Helen Dakin

Rationing of total knee replacement: a cost-effectiveness analysis on a large trial data set.

Objectives Many UK primary care trusts have recently introduced eligibility criteria restricting total knee replacement (TKR) to patients with low pre-operative Oxford Knee Scores (OKS) to cut expenditure. We evaluate these criteria by assessing the cost-effectiveness of TKR compared with no knee replacement for patients with different baseline characteristics from an NHS perspective. Design The cost-effectiveness of TKR in different patient subgroups was assessed using regression analyses of patient-level data from the Knee Arthroplasty Trial, a large, pragmatic randomised trial comparing knee prostheses. Setting 34 UK hospitals. Participants 2131 osteoarthritis patients undergoing TKR. Interventions and outcome measures Costs and quality-adjusted life years (QALYs) observed in the Knee Arthroplasty Trial within 5 years of TKR were compared with conservative assumptions about the costs and outcomes that would have been accrued had TKR not been performed. Results On average, primary TKR and 5 years of subsequent care cost £7458 per patient (SD: £4058), and patients gained an average of 1.33 (SD: 1.43) QALYs. As a result, TKR cost £5623/QALY gained. Although costs and health outcomes varied with age and sex, TKR cost <£20 000/QALY gained for patients with American Society of Anaesthesiologists grades 1–2 who had baseline OKS <40 and for American Society of Anaesthesiologists grade 3 patients with OKS <35, even with highly conservative assumptions about costs and outcomes without TKR. Body mass index had no significant effect on costs or outcomes. Restricting TKR to patients with pre-operative OKS <27 would inappropriately deny a highly cost-effective treatment to >10 000 patients annually. Conclusions TKR is highly cost-effective for most current patients if the NHS is willing to pay £20 000–£30 000/QALY gained. At least 97% of TKR patients in England have more severe symptoms than the thresholds we have identified, suggesting that further rationing by OKS is probably unjustified. Trial registration number ISRCTN 45837371.

The Influence of Cost‐Effectiveness and Other Factors on Nice Decisions

The National Institute for Health and Care Excellence (NICE) emphasises that cost-effectiveness is not the only consideration in health technology appraisal and is increasingly explicit about other factors considered relevant but not the weight attached to each. The objective of this study is to investigate the influence of cost-effectiveness and other factors on NICE decisions and whether NICEs decision-making has changed over time. We model NICEs decisions as binary choices for or against a health care technology in a specific patient group. Independent variables comprised of the following: clinical and economic evidence; characteristics of patients, disease or treatment; and contextual factors potentially affecting decision-making. Data on all NICE decisions published by December 2011 were obtained from HTAinSite [www.htainsite.com]. Cost-effectiveness alone correctly predicted 82% of decisions; few other variables were significant and alternative model specifications had similar performance. There was no evidence that the threshold has changed significantly over time. The model with highest prediction accuracy suggested that technologies costing £40 000 per quality-adjusted life-year (QALY) have a 50% chance of NICE rejection (75% at £52 000/QALY; 25% at £27 000/QALY). Past NICE decisions appear to have been based on a higher threshold than £20 000-£30 000/QALY. However, this may reflect consideration of other factors that cannot be easily quantified.

Review of studies mapping from quality of life or clinical measures to EQ-5D: an online database.

Systematic literature searches were conducted to identify studies that conducted statistical mapping to predict EQ-5D utilities or responses from any source instrument and reported the estimated algorithms in sufficient detail to allow other researchers to use them to predict EQ-5D in other studies. Ninety studies reporting 121 mapping algorithms met the inclusion criteria. The studies estimated EQ-5D utilities from 80 source instruments. All but two studies included direct utility mapping to predict EQ-5D utilities, while 20 studies (22%) conducted response mapping to predict responses to each EQ-5D domain. Seventy-two studies (80%) explored ordinary least squares regression and 16 (18%) used censored least absolute deviations (CLAD) models. The details of the studies identified are made available in an online database, which will be updated regularly to enable researchers to easily identify studies that can help them to estimate utilities for economic evaluation.

Patellar resurfacing in total knee replacement: five-year clinical and economic results of a large randomized controlled trial.

