The best of the UK? A report on the value and future of UK databases in the health and social care fields: a systematic map protocol

INTRODUCTION: This protocol covers the first part of a two-part project funded by the Health Libraries Group and the University Health and Medical Librarians Group. It details the proposed methodology for a systematic map of the literature relating to UK bibliographic databases in the fields of health and social care. The aim of this mapping exercise is to consider ways in which UK bibliographic databases are described, considered and discussed in the published and unpublished literature. In doing so, we hope to gain a clearer sense of the ways in which UK bibliographic databases are used and viewed by the research community. It also enables the identification of any gaps in the literature for further research and discussion. This topic is important because UK databases are generally underused by researchers in the UK context and some databases are at risk of closure. A lack of access to UK databases means that researchers may miss relevant UK evidence when identifying an evidence base. / METHOD: Systematic Map. / ANALYSIS: The authors will present a narrative description of the literature relating to UK bibliographic databases in the fields of health and social care. They will use tables to present descriptive information about the literature (eg, frequency tables) and use cross-tabulations to demonstrate intersecting themes. Separately, guidance on how to use the resources (eg, areas of unique content, updating frequencies, unique truncation symbols) will be sought from stakeholders and reported alongside the report narrative as a guide to usage

The clinical effectiveness and cost-effectiveness of cetuximab (mono- or combination chemotherapy), bevacizumab (combination with non-oxaliplatin chemotherapy) and panitumumab (monotherapy) for the treatment of metastatic colorectal cancer after first-line chemotherapy (review of technology appraisal No.150 and part review of technology appraisal No. 118): a systematic review and economic model

Colorectal cancer is the third most commonly diagnosed cancer in the UK after breast and lung cancer. People with metastatic disease who are sufficiently fit are usually treated with active chemotherapy as first- or second-line therapy. Recently, targeted agents have become available including anti-epidermal growth factor receptor (EGFR) agents, for example cetuximab and panitumumab, and anti-vascular endothelial growth factor (VEGF) receptor agents, for example bevacizumab

Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: An Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal

The UK National Institute for Health and Care Excellence (NICE) considered evidence for voretigene neparvovec (VN; Luxturna®) for the treatment of RPE65-mediated inherited retinal dystrophies (IRD) within its highly specialised technology programme. This paper summarises the evidence provided by the company; the appraisal of the evidence by the Peninsula Technology Appraisal Group, who were commissioned to act as the independent evidence review group (ERG); and the development of the NICE guidance by the appraisal committee. The evidence presented by the company highlighted the significant lifelong burden of IRD for patients and carers. Evidence to support the effectiveness of VN was lacking, but the available evidence showed a modest, sustained improvement across a variety of vision-related outcomes. While patients would remain visually impaired, the committee considered that VN would prevent further deterioration in vision. The modelling approach used by the company had a number of limitations and relied heavily upon a large volume of clinical expert input to produce cost-effectiveness estimates with large uncertainty around long-term effectiveness. The ERG’s main concerns revolved around these long-term outcomes and the plausibility of utility values. The NICE committee were convinced that the clinical benefits of VN were important and an appropriate use of national health service resources within a specialised service. The committee concluded that a high unmet need existed in patients with RPE65-mediated IRD and that VN represents a step change in the management of this condition

The effectiveness and cost-effectiveness of erythropoiesis-stimulating agents (epoetin and darbepoetin) for treating cancer-treatment induced anaemia (including review of TA142): a systematic review and economic model

Background: Anaemia is a common side-effect of cancer treatments and can lead to a reduction in quality of life. Erythropoiesis-stimulating agents (ESAs) are licensed for use in conjunction with red blood cell transfusions (RBCTs) to improve cancer treatment-induced anaemia (CIA). Methods: The clinical effectiveness review followed principles published by NHS CRD. Randomised controlled trials (RCTs), or systematic reviews of RCTs, of ESAs (epoetin or darbepoetin) for treating people with CIA were eligible for inclusion in the review. Comparators were best supportive care (BSC), placebo, or other ESA. Anaemia- and malignancy-related outcomes, health-related quality of life (HRQoL), and adverse events (AEs) were evaluated. Where appropriate, data were pooled using meta-analysis. An empirical health economic model was developed comparing ESA treatment to no ESA treatment. The model has two components: one evaluating short-term costs and QALYs (while patients are anaemic); and one evaluating long-term QALYs. Costs and benefits were discounted at 3.5% pa. Probabilistic and univariate deterministic sensitivity analyses were performed. Results: Twenty-three studies assessing ESAs within their licensed indication (based on start dose administered) were included. None of the RCTs were completely aligned with current EU licenses. Results suggest that there is clinical benefit from ESAs for anaemia-related outcomes. Data suggest improvement in HRQoL scores. The impact of ESAs on AEs and survival remains highly uncertain; although point estimates are lower confidence intervals are wide and not statistically significant. Base case incremental cost-effectiveness ratios (ICERs) for ESA treatment versus no ESA treatment ranged from £19,429–£35,018 per quality-adjusted life year (QALY) gained, but sensitivity and scenario analyses demonstrate considerable uncertainty in these ICERs, including the possibility of overall health disbenefit. All ICERs were sensitive to survival and cost. Conclusions: ESAs could be cost-effective when used closer to licence but there is considerable uncertainty mainly due to unknown impacts on overall survival

