping instruments moist was estimated to on average result in 2.36 (range 0–47) surgically transmitted Creutzfeldt–Jakob disease cases (across England) caused by infection occurring between 2019 and 2023. Prohibiting set migration or employing single-use instruments reduced the estimated risk of surgically transmitted Creutzfeldt–Jakob disease cases further, but at considerable cost. The estimated costs per quality-adjusted life-year gained of these strategies in addition to keeping instruments moist were in excess of £1M. It was estimated that single-use instrument sets (currently £350–500) or completely effective cleaning solutions would need to cost approximately £12 per patient to be cost-effective using a £30,000 per quality-adjusted life-year gained value. Limitations As no direct published evidence to implicate surgery as a cause of Creutzfeldt–Jakob disease has been found since 2005, the estimations of potential cases from elicitation are still speculative. A particular source of uncertainty was in the number of potential surgically transmitted Creutzfeldt–Jakob disease cases that may have occurred between 2005 and 2018. Conclusions Keeping instruments moist is estimated to reduce the risk of surgically transmitted Creutzfeldt–Jakob disease cases and associated costs. Further surgical management strategies can reduce the risks of surgically transmitted Creutzfeldt–Jakob disease but have considerable associated costs. Study registration This study is registered as PROSPERO CRD42017071807. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 11. See the NIHR Journals Library website for further project information.

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Health technology assessment and policy from the economic perspective

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462304000807 ◽

2004 ◽

Vol 20 (1) ◽

pp. 67-70 ◽

Cited By ~ 9

Author(s):

Frans Rutten

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Health Technology ◽

Quality Adjusted Life Year ◽

Decision Making Process ◽

Economic Perspective ◽

Cross Country ◽

Evidence For Policy ◽

Economic Considerations ◽

Quality Adjusted Life

This article comments on the four country papers in this volume from an economic perspective. Different phases of the decision-making process, which can be supported by Health Technology Assessment (HTA), are considered. For each of these, there is large cross-country variation in the way in which HTA influences policy. Furthermore, economic themes regarding the relevance of HTA evidence for policy making, the position of cost-effectiveness in relation to other criteria vis-à-vis reimbursement decisions, the use of a cost per quality-adjusted life year threshold, and the incorporation of economic considerations in practice guidelines are discussed.

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Open urethroplasty versus endoscopic urethrotomy for recurrent urethral stricture in men: the OPEN RCT

Health Technology Assessment ◽

10.3310/hta24610 ◽

2020 ◽

Vol 24 (61) ◽

pp. 1-110

Author(s):

Robert Pickard ◽

Beatriz Goulao ◽

Sonya Carnell ◽

Jing Shen ◽

Graeme MacLennan ◽

...

Keyword(s):

