Abstract

Health economics allows decision-makers to identify the most efficient allocation of limited resources. It involves measuring the benefits of different health care interventions in such a way that they can be compared across the whole health system. Quality-adjusted life years (QALYS) allow us to measure the benefires that acrue from both increased length of life (i.e. life expectancy) and improved quality of life - or even a combination of both. Once we have estimated the QALYS gains from a particular intervention, these can be compared against the QALYS that must be lost elsewhere in the health system in order to release funds to pay for a new intervention. If the QALY gains from a new intervention outweigh the opportunity costs of moving that money form where it is currently spent, then the health care system as a whole will benefit from the new intervention.

Abstract

OBJECTIVES: To determine the effectiveness and cost implications of using antiseptic passive disinfecting caps (disinfecting alcohol-containing cap that twists onto needle free connector intravenous access devices) as an adjunct to standard care in patients with central or peripheral lines in a NHS hospital setting compared with standard care alone.

METHODS: A systematic review identified studies reporting on the use of antiseptic barrier caps for the reduction of central and peripheral line associated infections. Studies reporting on central line associated bloodstream infections (CLABSI) in general hospital patients and patients in an intensive care setting were pooled using meta-analysis to calculate incidence rate ratios (IRR). A decision-analytic model estimated the costs and consequences associated with the use of an antiseptic barrier cap in these groups of patients from a UK NHS perspective, using the IRRs. Other parameter inputs were obtained from published literature using UK specific sources where possible. Deterministic sensitivity analyses (DSA) and probabilistic sensitivity analyses (PSA) were conducted to assess the robustness of results.

RESULTS: The meta-analyses produced IRRs in favour of the passive disinfecting cap of 0.43 (95% CI 0.22, 0.82) in general hospital patients and 0.29 (95% CI 0.09, 0.97) in intensive care patients. The adjunctive use of the passive disinfecting cap dominated standard care alone in both settings in the economic model, as it was both cost saving and reduced the number of CLABSIs. DSA indicated these results were robust to changes in individual input parameters, and PSA estimated the results to be cost saving in 96.4% of iterations in the general hospital setting and 86.3% in the intensive care setting.

CONCLUSIONS: Based on currently available evidence, the analyses suggest that using passive disinfecting caps to protect needle free connector intravenous access devices is a cost-effective use of resources for patients with central or peripheral line catheters in the UK NHS.

Abstract

OBJECTIVES: To estimate the relative effectiveness of enzalutamide in chemotherapy-naive metastatic castration-resistant prostate cancer by conducting a systematic literature review and a network meta-analysis (NMA).

METHODS: A systematic literature review identified randomized controlled trials comparing enzalutamide, abiraterone/prednisone, radium-223, sipuleucel-T, or docetaxel with each other or placebo in chemotherapy-naive or mixed populations (with and without prior chemotherapy) with asymptomatic/mildly symptomatic metastatic castration-resistant prostate cancer. Feasibility assessment evaluated the trials' suitability for NMA inclusion. The main outcomes were hazard ratios (HRs) for overall survival (OS) and radiographic progression-free survival (rPFS).

RESULTS: Searches of relevant bibliographic databases, trial registers, Web sites, and conference abstracts conducted in October 2014 identified 25,712 records. Ten randomized controlled trials were eligible for the NMA. Enzalutamide was superior to placebo for OS and rPFS (fixed-effects model). NMA results (fixed-effects model) showed no evidence of a difference between enzalutamide and abiraterone/prednisone (HR 0.95 [95% CrI 0.77–1.16]), sipuleucel-T (HR 1.07 [95% CrI 0.84–1.37]), or radium-223 (HR 1.10 [95% CrI 0.87–1.37]) for OS. HRs were similar for the random-effects model. Nevertheless, results (fixed-effects model) suggested that enzalutamide was superior to abiraterone/prednisone (HR 0.59 [95% CrI 0.48–0.72]) and sipuleucel-T (HR 0.32 [95% CrI 0.25–0.42]) for rPFS. Results also suggested superiority of enzalutamide versus placebo, abiraterone/prednisone, or sipuleucel-T for time to chemotherapy.

