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Applying Trial-Derived Treatment Effects to Real-World Populations: Generalizing Cost-Effectiveness Estimates When Modeling Complex Hazards.
OBJECTIVES: Generalizability of trial-based cost-effectiveness estimates to real-world target populations is important for decision making. In the context of independent aggregate time-to-event baseline and relative effects data, complex hazards can make modeling of data for use in economic evaluation challenging. Our article provides an overview of methods that can be used to apply trial-derived relative treatment effects to external real-world baselines when faced with complex hazards and follows with a motivating example. METHODS: Approaches for applying trial-derived relative effects to real-world baselines are presented in the context of complex hazards. Appropriate methods are applied in a cost-effectiveness analysis using data from a previously published study assessing the real-world cost-effectiveness of a treatment for carcinoma of the head and neck as a motivating example. RESULTS: Lack of common hazards between the trial and target real-world population, a complex baseline hazard function, and nonproportional relative effects made the use of flexible models necessary to adequately estimate survival. Assuming common distributions between trial and real-world reference survival substantially affected survival and cost-effectiveness estimates. Modeling time-dependent vs proportional relative effects affected estimates to a lesser extent, dependent on assumptions used in cost-effectiveness modeling. CONCLUSIONS: Appropriately capturing reference treatment survival when attempting to generalize trial-derived relative treatment effects to real-world target populations can have important impacts on cost-effectiveness estimates. A balance between model complexity and adequacy for decision making should be considered where multiple data sources with complex hazards are being evaluated.
Physical activity and health-related quality of life of patients with chronic knee pain after total knee replacement: Analysis of the PEP-TALK trial.
BACKGROUND: Chronic pain is a major challenge for some people after total knee replacement (TKR). The changing impact of this complication during the first post-operative year remains unclear. This analysis aimed to examine how physical activity and health-related quality of life (HRQoL) evolved over the first year after TKR for patients with and without post-operative chronic knee pain. METHODS: We conducted a secondary analysis of data from a randomised controlled trial (PEP-TALK), which tested the effectiveness of a behaviour change physiotherapy intervention compared with usual rehabilitation after TKR. Mean UCLA Activity Score and EQ-5D-5L for participants with and without chronic knee pain (14 points or lower in the Oxford Knee Score Pain Subscale (OKS-PS) at six months post-TKR) were compared at six and 12 months post-TKR. RESULTS: Data from 83 participants were analysed. For those with chronic knee pain, UCLA Activity Score remained unchanged between baseline to six months (mean: 3.8 to 3.8), decreasing at 12 months (3.0). Those without post-operative chronic knee pain reported improved physical activity from baseline to six months (4.0 vs 4.9), plateauing at 12 months (4.9). Participants with chronic knee pain reported lower baseline HRQoL (0.28 vs 0.48). Both groups improved health utility over one year. Of those without chronic pain at six months, 8.5% returned to chronic pain by 12 months. CONCLUSIONS: Monitoring clinical outcomes after six months may be indicated for those at risk of chronic pain post-TKR. Further, sufficiently powered analyses are warranted to increase the generalisability of this exploratory analyses' results.
Patient-reported outcomes measures of X-linked hypophosphataemia participants: findings from a prospective cohort study in the UK.
BACKGROUND: X-linked hypophosphataemia (XLH) is a rare genetic condition passed on through the X chromosome which causes multiple symptoms including weakened teeth, bones, and muscles. Due to the rarity of the condition, little is known about the health outcomes as reported by people with the disease. The objectives of this study were threefold: to characterise key patient reported outcome measures (PROMs) in adults with XLH, to identify clusters of symptom-severity groups based on PROMs, and to analyse the longitudinal progression of available PROMs. METHODS: Data from 48 participants from the Rare and Undiagnosed Diseases cohort Study (RUDY) was used to analyse both cross-sectional and longitudinal patient-reported outcomes. We analysed data for health-related quality of life (HRQL): EuroQol 5 dimensions-5 levels (EQ-5D-5L), Short-form 36 (SF-36) Physical Component Score (PCS), and SF-36 Mental Component Score (MCS), sleep: Pittsburgh sleep quality index (PSQI) and Epworth Sleepiness scale (ESS), fatigue: Fatigue Severity Scale (FSS) and Functional assessment of chronic illness therapy-fatigue (FACIT-F), pain: Short form McGill pain questionnaire version 2 (SF-MPQ-2) and PainDETECT, and mental well-being: Hospital anxiety and depression scale (HADS) anxiety and depression. Summary statistics, tests of mean differences, mixed-effects models, and cluster analysis were used to describe and examine the various health dimensions of individuals with XLH. RESULTS: Overall mean scores were EQ-5D-5L = 0.65, SF-36-PCS = 32.7, and SF-36-MCS = 48.4 for HRQL, ESS = 5.9 and PSQI = 8.9 for sleep, FSS = 32.8 and FACIT-F = 104.4 for fatigue, SF-MPQ-2 = 1.9 for pain, and HADS-depression = 4.7 and HADS-anxiety = 6.2 for mental well-being. 7% reported neuropathic pain (PainDETECT). Whilst many adults with XLH reported good outcomes, extreme or severe problems were reported across all outcomes. Cluster analysis identified that adults with XLH could be divided into two distinct groups, one reporting worse (35.3%) and the other better outcomes (64.7%) (less pain, fatigue, depression, and higher levels of sleep). Longitudinal analysis showed that FACIT-F and HADS-anxiety scores worsened slightly over two years with statistically significant (p
Worse off after knee replacements: How likely is it and what role does the pre-operative oxford knee score play? evidence from the English NHS
Knee replacements are extremely effective surgical interventions associated with significant improvements in pain, function and quality of life. Although significant improvements have been reported in outcomes measures when comparing pre- and post-operative scores, little is known about how this improvement varies for different groups depending on the starting pain and function level. We aim to describe the changes in the Oxford Knee Score (OKS) and health-related quality of life (HRQL), and in particular to estimate the probability of a worse outcome, by decile of baseline OKS for patients in the English NHS.
