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Published on: September 20, 2024
Health economic modelling in Cystic Fibrosis: A systematic review
Bishal Mohindru1, David Turner1, Tracey Sach1
1Norwich Medical School, Norwich Research Park, University of East Anglia, Norwich, Norfolk NR4 7TJ, UK.
Insights
Health economic models for Cystic Fibrosis (CF) treatment are crucial for decision-making. This review highlights gaps in current CF modeling practices, particularly regarding significant health events and guideline adherence, to inform future research.
Area of Science:
- Health Economics
- Pharmacoeconomics
- Biostatistics
Background:
- Cystic Fibrosis (CF) is a genetic, chronic condition with significant treatment costs in the UK, averaging €49,000-€76,000 annually per patient.
- A comprehensive understanding of health economic modeling practices in CF is needed for effective decision-making and research guidance.
Purpose of the Study:
- To review and analyze health economic modeling studies for Cystic Fibrosis (CF) management.
- To identify current modeling practices, their strengths, and limitations.
Main Methods:
- A systematic online search of five databases was conducted.
- Studies included were model-based economic evaluations for CF management, restricted to English language publications.
Main Results:
- Nine studies were reviewed, predominantly using Markov cohort models to evaluate pharmaceutical interventions and drug adherence.
- Most models shared consistent structures but varied in data sources and utility elicitation methods.
- A significant limitation identified was the failure of most models to incorporate major health events impacting cost and disease progression.
Conclusions:
- Current CF economic models often lack adherence to European Medicines Agency (EMA) guidelines for clinical trial endpoints and justification for model structures.
- There is a need for improved utility data related to significant health events.
- Future research should focus on conceptualizing CF progression, valuing key health events, and aligning with EMA reporting guidelines.
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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