Cost Effectiveness ◽

Health Technology Assessment ◽

Urethral Stricture ◽

Technology Assessment ◽

Cost Effective ◽

Health Technology ◽

Urinary Symptoms ◽

Current Evidence ◽

Endoscopic Urethrotomy

Background Men who suffer recurrence of bulbar urethral stricture have to decide between endoscopic urethrotomy and open urethroplasty to manage their urinary symptoms. Evidence of relative clinical effectiveness and cost-effectiveness is lacking. Objectives To assess benefit, harms and cost-effectiveness of open urethroplasty compared with endoscopic urethrotomy as treatment for recurrent urethral stricture in men. Design Parallel-group, open-label, patient-randomised trial of allocated intervention with 6-monthly follow-ups over 24 months. Target sample size was 210 participants providing outcome data. Participants, clinicians and local research staff could not be blinded to allocation. Central trial staff were blinded when needed. Setting UK NHS with recruitment from 38 hospital sites. Participants A total of 222 men requiring operative treatment for recurrence of bulbar urethral stricture who had received at least one previous intervention for stricture. Interventions A centralised randomisation system using random blocks allocated participants 1 : 1 to open urethroplasty (experimental group) or endoscopic urethrotomy (control group). Main outcome measures The primary clinical outcome was control of urinary symptoms. Cost-effectiveness was assessed by cost per quality-adjusted life-year (QALY) gained over 24 months. The main secondary outcome was the need for reintervention for stricture recurrence. Results The mean difference in the area under the curve of repeated measurement of voiding symptoms scored from 0 (no symptoms) to 24 (severe symptoms) between the two groups was –0.36 [95% confidence interval (CI) –1.78 to 1.02; p = 0.6]. Mean voiding symptom scores improved between baseline and 24 months after randomisation from 13.4 [standard deviation (SD) 4.5] to 6 (SD 5.5) for urethroplasty group and from 13.2 (SD 4.7) to 6.4 (SD 5.3) for urethrotomy. Reintervention was less frequent and occurred earlier in the urethroplasty group (hazard ratio 0.52, 95% CI 0.31 to 0.89; p = 0.02). There were two postoperative complications requiring reinterventions in the group that received urethroplasty and five, including one death from pulmonary embolism, in the group that received urethrotomy. Over 24 months, urethroplasty cost on average more than urethrotomy (cost difference £2148, 95% CI £689 to £3606) and resulted in a similar number of QALYs (QALY difference –0.01, 95% CI –0.17 to 0.14). Therefore, based on current evidence, urethrotomy is considered to be cost-effective. Limitations We were able to include only 69 (63%) of the 109 men allocated to urethroplasty and 90 (80%) of the 113 men allocated to urethrotomy in the primary complete-case intention-to-treat analysis. Conclusions The similar magnitude of symptom improvement seen for the two procedures over 24 months of follow-up shows that both provide effective symptom control. The lower likelihood of further intervention favours urethroplasty, but this had a higher cost over the 24 months of follow-up and was unlikely to be considered cost-effective. Future work Formulate methods to incorporate short-term disutility data into cost-effectiveness analysis. Survey pathways of care for men with urethral stricture, including the use of enhanced recovery after urethroplasty. Establish a pragmatic follow-up schedule to allow national audit of outcomes following urethral surgery with linkage to NHS Hospital Episode Statistics. Trial registration Current Controlled Trials ISRCTN98009168. Funding This project was funded by the NIHR Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 61. See the NIHR Journals Library website for further project information.

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Developing a reference protocol for structured expert elicitation in health-care decision-making: a mixed-methods study

Health Technology Assessment ◽

10.3310/hta25370 ◽

2021 ◽

Vol 25 (37) ◽

pp. 1-124

Author(s):

Laura Bojke ◽

Marta Soares ◽

Karl Claxton ◽

Abigail Colson ◽

Aimée Fox ◽

...

Keyword(s):

