154P - Clinical trial designs for the approval of rare cancer drugs in Japan

Date 17 December 2016
Event ESMO Asia 2016 Congress
Session Poster lunch
Topics Clinical research
Presenter Emi Noguchi
Citation Annals of Oncology (2016) 27 (suppl_9): ix46-ix51. 10.1093/annonc/mdw579
Authors E. Noguchi1, K. Yonemori1, T. Shimoi1, M. Yunokawa1, H.S. Okuma2, A. Kawachi2, A. Kitano3, T. Nishikawa2, A. Shimomura4, C. Shimizu4, Y. Takiguchi5, A. Kawai6, Y. Fujiwara2, K. Tamura2
  • 1Breast And Medical Oncology / Rare Cancer Center, National Cancer Center Hospital, 104-0045 - Tokyo/JP
  • 2Breast And Medical Oncology, National Cancer Center Hospital, 104-0045 - Tokyo/JP
  • 3Department Of Breast And Medical Oncology, National Cancer Center Hospital, 104-0045 - Tokyo/JP
  • 4Breast And Medical Oncology, National Cancer Center Hospital, Tokyo/JP
  • 5Medical Oncology, Graduate School of Medicine, Chiba University, 260-8670 - Chiba/JP
  • 6Rare Cancer Center, National Cancer Center Hospital, 104-0045 - Tokyo/JP

Abstract

Background

More attention has been focused on rare cancers in Japan, since the Japanese Cabinet launched the second term of the “Basic Plan for Promotion of Cancer Measures” in 2012. Patients with rare cancers are confronted with the challenge of diagnosis, treatment, research and drug development. This study aimed to characterize the clinical trial designs supporting drug approval for rare cancers.

Methods

We examined the Japanese New Drug Applications (JNDA) for oncology indications which were submitted between April 2006 and March 2016 and were approved by the Ministry of Health, Labour and Welfare (MHLW). Pharmaceuticals indicated for symptom management and supportive care were excluded. The information concerning clinical data package for JNDA was obtained from publicly accessible review reports of the Pharmaceuticals and Medical Devices Agency. We defined rare cancers as those with an annual incidence of 

Results

A total of 182 oncology indications for 106 drugs were included. Ninety rare cancer indications (67 drugs) granted regulatory approval. Among them, 20 indications were approved based on available evidence without conducting clinical trials additionally. For remaining 70 indications were as follows: 39 for hematologic diseases, 8 for sarcoma, and malignant melanoma, 5 for central nervous system tumors, 3 for neuroendocrine tumors, 2 for ovarian cancer, cervical cancer, and malignant pleural mesothelioma, 1 for urothelial cancer, and medullary thyroid cancer (overlapping). Fifty indications were granted orphan designations and 15 indications developed at the request of the MHLW. There were 24 indications approved based on the results of non-comparative phase II trials, 3 based on randomized phase II trials, and 43 based on phase III trials. Thirteen out of 43 phase III trials were international clinical trials. Both endpoint measuring time to event endpoint (e.g. progression-free survival) and tumor shrinkage (e.g. response rate) were commonly used (34 and 31 indications, respectively).

Conclusions

Wide variation exists in clinical trial designs supporting drug approval for rare cancers. Detailed investigation will give impetus for drug development in the area on rare cancers.

Clinical trial indentification

Legal entity responsible for the study

N/A

Funding

National Cancer Center

Disclosure

All authors have declared no conflicts of interest.