BACKGROUND There is conflicting evidence regarding the merits of patellar resurfacing during total knee arthroplasty, as many of the previous randomized controlled trials have not been adequately powered. METHODS A pragmatic, multicenter, randomized controlled trial was initiated in 1999 in the United Kingdom. Within a partial factorial design, 1715 patients were randomly allocated to receive or not receive patellar resurfacing during total knee arthroplasty. The primary outcome measure was the Oxford Knee Score; secondary measures included the Short Form-12, the EuroQoL 5D, cost, cost-effectiveness, and the need for subsequent knee surgery. RESULTS The mean Oxford Knee Score was 35 points at five years postoperatively in both groups. There was no significant difference between the groups with respect to the mean Oxford Knee Score (difference, 0.59 point; 95% confidence interval, -0.58 to 1.76 points) or any other outcome measure at five years postoperatively. The outcome was not affected by whether the patella was domed or anatomic. There was no significant difference between the two groups with respect to the prevalence of knee-related readmission, of minor or intermediate reoperation, or of subsequent patella-related surgery. The total health care cost for the primary arthroplasty, subsequent monitoring, and any revision surgery did not differ significantly between the two groups. CONCLUSIONS In the largest randomized controlled trial of patellar resurfacing reported to date, the functional outcome, reoperation rate, and total health care cost five years after primary total knee arthroplasty were not significantly affected by the addition of patellar resurfacing to the surgical procedure.

Mapping analyses to estimate health utilities based on responses to the OM8-30 otitis media questionnaire

PurposeTo investigate the statistical relationship between the OM8-30 health-related quality of life measure for children with otitis media with effusion (OME) and measures of health utility (Health Utilities Index [HUI] Mark 3 and Mark 2) and to develop models to estimate HUI3 and HUI2 health utilities from OM8-30 scores.MethodsA placebo-controlled, randomised trial (GNOME) evaluating intranasal mometasone in 217 children with OME provided concurrent responses to OM8-30 and HUI at three time points. Ordinary least squares (OLS), generalised linear models and two-step regression analyses were used to predict HUI3 and HUI2 utilities based on OM8-30 facet and domain scores.ResultsOLS models including all nine OM8-30 facets with or without predicted hearing level (HL) produced the best predictions of HUI3 utilities (mean absolute error: 0.134 with HL and 0.132 without; R2: 0.63 with HL and 0.596 without). An OLS model predicting HUI3 utilities based on the two OM8-30 domain scores, reported hearing difficulties, predicted HL, age and sex also produced accurate predictions.ConclusionRegression equations predicting HUI3 and HUI2 utilities based on OM8-30 facet and domain scores have been developed. These provide an empirical basis for estimating quality-adjusted life years (QALYs) for interventions in children with OME.

Cost-effectiveness of ranibizumab and bevacizumab for age-related macular degeneration: 2-year findings from the IVAN randomised trial

Objective To assess the incremental cost and cost-effectiveness of continuous and discontinuous regimens of bevacizumab (Avastin) and ranibizumab (Lucentis) for neovascular age-related macular degeneration (nAMD) from a UK National Health Service (NHS) perspective. Design A within-trial cost-utility analysis with a 2-year time horizon, based on a multicentre factorial, non-inferiority randomised controlled trial. Setting 23 hospital ophthalmology clinics. Participants 610 patients aged ≥50 years with untreated nAMD in the study eye. Interventions 0.5 mg ranibizumab or 1.25 mg bevacizumab given continuously (monthly) or discontinuously (as-needed) for 2 years. Main outcome measures Quality-adjusted life-years (QALYs). Results Total 2-year costs ranged from £3002/patient (

Cost–Utility Analysis of Tenofovir Disoproxil Fumarate in the Treatment of Chronic Hepatitis B

4700; 95% CI £2601 to £3403) for discontinuous bevacizumab to £18 590/patient (

A double-blind randomised placebo-controlled trial of topical intranasal corticosteroids in 4- to 11-year-old children with persistent bilateral otitis media with effusion in primary care.

29 106; 95% CI £18 258 to £18 922) for continuous ranibizumab. Ranibizumab was significantly more costly than bevacizumab for both continuous (+£14 989/patient (

Mapping analyses to estimate EQ-5D utilities and responses based on Oxford Knee Score

23 468); 95% CI £14 522 to £15 456; p<0.001) and discontinuous treatment (+£8498 (

Mixed Treatment Comparison Meta‐Analysis Evaluating the Relative Efficacy of Nucleos(t)ides for Treatment of Nucleos(t)ide‐Naive Patients with Chronic Hepatitis B

13 305); 95% CI £7700 to £9295; p<0.001), with negligible difference in QALYs. Continuous ranibizumab would only be cost-effective compared with continuous bevacizumab if the NHS were willing to pay £3.5 million (