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Before an intervention is publicly funded within the United Kingdom, the cost-effectiveness is assessed by the National Institute of Health and Care Excellence (NICE). The efficacy of an intervention across the patients’ lifetime is often influential of the cost-effectiveness analyses, but is associated with large uncertainties. We reviewed committee documents containing company submissions and evidence review group (ERG) reports to establish the methods used when extrapolating survival data, whether these adhered to NICE Technical Support Document (TSD) 14, and how uncertainty was addressed.
A systematic search was completed on the NHS Evidence Search webpage limited to single technology appraisals of cancer interventions published in 2017, with information obtained from the NICE Web site.
Twenty-eight appraisals were identified, covering twenty-two interventions across eighteen diseases. Every economic model used parametric curves to model survival. All submissions used goodness-of-fit statistics and plausibility of extrapolations when selecting a parametric curve. Twenty-five submissions considered alternate parametric curves in scenario analyses. Six submissions reported including the parameters of the survival curves in the probabilistic sensitivity analysis. ERGs agreed with the company's choice of parametric curve in nine appraisals, and agreed with all major survival-related assumptions in two appraisals.
TSD 14 on survival extrapolation was followed in all appraisals. Despite this, the choice of parametric curve remains subjective. Recent developments in Bayesian approaches to extrapolation are not implemented. More precise guidance on the selection of curves and modelling of uncertainty may reduce subjectivity, accelerating the appraisal process.
Microfracture (MF) has been the main intervention in symptomatic articular cartilage knee defects. Autologous chondrocyte implantation (ACI) has looked promising, but was not recommended by the UK National Institute for Health and Care Excellence (NICE) in 2015 due to the short-term follow-up data from trials.
Most long-term data comes from observational studies. We provided new unpublished analyses to NICE based on survival data of these studies, with appropriate caveats. They included: a large ACI study by Nawaz with useful subgroup data by osteoarthritis Kellgren-Lawrence stage and previous repair attempts; a very large MF study by Layton, and a small RCT by Knutsen indicating MF was as ‘good’ as ACI. A Markov model explored the cost-effectiveness of ACI vs. MF. Different scenarios were explored: ACI or MF as a first procedure, followed by ACI or MF in those needing a second repair. A NHS England perspective was adopted. Health outcomes were expressed as quality-adjusted life-years (QALYs).
The revised base-case analysis, used a list price of £16,000 (EUR 17,380 in 2013 prices) for cells, used ACI failure data from Nawaz with no previous procedures for ACI, and pooled MF failure data from two studies-Saris and Knutsen. ACI was more expensive but provided more QALYs. The incremental cost-effectiveness ratio comparing ACI then MF with MF then ACI was £8,000 (EUR 8,690) per QALY. Various sensitivity analyses were conducted assuming a threshold of £20,000 (EUR 21,730) per QALY: previous repair attempts reduced success of ACI (£22,000 (EUR 23,900) per QALY); reducing cell costs, ACI improved its cost-effectiveness; and limiting intervention to patients with higher Kellgren-Lawrence score did not appear cost-effectiveness.
The final NICE guidance published in October 2017 approved the use of ACI for patients who had no previous knee repairs, for people with minimal osteoarthritic damage to the knee, and for people with articular defects of over 2cm2.
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