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BMC Pharmacology and Toxicology

, 15:63

Clinical pharmacology

Abstract

BackgroundIdiopathic pulmonary fibrosis IPF is a life-limiting lung disease with considerable impact on patients and carers as the disease progresses. Currently few treatments are available. We aimed to evaluate the clinical and cost-effectiveness of available treatments for IPF.

MethodsSystematic reviews of clinical effectiveness, quality of life and cost effectiveness were undertaken. Eleven bibliographic databases were searched from inception to July 2013 and studies were assessed for eligibility against a set of pre-defined criteria. Two reviewers screened references, extracted data from included studies and appraised their quality. An advisory group was consulted about the choice of interventions. A narrative review was undertaken and where feasible fixed effect and random effects meta-analysis were undertaken including a network meta-analysis NMA.

A decision-analytic Markov model was developed to estimate cost-effectiveness of pharmacological treatments for IPF. Following best practice recommendations, the model perspective was of the national health service and personal social services, a discount rate of 3.5% for costs and health benefits was applied and outcomes were expressed as cost per quality adjusted life-year gained. Parameter values were obtained from the NMA and systematic reviews. Sensitivity analyses were undertaken.

ResultsFourteen studies were included in the review of clinical effectiveness, of which one evaluated azathioprine, three N-acetylcysteine NAC alone or in combination, four pirfenidone, one nintedanib, one sildenafil, one thalidomide, two pulmonary rehabilitation, and one a disease management programme. Study quality was generally good. Evidence suggests that some effective treatments are available. In NMA only nintedanib and pirfenidone show statistically significant improvements. The model results show increased survival for five pharmacological treatments NAC triple therapy, inhaled NAC, nintedanib, pirfenidone, and sildenafil compared with best supportive care, at increased cost. Only inhaled NAC was cost-effective at current willingness to pay thresholds but it may not be clinically effective.

ConclusionsFew interventions have any statistically significant effect and the cost-effectiveness of treatments is uncertain. A lack of studies on palliative care approaches was identified and there is a need for further research into pulmonary rehabilitation and thalidomide in particular. A well conducted RCT on inhaled NAC therapy should also be considered.

KeywordsIdiopathic pulmonary fibrosis Systematic review Meta-analysis Cost-effectiveness AbbreviationsBSCBest supportive care

CCTControlled clinical trial

EQ-5DEuroQol five dimensions

FVCForced vital capacity

HRQoLHealth related quality of life

ICERIncremental cost-effectiveness ratio

IPFIdiopathic pulmonary fibrosis

UKUnited Kingdom

NACN-acetylcysteine

NHSNational health service

NICENational institute for health and care excellence

NMANetwork meta-analysis

QALYQuality adjusted life Year

RCTRandomised controlled trial

SDStandard deviation

SF-36Short Form-36

SMDStandardised mean difference

VCVital capacity

WTPWillingness to pay.

Electronic supplementary materialThe online version of this article doi:10.1186-2050-6511-15-63 contains supplementary material, which is available to authorized users.

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Author: Emma Loveman - Vicky R Copley - Jill L Colquitt - David A Scott - Andy J Clegg - Jeremy Jones - Katherine MA O’Reill

Source: https://link.springer.com/



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