Can Rare Cancer Drugs Expect Sales in Japan?: A Prescription Pattern Analysis of Drugs for Chronic Myelogenous Leukemia and Neuroendocrine Tumor

Shoyo Shibata, Emi Noguchi, Maiko Matsushita, Takeshi Suzuki, Koken Ozaki


Despite high unmet medical needs, investment in rare cancer drug development has stagnated, likely because the potential market for such drugs is small. In this context, we hypothesized that rare cancer drugs could achieve a higher sales margin. A dataset was created from publicly available information obtained from the IQVIA Solutions Japan K.K. Pharmaceutical Market database on the website of the Pharmaceuticals and Medical Devices Agency/Ministry of Health, Labour and Welfare of Japan. The total amount of sales and prescription volumes between 2010 and 2016 for drugs whose indications include chronic myelogenous leukemia (CML) and neuroendocrine tumor (NET) were investigated. Regarding drugs for CML, the sales and prescription volumes of imatinib have been decreasing every year, whereas those of dasatinib and nilotinib have been increasing. Regarding drugs for NET, the sales and prescription volumes of sunitinib, everolimus, and streptozocin have been increasing every year. The present study revealed two sales models for the development of rare cancer drugs. First, sales amounts can be assured if clinical positioning with other existing drugs is sufficiently clear. Second, obtaining a label for rare cancers can stimulate drug development for more common cancers. These findings suggest that rare cancer drugs can offer high market value and profit potential; thus, to meet high unmet medical needs, clinical development programs for the development of rare cancer drugs should be promoted.


anticancer drug, marketing strategy, health economics, drug development, drug pricing

Full Text:



Ashley, D., Thomas, D., Gore, L., Carter, R., Zalcberg, J. R., Omtar, R., & Savulescu, J. (2015). Accepting risk in the acceleration of drug development for rare cancers. The Lancet Oncology, 16(4), e190-e194.

Fukuda, A. & Igarashi, A. (2016). Universal Health Coverage and Cancer Drugs - A Cost-Effectiveness Perspective (in Japanese). Gan To Kagaku Ryoho, 43(11), 1311-1315.

Fukumoto, D., Tsuyuki, A. & Suzuki, T. (2017). Drugs Targeted for Price Cutting in Japan: The Case of Price Revisions Based on the Divergence of Official Versus Delivery Prices. Therapeutic Innovation & Regulatory Science, 51(5), 597-603.

Gaddipati, H., Liu, K., Pariser, A., & Pazdur, R. (2012). Rare Cancer Trial Design: Lessons from FDA Approvals. Clinical Cancer Research, 18(19), 5172-5178.

Gatta, G., Van Der Zwan, J. M., Casali, P. G., Siesling, S., Dei Tos, A. P., Otter, R., . . . Capocaccoa, R. (2011). Rare cancers are not so rare: the rare cancer burden in Europe. European Journal of Cancer, 47(17), 2493-2511.

Hirakawa, A., Asano, J., Sato, H., & Teramukai, S. (2018). Master protocol trials in oncology: Review and new trial designs. Contemporary Clinical Trials Communications, 12, 1-8.

Kantarjian, H. M., Hochhaus, A., Saglio, G. De Souza, C., Flinn, I. W., Stenke, L., . . . Hughes, T. P. (2011). Nilotinib versus imatinib for the treatment of patients with newly diagnosed chronic phase, Philadelphia chromosome-positive, chronic myeloid leukaemia: 24-month minimum follow-up of the phase 3 randomised ENESTnd trial. The Lancet Oncology,12(9), 841-851.

Kawai A. (2015). Rare cancers, the clinical presentation and issues (in Japanese). Journal of Clinical and Experimental Medicine, 254(9), 621-627.

Kawai, A., Goto, T., Shibata, T., Tani, K., Mizutani, S., Nishikawa, A., . . . Ueda, R. (2018). Current state of therapeutic development for rare cancers in Japan, and proposals for improvement. Cancer Science, 109(5), 1731-1737.

Lipton, J. H., Chuah, C., Guerci-Bresler, A., Rosti, G., Simpson, D., Assouline, S., . . . Deininger, M. W. (2016). Ponatinib versus imatinib for newly diagnosed chronic myeloid leukaemia: an international, randomised, open-label, phase 3 trial. The Lancet Oncology, 17(5), 612-621.

Maeda, K., Kaneko, M., Narukawa, M., & Arato, T. (2017). Points to consider: efficacy and safety evaluations in the clinical development of ultra-orphan drugs. Orphanet Journal of Rare Diseases, 12(1), 143.

Ministry of Health, Labour and Welfare. Retrieved February 3, 2019 from

Nakamae, H., Fujisawa, S., Ogura, M., Uchida, T., Onishi, Y., Taniwaki, M., . . . Miyoshi, M. (2017). Dasatinib versus imatinib in Japanese patients with newly diagnosed chronic phase chronic myeloid leukemia: a subanalysis of the DASISION 5-year final report. International Journal of Hematology, 105(6), 792-804.

Ohtsu, A., Goto, K., Yoshino, T., Okamoto, W., & Tsuchihara, K. (2017). Current Status and Future Perspectives of SCRUM-Japan (in Japanese). Gan To Kagaku Ryoho, 44(8), 621-626.

Okuma, H. S., Yonemori, K., Shimizu, T., Yashushi, G., Honma, Y., Morizane, C., . . . Fujiwara, Y. (2018). MASTER KEY project: A basket/umbrella trial for rare cancers in Japan. Journal of Clinical Oncology, 36:15_suppl, TPS2598-TPS2598.

Oliver, A. (2003). Health economic evaluation in Japan: a case study of one aspect of health technology assessment. Health Policy, 63(2), 197-204.

Pharmaceutical and Medical Devices Agency. Retrieved February 3, 2019 from

Redig, A. J. & Janne, P. A. (2015). Basket trials and the evolution of clinical trial design in an era of genomic medicine. Journal of Clinical Oncology, 33(9), 975-977.

Renfro, L. A. & Sargent, D. J. (2016). Statistical controversies in clinical research: basket trials, umbrella trials, and other master protocols: a review and examples. Annals of Oncology, 28(1), 34-43.

Rotte, A., Jin, J. Y. & Lemaire, V. (2017). Mechanistic overview of immune checkpoints to support the rational design of their combinations in cancer immunotherapy. Annals of Oncology, 29(1), 71-83.

Shibata, S., Uemura, R., & Suzuki, T. (2016). Factors that Affect the Acquisition of Reward Premiums for Promotion of Innovative Drug Discovery in Japan. Therapeutic Innovation & Regulatory Science, 50(1), 56-65.

Shibata, S., Uemura, R., & Suzuki, T. (2016). Impact of Premium Rewards for the Promotion of Innovative Drug Discovery on the Japanese Pharmaceutical Market: An Analysis by Therapeutic Area. Therapeutic Innovation & Regulatory Science, 50(1), 49-55.

Shibata, S., Uemura, R., & Suzuki, T. (2016). Evaluating the Effectiveness of Repricing for Market Expansion in the Japanese Drug Pricing System. Therapeutic Innovation & Regulatory Science, 50(6), 751-758.

Shibata, S., Uemura, R., & Suzuki, T. (2016). Comparative Analysis Between the Top-Selling Drugs in the Japanese Pharmaceutical Market and Those in the United States, the United Kingdom, France, and Germany. Therapeutic Innovation & Regulatory Science, 50(2), 221-227.

Shibata, S., Uemura, R., Chiba, K., & Suzuki, T. (2016). A comprehensive analysis of factors that contribute to conditional approval and all-case surveillance designations that subsequently lead to shortening of review times in Japan. Journal of Regulatory Science, 4(1), 1-9.

Shibata, S., Kawaguchi, H., Uemura, R., & Suzuki, T. (2016). Emerging Growth of Orphan Drugs for Neurological Diseases in Japan: Potential Benefits for Both Patients and Pharmaceutical Companies. Journal of Regulatory Science, 4(3), 7-13.

Shibata, S., Matsushita, M., Saito, Y., & Suzuki, T. (2017). Optimal Anti-cancer Drug Profiles for Effective Penetration of the Anti-cancer Drug Market by Generic Drugs in Japan. Therapeutic Innovation & Regulatory Science, 52(4), 442-448.

Shibata, S., Wayama, Y., Tsuyuki, A., Matsushita, M., Chiba, K., Matsuki, E., . . . Suzuki, T. (2017). An empirical study of the prescription pattern of drugs for hematological malignancies in Japan from 2010–2014. Biological and Pharmaceutical Bulletin, 40(6), 894-901.

Shibata, S. & Suzuki, T. (2018) The pharmaceutical market and drug development prognosis in Japan: Current and future perspectives according to pharmacological classes. Journal of Generic Medicines, 14(2), 70-80.

Shibata, S., Matsushita, M., Saito, Y., & Suzuki, T. (2018). Anticancer Drug Prescription Patterns in Japan: Future Directions in Cancer Therapy. Therapeutic Innovation & Regulatory Science, 52(6), 718-723.

Simon, R. (2017). Critical Review of Umbrella, Basket, and Platform Designs for Oncology Clinical Trials. Clinical Pharmacology & Therapeutics, 102(6), 934-941.

Tamaki, T., Dong, Y., Ohno, Y., Sobue, T., Nishimoto, H. & Shibata, A. (2014). The burden of rare cancer in Japan: application of the RARECARE definition. Cancer Epidemiology, 38(5), 490-495.

Tang, J., Shalabi, A. & Hubbard-Lucey, V. M. (2017). Comprehensive analysis of the clinical immuno-oncology landscape. Annals of Oncology, 29(1), 84-91.

Tojo, A., Usuki, K., Urabe, A., Maeda, Y., Kobayashi, Y., Jinnai, I., . . . Naoe, T. (2009). A Phase I/II study of nilotinib in Japanese patients with imatinib-resistant or-intolerant Ph+ CML or relapsed/refractory Ph+ ALL. International Journal of Hematology, 89(5), 679-688.

Tojo, A., Kyo, T., Yamamoto, K., Nakamae, H., Takahashi, N., Kobayashi, Y., . . . Ohyashiki, K. (2017). Ponatinib in Japanese patients with Philadelphia chromosome-positive leukemia, a phase 1/2 study. International Journal of Hematology, 106(3), 385-397.

Wakutsu, N. & Nakamura, H. (2015). Analyzing the Benefits from New NHI Drug Pricing System in Japan: Factor Decomposition and Simulation (in Japanese). Iryo To Shakai, 25(2), 205-220.

Zafar, S. Y., Peppercorn, J. M., Schrag, D., Taylor, D. H., Goetzinger, A. M., Zhong, X. & Abernethy, A. P. (2013). The financial toxicity of cancer treatment: a pilot study assessing out-of-pocket expenses and the insured cancer patient's experience. The Oncologist, 18(4), 381-390.