Health Care ◽

Decision Making ◽

Mixed Methods ◽

Cost Effectiveness ◽

Health Technology Assessment ◽

Technology Assessment ◽

Health Technology ◽

Expert Elicitation ◽

Health Care Decision ◽

Care Decision

Background Many decisions in health care aim to maximise health, requiring judgements about interventions that may have higher health effects but potentially incur additional costs (cost-effectiveness framework). The evidence used to establish cost-effectiveness is typically uncertain and it is important that this uncertainty is characterised. In situations in which evidence is uncertain, the experience of experts is essential. The process by which the beliefs of experts can be formally collected in a quantitative manner is structured expert elicitation. There is heterogeneity in the existing methodology used in health-care decision-making. A number of guidelines are available for structured expert elicitation; however, it is not clear if any of these are appropriate for health-care decision-making. Objectives The overall aim was to establish a protocol for structured expert elicitation to inform health-care decision-making. The objectives are to (1) provide clarity on methods for collecting and using experts’ judgements, (2) consider when alternative methodology may be required in particular contexts, (3) establish preferred approaches for elicitation on a range of parameters, (4) determine which elicitation methods allow experts to express uncertainty and (5) determine the usefulness of the reference protocol developed. Methods A mixed-methods approach was used: systemic review, targeted searches, experimental work and narrative synthesis. A review of the existing guidelines for structured expert elicitation was conducted. This identified the approaches used in existing guidelines (the ‘choices’) and determined if dominant approaches exist. Targeted review searches were conducted for selection of experts, level of elicitation, fitting and aggregation, assessing accuracy of judgements and heuristics and biases. To sift through the available choices, a set of principles that underpin the use of structured expert elicitation in health-care decision-making was defined using evidence generated from the targeted searches, quantities to elicit experimental evidence and consideration of constraints in health-care decision-making. These principles, including fitness for purpose and reflecting individual expert uncertainty, were applied to the set of choices to establish a reference protocol. An applied evaluation of the developed reference protocol was also undertaken. Results For many elements of structured expert elicitation, there was a lack of consistency across the existing guidelines. In almost all choices, there was a lack of empirical evidence supporting recommendations, and in some circumstances the principles are unable to provide sufficient justification for discounting particular choices. It is possible to define reference methods for health technology assessment. These include a focus on gathering experts with substantive skills, eliciting observable quantities and individual elicitation of beliefs. Additional considerations are required for decision-makers outside health technology assessment, for example at a local level, or for early technologies. Access to experts may be limited and in some circumstances group discussion may be needed to generate a distribution. Limitations The major limitation of the work conducted here lies not in the methods employed in the current work but in the evidence available from the wider literature relating to how appropriate particular methodological choices are. Conclusions The reference protocol is flexible in many choices. This may be a useful characteristic, as it is possible to apply this reference protocol across different settings. Further applied studies, which use the choices specified in this reference protocol, are required. Funding This project was funded by the NIHR Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 25, No. 37. See the NIHR Journals Library website for further project information. This work was also funded by the Medical Research Council (reference MR/N028511/1).

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MRI-guided pulmonary vein isolation for atrial fibrillation: what is good enough? An early health technology assessment

Open Heart ◽

10.1136/openhrt-2019-001014 ◽

2019 ◽

Vol 6 (2) ◽

pp. e001014

Author(s):

Steven Wenker ◽

Chris van Lieshout ◽

Geert Frederix ◽

Jeroen van der Heijden ◽

Peter Loh ◽

...

Keyword(s):

Atrial Fibrillation ◽

Cost Effectiveness ◽

Health Technology Assessment ◽

Pulmonary Vein ◽

Technology Assessment ◽

Clinical Effectiveness ◽

Cost Effective ◽

Health Technology ◽

Mri Guided ◽

Effectiveness Threshold

Next to anticoagulation, pulmonary vein isolation (PVI) is the most important interventional procedure in the treatment of atrial fibrillation (AF). Despite widespread clinical application of this therapy, patients often require multiple procedures to reach clinical success. In contrast to conventional imaging modalities, MRI allows direct visualisation of the ablation lesion. Therefore, the use of real-time MRI to guide cardiac electrophysiology procedures may increase clinical effectiveness. An essential aspect, from a decision-making point of view, is the effect on costs and the potential cost-effectiveness of new technologies. Generally, health technology assessment (HTA) studies are performed when innovations are close to clinical application. However, early stage HTA can inform users, researchers and funders about the ultimate clinical and economic potential of a future innovation. Ultimately, this can guide funding allocation. In this study, we performed an early HTA evaluate MRI-guided PVIs.MethodsWe performed an economic evaluation using a decision tree with a time-horizon of 1 year. We calculated the clinical effectiveness (defined as the proportion of patients that is long-term free of AF after a single procedure) required for MRI-guided PVI to be cost-effective compared with conventional treatment.ResultsDepending on the cost-effectiveness threshold (willingness to pay for one additional quality-of-life adjusted life year (QALY), interventional MRI (iMRI) guidance for PVI can be cost-effective if clinical effectiveness is 69.8% (at €80 000/QALY) and 77.1% (at €20 000/QALY), compared with 64% for fluoroscopy-guided procedures.ConclusionUsing an early HTA, we established a clinical effectiveness threshold for interventional MRI-guided PVIs that can inform a clinical implementation strategy. If crucial technologies are developed, it seems plausible that iMRI-guided PVIs will be able to reach this threshold.

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The National Pharmaceutical Council: Endorsing the Construction of Imaginary Worlds in Health Technology Assessment

Pharmacy ◽

10.3390/pharmacy8030119 ◽

2020 ◽

Vol 8 (3) ◽

pp. 119

Author(s):

Paul C Langley

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Health Technology ◽

Quality Adjusted Life Year ◽

Normal Science ◽

Value Assessment ◽

Assessment Framework ◽

Professional Groups ◽

A Value ◽

Quality Adjusted Life

All too often, organizations embrace standards for health technology assessment that fail to meet those of normal science. A value assessment framework has been endorsed that is patently in the realm of pseudoscience. If a value assessment framework is to be accepted, then claims for the value of competing products must be credible, evaluable and replicable. If not, for example, when the assessment relies on the construction of an imaginary lifetime incremental cost-per-quality-adjusted-life-year (QALY) world, then that assessment should be rejected. Such an assessment would fail one of the central roles of normal science: the discovery of new facts through an ongoing process of conjecture and refutation where provisional claims can be continually challenged. It is no good defending an endorsement of a value framework that fails expected standards on the grounds that it has been endorsed by professional groups and reflects decades of development. This is intellectually lazy. If this is the case, then the scientific revolution of the 17th century need not have happened. The purpose of this commentary is to consider the recommended standards for health technology assessment of the National Pharmaceutical Council (NPC), with particular reference to proposed methodological standards in value assessment and the commitment to mathematically impossible QALYs.

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The Australia and New Zealand Horizon Scanning Network

Australian Health Review ◽

10.1071/ah050395 ◽

2005 ◽

Vol 29 (4) ◽

pp. 395 ◽

Cited By ~ 7

Author(s):

Linda Mundy ◽

Tracy L Merlin ◽

Adriana Parrella ◽

Wendy J Babidge ◽

Dianne E Roberts ◽

...

Keyword(s):

Cost Effectiveness ◽

Health Technology Assessment ◽

Technology Assessment ◽

Cost Effective ◽

Health Technology ◽

Public Health Care ◽

Primary Role ◽

Health Technologies ◽

Horizon Scanning ◽

Safety Effectiveness

UP UNTIL 1982, new health technologies, procedures or services were introduced into the health system in an uncontrolled, unregulated manner. This had the potential for wide-ranging impact on the public health care system including ballooning costs, a lack of preparedness by training and accreditation organisations, and consequent patient safety concerns. Health technology assessment was introduced into Australia in 1982 when the National Health Technology Assessment Panel was formed. This original panel has undergone numerous name changes and evolved into the Australian Government-funded Medical Services Advisory Committee (MSAC).1 The primary role of the MSAC is to inform the Federal Minister for Health and Ageing on the safety, effectiveness and costeffectiveness of new medical technologies and procedures using the available evidence.2 Assessments of the safety, effectiveness and cost-effectiveness of these technologies may occur only after the technology has diffused and is practised widely.3 Early identification of such technologies may avoid the detrimental consequences of their indiscriminate introduction and could result in the adoption of beneficial and cost-effective technologies and the elimination of technologies that are unsafe or for which there is no evidence of cost-effectiveness.4

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Cost–effectiveness analysis of ribociclib plus fulvestrant for hormone receptor-positive/human EGF receptor 2-negative breast cancer

Immunotherapy ◽

10.2217/imt-2020-0237 ◽

2021 ◽

Author(s):

Wei Jiang ◽

Zhichao He ◽

Tiantian Zhang ◽

Chongchong Guo ◽

Jianli Zhao ◽

...