CONCLUSIONS: For rPFS, the NMA suggests that enzalutamide is superior to abiraterone/prednisone and sipuleucel-T. There is no evidence of a statistically significant difference in OS between enzalutamide and abiraterone/prednisone, sipuleucel-T, or radium-223. Given the limitations in network construction and underlying assumptions made to complete these analyses, results should be interpreted with caution.

Abstract

PURPOSE: This paper aims to develop a behavioural science informed communication strategy aimed at health professionals and patients promoting best practice recommendations regarding the use of specialist liquid medicines for elderly people with swallowing difficulties.

DESIGN / METHODOLOGY / APPROACH: The medicine prescribing, formulation and administration related challenges and experiences of health professionals and elderly patients with swallowing difficulties were identified through a pragmatic literature search. Key findings across the papers were synthesised into themes, before being linked to domains from a behavioural science framework. Published recommendations for behaviour change techniques that can be used to target the domains were then mapped to the domains. Guidance on how to develop a communication strategy, drawing on the insight gained from the literature review and the behavioural science recommendations, and designed to stimulate change in health-care professionals' and patients' behaviours, was then developed.

FINDINGS: In total, 13 themes emerged across 15 papers, including "patient and health professional roles and remits". These themes were linked to nine domains from the framework, highlighting the range of individual, social and environmental factors influencing patients' and health professionals' perceptions and experiences. A summary table, mapping the domains and underpinning themes to recommended behaviour change techniques, was used to develop the subsequent communication strategy recommendations. Recommendations include using techniques such as providing social processes of encourage, pressure and support to change patients' and health professionals' perceptions of their roles/responsibilities in medicines prescribing and administration, delivered via, for example, an educational leaflet and/or online training.

PRACTICAL IMPLICATIONS: The summary table and guidance can inform development of an evidence-based strategy for communicating best practice recommendations regarding the use of liquid medicines for elderly patients with swallowing difficulties, tailored to the perceptions and challenges identified.

ORIGINALITY / VALUE: The behavioural science approach is less established within the pharmaceutical industry for promotion of best practice recommendations and related products, yet it offers a framework for an evidence-based and systematic approach that goes beyond a literature review or focus group.

Abstract

BACKGROUND AND OBJECTIVES: Rheumatoid arthritis (RA) is a chronic, debilitating disease affecting an estimated 1.5 million patients in the US. The condition is associated with a substantial health and economic burden. An economic model was developed to evaluate the cost-effectiveness of tofacitinib (a novel oral Janus kinase inhibitor) versus biologic therapies commonly prescribed in the US for the treatment of RA.

METHODS: A cost-utility model was developed whereby sequences of treatments were evaluated. Response to treatment was modeled by HAQ change, and informed by a network meta-analysis. Mortality, resource use and quality of life were captured in the model using published regression analyses based on HAQ score. Treatment discontinuation was linked to response to treatment and to adverse events. Patients were modeled as having had an inadequate response to methotrexate (MTX-IR), or to a first biologic therapy (TNFi-IR).

RESULTS: The tofacitinib strategy was associated with cost savings compared with alternative treatment sequences across all modeled scenarios (i.e. in both the MTX-IR and TNFi-IR scenarios), with lifetime cost savings per patient ranging from $65,205 to $93,959 (2015 costs). Cost savings arose due to improved functioning and the resulting savings in healthcare expenditure, and lower drug and administration costs. The tofacitinib strategies all resulted in an increase in quality-adjusted life years (QALYs), with additional QALYs per patient ranging from 0.01 to 0.22.

CONCLUSIONS: Tofacitinib as a second-line therapy following methotrexate failure and as a third-line therapy following a biologic failure produces lower costs and improved quality of life compared with the current pathway of care.

Abstract

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.

Abstract

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.