Cost-Effectiveness Analysis Methods Used in Evaluations of Treatment for Cystic Fibrosis: A Scoping Review

Dominique Seo (), David C. Young, Eberechukwu Onukwugha and T. Joseph Mattingly
Additional contact information
Dominique Seo: University of Maryland School of Pharmacy
David C. Young: University of Utah College of Pharmacy
Eberechukwu Onukwugha: University of Maryland School of Pharmacy
T. Joseph Mattingly: University of Utah College of Pharmacy

PharmacoEconomics, 2025, vol. 43, issue 7, No 2, 721 pages

Abstract: Abstract Background Cystic fibrosis (CF) is a rare genetic condition requiring extensive medical care, which has a significant impact on people with CF. Advances in treatment have extended life expectancy, yet there remains a significant economic burden to manage CF. Cost-effectiveness analysis (CEA) is crucial for evaluating the economic value of treatments and screening for CF. This scoping review seeks to highlight the best practices and gaps in the current evidence base, contributing to robust and comparable CEAs in CF research. Methods A scoping review was conducted using PubMed and Embase. Studies were included if they featured a CEA focused on CF treatment. Data extraction covered study characteristics, model inputs, and modeling assumptions. A qualitative synthesis was conducted to assess the inclusion of considerations for both healthcare and societal impacts. Results In total, 11 studies were included. Of these, six focused on evaluations of supportive therapies for CF and five focused on evaluation of cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Heterogeneity in comparators and drug costing methods complicated cross-study comparisons. A qualitative review revealed differences in the types of costs and outcomes considered. Studies captured long-term disease progression, health-related quality-of-life effects, and direct medical costs. Conclusions This review highlights the complexity of CEAs for CF treatment and underscores the need for standardized methodologies and comprehensive evaluations, including broader economic impacts, to support more robust analyses and better-informed decision-making in CF treatment.

Date: 2025
References: Add references at CitEc
Citations:

Downloads: (external link)
http://link.springer.com/10.1007/s40273-025-01497-w Abstract (text/html)
Access to the full text of the articles in this series is restricted.

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:spr:pharme:v:43:y:2025:i:7:d:10.1007_s40273-025-01497-w

Ordering information: This journal article can be ordered from
http://www.springer.com/economics/journal/40273

DOI: 10.1007/s40273-025-01497-w

Access Statistics for this article

PharmacoEconomics is currently edited by Timothy Wrightson and Christopher I. Carswell

More articles in PharmacoEconomics from Springer
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().