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Health economic modelling in Cystic Fibrosis: A systematic review

Health economic modelling in Cystic Fibrosis: A systematic review
Health economic modelling in Cystic Fibrosis: A systematic review

INTRODUCTION: Cystic Fibrosis (CF) is a heritable chronic condition. Due to the genetic and progressive nature of CF, a number of interventions are available for the condition. In the United Kingdom (U.K.) average annual cost of CF treatment is between €49,000 to €76,000 (2012) per patient [1]. A review of health economic modelling studies is warranted to provide decision makers and researchers with an in depth understanding of modelling practices in CF and guidance for future research.

METHODS: Online searches were performed in the 5 databases, studies were included if they were: a) Model based economic evaluation for management of Cystic Fibrosis. Articles were restricted to English language only, but no restriction was applied on publication year.

RESULTS: Nine studies were reviewed, most were Markov cohort models. Models evaluated pharmaceutical interventions and drug adherence. Modelling structure was consistent across most articles and a range of sources were used to populate the models. Cost and utility data were based on different sources and elicitation methods respectively. The majority of models failed to incorporate significant health events which impact both cost and disease progression.

CONCLUSION: In our review we observed a lack of, application of European Medicines Agency (EMA) guidelines for clinical trial endpoints, model structure justifications and lastly, health-related quality of life derived utility information around important clinical events. Future work around conceptual modelling of CF progression, utility valuation of significant health events and meeting EMA guidelines for trial reporting is encouraged.

Cost-Benefit Analysis, Cystic Fibrosis/economics, Health Care Costs, Humans, Models, Economic
1569-1993
452-460
Mohindru, Bishal
752b0309-8bb0-4094-9ec7-38f849e2697d
Turner, David
407ea6bc-44cc-4afd-927d-ea953190e60a
Sach, Tracey
5c09256f-ebed-4d14-853a-181f6c92d6f2
Bilton, Diana
46bbb90e-5d96-4395-b708-455b9d906b54
Carr, Siobhan
25b470a5-9227-4d9b-b397-f1ae4529b737
Archangelidi, Olga
0ac00a72-1e2a-4bc4-9fad-105801545611
Bhadhuri, Arjun
538e68a1-3855-4d35-91ae-863d0afaf291
Whitty, Jennifer A
c8c07592-aaeb-4952-9544-4ab79c03204e
Mohindru, Bishal
752b0309-8bb0-4094-9ec7-38f849e2697d
Turner, David
407ea6bc-44cc-4afd-927d-ea953190e60a
Sach, Tracey
5c09256f-ebed-4d14-853a-181f6c92d6f2
Bilton, Diana
46bbb90e-5d96-4395-b708-455b9d906b54
Carr, Siobhan
25b470a5-9227-4d9b-b397-f1ae4529b737
Archangelidi, Olga
0ac00a72-1e2a-4bc4-9fad-105801545611
Bhadhuri, Arjun
538e68a1-3855-4d35-91ae-863d0afaf291
Whitty, Jennifer A
c8c07592-aaeb-4952-9544-4ab79c03204e

Mohindru, Bishal, Turner, David, Sach, Tracey, Bilton, Diana, Carr, Siobhan, Archangelidi, Olga, Bhadhuri, Arjun and Whitty, Jennifer A (2019) Health economic modelling in Cystic Fibrosis: A systematic review. Journal of Cystic Fibrosis, 18 (4), 452-460. (doi:10.1016/j.jcf.2019.01.007).

Record type: Article

Abstract

INTRODUCTION: Cystic Fibrosis (CF) is a heritable chronic condition. Due to the genetic and progressive nature of CF, a number of interventions are available for the condition. In the United Kingdom (U.K.) average annual cost of CF treatment is between €49,000 to €76,000 (2012) per patient [1]. A review of health economic modelling studies is warranted to provide decision makers and researchers with an in depth understanding of modelling practices in CF and guidance for future research.

METHODS: Online searches were performed in the 5 databases, studies were included if they were: a) Model based economic evaluation for management of Cystic Fibrosis. Articles were restricted to English language only, but no restriction was applied on publication year.

RESULTS: Nine studies were reviewed, most were Markov cohort models. Models evaluated pharmaceutical interventions and drug adherence. Modelling structure was consistent across most articles and a range of sources were used to populate the models. Cost and utility data were based on different sources and elicitation methods respectively. The majority of models failed to incorporate significant health events which impact both cost and disease progression.

CONCLUSION: In our review we observed a lack of, application of European Medicines Agency (EMA) guidelines for clinical trial endpoints, model structure justifications and lastly, health-related quality of life derived utility information around important clinical events. Future work around conceptual modelling of CF progression, utility valuation of significant health events and meeting EMA guidelines for trial reporting is encouraged.

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Accepted/In Press date: 21 January 2019
e-pub ahead of print date: 7 February 2019
Published date: 20 June 2019
Additional Information: Copyright © 2019. Published by Elsevier B.V.
Keywords: Cost-Benefit Analysis, Cystic Fibrosis/economics, Health Care Costs, Humans, Models, Economic

Identifiers

Local EPrints ID: 480836
URI: http://eprints.soton.ac.uk/id/eprint/480836
ISSN: 1569-1993
PURE UUID: 0dbfac65-2600-4334-8909-a26b35be2151
ORCID for Tracey Sach: ORCID iD orcid.org/0000-0002-8098-9220

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Date deposited: 10 Aug 2023 16:37
Last modified: 17 Mar 2024 04:20

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Contributors

Author: Bishal Mohindru
Author: David Turner
Author: Tracey Sach ORCID iD
Author: Diana Bilton
Author: Siobhan Carr
Author: Olga Archangelidi
Author: Arjun Bhadhuri
Author: Jennifer A Whitty

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