Journal: medRxiv

Article Title: Early economic evaluation of a predictive tool to test CFTR modulator treatment response in people with cystic fibrosis

doi: 10.64898/2026.01.12.26343787

Figure Lengend Snippet: A) Decision tree design depicting treatment sequence of the treat-all and predict-response strategies. Patients are allocated to either standard of care (SoC) or cystic fibrosis transmembrane conductance regulator (CFTR) modulators. B) Schematic representation of the Markov model structure in which pwCF either receive SoC or CFTR modulators. PwCF enter the model in the mild, moderate or severe health state, whereafter they move through model in yearly cycles. Solid arrows indicate the possible directions of movement. Dotted arrows represent the possibility of moving to a less severe health state which is only possible in the first cycle in the arm receiving CFTR modulators.

Article Snippet: Costs for SoC, CFTR modulator treatment (ETI, Trikafta®/Kaftrio®) by Vertex Pharmaceuticals Incorporated, Boston, MA, USA) and PEX-events were calculated using public list prices and reference prices from the Dutch Healthcare Institute and the Dutch Healthcare Authority [ - ].

Techniques: Sequencing

Journal: medRxiv

Article Title: Early economic evaluation of a predictive tool to test CFTR modulator treatment response in people with cystic fibrosis

doi: 10.64898/2026.01.12.26343787

Figure Lengend Snippet:

Article Snippet: Costs for SoC, CFTR modulator treatment (ETI, Trikafta®/Kaftrio®) by Vertex Pharmaceuticals Incorporated, Boston, MA, USA) and PEX-events were calculated using public list prices and reference prices from the Dutch Healthcare Institute and the Dutch Healthcare Authority [ - ].

Techniques:

Journal: medRxiv

Article Title: Early economic evaluation of a predictive tool to test CFTR modulator treatment response in people with cystic fibrosis

doi: 10.64898/2026.01.12.26343787

Figure Lengend Snippet: The plot visualizes the impact of variation of individual parameters on the incremental cost-eAectiveness ratio by alternately varying input values between 80% (min, blue) and 120% (max, red) of their original value. CFTR = Cystic Fibrosis Transmembrane conductance Regulator, PEX = pulmonary exacerbation, ICER = Incremental Cost-EAectiveness Ratio.

Article Snippet: Costs for SoC, CFTR modulator treatment (ETI, Trikafta®/Kaftrio®) by Vertex Pharmaceuticals Incorporated, Boston, MA, USA) and PEX-events were calculated using public list prices and reference prices from the Dutch Healthcare Institute and the Dutch Healthcare Authority [ - ].

Techniques: