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OCTRU/CSM have recruited a new lead statistician to join the team.
Imminent fracture risk assessments in the UK FLS setting: implications and challenges.
With the recognition that a sentinel fracture leads to a high imminent risk of fracture, we discuss the implications and challenges of using imminent fracture risk in the secondary fracture prevention setting.
Potential lifetime quality of life benefits of choroideremia gene therapy: projections from a clinically informed decision model.
BACKGROUND: The first gene therapy for an inherited retinal dystrophy recently received market approval in the United States; multiple other gene therapies are in the clinical pipeline. Thus far, gene therapy has commanded prices in the range of $500,000 to over $1,000,000 for the one-time doses and have been indicated for highly orphan diseases where there is no other viable treatment option. To be adopted by healthcare systems, gene therapy will need to show clinical benefit in line with its increased costs. Before longitudinal patient studies are available, model-based estimations will be necessary to project the full clinical benefit of gene therapy. METHODS: To investigate the lifetime benefit of gene therapy for the retinal dystrophy choroideremia, we have built a Markov model of disease progression informed by clinical data of AAV.REP1 and voretigene neparvovec (Luxturna, Spark Therapeutics). Gene therapy patient benefit was estimated by quality-adjusted life years (QALYs) in three hypothetical disease severity patient groups. The severity of disease was defined by the combined effect of remaining retinal area and visual acuity and assigned corresponding health utility values. RESULTS: Early-stage patients treated with gene therapy were estimated to gain, in average, 14.30 QALYs over standard-of-care, mid-stage patients 6.22 QALYs, and late-stage patients 1.48 QALYs over untreated patients during their lifetime owing to treatment. Cost-effectiveness was not assessed as AAV.REP1 is still in clinical trials. CONCLUSIONS: In young adults in the earlier stages of choroideremia, successful gene therapy is expected to provide a significant increase in health-related quality of life.
Threshold prices for computer and robot-assisted knee and hip replacements in the English NHS
Objectives To estimate threshold prices for computer- and robot-assisted knee and hip replacement. Methods A lifetime cohort Markov model provided the framework for analysis. Linked primary care and inpatient hospital records informed estimates of outcomes under current practice. Outcomes were estimated under a range of hypothetical relative improvements in quality of life if unrevised and in revision risk after computer or robot-assisted surgery. Threshold prices, a price at which the net health benefit from funding the intervention would be zero, for these improvements were estimated for a cost-effectiveness threshold of £20 000 per additional quality-adjusted life-year (QALY) gained. Results For average patient profiles under current knee and hip replacement practice, lifetime QALYs were 10.3 (9.9 to 10.7) and 11.0 (10.6 to 11.4), with costs of £6060 (£5947 to £6203) and £6506 (£6335 to £6710) for knee and hip replacement, respectively. A combined 50% relative reduction in risk of revision and 5% improvement in postoperative quality of life if unrevised would, for example, result in QALYs increasing to 10.9 (10.4 to 11.3) and 11.6 (11.2 to 12.0), and costs falling to £5880 (£5816 to £5956) and £6258 (£6149 to £6376) after knee and hip replacement, respectively. These particular improvements would have an associated threshold price of £11 182 (£10 691 to £11 721) for knee replacement and £12 134 (£11 616 to £12 701) for hip replacement. The 50% reduction in revision rate alone would have associated threshold prices of £1094 (£788 to £1488) and £1347 (£961 to £1842), and the 5% improvement in quality of life alone would have associated threshold prices of £9911 (£9476 to £10 296) and £10 578 (£10 171 to £10 982). Conclusions At current prices, computer- and robot-assisted knee and hip replacement will likely need to lead to improvements in patient-reported outcomes in addition to any reduction in the risk revision.
The World Hip Trauma Evaluation (WHiTE) platform trial: a framework for randomized comparisons of interventions for fragility hip fracture.
AIMS: Hip fracture is one of the biggest challenges facing patients and healthcare systems. Worldwide, there are currently 1.3 million hip fractures per year, projected to rise to more than six million by 2050. This protocol describes a platform trial framework, designed to efficiently deliver multiple randomized comparisons of interventions for patients with a fragility hip fracture. METHODS: All patients aged 60 years and over with a hip fracture presenting to the World Hip Trauma Evaluation (WHiTE) recruitment centres will be considered for eligibility for each of the randomized comparisons appended to the platform at the time of recruitment. They will be offered the opportunity to take part in any or all of the randomized comparisons for which they are eligible. Comparisons may be contemporaneous or distributed throughout the treatment pathway. This master protocol describes the trial procedures, core dataset, and documentation. It describes those components of the research process which will be consistent between randomized comparisons. Where additional procedures are planned, specific to a randomized comparison, these will be described in a separate appendix protocol for that randomized comparison. CONCLUSION: The WHiTE platform trial will provide randomized evidence regarding the clinical and cost-effectiveness of interventions to improve outcomes for patients with fragility hip fracture. Findings will inform national and international policy and practice guidelines for the management of patients with a hip fracture.