Cost-utility of unicompartmental compared to total knee replacements: an analysis based on matched routinely-collected data from the United Kingdom
OBJECTIVES: To estimate and compare the lifetime costs and health consequences associated with unicompartmental knee replacement (UKR) and total knee replacement (TKR). METHODS: Various sources of routinely-collected data from the English National Health System were used to inform the analysis. Propensity score matching was used to identify comparable patients who received a UKR or TKR. Health outcomes, measured in Quality Adjusted Life Years (QALYs), as well as hospital and primary care costs to the health system were estimated and compared for subgroups based on age (&60, 60-75 and 75+) and gender, and for two subgroups based on surgeon usage of UKR (whether &10% or ≥10% of knee replacements performed by a surgeon were UKRs). These estimates and their distributions were used to populate a lifetime Markov model. RESULTS: For all age and gender subgroups the provision of UKR is expected to lead to a gain in QALYs compared to TKR (Male &60: 0.11, 60-75: 0.27, 75+: 0.19, Female &60: 0.06, 60-75: 0.26, 75+: 0.39) and a reduction in costs (Male &60: -£1,070, 60-75: -£1,676, 75+: -£1,903, Female &60: -£804, 60-75: -£1,249, 75+: -£1,215 per patient over the lifetime). UKR is expected to lead to a reduction in QALYs compared to TKR when performed by surgeons with low UKR utilisation but an increase among those with high utilisation (&10%: -0.19, ≥10%: 0.17). Regardless of surgeon usage, costs associated with UKR are expected to be lower than those of TKR (&10%: -£886, ≥10%: -£1,258). CONCLUSIONS: Based on its current provision in the UK, UKR can be expected to generate better health outcomes and lower lifetime costs than TKR. However, health outcomes are worse for UKRs when performed by surgeons who use UKR for &10% of their knee replacements and these surgeons should consider broadening their indications.
World Hip Trauma Evaluation (WHiTE): framework for embedded comprehensive cohort studies.
INTRODUCTION: Osteoporotic hip fractures present a significant global challenge to patients, clinicians and healthcare systems. It is estimated that hip fracture accounts for 1.4% of total social and healthcare costs in the established market economies. METHODS AND ANALYSIS: The World Hip Trauma Evaluation (WHiTE) was set up to measure outcome in a comprehensive cohort of UK patients with hip fracture. All patients in the cohort are treated under a single comprehensive treatment pathway. A core outcome set, including health-related quality of life, is collected on all the patients. This protocol describes the current multicentre project that will be used as a vehicle to deliver a series of embedded observational studies. ETHICS AND DISSEMINATION: Research Ethics Committee approval was granted (Rec reference 11/LO/0927, approved 18/8/2011) and each hospital trust provided National Health Service (NHS) approvals. TRIAL REGISTRATION NUMBER: The study is registered with National Institute of Health Research Portfolio (UKCRN ID 12351) and the ISRCTN registry (ISRCTN63982700).
Importance of Time Point-Specific Indirect Treatment Comparisons of Osteoporosis Treatments: A Systematic Literature Review and Network Meta-Analyses.
