Cost-effectiveness Analysis of Genetic Testing and Tailored First-Line Therapy for Patients With Metastatic Gastrointestinal Stromal Tumors

Importance Gastrointestinal stromal tumor (GIST) is frequently driven by oncogenic KIT variations. Imatinib targeting of KIT marked a new era in GIST treatment and ushered in precision oncological treatment for all solid malignant neoplasms. However, studies on the molecular biological traits of GIST have found that tumors respond differentially to imatinib dosage based on the KIT exon with variation. Despite this knowledge, few patients undergo genetic testing at diagnosis, and empirical imatinib therapy remains routine. Barriers to genetic profiling include concerns about the cost and utility of testing. Objective To determine whether targeted gene testing (TGT) is a cost-effective diagnostic for patients with metastatic GIST from the US payer perspective. Design, Setting, and Participants This economic evaluation developed a Markov model to compare the cost-effectiveness of TGT and tailored first-line therapy compared with empirical imatinib therapy among patients with a new diagnosis of metastatic GIST. The main health outcome, quality-adjusted life years (QALYs), and costs were obtained from the literature, and transitional probabilities were modeled from disease progression and survival estimates from randomized clinical trials of patients with metastatic GIST. Data analyses were conducted October 2019 to January 2020. Exposure TGT and tailored first-line therapy. Main Outcomes and Measures The primary outcome was QALYs and cost. Cost-effectiveness was defined using an incremental cost-effectiveness ratio, with an incremental cost-effectiveness ratio less than $100000/QALY considered cost-effective. One-way and probabilistic sensitivity analyses were conducted to assess model stability. Results Therapy directed by TGT was associated with an increase of 0.10 QALYs at a cost of $9513 compared with the empirical imatinib approach, leading to an incremental cost-effectiveness ratio of $92100. These findings were sensitive to the costs of TGT, drugs, and health utility model inputs. Therapy directed by TGT remained cost-effective for genetic testing costs up to $3730. Probabilistic sensitivity analysis found that TGT-directed therapy was considered cost-effective 70% of the time. Conclusions and Relevance These findings suggest that using genetic testing to match treatment of KIT variations to imatinib dosing is a cost-effective approach compared with empirical imatinib. Question Is targeted gene testing cost-effective in patients with metastatic gastrointestinal stromal tumor? Findings This economic evaluation developed a Markov model to examine the cost-effectiveness of targeted gene testing with tailored imatinib dosing for patients with gastrointestinal stromal tumor based on KIT exon variation status. The model found that targeted gene testing was cost-effective, with an incremental cost-effectiveness ratio of $92100, compared with empirical treatment with imatinib. Meaning These findings support the cost-effectiveness of widespread adoption of genetic testing for patients newly diagnosed with metastatic gastrointestinal stromal tumor. This economic evaluation examines the cost-effectiveness of targeted gene testing for tailored first-line treatment among patients with gastrointestinal stromal tumors (GIST).