‘Drug lag’ has been a problem in Japan in the last two decades. The Pharmaceuticals and Medical Devices Agency (PMDA), the Japanese regulator, has taken initiatives to shorten the review period, and access to drug has dramatically improved. We surveyed the current status of unapproved oncology drugs as of July 3, 2017.
We examined the approval status of the US Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the PMDA between January 2000 and July 2017. Data was extracted from the documents published on each regulators’ website.
A total of 59 oncology drugs have been approved by either the FDA or EMA, but not by the PMDA: 8 drugs were submitted for Japanese New Drug Applications and under review; 14 drugs were undergoing clinical trials in Japan. Twenty-four drugs were approved by both the FDA and EMA, and 35 were approved by either the FDA or EMA. Furthermore, thirty-four drugs were approved in the FDA or EMA in 2015 and thereafter, followed by 13 from 2010 to 2014, 8 from 2005 to 2009, and 4 from 2000 to 2004. The FDA/EMA-approved indications of 59 drugs are as follow: 26 drugs were approved for hematological malignancies; 9 for genitourinary malignancies (5 for urothelial carcinoma, 3 for prostate cancer, and 1 for renal cell carcinoma); 6 for skin cancer (3 for basal cell carcinoma, 2 for malignant melanoma, and 1 for Merkel cell carcinoma); 4 for breast cancer; 3 each for soft tissue sarcoma, lung cancer and ovarian cancer; 1 each for head and neck, thyroid, pancreatic cancer, neuroblastoma and malignant ascites. Thirty-seven (63%) indications correspond to rare cancers in Japan (defined as those with an annual incidence of
‘Drug lag’ for oncology drugs has been solved considerably in Japan, but it still exists. Approximately 60% of the FDA/EMA-approved indications for PMDA-unapproved drugs were for rare cancers in Japan. Drug development for rare cancers, especially development for diseases with a lower incidence rate in Japan than the US and EU is a future subject.
Clinical trial identification
Legal entity responsible for the study
Office for Advanced Medical Care Evaluation and Health Technology Assessment, National Cancer Center
All authors have declared no conflicts of interest.