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
#MMPMID24290164
Tappenden P
; Harnan S
; Uttley L
; Mildred M
; Carroll C
; Cantrell A
Health Technol Assess
2013[Dec]; 17
(56
): v-xvii, 1-181
PMID24290164
show ga
BACKGROUND: Cystic fibrosis (CF) is an inherited condition characterised by the
abnormal transport of chloride ions across transporting epithelia. This leads to
the production of thick sticky mucus in the lungs, pancreas, liver, intestine and
reproductive tract, and an increase in the salt content in sweat. Among other
problems, people with CF experience recurrent respiratory infections and have
difficulties digesting food. CF affects over 9000 individuals in the UK. CF
shortens life expectancy and adversely affects quality of life. In 2010, CF was
recorded as the cause of 103 deaths in England and Wales. OBJECTIVE: To evaluate
the clinical effectiveness and cost-effectiveness of colistimethate sodium dry
powder for inhalation (DPI) (Colobreathe(®), Forest Laboratories) and tobramycin
DPI (TOBI Podhaler(®), Novartis Pharmaceuticals) for the treatment of Pseudomonas
aeruginosa lung infection in CF. DATA SOURCES: Electronic databases were searched
in February and March 2011 [MEDLINE, MEDLINE In-Process & Other Non-Indexed
citations, EMBASE, The Cochrane Library databases, Cumulative Index to Nursing
and Allied Health Literature (CINAHL), Web of Science, Conference Proceedings
Citation Index (CPCI) and Bioscience Information Service (BIOSIS) Previews].
Relevant databases were searched for ongoing and unpublished studies, and
bibliographies of relevant systematic reviews and the manufacturers' submissions
were also hand-searched. REVIEW METHODS: A systematic review of the clinical
effectiveness and cost-effectiveness of colistimethate sodium DPI and tobramycin
DPI for the treatment of chronic P. aeruginosa lung infection in CF was
conducted. Existing economic evidence within the literature was reviewed and a de
novo health economic model was also developed. RESULTS: Three randomised
controlled trials (RCTs) were included in the clinical effectiveness review. Both
colistimethate sodium DPI and tobramycin DPI were reported to be non-inferior to
nebulised tobramycin for the outcome forced expiratory volume in first second
percentage predicted (FEV1%). It was not possible to draw any firm conclusions as
to the relative efficacy of colistimethate sodium DPI compared with tobramycin
DPI. The economic analysis suggests that colistimethate sodium DPI produces fewer
quality-adjusted life-years (QALYs) than nebulised tobramycin. Given the
incremental discounted lifetime cost of tobramycin DPI compared with nebulised
tobramycin, it highly unlikely that tobramycin DPI has an incremental
cost-effectiveness ratio that is better than £30,000 per QALY gained. LIMITATION:
The uncertainty surrounding the short-term evidence base inevitably results in
uncertainty surrounding the long-term clinical effectiveness and
cost-effectiveness of colistimethate sodium DPI. CONCLUSIONS: Both DPI
formulations have been shown to be non-inferior to nebulised tobramycin as
measured by FEV1%. The results of these trials should be interpreted with caution
owing to the means by which the results were analysed, the length of follow-up,
and concerns about the ability of FEV1% to accurately represent changes in lung
health. Although the increase in QALYs is expected to be lower with
colistimethate sodium DPI than with nebulised tobramycin, a price for this
intervention had not been agreed at the time of the assessment. Depending on the
price of colistimethate sodium DPI, this results either in a situation whereby
colistimethate sodium DPI is dominated by nebulised tobramycin or in one whereby
the incremental cost-effectiveness of nebulised tobramycin compared with
colistimethate sodium DPI is in the range of £24,000-277,000 per QALY gained. The
economic analysis also suggests that, given its price, it is unlikely that
tobramycin DPI has a cost-effectiveness ratio of
|Administration, Inhalation
[MESH]
|Child
[MESH]
|Colistin/administration & dosage/*analogs & derivatives/economics/therapeutic use
[MESH]
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