Colistimethate sodium powder and tobramycin powder for inhalation for the treatment of chronic Pseudomonas aeruginosa lung infection in cystic fibrosis: systematic review and economic model

Tappenden, Paul; Harnan, S; Uttley, Lesley; Mildred, M; Cantrell, Anna

doi:10.3310/hta17560

Cited by 26 publications

(17 citation statements)

References 107 publications

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“…However, in order to undertake health economic modelling, there needs to be sufficient data to populate the model, which in turn should reflect disease progression [38]. Previous research [39] and models have highlighted a lack of HSU evidence to inform CF health economic models [15,40]. This is the first systematic review to describe the HSU data available in the literature for CF.…”

Section: Discussionmentioning

confidence: 99%

Health State Utility Data in Cystic Fibrosis: A Systematic Review

Mohindru

Turner

Sach

et al. 2019

PharmacoEconomics Open

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Introduction Cystic fibrosis (CF) is a life-limiting, hereditable condition, with the highest prevalence in Europe. CF treatments have led to improvements in clinical symptoms, disease management and decelerated disease progression. However, little is known about the health state utility (HSU) associated with CF disease states, adverse events, and changes in disease severity. Although HSU data have contributed to existing health economic modelling studies, a lack of such data have been highlighted. This systematic review aims to provide a summary of HSU-related research in CF and highlight related research gaps. Methods Online searches were performed in six databases and studies in any of the following categories were included: (1) estimation of HSUs in CF; (2) mapping studies between patient-reported outcome measures (PROMs) and HSUs; (3) economic evaluations on the management of CF that report primary HSU data; and (4) any CF clinical trial that reported HSU as an outcome. Results A total of 17 studies were reviewed, of which 12 provided HSU values for specific CF populations. The remaining five articles provided HSU data that were broken down by CF relevant health states, including lung transplantations, pulmonary exacerbation (PEx) events and forced expiratory volume in 1 s (FEV 1). Conclusion Current HSU data in CF are limited and there is considerable scope for further research, both in providing HSU values for CF and in investigating methods for HSU elicitation/evaluation in CF populations.

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Section: Discussionmentioning

confidence: 99%

Health State Utility Data in Cystic Fibrosis: A Systematic Review

Mohindru

Turner

Sach

et al. 2019

PharmacoEconomics Open

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“…The broader evidence network for a mixed treatment comparison was also examined, but was not included in this review as a network could not be constructed due to clinical heterogeneity between trials; for more details see [9]. The following electronic databases were searched for published trials and systematic reviews: MEDLINE, EMBASE, Cochrane Library, CINAHL, Web of Science Citation Index, Conference Proceedings Citation Index, and BIOSIS Previews.…”

Section: Methodsmentioning

confidence: 99%

Systematic review of the dry powder inhalers colistimethate sodium and tobramycin in cystic fibrosis

Uttley¹,

Harnan²,

Cantrell³

et al. 2013

European Respiratory Review

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This systematic review evaluated evidence for two dry powder formulations, colistimethate sodium and tobramycin, for the treatment of chronic Pseudomonas aeruginosa in cystic fibrosis, as part of the UK national recommendation process for new technologies. Electronic bibliographic databases were searched in May 2012 (MEDLINE, MEDLINE in-Process, EMBASE, Cochrane Library databases, CINAHL, Web of Science, Conference Proceedings Citation Index and BIOSIS Previews). Relevant outcomes included rate and extent of microbial response (e.g. sputum density of P. aeruginosa), lung function (e.g. forced expiratory volume in 1 s (FEV1)), frequency, severity of acute exacerbations and adverse events. Three trials were included, and both dry powder formulations were reported to be non-inferior in the short term to nebulised tobramycin for FEV1. However, long-term follow-up data were missing and the effect on exacerbation rates was not always reported. Whilst short-term results showed that both dry powder drugs were non-inferior to nebulised tobramycin, there was no long-term follow-up and no phase 3 trials compared nebulised and dry powder colistimethate sodium. The use of FEV1 as the primary end-point may not accurately represent changes in lung health. This review illustrates the difficulty in assessing new technologies where the evidence base is poor. @ERSpublications It is difficult to assess potential benefits of new, innovative technologies when the evidence base for efficacy is poor http://ow.ly/nVbCP

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“…In 2011, NICE undertook a technology appraisal [8,9 && ,10,11] of the antipseudomonal antibiotic DPIs (and corresponding inhaler devices): colistimethate sodium (Colobreathe and Turbospin device), and tobramycin (TOBI and Podhaler) for the treatment of chronic P. aeruginosa lung infection in patients with CF.…”

Section: Review Of Clinical Evidence Concerning Colistimethate Sodiummentioning

confidence: 99%