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AIMS: The objective of this paper is to assess whether cardiac contractility modulation (via the Optimizer System) plus standard of care (SoC) is a cost-effective treatment for people with heart failure [New York Heart Association (NYHA) III, left ventricular ejection fraction of 25-45%, and narrow QRS] compared against SoC alone from the perspective of the English National Health Service. METHODS AND RESULTS: We developed a regression equation-based cost-effectiveness model, using individual patient data from three randomized control trials (FIX-HF-5 Phases 1 and 2, and FIX-HF-5C) to populate the majority of parameters. A series of regression equations predicted NYHA class over time, mortality, all-cause hospitalization rates, and health-related quality of life. We conducted the analysis in line with the National Institute for Health and Care Excellence reference case, modelling costs from an English National Health Service perspective, and considering outcomes in quality-adjusted life years (QALYs) over a patient lifetime perspective. Our base case analysis produced an incremental cost per additional QALY of GBP22 988 (€25 750) when comparing Optimizer + SoC to SoC alone. This result was not sensitive to parameter uncertainty but was sensitive to the time horizon over which costs and QALYs were captured and the duration over which a survival benefit with Optimizer + SoC can be assumed to apply. CONCLUSIONS: Cardiac contractility modulation is likely to be cost-effective in people with heart failure with reduced ejection fraction, NYHA III, and narrow QRS, provided that the treatment benefit can be maintained beyond the duration of the existing clinical trial follow-up. This analysis supports the current recommendations of the European Society of Cardiology that this therapy may be considered for such patients. peerReviewed

1. The COVID-19 pandemic has highlighted the importance of efficient sampling strategies and statistical methods for monitoring infection prevalence, both in humans and reservoir hosts. Pooled testing can be an efficient tool for learning pathogen prevalence in a population. Typically pooled testing requires a second phase follow up procedure to identify infected individuals, but when the goal is solely to learn prevalence in a population, such as a reservoir host, there are more efficient methods for allocating the second phase samples. 2. To estimate pathogen prevalence in a population, this manuscript presents an approach for data integration with two-phased testing of pooled samples that allows more efficient estimation of prevalence with less samples than traditional methods. The first phase uses pooled samples to estimate the population prevalence and inform efficient strategies for the second phase. To combine information from both phases, we introduce a Bayesian data integration procedure that combines pooled samples with individual samples for joint inferences about the population prevalence. 3. Data integration procedures result in more efficient estimation of prevalence than traditional procedures that only use individual samples or a single phase of pooled sampling. 4. The manuscript presents guidance on implementing the first phase and second phase sampling plans using data integration. Such methods can be used to assess the risk of pathogen spillover from reservoir hosts to humans, or to track pathogens such as SARS-CoV-2 in populations.

Objective Precision medicine allows healthcare interventions to be tailored to groups of patients based on their disease susceptibility, diagnostic or prognostic information, or treatment response. We analysed what developments are expected in precision medicine over the next decade and considered the implications for health technology assessment (HTA) agencies. Methods We performed a pragmatic literature search to account for the large size and wide scope of the precision medicine literature. We refined and enriched these results with a series of expert interviews up to 1 h in length, including representatives from HTA agencies, research councils and researchers designed to cover a wide spectrum of precision medicine applications and research. Results We identified 31 relevant papers and interviewed 13 experts. We found that three types of precision medicine are expected to emerge in clinical practice: complex algorithms, digital health applications and ‘omics’-based tests. These are expected to impact upon each stage of the HTA process, from scoping and modelling through to decision-making and review. The complex and uncertain treatment pathways associated with patient stratification and fast-paced technological innovation are central to these effects. Discussion Innovation in precision medicine promises substantial benefits but will change the way in which some health services are delivered and evaluated. The shelf life of guidance may decrease, structural uncertainty may increase and new equity considerations will emerge. As biomarker discovery accelerates and artificial intelligence-based technologies emerge, refinements to the methods and processes of evidence assessments will help to adapt and maintain the objective of investing in healthcare that is value for money. Electronic supplementary material The online version of this article (10.1007/s40273-018-0686-6) contains supplementary material, which is available to authorized users.

Mepilex Border Sacrum and Heel dressings are self-adherent, multilayer foam dressings designed for use on the heel and sacrum aiming to prevent pressure ulcers. The dressings are used in addition to standard care protocols for pressure ulcer prevention. The National Institute for Health and Care Excellence (NICE) selected Mepilex Border Sacrum and Heel dressings for evaluation. The External Assessment Centre (EAC) critiqued the company's submission. Thirteen studies (four randomised controlled trials and nine nonrandomised comparative studies) were included. The majority of studies compared Mepilex Border Sacrum dressings (plus standard care) with standard care alone. Comparative evidence for Mepilex Border Heel dressings was limited. A meta-analysis indicated a non-statistically significant difference in favour of Mepilex Border Sacrum dressings for pressure ulcer incidence [RR 0.51 (95% CI 0.22-1.18)]. The company produced a de novo cost model, which was critiqued by the EAC. After the EAC updated input parameters, cost savings of £19 per patient compared with standard care alone for pressure ulcer prevention were estimated with Mepilex Border dressings predicted to be cost saving in 57% of iterations. The Medical Technologies Advisory Committee reviewed the evidence and judged that, although Mepilex Border Heel and Sacrum dressings have potential to prevent pressure ulcers in people who are considered to be at risk in acute care settings, further evidence is required to address uncertainties around the claimed benefits of the dressings and the incidence of pressure ulcers in an NHS acute-care setting. After a public consultation, NICE published this as Medical Technology Guidance 40.

