Impact of CFTR Modulator Initiation on Direct Medical Costs for People With Cystic Fibrosis
Author(s)
Dominique Seo, MPH1, T. Joseph Mattingly, II, MBA, PharmD, PhD2, C. Daniel Mullins, PhD3, Kristine A. Parbuoni, PharmD, BCPPS4, Roumen M. Vesselinov, PhD3, Dave Young, PharmD5, Eberechukwu Onukwugha, MSc, PhD6.
1Graduate Student, University of Maryland, Baltimore, Baltimore, MD, USA, 2University of Utah, Park City, UT, USA, 3University of Maryland School of Medicine, Baltimore, MD, USA, 4University of Maryland School of Pharmacy, Baltimore, MD, USA, 5University of Utah, Salt Lake City, UT, USA, 6University of Maryland, School of Pharmacy, Baltimore, MD, USA.
1Graduate Student, University of Maryland, Baltimore, Baltimore, MD, USA, 2University of Utah, Park City, UT, USA, 3University of Maryland School of Medicine, Baltimore, MD, USA, 4University of Maryland School of Pharmacy, Baltimore, MD, USA, 5University of Utah, Salt Lake City, UT, USA, 6University of Maryland, School of Pharmacy, Baltimore, MD, USA.
OBJECTIVES: CFTR modulators (CFTRm) have transformed treatment and improved quality of life for people with Cystic Fibrosis (pwCF). However, there is limited evidence on cost impacts across healthcare settings, which provides important context for value assessment. This analysis evaluates the impact of CFTRm initiation on direct medical costs overall and across healthcare settings.
METHODS: This study used a 25% random sample of the IQVIA PharMetrics® Plus for Academics data; a comprehensive database composed of fully adjudicated insurance claims data and enrollment information for mostly commercial individuals. We included patients aged 0-64 who initiated a CFTRm between 2012-2021 and were continuously enrolled in a health plan for 12 months before and after treatment initiation. A pre-post design was used to compare direct medical costs in the year before and after treatment. We calculated total, inpatient, outpatient, emergency department (ED), and pharmacy costs in the 12 months before and after treatment initiation. Regression models adjusted for age, sex, census region, primary payer and plan type. We report incremental costs, estimated using a generalized linear model.
RESULTS: The sample consisted of 136 pwCF. Compared to the year before CFTRm initiation, in the year following treatment initiation the average total costs increased 3.2x or by $261,056. Pharmacy costs rose 5.8x from $49,542 to $289,313 (p < 0.01). ED costs decreased about 25% from $805 to $599, but this change was not statistically significant (p=0.43). Pre-post differences in inpatient and outpatient costs were also not statistically significant.
CONCLUSIONS: The substantial rise in direct medical costs after CFTRm initiation is driven by increased pharmacy spending. While ED costs decreased, the change was not statistically significant, and savings were insufficient to offset the surge in drug costs. The implications of the heterogeneity in cost can have significant impacts on patient access and value assessment and warrant further study.
METHODS: This study used a 25% random sample of the IQVIA PharMetrics® Plus for Academics data; a comprehensive database composed of fully adjudicated insurance claims data and enrollment information for mostly commercial individuals. We included patients aged 0-64 who initiated a CFTRm between 2012-2021 and were continuously enrolled in a health plan for 12 months before and after treatment initiation. A pre-post design was used to compare direct medical costs in the year before and after treatment. We calculated total, inpatient, outpatient, emergency department (ED), and pharmacy costs in the 12 months before and after treatment initiation. Regression models adjusted for age, sex, census region, primary payer and plan type. We report incremental costs, estimated using a generalized linear model.
RESULTS: The sample consisted of 136 pwCF. Compared to the year before CFTRm initiation, in the year following treatment initiation the average total costs increased 3.2x or by $261,056. Pharmacy costs rose 5.8x from $49,542 to $289,313 (p < 0.01). ED costs decreased about 25% from $805 to $599, but this change was not statistically significant (p=0.43). Pre-post differences in inpatient and outpatient costs were also not statistically significant.
CONCLUSIONS: The substantial rise in direct medical costs after CFTRm initiation is driven by increased pharmacy spending. While ED costs decreased, the change was not statistically significant, and savings were insufficient to offset the surge in drug costs. The implications of the heterogeneity in cost can have significant impacts on patient access and value assessment and warrant further study.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE527
Topic
Economic Evaluation, Real World Data & Information Systems
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Rare & Orphan Diseases