Cost-Effectiveness Analysis of Treating Patients With NTRK-Positive Cancer With the Histology-Independent Therapy Entrectinib

HEcoPerMed consortium, Simone Huygens, Heleen Vellekoop, Matthijs Versteegh, Irene Santi, László Szilberhorn, Tamás Zelei, Balázs Nagy, Apostolos Tsiachristas, Rositsa Koleva-Kolarova, Sarah Wordsworth, Maureen Rutten-van Mölken*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

7 Citations (Scopus)
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Objectives: This study tackles several challenges of evaluating histology-independent treatments using entrectinib as an example. Histology-independent treatments are provided based on genetic marker(s) of tumors, regardless of the tumor type. We evaluated the lifetime cost-effectiveness of testing all patients for NTRK fusions and treating the positive cases with entrectinib compared with no testing and standard of care (SoC) for all patients. Methods: The health economic model consisted of a decision tree reflecting the NTRK testing phase followed by a microsimulation model reflecting treatment with either entrectinib or SoC. Efficacy of entrectinib was based on data from basket trials, whereas historical data from NTRK-negative patients were corrected for the prognostic value of NTRK fusions to model SoC. Results: “Testing” (testing for NTRK fusions, with subsequent entrectinib treatment in NTRK-positive patients and SoC in NTRK-negative patients) had higher per-patient quality-adjusted life-years (QALYs) and costs than “No testing” (SoC for all patients), with a difference of 0.0043 and €732, respectively. This corresponded to an incremental cost-effectiveness ratio (ICER) of €169 957/QALY and, using a cost-effectiveness threshold of €80 000/QALY, an incremental net monetary benefit of −€388. When excluding the costs of genetic testing for NTRK fusions, the ICER was reduced to €36 290/QALY and the incremental net monetary benefit increased to €188. Conclusions: When treatment requires the identification of a genetic marker, the associated costs and effects need to be accounted for. Because of the low prevalence of NTRK fusions, the number needed-to-test to identify patients eligible for entrectinib is large. Excluding the testing phase reduces the ICER substantially.

Original languageEnglish
Pages (from-to)193-203
Number of pages11
JournalValue in Health
Issue number2
Early online date10 Oct 2022
Publication statusPublished - Feb 2023

Bibliographical note

Funding/Support: The HEcoPerMed project has received funding from the European Union’s Horizon 2020 research and innovation program under
grant agreement number 824997.

Publisher Copyright: © 2022 International Society for Pharmacoeconomics and Outcomes Research, Inc.


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