Objectives This study aimed to review current use of experts within National Institute for Health and Care Excellence (NICE) guidance-making programmes, identify improvements in use of expert judgement, and to assess tools and protocols to support the elicitation of information from experts for use by NICE. Methods The study comprised a review of NICE process guides; semi-structured interviews with individuals representing each NICE guidance-making programme and a comparison of the suitability of currently available tools and protocols for expert elicitation to the requirements of NICE programmes. Results Information elicited from experts and the way in which it is used varies across NICE guidance-making programmes. Experts’ involvement can be as intensive as being a member of a committee and thus having direct influence on recommendations or limited one-off consultations on specific parameters. We identified 16 tools for expert elicitation that were potentially relevant. None fully met the requirements of NICE, although an existing tool could be potentially adapted. Ongoing research to develop a reference protocol for expert elicitation in healthcare decision making may be of use in future. Conclusions NICE uses expert judgement across all its guidance-making programmes, but its uses vary considerably. There is no currently available tool for expert elicitation suitable for use by NICE. However, adaptation of an existing tool or ongoing research in the area could address this in the future.

Memokath-051 is a thermo-expandable, nickel-titanium alloy spiral stent used to treat ureteric obstruction resulting from malignant or benign strictures. The National Institute for Health and Care Excellence (NICE) selected Memokath-051 for evaluation. The company, PNN Medical, claimed Memokath-051 has clinical superiority and cost savings compared with double-J stents. It identified five studies reporting clinical evidence on Memokath-051 and constructed a de novo cost model comparing Memokath-051 to double-J stents. Results indicated that Memokath-051 generated cost savings of £4156 per patient over 2.5 years. The External Assessment Centre (EAC) critiqued the company’s submission and completed substantial additional work. Sixteen studies were identified assessing Memokath-051 and all listed comparators in the scope (double-J stents, reconstructive surgery and metallic and alloy stents) except nephrostomy. Similar success rates were reported for Memokath-051 compared with double-J and Resonance stents and worse outcomes compared with other options with evidence available. The EAC updated the company’s cost model structure and modified several inputs. The EAC’s model estimated that Memokath-051 generated savings of at least £1619 per patient over 5 years compared with double-J stents, was cost neutral compared with other metallic stents and was cost saving compared with surgery up to month 55. Overall, Memokath-051 is likely to be cost saving in patients not indicated for reconstructive surgery and those expected to require a ureteral stent for at least 30 months. The Medical Technologies Advisory Committee (MTAC) reviewed the evidence and supported the case for adoption, issuing partially supportive recommendations published after public consultation as Medical Technologies Guidance 35. Electronic supplementary material The online version of this article (10.1007/s40258-018-0389-3) contains supplementary material, which is available to authorized users.

Precision medicine allows healthcare interventions to be tailored to groups of patients based on their disease susceptibility, diagnostic or prognostic information, or treatment response. We analysed what developments are expected in precision medicine over the next decade and considered the implications for health technology assessment (HTA) agencies.We performed a pragmatic literature search to account for the large size and wide scope of the precision medicine literature. We refined and enriched these results with a series of expert interviews up to 1 h in length, including representatives from HTA agencies, research councils and researchers designed to cover a wide spectrum of precision medicine applications and research.We identified 31 relevant papers and interviewed 13 experts. We found that three types of precision medicine are expected to emerge in clinical practice: complex algorithms, digital health applications and 'omics'-based tests. These are expected to impact upon each stage of the HTA process, from scoping and modelling through to decision-making and review. The complex and uncertain treatment pathways associated with patient stratification and fast-paced technological innovation are central to these effects.Innovation in precision medicine promises substantial benefits but will change the way in which some health services are delivered and evaluated. The shelf life of guidance may decrease, structural uncertainty may increase and new equity considerations will emerge. As biomarker discovery accelerates and artificial intelligence-based technologies emerge, refinements to the methods and processes of evidence assessments will help to adapt and maintain the objective of investing in healthcare that is value for money.

Interstitial ectopic pregnancy is a medical emergency affecting women of reproductive age. Its presentation is late and is often misinterpreted as early miscarriage. If undiagnosed and allowed to progress to a significant gestation, rupture will occur with devastating maternal consequences. There are several treatment options available depending on a number of clinical factors; surgical, medical and expectant management. The most significant outcome is from early treatment, resulting in reduced maternal morbidity and preservation of fertility. The key to successful treatment is early detection. Ultrasound plays a major role in detection of this life threatening condition.