The relationship between cost and the recommendation, refusal, and discontinuation of treatment for chronic myeloid leukemia and multiple myeloma in Japan: a cross-sectional exploratory survey

• Published in: Journal of medical economics Vol. 25; no. 1; pp. 552 - 560
• Main Authors: Munakata, Taro, Handa, Yutaka, Mizuno, Toru, Tomiuchi, Nobuko, LoPresti, Michael, Shimizu, Junichi
• Format: Journal Article
• Language: English
• Published: England Taylor & Francis 31.12.2022
• Subjects: Cross-Sectional Studies; health economics; Hematology; Humans; Japan; Leukemia, Myelogenous, Chronic, BCR-ABL Positive - drug therapy; Multiple Myeloma - drug therapy; Protein Kinase Inhibitors - therapeutic use; treatment cost; treatment decisions; Japan; I19; Hematology; Japan; I1; I; I12; treatment decisions; treatment cost; health economics
• ISSN: 1369-6998; 1941-837X
• DOI: 10.1080/13696998.2022.2062951

This study aimed to ascertain the number of patients with chronic myelogenous leukemia (CML) and transplant-ineligible patients with multiple myeloma (MM) not recommended by their physicians for optimal drug treatment or who refuse, discontinue, reduce, or skip treatment owing to cost in Japan. A cross-sectional survey was conducted among hematologists, hematologic oncologists, and oncologists in Japan treating ≥1 patient with CML or ≥5 transplant-ineligible patients with MM per year. A total of 212 physicians participated: 105 treating patients with CML and 107 treating transplant-ineligible patients with MM. While treatment cost did not lead to non-optimal treatment most patients, physicians reported that they recommended non-optimal treatment to 6.53% of their patients with CML and 1.41% of their transplant-ineligible patients with MM, that 1.51 and 0.35% of their patients, respectively, refused treatment and that 1.97 and 0.71% discontinued treatment owing to treatment cost. However, no significant differences in the effect of treatment cost on recommendation, discontinuation, refusal, or reduction of treatment were observed. Non-recommendation of optimal treatment owing to treatment cost was most common for third-line CML and fourth-line transplant-ineligible MM treatment. Discontinuation due to treatment cost was most common in third-line treatment for both. Our results show that non-optimal treatment due to treatment cost occurs among some physicians in Japan for patients with CML and transplant-ineligible patients with MM, but it may be limited to a small percentage of patients. Further research is needed to identify the drivers of treatment decisions for physicians and patients, including those involving treatment cost.