An Early Health Economic Analysis of the Potential Cost Effectiveness of an Adherence Intervention to Improve Outcomes for Patients with Cystic Fibrosis
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Cystic fibrosis (CF) negatively impacts upon health-related quality of life and survival. Adherence to nebulised treatments is low; improving adherence is hypothesised to reduce rates of exacerbation requiring intravenous antibiotics and lung function decline.
A state transition model was developed to assess the cost effectiveness of an intervention aimed at increasing patient adherence to nebulised and inhaled antibiotics compared with current CF care, in advance of the forthcoming CFHealthHub randomised controlled trial (RCT).
The model estimated the costs and health outcomes for each option from the perspective of the UK National Health Service and Personal Social Services over a lifetime horizon. Health gains were valued in terms of quality-adjusted life-years (QALYs) gained. Forced expiratory volume in 1 second (FEV1) trajectories were predicted over three lung function strata: (1) FEV1 ≥70%, (2) FEV1 40–69% and (3) FEV1 <40%. Additional states were included to represent ‘post-lung transplantation’ and ‘dead’. The model was populated using CF Registry data, literature and expert opinion. Costs were presented at 2016 values. Uncertainty was assessed using deterministic and probabilistic sensitivity analyses.
If effective, the adherence intervention is expected to produce an additional 0.19 QALYs and cost savings of £64,078 per patient. Across all analyses, the intervention dominated current care. Over a 5-year period, the intervention is expected to generate cost savings of £49.5 million for the estimated 2979 patients with CF with Pseudomonas aeruginosa currently aged ≥16 years in the UK. If applied to a broader population of adult patients with CF receiving any nebulised therapy, the expected savings could be considerably greater.
If effective, the adherence intervention is expected to produce additional health gains at a lower cost than current CF care. However, the economic analysis should be revisited upon completion of the full RCT. More generally, the analysis suggests that considerable gains could be accrued through the implementation of adherence interventions that shift care from expensive hospital-based rescue to community-based prevention.
This study represents an early evaluation undertaken as part of the CFHealthHub ACtiF programme, funded by the National Institute for Health Research (NIHR) Health Technology Assessment (HTA) Programme (grant number RP-PG-1212-20015). The authors thank the CF Registry for providing access to data.
Paul Tappenden developed the health economic model. Susannah Sadler undertook the analyses of the CF Registry dataset. Martin Wildman advised on the design of the study and the evidence used to inform the model. All authors contributed to the preparation of this manuscript. Paul Tappenden will act as the overall guarantor for this work.
Compliance with Ethical Standards
Dr Wildman has received support from Pari to speak at conferences about the importance of adherence and to travel to meetings with Pari about setting up a trial to understand whether increasing adherence improves outcomes in CF. He has also received funding from Philips to support research using the Ineb nebuliser to understand how the device can be used to measure adherence and received speaker fees from Forest to give independent talks at CF meetings around the UK about the importance of adherence. Paul Tappenden and Susannah Sadler have no conflicts of interest.
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