Adjusting for unmeasured confounding in nonrandomized longitudinal studies: a methodological review

OBJECTIVE: Motivated by recent calls to use electronic health records for research, we reviewed the application and development of methods for addressing the bias from unmeasured confounding in longitudinal data. DESIGN: Methodological review of existing literature SETTING: We searched MEDLINE and EMBASE for articles addressing the threat to causal inference from unmeasured confounding in nonrandomised longitudinal health data through quasi-experimental analysis. RESULTS: Among the 121 studies included for review, 84 used instrumental variable analysis (IVA), of which 36 used lagged or historical instruments. Difference-in-differences (DiD) and fixed effects (FE) models were found in 29 studies. Five of these combined IVA with DiD or FE to try to mitigate for time-dependent confounding. Other less frequently used methods included prior event rate ratio adjustment, regression discontinuity nested within pre-post studies, propensity score calibration, perturbation analysis and negative control outcomes. CONCLUSIONS: Well-established econometric methods such as DiD and IVA are commonly used to address unmeasured confounding in non-randomised, longitudinal studies, but researchers often fail to take full advantage of available longitudinal information. A range of promising new methods have been developed, but further studies are needed to understand their relative performance in different contexts before they can be recommended for widespread use

Cost-effectiveness of cetuximab, cetuximab plus irinotecan, and panitumumab for third and further lines of treatment for KRAS wild-type patients with metastatic colorectal cancer

AbstractObjectivesTo estimate the cost-effectiveness of cetuximab monotherapy, cetuximab plus irinotecan, and panitumumab monotherapy compared with best supportive care (BSC) for the third and subsequent lines of treatment of patients with Kirsten rat sarcoma wild-type metastatic colorectal cancer from the perspective of the UK National Health Service.MethodsAn “an area under the curve” cost-effectiveness model was developed. The clinical effectiveness evidence for both cetuximab and panitumumab was taken from a single randomized controlled trial (RCT) in each case and for cetuximab plus irinotecan from several sources.ResultsPatients are predicted to survive for approximately 6 months on BSC, 8.5 months on panitumumab, 10 months on cetuximab, and 16.5 months on cetuximab plus irinotecan.Panitumumab is dominated, and cetuximab is extended dominated. An incremental cost-effectiveness ratio (ICER) of £95,000 per quality-adjusted life-year (QALY) was estimated for cetuximab versus BSC and is likely to be relatively accurate, because the relevant clinical evidence is taken from a high-quality RCT. The estimated ICER for panitumumab versus BSC, at £187,000 per QALY, is less certain due to assumptions in the adjustment for the substantial crossing-over of patients in the RCT. The ICER for cetuximab plus irinotecan versus BSC, at £88,000 per QALY, is least certain due to substantial uncertainty about progression-free survival, treatment duration, and overall survival. Nonetheless, when key parameters are varied within plausible ranges, all three treatments always remain poor value for money.ConclusionsAll three treatments are highly unlikely to be considered cost-effective in this patient population in the United Kingdom. We explain how the reader can adapt the model for other countries

Hoitotyön kirjaamisen kehittämisen käynnistäminen tehostetun palveluasumisen yksikössä

Opinnäytetyön tarkoituksena oli käynnistää hoitohenkilöstön kirjaamisen kehittäminen ja luoda helppolukuinen kirjaamisohje rakenteisen kirjaamisen tueksi tehostetun palveluasumisen yksikköön. Tarkoituksena oli luoda apuväline kirjaamisen tueksi ja tukea tällä kirjaamisen kehittämistä niin, että hoitotyön kirjaaminen olisi yhtenäistä ja kirjaamisen perusteella asukkaan voinnista saisi mahdollisimman selkeän kuvan. Tavoitteena oli kirjaamisen kehittämisen käynnistäminen asukas- ja voimavaralähtöiseksi niin, että hoitohenkilökunta kiinnittää huomiota nykyiseen kirjaamiseen ja kehittää sitä jatkossa asiakaslähtöisemmäksi. Yksikössä tullaan siirtymään hiljaiseen raportointiin ja kirjaukset tulevat siirtymään myös Kanta-arkistoon tulevaisuudessa, joten vaatimukset kirjaamisessa kasvavat. Kirjaamisen kehittämisen käynnistämiseksi laadittiin helppolukuinen kirjaamisohje ja järjestettiin infotilaisuus palvelutalon hoitohenkilökunnalle. Infotilaisuuden tarkoituksena oli nostaa esiin asiakaslähtöisen kirjaamisen lähtökohdat ja tarkastella case- esimerkkien valossa, millaista on asiakaslähtöinen kirjaaminen. Luovutimme tässä tilanteessa kirjaamisohjeen palvelutalolle. Kirjaamisohjeen rakenne noudatteli palvelutalossa käytettävän raportointijärjestelmän hoidon komponentteja. Opinnäytetyö palveli kirjaamisen kehittämisen käynnistäjänä palvelutalossa, jossa tarkoituksena on jatkaa asiakaslähtöisen kirjaamisen kehittämistä niin, että kirjaamiskäytäntö jalkautuu osaksi hoitotyötä.The purpose of this thesis was to launch a development program for nursing documentation and to create an easy to use documentation instruction to support structured nursing documentation in the assisted living unit with 24-hour assistance. The purpose was to create an aid to support structured nursing documentation and on this to support the development of nursing documentation in a way, that the documentation would be unified, and one could get a clear picture of the residents’ status based on the documentation. The goal was to launch the development of documentation to be resident and resource based in a way that the nursing staff will focus on the documentation as it is and will continue to develop it towards resident-oriented documentation. In the future, reporting in the unit will be altered from oral reporting to silent reporting and the nursing documents will also be transferred to Kanta-archives, thus the demands on the nursing documentation will grow. In order to begin the development of the nursing documentation, an easy to read guide was created, and an information session at the unit was arranged. The aim of the session was to bring up baselines of resident oriented documentation and to view it through case examples. The guide was handed over at the same time. The documentation guide follows the components of the electric documentation program in use at the assisted living unit. The launching of the development program for nursing documentation in the assisted living unit with 24-hour assistance was brought about by this thesis. The aim is to continue to develop the resident based nursing documentation and to include it as a part of nursing

