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

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.