PURPOSE: The efficacy comparison of osteoporosis treatments can be hindered by the absence of head-to-head trials; instead, network meta-analyses (NMAs) have been used to determine comparative effectiveness. This study was the first to investigate the impact of time point-specific NMAs of osteoporosis treatments on variability in treatments' onset of action caused by their different mechanisms of actions and trial designs. METHODS: A systematic literature review was conducted to identify randomized controlled trials (RCTs) of treatments for postmenopausal women with osteoporosis, including romosozumab (ROMO), teriparatide (TPTD), abaloparatide (ABL), alendronate (ALN), risedronate (RIS), ibandronate (IB), zoledronic acid/zoledronate (ZOL), denosumab (DEN), and raloxifene (RLX), on at least 1 fracture or bone mineral density (BMD) outcome. Of 100 RCTs identified in 5 databases, 27 RCTs were included for NMAs of new vertebral, nonvertebral, and hip fracture outcomes at 12, 24, and 36 months, and 47 RCTs were included for NMAs of BMD outcomes at lumbar spine, total hip, and femoral neck to compare the relative efficacy of osteoporosis treatments. Quality of included studies was assessed using the Cochrane Risk of Bias tool. FINDINGS: For vertebral fractures, TPTD (83.63%), ABL (69.11%), and ROMO/ALN (78.70%) had the highest probability to be the most effective treatment at 12, 24, and 36 months, respectively. ROMO/ALN had the highest probability (54.4%, 64.69%, and 90.29%, respectively) to be the most effective treatment for nonvertebral fractures at 12, 24, and 36 months. For hip fractures, ROMO/ALN (46.31%), ABL (61.1%), and DEN (55.21%) had the highest probability to be the most effective treatment at 12, 24, and 36 months, respectively. ROMO had the highest probability (76.06%, 44.19%, and 51.78%, respectively) to be the most effective treatment for BMD outcomes at lumbar spine, total hip, and femoral neck. IMPLICATIONS: The importance of indirectly comparing available osteoporosis treatments using time point-specific NMAs was confirmed because indirect comparison results differed substantially across time points.
UK podiatrists' experiences of podiatry services for people living with arthritis: a qualitative investigation.
BACKGROUND: Provision of podiatry services, like other therapies in the UK, is an area that lacks guidance by the National Institute for Health and Care Excellence. Many individuals living with arthritis in the UK are not eligible to access NHS podiatry services. The primary aim of this investigation was to understand the views of podiatry clinicians on their experiences of referral, access, provision and treatment for foot problems for patients who have arthritis. METHODS: Focus groups were undertaken to explore, in-depth, individual views of podiatrists working in the UK to gain feedback on experiences of barriers and facilitators to referral, access, provision and treatment for foot problems for individuals living with arthritis. A purposive sampling strategy was adopted and two, semi-structured, focus group interviews conducted, involving 12 podiatrists from both NHS and independent sectors. To account for geographical variations one focus group took place in each of 2 predetermined 'zones' of the UK; Yorkshire and Hampshire. Thematic analysis was employed to identify key meanings and report patterns within the data. RESULTS: The key themes derived from the podiatry clinician focus groups suggest a variety of factors influencing demand for, and burden of, foot pain within the UK. Participants expressed frustration on having a service that accepts and treats patients according to their condition, rather than their complaint. Additionally, concern was conveyed over variations in the understanding of stakeholders' views of what podiatry is and what podiatrists aim to achieve for patients. CONCLUSION: Podiatrists interviewed believed that many individuals living with arthritis in the UK are not eligible to access NHS podiatry services and that this may be, in part, due to confusion over what is known about podiatry and access criteria. Essentially, podiatrists interviewed called for a timely renaissance of current systems, to newer models of care that meet the foot care needs of individual patients' circumstances and incorporate national multi-disciplinary guidance. Through this project, we have formulated key recommendations that are directed towards improving what other stakeholders (including GPs, commissioners and users of podiatry services) know about the effectiveness of podiatry and also to futureproof the profession of podiatry.
A qualitative study of GP, NP and patient views about the use of rapid streptococcal antigen detection tests (RADTs) in primary care: 'swamped with sore throats?'.
OBJECTIVE: To explore patient and healthcare professionals' (HCP) views of clinical scores and rapid streptococcal antigen detection tests (RADTs) for acute sore throat. DESIGN: Qualitative semistructured interview study. SETTING: UK primary care. PARTICIPANTS: General practitioners (GPs), nurse practitioners (NPs) and patients from general practices across Hampshire, Oxfordshire and the West Midlands who were participating in the Primary Care Streptococcal Management (PRISM) study. METHOD: Semistructured, face-to-face and phone interviews were conducted with GPs, NPs and patients from general practices across Hampshire, Oxfordshire and the West Midlands. RESULTS: 51 participants took part in the study. Of these, 42 were HCPs (29 GPs and 13 NPs) and 9 were patients. HCPs could see a positive role for RADTs in terms of reassurance, as an educational tool for patients, and for aiding inexperienced practitioners, but also had major concerns about RADT use in clinical practice. Particular concerns included the validity of the tests (the role of other bacteria, and carrier states), the tension and possible disconnect with clinical assessment and intuition, the issues of time and resource use and the potential for medicalisation of self-limiting illness. In contrast, however, experience of using RADTs over time seemed to make some participants more positive about using the tests. Moreover, patients were much more positive about the place of RADTs in providing reassurance and in limiting their antibiotic use. CONCLUSIONS: It is unlikely that RADTs will have a (comfortable) place in clinical practice in the near future until health professionals' concerns are met, and they have direct experience of using them. The routine use of clinical scoring systems for acute upper respiratory illness also face important barriers related to clinicians' perceptions of their utility in the face of clinician experience and intuition.