Cost-effectiveness of diagnostic and therapeutic interventions for chronic hepatitis C: a systematic review of model-based analyses

Abstract Background Decisions about which subgroup of chronic hepatitis C (CHC) patients should be treated with direct acting anti-viral agents (DAAs) have economic importance due to high drug prices. Treat-all DAA strategies for CHC have gained acceptance despite high drug acquisition costs. However, there are also costs associated with the surveillance of CHC to determine a subgroup of patients with significant impairment. The aim of this systematic review was to describe the modelling methods used and summarise results in cost-effectiveness analyses (CEAs) of both CHC treatment with DAAs and surveillance of liver disease. Methods Electronic databases including Embase and Medline were searched from inception to May 2015. Eligible studies included models predicting costs and/or outcomes for interventions, surveillance, or management of people with CHC. Narrative and quantitative synthesis were conducted. Quality appraisal was conducted using validated checklists. The review was conducted following principles published by NHS Centre for Research and Dissemination. Results Forty-one CEAs met the eligibility criteria for the review; 37 evaluated an intervention and four evaluated surveillance strategies for targeting DAA treatment to those likely to gain most benefit. Included studies were of variable quality mostly due to reporting omissions. Of the 37 CEAs, eight models that enabled comparative analysis were fully appraised and synthesized. These models provided non-unique cost-effectiveness estimates in a specific DAA comparison in a specific population defined in terms of genotype, prior treatment status, and presence or absence of cirrhosis. Marked heterogeneity in cost-effectiveness estimates was observed despite this stratification. Approximately half of the estimates suggested that DAAs were cost-effective considering a threshold of US$30,000 and 73$50,000. Two models evaluating surveillance strategies suggested that treating all CHC patients regardless of the staging of liver disease could be cost-effective. Conclusions CEAs of CHC treatments need to better account for variability in their estimates. This analysis suggested that there are still circumstances where DAAs are not cost-effective. Surveillance in place of a treat-all strategy may still need to be considered as an option for deploying DAAs, particularly where acquisition cost is at the limit of affordability for a given health system

Role of autoantibody levels as biomarkers in the management of patients with myasthenia gravis : A systematic review and expert appraisal

Background and purpose Although myasthenia gravis (MG) is recognized as an immunoglobulin G autoantibody-mediated disease, the relationship between autoantibody levels and disease activity in MG is unclear. We sought to evaluate this landscape through systematically assessing the evidence, testing the impact of predefined variables on any relationship, and augmenting with expert opinion. Methods In October 2020, a forum of leading clinicians and researchers in neurology from across Europe (Expert Forum for Rare Autoantibodies in Neurology in Myasthenia Gravis) participated in a series of virtual meetings that took place alongside the conduct of a systematic literature review (SLR). Results Forty-two studies were identified meeting inclusion criteria. Of these, 10 reported some correlation between a patient's autoantibody level and disease severity. Generally, decreased autoantibody levels (acetylcholine receptor, muscle-specific kinase, and titin) were positively and significantly correlated with improvements in disease severity (Quantitative Myasthenia Gravis score, Myasthenia Gravis Composite score, Myasthenia Gravis Activities of Daily Living score, Myasthenia Gravis Foundation of America classification). Given the limited evidence, testing the impact of predefined variables was not feasible. Conclusions This first SLR to assess whether a correlation exists between autoantibody levels and disease activity in patients with MG has indicated a potential positive correlation, which could have clinical implications in guiding treatment decisions. However, in light of the limited and variable evidence, we cannot currently recommend routine clinical use of autoantibody level testing in this context. For now, patient's characteristics, clinical disease course, and laboratory data (e.g., autoantibody status, thymus histology) should inform management, alongside patient-reported outcomes. We highlight the need for future studies to reach more definitive conclusions on this relationship