Keyword(s):

Breast Cancer ◽

Cost Effectiveness ◽

Hormone Receptor ◽

Egf Receptor ◽

Cost Effective ◽

Quality Adjusted Life Year ◽

Cost Effectiveness Ratio ◽

Hormone Receptor Positive ◽

The Cost ◽

Quality Adjusted Life

Aim: To evaluate the cost–effectiveness of ribociclib plus fulvestrant versus fulvestrant in hormone receptor-positive/human EGF receptor 2-negative advanced breast cancer. Materials & methods: A three-state Markov model was developed to evaluate the costs and effectiveness over 10 years. Direct costs and utility values were obtained from previously published studies. We calculated incremental cost–effectiveness ratio to evaluate the cost–effectiveness at a willingness-to-pay threshold of $150,000 per additional quality-adjusted life year. Results: The incremental cost–effectiveness ratio was $1,073,526 per quality-adjusted life year of ribociclib plus fulvestrant versus fulvestrant. Conclusions: Ribociclib plus fulvestrant is not cost-effective versus fulvestrant in the treatment of advanced hormone receptor-positive/human EGF receptor 2-negative breast cancer. When ribociclib is at 10% of the full price, ribociclib plus fulvestrant could be cost-effective.

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A one-year cost–utility analysis of REBOA versus RTACC for non-compressible torso haemorrhage

Trauma ◽

10.1177/1460408617738810 ◽

2017 ◽

Vol 21 (1) ◽

pp. 45-54 ◽

Cited By ~ 1

Author(s):

Maxwell S Renna ◽

Cristiano van Zeller ◽

Farah Abu-Hijleh ◽

Cherlyn Tong ◽

Jasmine Gambini ◽

...

Keyword(s):

Cost Effectiveness ◽

Cost Effective ◽

Quality Adjusted Life Year ◽

Cost Utility ◽

Incremental Cost Effectiveness Ratio ◽

Cost Utility Analysis ◽

Utility Analysis ◽

Cost Effectiveness Ratio ◽

One Year ◽

Quality Adjusted Life

Introduction Major trauma is a leading cause of death and disability in young adults, especially from massive non-compressible torso haemorrhage. The standard technique to control distal haemorrhage and maximise central perfusion is resuscitative thoracotomy with aortic cross-clamping (RTACC). More recently, the minimally invasive technique of resuscitative endovascular balloon occlusion of the aorta (REBOA) has been developed to similarly limit distal haemorrhage without the morbidity of thoracotomy; cost–utility studies on this intervention, however, are still lacking. The aim of this study was to perform a one-year cost–utility analysis of REBOA as an intervention for patients with major traumatic non-compressible abdominal haemorrhage, compared to RTACC within the U.K.’s National Health Service. Methods A retrospective analysis of the outcomes following REBOA and RTACC was conducted based on the published literature of survival and complication rates after intervention. Utility was obtained from studies that used the EQ-5D index and from self-conducted surveys. Costs were calculated using 2016/2017 National Health Service tariff data and supplemented from further literature. A cost–utility analysis was then conducted. Results A total of 12 studies for REBOA and 20 studies for RTACC were included. The mean injury severity scores for RTACC and REBOA were 34 and 39, and mean probability of death was 9.7 and 54%, respectively. The incremental cost-effectiveness ratio of REBOA when compared to RTACC was £44,617.44 per quality-adjusted life year. The incremental cost-effectiveness ratio, by exceeding the National Institute for Health and Clinical Effectiveness’s willingness-to-pay threshold of £30,000/quality-adjusted life year, suggests that this intervention is not cost-effective in comparison to RTACC. However, REBOA yielded a 157% improvement in utility with a comparatively small cost increase of 31.5%. Conclusion Although REBOA has not been found to be cost-effective when compared to RTACC, ultimately, clinical experience and expertise should be the main factor in driving the decision over which intervention to prioritise in the emergency context.

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